Study of ACE-083 in Patients With Charcot-Marie-Tooth Dis... | NCT03124459 | Trialant
NCT03124459
Sponsor
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Status
Terminated
Last Update Posted
Sep 26, 2022Actual
Enrollment
63Actual
Phase
Phase 2
Conditions
Charcot-Marie-Tooth Disease
Interventions
ACE-083
Placebo
Countries
United States
Protocol Section
Identification Module
NCT ID
NCT03124459
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
A083-03
Secondary IDs
ID
Type
Description
Link
ACE-083
Other Identifier
Acceleron Pharma Inc.
Brief Title
Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease
Official Title
A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease Types 1 and X
Acronym
Not provided
Organization
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USAINDUSTRY
Status Module
Record Verification Date
Sep 2022
Overall Recruitment Status or Expanded Access Status
Terminated
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
(Investigation of ACE-083 for use in patients with CMT is being discontinued as it did not achieve functional secondary endpoints in the A083-03 trial.
Expanded Access Info
No
Start Date
Jul 31, 2017Actual
Primary Completion Date
Mar 11, 2020Actual
Completion Date
Mar 11, 2020Actual
First Submitted Date
Apr 12, 2017
First Submission Date that Met QC Criteria
Apr 20, 2017
First Posted Date
Apr 21, 2017Actual
Results Waived
Not provided
Results First Submitted Date
Mar 11, 2021
Results First Submitted that Met QC Criteria
Jul 8, 2021
Results First Posted Date
Jul 28, 2021Actual
Certification/Extension (aka Delayed Results) First Submitted Date
Not provided
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Not provided
Last Update Submitted Date
Sep 13, 2022
Last Update Posted Date
Sep 26, 2022Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USAINDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
Is FDA Regulated Drug
Yes
Is FDA Regulated Device
No
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
Brief Summary
This is a multicenter, phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of ACE-083 in patients with Charcot-Marie-Tooth Disease Type 1 and Type X (CMT1 and CMTX), to be conducted in two parts. Part 1 is non-randomized, open-label, dose-escalation and Part 2 is randomized, double-blind, and placebo-controlled.
Detailed Description
Part 1 (non-randomized, open-label, dose-escalation)
Part 1 will consist of up to 3 cohorts of 6 patients each and will evaluate multiple ascending dose levels of ACE-083 administered bilaterally once every 3 weeks for up to 5 doses in the tibialis anterior (TA) muscle. Patients in each cohort will be enrolled in a 4-week screening period before beginning treatment.
Part 2 (randomized, double-blind, placebo-controlled) Prior to the initiation of Part 2, a review of safety and efficacy data from Part 1 will be conducted by the Safety Review Team (SRT) to determine the recommended dose level (maximum 250 mg/muscle). A total of up to 40 new patients may be enrolled and randomized (1:1 randomization) to receive either ACE 083 (n=20) or placebo (n=20) bilaterally by injection into both TA muscles once every 3 weeks for up to 17 doses.
Study duration for Parts 1 and 2 for each patient will be approximately 24 weeks, including a 4-week screening period, a 12-week treatment period, and an 8-week follow-up period after the last dose.
Study duration for Part 2 will be 15 months, including 4-week screening, 6 months double blind placebo-controlled, 6 months open-label and 8 week follow-up.
Conditions Module
Conditions
Charcot-Marie-Tooth Disease
Keywords
CMT1 / CMTX
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 2
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
63Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
Part 1 Cohort 1
Experimental
ACE-083 150 mg intramuscular (IM) (tibialis anterior muscle), once every 3 weeks for up to 5 doses.
Drug: ACE-083
Part 1 Cohort 2
Experimental
ACE-083 200 mg IM (tibialis anterior muscle), once every 3 weeks for up to 5 doses.
Drug: ACE-083
Part 1 Cohort 3
Experimental
ACE-083 up to 250 mg IM (tibialis anterior muscle), once every 3 weeks for up to 5 doses.
Drug: ACE-083
Part 2 (double-blind placebo controlled)
Experimental
ACE-083 up to 250 mg IM (tibialis anterior muscle) or placebo, once every 3 weeks for up to 9 doses
Drug: ACE-083
Drug: Placebo
Part 2 (open label)
Experimental
ACE-083 up to 250 mg IM (tibialis anterior muscle), once every 3 weeks for up to 8 doses
Drug: ACE-083
Interventions
Name
Type
Description
Arm Group Labels
Other Names
ACE-083
Drug
Part 1 - Recombinant fusion protein. Part 2 - Recombinant fusion protein or buffer solution.
Part 1 Cohort 1
Part 1 Cohort 2
Part 1 Cohort 3
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Part 1: Frequency of Adverse Events
Number of subjects with at least one adverse event related to treatment intervention from Part 1 of this study. Since this outcome measure was only pre-specified for Part 1, only data from the Part 1 participants is reported.
From initiation of treatment (Study Day 1) to end of follow-up period for Part 1 (Study Day 141).
Part 2: Percent Change in Muscle Volume to the End of the Double-blind Placebo-controlled Portion of the Study.
The percent change from baseline in volume of injected muscle, by MRI compared to the Day 190 Assessment is reported. The pre-specified timepoint for this outcome was only for data collected from participants to the end of the double-blind placebo controlled portion of the study, therefore data for the open-label arm of the study is not reported.
From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190).
Secondary Outcomes
Measure
Description
Time Frame
Part 2: Absolute Change in Amount of Intramuscular Fat Tissue to the End of the Double-blind Placebo-controlled Portion of the Study
The absolute change from baseline in intramuscular fat fraction of the injected muscle, by MRI compared to Day 190 Assessment is reported. The pre-specified timepoint for this outcome was only for data collected from participants to the end of the double-blind placebo controlled portion of the study, therefore data for the open-label arm of the study is not reported.
Other Outcomes
Not provided
Eligibility Module
Eligibility Criteria
Key Inclusion Criteria
Age ≥ 18 years
Diagnosis of CMT1 or CMTX confirmed by:
Clinical presentation and electrodiagnostics
Genetically-confirmed CMT1 or CMTX for the patient or first-degree relative
Part 1:
Six-minute walk distance (6MWD) of at least 150 meters (without a brace or walker)
Independent ambulation for at least 10 meters, without a brace
Left and right ankle plantar flexion MRC grade 4+ to 5, inclusive
Part 2:
6MWD ≥ 150 and ≤ 500 meters (without a brace or walker); a maximum of 20% of enrolled patients with 6MWD ≥ 450 meters will be included
Left and right ankle plantar flexion MRC grade 4- to 5, inclusive
Left and right ankle dorsiflexion Medical Research Council (MRC) manual muscle testing (MMT) grade 3 to 4+ inclusive. No more than 12 of the 40 subjects may have a grade of 3 or 3+ on one or both sides.
