Safety, Tolerability, Pharmacokinetics, and Pharmacodynam... | NCT02519036

NCT02519036

ID	Type	Description	Link
2015-000381-66	EudraCT Number

Status Module

Sponsor/Collaborators Module

Oversight Module

Description Module

Conditions Module

Design Module

Interventional Study Design

Biospecimen

No data available

No data is available for this block.

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
ISIS 443139 10 mg	Experimental	Participants received ISIS 443139, 10 milligrams (mg), by intrathecal injection, on Study Days 1, 29, 57, and 85.	Drug: ISIS 443139 10 mg
ISIS 443139 30 mg	Experimental	Participants received ISIS 443139, 30 mg, by intrathecal injection, on Study Days 1, 29, 57, and 85.	Drug: ISIS 443139 30 mg
ISIS 443139 60 mg	Experimental	Participants received ISIS 443139, 60 mg, by intrathecal injection, on Study Days 1, 29, 57, and 85.	Drug: ISIS 443139 60 mg
ISIS 443139 90 mg	Experimental	Participants received ISIS 443139, 90 mg, by intrathecal injection, on Study Days 1, 29, 57, and 85.	Drug: ISIS 443139 90 mg
ISIS 443139 120 mg	Experimental	Participants received ISIS 443139, 120 mg, by intrathecal injection, on Study Days 1, 29, 57, and 85.	Drug: ISIS 443139 120 mg
Placebo

Name	Type	Description	Arm Group Labels	Other Names
ISIS 443139 10 mg	Drug	ISIS 443139, 10 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85.	ISIS 443139 10 mg

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Number of Participants With Treatment-related Adverse Events (TEAEs)	An adverse event (AE) was any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered related to the investigational drug product. An AE was to be regarded as a TEAE if it was present prior to receiving the first dose of Study Drug and subsequently worsened or was not present prior to receiving the first dose of Study Drug but subsequently appeared.	Up to approximately 28 weeks

Measure	Description	Time Frame
Observed Cerebrospinal Fluid (CSF) Concentration for ISIS 443139		Days 1, 29, 57, 85, and 113 or 141

Other Outcomes

Measure	Description	Time Frame
Maximum Plasma Concentration (Cmax) for ISIS 443139		Days 1 and 85
Time to Maximum Plasma Concentration (Tmax) for ISIS 443139		Days 1 and 85
Change From Baseline in CSF Mutant Huntingtin (fM) Protein Concentration

Eligibility Module

Contacts/Locations Module

Facility	Status	City	State	ZIP	Country	Contacts
University of British Columbia		Vancouver	British Columbia	V6T 2B5	Canada
Charite University Berlin

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
31381524	Derived	Rodrigues FB, Ferreira JJ, Wild EJ. Huntington's Disease Clinical Trials Corner: June 2019. J Huntingtons Dis. 2019;8(3):363-371. doi: 10.3233/JHD-199003.
31059641	Derived	Tabrizi SJ, Leavitt BR, Landwehrmeyer GB, Wild EJ, Saft C, Barker RA, Blair NF, Craufurd D, Priller J, Rickards H, Rosser A, Kordasiewicz HB, Czech C, Swayze EE, Norris DA, Baumann T, Gerlach I, Schobel SA, Paz E, Smith AV, Bennett CF, Lane RM; Phase 1-2a IONIS-HTTRx Study Site Teams. Targeting Huntingtin Expression in Patients with Huntington's Disease. N Engl J Med. 2019 Jun 13;380(24):2307-2316. doi: 10.1056/NEJMoa1900907. Epub 2019 May 6.

Label	URL
Ionis Pharmaceuticals, Inc. home page	View source

IPD Sharing Statement Module

No data available

No data is available for this block.

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	Placebo	Participants received placebo, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
FG001	ISIS 443139 10 mg	Participants received ISIS 443139, 10 milligrams (mg), by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Annotation Section

No data available

No data is available for this block.

Document Section

Large Document Module

Uploaded Document Information

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot_SAP	Yes	Yes	No	Study Protocol and Statistical Analysis Plan	Mar 15, 2018

Derived Section

Miscellaneous Info Module

Submission Tracking

No data available

No data is available for this block.