Females of childbearing potential must have negative urine pregnancy test prior to enrollment and use highly effective birth control methods during study participation and for 8 weeks following the last dose of ACE-083. Males must agree to use a condom during any sexual contact with females of childbearing potential while participating in the study and for 8 weeks following the last dose of ACE-083, even if he has undergone a successful vasectomy.
Ability to adhere to the study visit schedule/procedures, and to understand and comply with protocol requirements
Signed written informed consent
Key Exclusion Criteria
History of active malignancy, with the exception of fully excised or treated basal cell carcinoma, cervical carcinoma in-situ, or ≤ 2 squamous cell carcinomas of the skin
Symptomatic cardiopulmonary disease, significant functional impairment, significant orthopedic or neuropathic pain, or other co morbidities that in the opinion of the investigator would limit a patient's ability to complete strength and/or functional assessments on study
Type 1 or type 2 diabetes mellitus
Thyroid disorder unless condition is stable with no change in treatment for at least 4 weeks before the first dose and no expected change for duration of study
Renal impairment (serum creatinine ≥ 2 times the upper limit of normal (ULN])
Increased risk of bleeding (i.e., due to hemophilia, platelet disorders, or use of any anticoagulation/platelet modifying therapies up to 2 weeks prior to Study Day 1 and for duration of study; low dose aspirin [≤ 100 mg daily] is permitted)
Severe deformity or ankle fixation that would sufficiently limit passive range of motion to affect assessment of dorsiflexion strength
Major surgery within 4 weeks prior to Study Day 1
Chronic pharmacologic doses of systemic corticosteroids (≥ 2 weeks) within 4 weeks before Study Day 1 and for duration of study; intra-articular/topical/inhaled/intranasal physiologic doses of systemic corticosteroids are permitted
Androgens, growth hormone, insulin or oral hormone replacement therapy within 6 months before Study Day 1 and for duration of study; topical physiologic androgen replacement is permitted
Any change in medications potentially affecting muscle strength or function within 4 weeks of Study Day 1 and for duration of study (e.g., creatinine, CoQ10, systemic beta-adrenergic agonists)
Previous exposure to any investigational agent potentially affecting muscle volume, muscle strength, or muscle or nerve function within 5 half-lives of last dose plus an additional 8-week washout period (or 12 weeks prior to Study Day 1 if half-life is unknown)
Any previous or current exposure to ACE-083
Significant change in physical activity or exercise (e.g., significant increase or decrease in intensity or frequency) within 8 weeks before Study Day 1 or inability to maintain the baseline level of physical activity throughout the study
Any condition that would prevent MRI scanning or compromise the ability to obtain a clear and interpretable scan of the lower leg, as applicable (e.g., knee/hip replacement metallic implants)
Known active substance abuse, including alcohol
History of sensitivity to protein pharmaceuticals
Female that is lactating/breast-feeding
Accepts Healthy Volunteers
No
Sex
All
Sex/Gender Based
Not provided
Sex/Gender Description
Not provided
Minimum Age
18 Years
Maximum Age
Not provided
Standard Ages
AdultOlder Adult
Study Population
Not provided
Sampling Method
Not provided
Contacts/Locations Module
Central Contacts
Not provided
Overall Officials
Name
Affiliation
Role
Jay Backstrom, MD
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Thomas FP, Brannagan TH 3rd, Butterfield RJ, Desai U, Habib AA, Herrmann DN, Eichinger KJ, Johnson NE, Karam C, Pestronk A, Quinn C, Shy ME, Statland JM, Subramony SH, Walk D, Stevens-Favorite K, Miller B, Leneus A, Fowler M, van de Rijn M, Attie KM. Randomized Phase 2 Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease. Neurology. 2022 Jun 6;98(23):e2356-e2367. doi: 10.1212/WNL.0000000000200325.
See Also Links
Not provided
Available IPD Information
Not provided
IPD Sharing Statement Module
Plan to Share IPD
Undecided
Description
Not provided
Types
Not provided
Time Frame
Not provided
Access Criteria
Not provided
URL
Not provided
Results Section
Participant Flow Module
Pre-assignment Details
Not provided
Recruitment Details
Not provided
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
Part 1 Cohort 1 (150 mg)
ACE-083 150 mg intramuscular (IM; tibialis anterior muscle), once every 3 weeks for up to 5 doses.
ACE-083: Part 1 - Recombinant fusion protein.
FG001
Part 1 Cohort 2 (200 mg)
Periods
Title
Milestones
Reasons Not Completed
Part 1-Open Label Dose Escalation
Type
Comment
Milestone Data
STARTED
Participants in the Part 2 double-blind placebo controlled portion of the study were not the same participants that participated in the Part 1 Open-Label Dose escalation portion of the study.
From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190).
Part 2: Percent Change in Muscle Strength to the End of the Double-blind Placebo-controlled Portion of the Study
Percent change from baseline in strength of the injected muscle, by Quantitative Muscle Testing (QMT) compared to Day 190 Assessment is reported. The pre-specified timepoint for this outcome was only for data collected from participants to the end of the double-blind placebo controlled portion of the study, therefore data for the open-label arm of the study is not reported.
From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190).
Part 2: Percent Change in Muscle Function - Walk/Run Time to the End of the Double-blind Placebo-controlled Portion of the Study
The percent change from baseline in functional assessments, as measured by 10-meter walk/run time when compared to Day 190 Assessment is reported. The pre-specified timepoint for this outcome was only for data collected from participants to the end of the double-blind placebo controlled portion of the study, therefore data for the open-label arm of the study is not reported.
From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190).
Part 2: Percent Change in Muscle Function - Walk Distance Assessed at the End of the Double-blind Placebo-controlled Portion of the Study
Percent change from baseline in functional assessments, as measured by 6-minute walk distance when compared to Day 190 Assessment, is reported. The pre-specified timepoint for this outcome was only for data collected from participants to the end of the double-blind placebo controlled portion of the study, therefore data for the open-label arm of the study is not reported.
From initiation of treatment (Study Day 1) to end of follow-up period for the double-blind placebo-controlled portion of the study (Study Day 190).
Part 2: Change in Balance and Fall Risk at the End of the Double-blind Placebo-controlled Portion of the Study.
Change from baseline in static and dynamic balance, as measured by the Berg Balance Scale, a 14-item scoring system to assess balance and fall risk in adults. The Berg balance scale is used to objectively determine a patient's ability (or inability) to safely balance during a series of predetermined tasks. It is a 14 item list with each item consisting of a five-point ordinal scale ranging from 0 to 4, with 0 indicating the lowest level of function and 4 the highest level of function. Total scores are used for reporting, with a range of 0-56, with higher scores mean a better demonstration of function. A score of 56 indicates functional balance. A score of < 45 indicates individuals may be at greater risk of falling. The pre-specified timepoints for reporting are baseline Berg scale score and Day 190 score, therefore data for the open-label arm of the study is not reported.
From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190).
Part 2: Percent Change in Balance and Fall Risk From Baseline to the End of the Double-blind Placebo-controlled Portion of the Study
Percent change was calculated for the difference from baseline and Day 190 Assessment scores on the Berg Balance Scale. The Berg Balance Scale, is a 14-item scoring system to assess balance and fall risk in adults. The Berg balance scale is used to objectively determine a patient's ability (or inability) to safely balance during a series of predetermined tasks. It is a 14 item list with each item consisting of a five-point ordinal scale ranging from 0 to 4, with 0 indicating the lowest level of function and 4 the highest level of function. Total scores are used for reporting, with a range of 0-56, with higher scores mean a better demonstration of function. A score of 56 indicates functional balance. A score of < 45 indicates individuals may be at greater risk of falling. The pre-specified timepoints for reporting are baseline Berg scale score and Day 190 score, therefore data for the open-label arm of the study is not reported.
From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190).
Part 2: Change in Clinical Examination Score From Baseline to End of the Double-blind Placebo-controlled Portion of the Study
Change from baseline in the Charcot-Marie-Tooth (CMT) Examination Score, version 2 (CMTES2), a composite scoring system to assess sensory and motor impairment in subjects with CMT. The total score is a subset of the following items from the CMT neuropathy score instrument: Sensory symptoms, Motor symptoms (legs), Motor symptoms (arms), Pinprick Sensibility, Vibration, Strength (legs), and Strength (arms). Each individual item is assessed using a rating from 0 to 4 inclusive. The range of CMTES2 scores is from 0 to 28 inclusive. A higher score means a greater degree of symptom severity. The Baseline score and score on the Day 190 Assessment are reported. The pre-specified timepoints for reporting are baseline Berg scale score and Day 190 score, therefore data for the open-label arm of the study is not reported.
From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190).
Part 2: Percent Change in Clinical Examination Score in Baseline to End of the Double-blind Placebo-controlled Portion of the Study
Percent change was calculated for the difference in the Charcot-Marie-Tooth (CMT) Examination Score (CMTES2) from baseline and Day 190 Assessment scores. The pre-specified timepoints for reporting are baseline Berg scale score and Day 190 score, therefore data for the open-label arm of the study is not reported.
From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190).
Part 2: Change in Patient-reported Quality of Life From Baseline to the End of the Double-blind Placebo-controlled Portion of the Study
The absolute change from baseline in Charcot-Marie-Tooth Health Index (CMT-HI), a disease-specific, patient-reported health index score from baseline and Day 190 Assessment scores. The pre-specified timepoints for reporting are baseline Berg scale score and Day 190 score, therefore data for the open-label arm of the study is not reported.
From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190).
Aurora
Colorado
80045
United States
University of Florida
Gainesville
Florida
32610
United States
University of Iowa Hospitals and Clinics
Iowa City
Iowa
52242
United States
University of Kansas Medical Center - Neurology Department
Kansas City
Kansas
66160
United States
University of Minnesota, Neurology Department
Minneapolis
Minnesota
55455
United States
Washington University School of Medicine
St Louis
Missouri
63110
United States
Hackensack University Medical Center
Hackensack
New Jersey
07601
United States
Columbia University
New York
New York
10032
United States
University of Rochester Medical Center, Neurology
Rochester
New York
14642
United States
Carolinas Healthcare System Neurosciences Institute
Charlotte
North Carolina
28203
United States
Oregon Health & Science University
Portland
Oregon
97239
United States
University of Pennsylvania
Philadelphia
Pennsylvania
19104
United States
University of Utah
Salt Lake City
Utah
84112
United States
University of Vermont
Burlington
Vermont
05401
United States
Virginia Commonwealth University
Richmond
Virginia
23298
United States
ACE-083 200 mg IM, (tibialis anterior muscle), once every 3 weeks for up to 5 doses.
ACE-083: Part 1 - Recombinant fusion protein.
FG002
Part 1 Cohort 3 (240 mg)
ACE-083 240 mg IM (tibialis anterior muscle), once every 3 weeks for up to 5 doses.
ACE-083: Part 1 - Recombinant fusion protein.
FG003
Part 2 (Double-blind Placebo Controlled) Placebo Arm
Placebo once every 3 weeks for up to 9 doses
ACE-083: Part 2 -buffer solution
FG004
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
ACE-083 240 mg IM (tibialis anterior muscle) once every 3 weeks for up to 9 doses
ACE-083: Part 2 - Recombinant fusion protein
FG005
Part 2 (Open-label) ACE-083 Arm
ACE-083 240 mg IM (tibialis anterior muscle) once every 3 weeks for up to 8 doses
ACE-083: Part 2 - Recombinant fusion protein
FG0006 subjects
FG0016 subjects
FG0026 subjects
FG0030 subjectsPatients in the double-blind placebo controlled study were not the same participants that participated in the Part 1 Open-Label Dose escalation portion of the study.
FG0040 subjectsPatients in the double-blind placebo controlled study were not the same participants that participated in the Part 1 Open-Label Dose escalation portion of the study.
FG0050 subjectsPatients in the double-blind placebo controlled study were not the same participants that participated in the Part 1 Open-Label Dose escalation portion of the study. Participants that completed the double-blind portion of the study then moved into this Part 2 open-label portion of the study.
COMPLETED
FG0006 subjects
FG0016 subjects
FG0026 subjects
FG0030 subjectsPatients in the double-blind placebo controlled study were not the same participants that participated in the Part 1 Open-Label Dose escalation portion of the study.
FG0040 subjectsPatients in the double-blind placebo controlled study were not the same participants that participated in the Part 1 Open-Label Dose escalation portion of the study.
FG0050 subjectsPatients in the double-blind placebo controlled study were not the same participants that participated in the Part 1 Open-Label Dose escalation portion of the study. Participants that completed the double-blind portion of the study then moved into this Part 2 open-label portion of the study.
NOT COMPLETED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG0040 subjects
FG0050 subjects
Phase 2- Randomized Doubled-blind
Type
Comment
Milestone Data
STARTED
Participants in Part 1 Open-Label Dose escalation portion of the study were not the same participants that participated in the double-blind placebo controlled study.
FG0000 subjectsParticipants in Part 1 Open-Label Dose escalation portion of the study were not the same participants that participated in the double-blind placebo controlled study.
FG0010 subjectsParticipants in Part 1 Open-Label Dose escalation portion of the study were not the same participants that participated in the double-blind placebo controlled study.
FG0020 subjectsParticipants in Part 1 Open-Label Dose escalation portion of the study were not the same participants that participated in the double-blind placebo controlled study.
FG00321 subjectsParticipants in the double-blind placebo controlled study were not the same participants that participated in the Part 1 Open-Label Dose escalation portion of the study.
FG00424 subjectsParticipants in the double-blind placebo controlled study were not the same participants that participated in the Part 1 Open-Label Dose escalation portion of the study.
FG0050 subjectsParticipants in the double-blind placebo controlled study were not the same participants that participated in the Part 1 Open-Label Dose escalation portion of the study. Participants that completed the double-blind portion of the study then moved into this Part 2 open-label portion of the study.
COMPLETED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0039 subjects
FG004
NOT COMPLETED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG00312 subjects
FG004
Type
Comment
Reasons
Adverse Event
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG003
Part 2- Open Label ACE-083
Type
Comment
Milestone Data
STARTED
Participants in Part 1 Open-Label Dose escalation portion of the study were not the same participants that participated in the double-blind placebo controlled study. Participants that completed the Part 2 double-blind placebo controlled portion of the study were able to move into the open-label portion of the study.
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjectsParticipants in the double-blind placebo controlled study were not the same participants that participated in the Part 1 Open-Label Dose escalation portion of the study. Participants that completed the double-blind placebo controlled portion of Part 2, moved into the open-label portion part 2 study.
FG0040 subjectsParticipants in the double-blind placebo controlled study were not the same participants that participated in the Part 1 Open-Label Dose escalation portion of the study. Participants that completed the double-blind placebo controlled portion of Part 2, moved into the open-label portion part 2 study.
FG00540 subjectsThe open-label portion of Part 2 are the participants that were enrolled in and completed the double-blind placebo-controlled portion of Part 2.
COMPLETED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
NOT COMPLETED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
Type
Comment
Reasons
Study terminated by Sponsor
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG003
The Full Analysis Set includes all randomized patients who have received at least 1 dose of study drug (includes placebo). Additionally, the participants in the Open-Label portion of Part 2 are a sub-set of the participants that enrolled and then subsequently completed the double-blind placebo controlled portion of Part 2. The open-label baseline measures are captured separately.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Part 1 Cohort 1
ACE-083 150 mg IM (tibialis anterior muscle), once every 3 weeks for up to 5 doses.
ACE-083: Part 1 - Recombinant fusion protein.
BG001
Part 1 Cohort 2
ACE-083 200 mg IM, (tibialis anterior muscle), once every 3 weeks for up to 5 doses.
ACE-083: Part 1 - Recombinant fusion protein.
BG002
Part 1 Cohort 3
ACE-083 240 mg IM (tibialis anterior muscle), once every 3 weeks for up to 5 doses.
ACE-083: Part 1 - Recombinant fusion protein.
BG003
Part 2 (Double-blind Placebo Controlled) Placebo
Placebo once every 3 weeks for up to 9 doses
ACE-083: Part 2 - buffer solution.
BG004
Part 2 (Double-blind Placebo Controlled) ACE-083
ACE-083 up to 250 mg IM (tibialis anterior muscle) once every 3 weeks for up to 9 doses
ACE-083 Part 2 - Recombinant fusion protein
BG005
Part 2 (Open-label) ACE 083-03 Arm
ACE-083 up to 250 mg IM (tibialis anterior muscle) once every 3 weeks for up to 8 doses
ACE-083 Part 2 - Recombinant fusion protein
BG006
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG0006
BG0016
BG0026
BG00321
BG00424
BG00540
BG006103
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Categorical
The participants in the Open-Label portion of Part 2 are a sub-set of the participants that enrolled and then subsequently completed the double-blind placebo controlled portion of Part 2. The open-label baseline measures are captured separately.
Count of Participants
Participants
Title
Denominators
Categories
ParticipantsBG0006
ParticipantsBG0016
ParticipantsBG0026
ParticipantsBG003
Age, Categorical
The participants in the Open-Label portion of Part 2 are a sub-set of the participants that enrolled and then subsequently completed the double-blind placebo controlled portion of Part 2.
Count of Participants
Participants
Title
Denominators
Categories
ParticipantsBG0000
ParticipantsBG0010
ParticipantsBG002
Age, Continuous
One patient in the Phase 2 randomized portion of the study was randomized (to the ACE-083 arm) but not dosed.
Mean
Standard Deviation
years
Title
Denominators
Categories
ParticipantsBG0006
ParticipantsBG0016
ParticipantsBG002
Age, Continuous
The participants in the Open-Label portion of Part 2 are a sub-set of the participants that enrolled and then subsequently completed the double-blind placebo controlled portion of Part 2.
Mean
Standard Deviation
years
Title
Denominators
Categories
ParticipantsBG0000
ParticipantsBG0010
ParticipantsBG002
Sex: Female, Male
One patient in the Phase 2 randomized portion of the study was randomized (to the ACE-083 arm) but not dosed.
Count of Participants
Participants
Title
Denominators
Categories
ParticipantsBG0006
ParticipantsBG0016
ParticipantsBG002
Sex: Female, Male
The participants in the Open-Label portion of Part 2 are a sub-set of the participants that enrolled and then subsequently completed the double-blind placebo controlled portion of Part 2.
Count of Participants
Participants
Title
Denominators
Categories
ParticipantsBG0000
ParticipantsBG0010
ParticipantsBG002
Ethnicity (NIH/OMB)
One patient in the Phase 2 randomized portion of the study was randomized (to the ACE-083 arm) but not dosed.
Count of Participants
Participants
Title
Denominators
Categories
ParticipantsBG0006
ParticipantsBG0016
ParticipantsBG002
Ethnicity (NIH/OMB)
The participants in the Open-Label portion of Part 2 are a sub-set of the participants that enrolled and then subsequently completed the double-blind placebo controlled portion of Part 2.
Count of Participants
Participants
Title
Denominators
Categories
ParticipantsBG0000
ParticipantsBG0010
ParticipantsBG002
Race (NIH/OMB)
One patient in the Phase 2 randomized portion of the study was randomized (to the ACE-083 arm) but not dosed.
Count of Participants
Participants
Title
Denominators
Categories
ParticipantsBG0006
ParticipantsBG0016
ParticipantsBG002
Race (NIH/OMB)
The participants in the Open-Label portion of Part 2 are a sub-set of the participants that enrolled and then subsequently completed the double-blind placebo controlled portion of Part 2.
Count of Participants
Participants
Title
Denominators
Categories
ParticipantsBG0000
ParticipantsBG0010
ParticipantsBG002
Region of Enrollment
The participants in the Open-Label portion of Part 2 are a sub-set of the participants that enrolled and then subsequently completed the double-blind placebo controlled portion of Part 2. The open-label baseline measures are captured separately.
Count of Participants
Participants
Title
Denominators
Categories
United States
ParticipantsBG0006
ParticipantsBG0016
ParticipantsBG002
Region of Enrollment
The participants in the Open-Label portion of Part 2 are a sub-set of the participants that enrolled and then subsequently completed the double-blind placebo controlled portion of Part 2.
Number
participants
Title
Denominators
Categories
United States
ParticipantsBG0000
ParticipantsBG0010
ParticipantsBG002
Type
Title
Description
Population Description
Reporting Status
Anticipated Posting Date
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Time Frame
Units Analyzed
Denominator Units Selected
Arm/Group Information
Denominators
Classes
Analyses
Primary
Part 1: Frequency of Adverse Events
Number of subjects with at least one adverse event related to treatment intervention from Part 1 of this study. Since this outcome measure was only pre-specified for Part 1, only data from the Part 1 participants is reported.
The Safety Set consists of all patients enrolled in the study who have received at least 1 dose of study drug.
Posted
Count of Participants
Participants
From initiation of treatment (Study Day 1) to end of follow-up period for Part 1 (Study Day 141).
ID
Title
Description
OG000
Part 1 Cohort 1 (150 mg)
ACE-083 150 mg IM (tibialis anterior muscle), once every 3 weeks for up to 5 doses.
ACE-083: Part 1 - Recombinant fusion protein.
OG001
Part 1 Cohort 2 (200 mg)
ACE-083 200 mg IM, (tibialis anterior muscle), once every 3 weeks for up to 5 doses.
ACE-083: Part 1 - Recombinant fusion protein.
OG002
Part 1 Cohort 3 (240 mg)
ACE-083 240 mg IM (tibialis anterior muscle), once every 3 weeks for up to 5 doses.
ACE-083: Part 1 - Recombinant fusion protein.
Units
Counts
Participants
OG0006
OG0016
OG0026
Title
Denominators
Categories
Title
Measurements
OG0006
OG0015
OG0026
Primary
Part 2: Percent Change in Muscle Volume to the End of the Double-blind Placebo-controlled Portion of the Study.
The percent change from baseline in volume of injected muscle, by MRI compared to the Day 190 Assessment is reported. The pre-specified timepoint for this outcome was only for data collected from participants to the end of the double-blind placebo controlled portion of the study, therefore data for the open-label arm of the study is not reported.
Per Protocol Set: All participants enrolled/randomized in the study who received at least 1 dose of the study drug (includes placebo) with no major protocol violations
Posted
Least Squares Mean
Standard Error
percent change
From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190).
ID
Title
Description
OG000
Part 2 (Double-blind Placebo Controlled) Placebo Arm
Placebo once every 3 weeks for up to 9 doses
ACE-083: Part 2 -buffer solution
OG001
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
ACE-083 240 mg IM (tibialis anterior muscle) once every 3 weeks for up to 9 doses
ACE-083: Part 2 - Recombinant fusion protein
Units
Secondary
Part 2: Absolute Change in Amount of Intramuscular Fat Tissue to the End of the Double-blind Placebo-controlled Portion of the Study
The absolute change from baseline in intramuscular fat fraction of the injected muscle, by MRI compared to Day 190 Assessment is reported. The pre-specified timepoint for this outcome was only for data collected from participants to the end of the double-blind placebo controlled portion of the study, therefore data for the open-label arm of the study is not reported.
Per Protocol Set: All participants enrolled/randomized in the study who received at least 1 dose of the study drug (includes placebo) with no major protocol violations
Posted
Least Squares Mean
Standard Error
mm3
From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190).
ID
Title
Description
OG000
Part 2 (Double-blind Placebo Controlled) Placebo Arm
Placebo once every 3 weeks for up to 9 doses
ACE-083: Part 2 -buffer solution
OG001
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
ACE-083 240 mg IM (tibialis anterior muscle) once every 3 weeks for up to 9 doses
ACE-083: Part 2 - Recombinant fusion protein
Secondary
Part 2: Percent Change in Muscle Strength to the End of the Double-blind Placebo-controlled Portion of the Study
Percent change from baseline in strength of the injected muscle, by Quantitative Muscle Testing (QMT) compared to Day 190 Assessment is reported. The pre-specified timepoint for this outcome was only for data collected from participants to the end of the double-blind placebo controlled portion of the study, therefore data for the open-label arm of the study is not reported.
Per Protocol Set: All participants enrolled/randomized in the study who received at least 1 dose of the study drug (includes placebo) with no major protocol violations
Posted
Least Squares Mean
Standard Error
percent change
From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190).
ID
Title
Description
OG000
Part 2 (Double-blind Placebo Controlled) Placebo Arm
Placebo once every 3 weeks for up to 9 doses
ACE-083: Part 2 -buffer solution
OG001
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
ACE-083 240 mg IM (tibialis anterior muscle) once every 3 weeks for up to 9 doses
ACE-083: Part 2 - Recombinant fusion protein
Secondary
Part 2: Percent Change in Muscle Function - Walk/Run Time to the End of the Double-blind Placebo-controlled Portion of the Study
The percent change from baseline in functional assessments, as measured by 10-meter walk/run time when compared to Day 190 Assessment is reported. The pre-specified timepoint for this outcome was only for data collected from participants to the end of the double-blind placebo controlled portion of the study, therefore data for the open-label arm of the study is not reported.
Per Protocol Set: All participants enrolled/randomized in the study who received at least 1 dose of the study drug (includes placebo) with no major protocol violations
Posted
Least Squares Mean
Standard Error
Percent change
From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190).
ID
Title
Description
OG000
Part 2 (Double-blind Placebo Controlled) Placebo Arm
Placebo once every 3 weeks for up to 9 doses
ACE-083: Part 2 -buffer solution
OG001
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
ACE-083 240 mg IM (tibialis anterior muscle) once every 3 weeks for up to 9 doses
ACE-083: Part 2 - Recombinant fusion protein
Secondary
Part 2: Percent Change in Muscle Function - Walk Distance Assessed at the End of the Double-blind Placebo-controlled Portion of the Study
Percent change from baseline in functional assessments, as measured by 6-minute walk distance when compared to Day 190 Assessment, is reported. The pre-specified timepoint for this outcome was only for data collected from participants to the end of the double-blind placebo controlled portion of the study, therefore data for the open-label arm of the study is not reported.
Per Protocol Set: All participants enrolled/randomized in the study who received at least 1 dose of the study drug (includes placebo) with no major protocol violations
Posted
Least Squares Mean
Standard Error
Percent change
From initiation of treatment (Study Day 1) to end of follow-up period for the double-blind placebo-controlled portion of the study (Study Day 190).
ID
Title
Description
OG000
Part 2 (Double-blind Placebo Controlled) Placebo Arm
Placebo once every 3 weeks for up to 9 doses
ACE-083: Part 2 -buffer solution
OG001
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
ACE-083 240 mg IM (tibialis anterior muscle) once every 3 weeks for up to 9 doses
ACE-083: Part 2 - Recombinant fusion protein
Secondary
Part 2: Change in Balance and Fall Risk at the End of the Double-blind Placebo-controlled Portion of the Study.
Change from baseline in static and dynamic balance, as measured by the Berg Balance Scale, a 14-item scoring system to assess balance and fall risk in adults. The Berg balance scale is used to objectively determine a patient's ability (or inability) to safely balance during a series of predetermined tasks. It is a 14 item list with each item consisting of a five-point ordinal scale ranging from 0 to 4, with 0 indicating the lowest level of function and 4 the highest level of function. Total scores are used for reporting, with a range of 0-56, with higher scores mean a better demonstration of function. A score of 56 indicates functional balance. A score of < 45 indicates individuals may be at greater risk of falling. The pre-specified timepoints for reporting are baseline Berg scale score and Day 190 score, therefore data for the open-label arm of the study is not reported.
Per Protocol Set: All participants enrolled/randomized in the study who received at least 1 dose of the study drug (includes placebo) with no major protocol violations
Posted
Mean
Standard Deviation
score on scale
From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190).
ID
Title
Description
OG000
Part 2 (Double-blind Placebo Controlled) Placebo Arm
Placebo once every 3 weeks for up to 9 doses
ACE-083: Part 2 -buffer solution
OG001
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
Secondary
Part 2: Percent Change in Balance and Fall Risk From Baseline to the End of the Double-blind Placebo-controlled Portion of the Study
Percent change was calculated for the difference from baseline and Day 190 Assessment scores on the Berg Balance Scale. The Berg Balance Scale, is a 14-item scoring system to assess balance and fall risk in adults. The Berg balance scale is used to objectively determine a patient's ability (or inability) to safely balance during a series of predetermined tasks. It is a 14 item list with each item consisting of a five-point ordinal scale ranging from 0 to 4, with 0 indicating the lowest level of function and 4 the highest level of function. Total scores are used for reporting, with a range of 0-56, with higher scores mean a better demonstration of function. A score of 56 indicates functional balance. A score of < 45 indicates individuals may be at greater risk of falling. The pre-specified timepoints for reporting are baseline Berg scale score and Day 190 score, therefore data for the open-label arm of the study is not reported.
Per Protocol Set: All participants enrolled/randomized in the study who received at least 1 dose of the study drug (includes placebo) with no major protocol violations
Posted
Mean
Standard Deviation
percent change in score on scale
From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190).
ID
Title
Description
OG000
Part 2 (Double-blind Placebo Controlled) Placebo Arm
Placebo once every 3 weeks for up to 9 doses
ACE-083: Part 2 -buffer solution
Secondary
Part 2: Change in Clinical Examination Score From Baseline to End of the Double-blind Placebo-controlled Portion of the Study
Change from baseline in the Charcot-Marie-Tooth (CMT) Examination Score, version 2 (CMTES2), a composite scoring system to assess sensory and motor impairment in subjects with CMT. The total score is a subset of the following items from the CMT neuropathy score instrument: Sensory symptoms, Motor symptoms (legs), Motor symptoms (arms), Pinprick Sensibility, Vibration, Strength (legs), and Strength (arms). Each individual item is assessed using a rating from 0 to 4 inclusive. The range of CMTES2 scores is from 0 to 28 inclusive. A higher score means a greater degree of symptom severity. The Baseline score and score on the Day 190 Assessment are reported. The pre-specified timepoints for reporting are baseline Berg scale score and Day 190 score, therefore data for the open-label arm of the study is not reported.
Per Protocol Set: All participants enrolled/randomized in the study who received at least 1 dose of the study drug (includes placebo) with no major protocol violations
Posted
Mean
Standard Deviation
score on scale
From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190).
ID
Title
Description
OG000
Part 2 (Double-blind Placebo Controlled) Placebo Arm
Placebo once every 3 weeks for up to 9 doses
ACE-083: Part 2 -buffer solution
OG001
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
Secondary
Part 2: Percent Change in Clinical Examination Score in Baseline to End of the Double-blind Placebo-controlled Portion of the Study
Percent change was calculated for the difference in the Charcot-Marie-Tooth (CMT) Examination Score (CMTES2) from baseline and Day 190 Assessment scores. The pre-specified timepoints for reporting are baseline Berg scale score and Day 190 score, therefore data for the open-label arm of the study is not reported.
Per Protocol Set: All participants enrolled/randomized in the study who received at least 1 dose of the study drug (includes placebo) with no major protocol violations.
Posted
Mean
Standard Deviation
percent change in score on scale
From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190).
ID
Title
Description
OG000
Part 2 (Double-blind Placebo Controlled) Placebo Arm
Placebo once every 3 weeks for up to 9 doses
ACE-083: Part 2 -buffer solution
OG001
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
ACE-083 240 mg IM (tibialis anterior muscle) once every 3 weeks for up to 9 doses
ACE-083: Part 2 - Recombinant fusion protein
Units
Secondary
Part 2: Change in Patient-reported Quality of Life From Baseline to the End of the Double-blind Placebo-controlled Portion of the Study
The absolute change from baseline in Charcot-Marie-Tooth Health Index (CMT-HI), a disease-specific, patient-reported health index score from baseline and Day 190 Assessment scores. The pre-specified timepoints for reporting are baseline Berg scale score and Day 190 score, therefore data for the open-label arm of the study is not reported.
Per Protocol Set: All participants enrolled/randomized in the study who received at least 1 dose of the study drug (includes placebo) with no major protocol violations
Posted
Least Squares Mean
Standard Deviation
absolute change in score on a scale
From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190).
ID
Title
Description
OG000
Part 2 (Double-blind Placebo Controlled) Placebo Arm
Placebo once every 3 weeks for up to 9 doses
ACE-083: Part 2 -buffer solution
OG001
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
ACE-083 240 mg IM (tibialis anterior muscle) once every 3 weeks for up to 9 doses
ACE-083: Part 2 - Recombinant fusion protein
Time Frame
Part 1 adverse events (AEs) were collected from baseline through Day 141. For Part 2, AEs were collected from on or after the first double-blind study drug administration up to the end of the double-blind period (Day 190). For the open-label period, adverse events are defined as AEs that started or worsened in intensity on or after the first open-label study drug administration up to the end of the study(Day 393/end-of-study).
Description
Not provided
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
Part 1 Cohort 1(150mg)
ACE-083 150 mg IM (tibialis anterior muscle), once every 3 weeks for up to 5 doses.
ACE-083: Part 1 - Recombinant fusion protein.
0
6
0
6
6
6
EG001
Part 1 Cohort 2 (200mg)
ACE-083 200 mg IM, (tibialis anterior muscle), once every 3 weeks for up to 5 doses.
ACE-083: Part 1 - Recombinant fusion protein.
0
6
0
6
6
6
EG002
Part 1 Cohort 3 (250mg)
ACE-083 up to 250 mg IM (tibialis anterior muscle), once every 3 weeks for up to 5 doses.
ACE-083: Part 1 - Recombinant fusion protein.
0
6
1
6
6
6
EG003
Part 2 (Double-blind Placebo Controlled) Placebo Arm
Placebo, once every 3 weeks for up to 9 doses
ACE-083:
Part 2 - Placebo: buffer solution
0
21
1
21
19
21
EG004
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
ACE-083 up to 250 mg IM (tibialis anterior muscle) once every 3 weeks for up to 9 doses
ACE-083:
Part 2 - Recombinant fusion protein
0
23
0
23
20
23
EG005
Part 2 (Open Label)
ACE-083 up to 250 mg IM (tibialis anterior muscle), once every 3 weeks for up to 8 doses
ACE-083: Part 1 - Recombinant fusion protein. Part 2 - Recombinant fusion protein or buffer solution.
0
40
1
40
36
40
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Hypokalaemia
Metabolism and nutrition disorders
MEdDRA 19.1
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG0030 affected21 at risk
EG0040 affected23 at risk
EG0050 affected40 at risk
Suicide attempt
Psychiatric disorders
MEdDRA 19.1
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
Cellulitis
Infections and infestations
MEdDRA 19.1
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
pain in extremity
Musculoskeletal and connective tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0003 affected6 at risk
EG0010 affected6 at risk
EG0022 affected6 at risk
EG0034 affected21 at risk
EG004
muscle spasm
Musculoskeletal and connective tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0012 affected6 at risk
EG0021 affected6 at risk
EG003
myalgia
Musculoskeletal and connective tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0011 affected6 at risk
EG0021 affected6 at risk
EG003
joint stiffness
Musculoskeletal and connective tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG003
muscle tightness
Musculoskeletal and connective tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG003
arthralgia
Musculoskeletal and connective tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0011 affected6 at risk
EG0020 affected6 at risk
EG003
back pain
Musculoskeletal and connective tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG003
limb discomfort
Musculoskeletal and connective tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
muscle twitching
Musculoskeletal and connective tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG003
muscular weakness
Musculoskeletal and connective tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
musculoskeletal stiffness
Musculoskeletal and connective tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
osteoarthritis
Musculoskeletal and connective tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0011 affected6 at risk
EG0020 affected6 at risk
EG003
musculoskeletal discomfort
Musculoskeletal and connective tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
neck pain
Musculoskeletal and connective tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
arthritis
Musculoskeletal and connective tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
flank pain
Musculoskeletal and connective tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
haemarthrosis
Musculoskeletal and connective tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
joint instability
Musculoskeletal and connective tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
systemic lupus erythematosus
Musculoskeletal and connective tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
injection site discomfort
General disorders
MedDRA (19.1)
Systematic Assessment
EG0003 affected6 at risk
EG0012 affected6 at risk
EG0023 affected6 at risk
EG003
injection site bruising
General disorders
MedDRA (19.1)
Systematic Assessment
EG0002 affected6 at risk
EG0012 affected6 at risk
EG0022 affected6 at risk
EG003
injection site erythema
General disorders
MedDRA (19.1)
Systematic Assessment
EG0002 affected6 at risk
EG0011 affected6 at risk
EG0021 affected6 at risk
EG003
injection site pain
General disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0011 affected6 at risk
EG0021 affected6 at risk
EG003
injection site swelling
General disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0011 affected6 at risk
EG0021 affected6 at risk
EG003
injection site pruritus
General disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG003
injection site discoloration
General disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0011 affected6 at risk
EG0020 affected6 at risk
EG003
injection site macule
General disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0011 affected6 at risk
EG0020 affected6 at risk
EG003
injection site reaction
General disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
injection site warmth
General disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
peripheral swelling
General disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG003
fatigue
General disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
vessel puncture site bruise
General disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
chest discomfort
General disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
injection site dysaethesia
General disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
injection site induration
General disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
injection site papule
General disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
injection site paraesthesia
General disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
local swelling
General disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
malaise
General disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
medical device site pain
General disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
pyrexia
General disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
influenza like illness
General disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
injection site haematoma
General disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
oedema peripheral
General disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
tenderness
General disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
influenza
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG003
nasopharyngitis
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG003
upper respiratory tract infection
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0011 affected6 at risk
EG0020 affected6 at risk
EG003
viral infection
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
viral upper respiratory tract infection
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG003
localised infection
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
urinary tract infection
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
cellulitis
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
gingivitis
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
onychomycosis
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
pharyngitis streptococcal
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
pneunomia
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
respiratory tract infection
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
sinobronchitis
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
sinusitis
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
conjunctivitis
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
ear infection
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
fungal skin infection
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
labyrinthitis
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
laryngitis
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
lyme disease
Infections and infestations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
fall
Injury, poisoning and procedural complications
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0012 affected6 at risk
EG0020 affected6 at risk
EG003
ligament sprain
Injury, poisoning and procedural complications
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0011 affected6 at risk
EG0020 affected6 at risk
EG003
thermal burn
Injury, poisoning and procedural complications
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
tibia fracture
Injury, poisoning and procedural complications
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG003
contusion
Injury, poisoning and procedural complications
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
skin abrasion
Injury, poisoning and procedural complications
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
arthropod bite
Injury, poisoning and procedural complications
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
arthropod sting
Injury, poisoning and procedural complications
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
bone contusion
Injury, poisoning and procedural complications
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
muscle strain
Injury, poisoning and procedural complications
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
post-traumatic pain
Injury, poisoning and procedural complications
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
exposure to communicable disease
Injury, poisoning and procedural complications
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
joint dislocation
Injury, poisoning and procedural complications
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
limb injury
Injury, poisoning and procedural complications
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
procedural pain
Injury, poisoning and procedural complications
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
headache
Nervous system disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG003
memory impairment
Nervous system disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG003
paraesthesia
Nervous system disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG003
restless legs syndrome
Nervous system disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
burning sensation
Nervous system disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
dizziness
Nervous system disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
migraine
Nervous system disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
sensory loss
Nervous system disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
disturbance in attention
Nervous system disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
hypoaesthesia
Nervous system disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
presyncope
Nervous system disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
syncope
Nervous system disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
dermatitis contact
Skin and subcutaneous tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG003
erythema
Skin and subcutaneous tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG003
nail discolouration
Skin and subcutaneous tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
pruritus
Skin and subcutaneous tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0011 affected6 at risk
EG0020 affected6 at risk
EG003
skin warm
Skin and subcutaneous tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG003
dermatosis
Skin and subcutaneous tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
eczema
Skin and subcutaneous tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
mechanical urticaria
Skin and subcutaneous tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
onychoclasis
Skin and subcutaneous tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
pseudofolliculitis barbae
Skin and subcutaneous tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
rash
Skin and subcutaneous tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
skin burning sensation
Skin and subcutaneous tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
skin discolouration
Skin and subcutaneous tissue disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
diarrhoea
Gastrointestinal disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG003
gingival swelling
Gastrointestinal disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
vomiting
Gastrointestinal disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
nausea
Gastrointestinal disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
gingival pain
Gastrointestinal disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
lip swelling
Gastrointestinal disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
oral pain
Gastrointestinal disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
toothache
Gastrointestinal disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
abdominal pain upper
Gastrointestinal disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
dysphagia
Gastrointestinal disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
gastrointestinal disorders
Gastrointestinal disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
cough
Respiratory, thoracic and mediastinal disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
dyspnoea
Respiratory, thoracic and mediastinal disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
dyspnoea exertional
Respiratory, thoracic and mediastinal disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
epistaxis
Respiratory, thoracic and mediastinal disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0011 affected6 at risk
EG0020 affected6 at risk
EG003
oropharyngeal pain
Respiratory, thoracic and mediastinal disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
rhinorrheoea
Respiratory, thoracic and mediastinal disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
upper respiratory tract congestion
Respiratory, thoracic and mediastinal disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
paranasal sinus discomfort
Respiratory, thoracic and mediastinal disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
snoring
Respiratory, thoracic and mediastinal disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
flushing
Vascular disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0012 affected6 at risk
EG0020 affected6 at risk
EG003
Behcet's syndrome
Vascular disorders
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
hypertension
Vascular disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0011 affected6 at risk
EG0020 affected6 at risk
EG003
haematoma
Vascular disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
blood creatine phosphokinase increased
Investigations
MedDRA (19.1)
Systematic Assessment
EG0001 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
crystal urine present
Investigations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
myoglobin blood increased
Investigations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
thyroxine free increased
Investigations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
urine analysis abnormal
Investigations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
bacterial test positive
Investigations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
gamma-glutamyltransferase increased
Investigations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
platelet count increased
Investigations
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
hypokalaemia
Metabolism and nutrition disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG003
hypercholesterolaemia
Metabolism and nutrition disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
decreased appetitie
Metabolism and nutrition disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
iron deficiency
Metabolism and nutrition disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
anxiety
Psychiatric disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0021 affected6 at risk
EG003
attention deficit/hyperactivity disorder
Psychiatric disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
confusional state
Psychiatric disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
goitre
Endocrine disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
chromaturia
Renal and urinary disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
urine odour abnormal
Renal and urinary disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
calculus urinary
Renal and urinary disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
nephrolithiasis
Renal and urinary disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
pelvic pain
Reproductive system and breast disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
menorrhagia
Reproductive system and breast disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
tooth extraction
Surgical and medical procedures
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
palpitations
Cardiac disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
tachycardia
Cardiac disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
inflammation of lacrimal passage
Eye disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
vitreous haemorrhage
Eye disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
hepatic cyst
Hepatobiliary disorders
MedDRA (19.1)
Systematic Assessment
EG0000 affected6 at risk
EG0010 affected6 at risk
EG0020 affected6 at risk
EG003
The study was terminated because the statistically significant increase in total muscle volume (TMV) failed to translate to statistically significant improvements in functional or disease-related quality of life secondary endpoints in the study.
Certain Agreements
Are all PI(s) employees of the sponsor?
No
Restriction Type
LTE60
Results Disclosure Restriction on PI(s)?
Yes
Other Details
Not provided
Point of Contact
Title
Organization
Phone
Extension
Email
Senior Vice President, Global Clinical Development