Condition Browse Module

Intervention Browse Module

Masking Info

Safety set included all participants who were randomized and received at least one dose of study drug.

Arm/Group Information

ID	Title	Description
BG000	Placebo	Participants received placebo, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
BG001	ISIS 443139 10 mg	Participants received ISIS 443139, 10 milligrams (mg), by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
BG002	ISIS 443139 30 mg	Participants received ISIS 443139, 30 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
BG003	ISIS 443139 60 mg	Participants received ISIS 443139, 60 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
BG004	ISIS 443139 90 mg	Participants received ISIS 443139, 90 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
BG005	ISIS 443139 120 mg	Participants received ISIS 443139, 120 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
BG006	Total	Total of all reporting groups

Units	Counts
Participants	BG00012 BG0013 BG0026 BG0036 BG0049 BG00510 BG00646

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Continuous

Mean

Standard Deviation

years

Title

Denominators

Categories

Title	Measurements
	BG00049± 10 BG00144± 17 BG00253± 7 BG003

Sex: Female, Male

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Female	BG0004 BG0011 BG002

Race/Ethnicity, Customized

Count of Participants

Participants

Title

Denominators

Categories

White

Title	Measurements
	BG00011 BG0013 BG002

Race/Ethnicity, Customized

Count of Participants

Participants

Title

Denominators

Categories

Not Hispanic or Latino

Title	Measurements
	BG00012 BG0013 BG002

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

Number of Participants With Treatment-related Adverse Events (TEAEs)

An adverse event (AE) was any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered related to the investigational drug product. An AE was to be regarded as a TEAE if it was present prior to receiving the first dose of Study Drug and subsequently worsened or was not present prior to receiving the first dose of Study Drug but subsequently appeared.

Safety set included all participants who were randomized and received at least one dose of study drug.

Posted

Count of Participants

Participants

Up to approximately 28 weeks

ID	Title	Description
OG000	Placebo	Participants received placebo, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG001	ISIS 443139 10 mg	Participants received ISIS 443139, 10 milligrams (mg), by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG002	ISIS 443139 30 mg	Participants received ISIS 443139, 30 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG003	ISIS 443139 60 mg	Participants received ISIS 443139, 60 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG004	ISIS 443139 90 mg	Participants received ISIS 443139, 90 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG005	ISIS 443139 120 mg	Participants received ISIS 443139, 120 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.

Units	Counts
Participants	OG00012 OG0013 OG0026 OG003

Title

Denominators

Categories

Title	Measurements
	OG00012 OG0013 OG0026 OG003

Secondary

Observed Cerebrospinal Fluid (CSF) Concentration for ISIS 443139

Pharmacokinetic (PK) population included all participants who were randomized to ISIS 443139, received at least one dose and had sufficient sampling to permit PK evaluation.

Posted

Mean

Standard Deviation

nanograms per milliliter (ng/mL)

Days 1, 29, 57, 85, and 113 or 141

ID	Title	Description
OG000	ISIS 443139 10 mg	Participants received ISIS 443139, 10 milligrams (mg), by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG001	ISIS 443139 30 mg	Participants received ISIS 443139, 30 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG002	ISIS 443139 60 mg	Participants received ISIS 443139, 60 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG003	ISIS 443139 90 mg	Participants received ISIS 443139, 90 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.

Other Pre-specified

Maximum Plasma Concentration (Cmax) for ISIS 443139

PK population included all participants who were randomized to ISIS 443139, received at least one dose and had sufficient sampling to permit PK evaluation.

Posted

Geometric Mean

Geometric Coefficient of Variation

ng/mL

Days 1 and 85

ID	Title	Description
OG000	ISIS 443139 10 mg	Participants received ISIS 443139, 10 milligrams (mg), by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG001	ISIS 443139 30 mg	Participants received ISIS 443139, 30 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG002	ISIS 443139 60 mg	Participants received ISIS 443139, 60 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG003	ISIS 443139 90 mg	Participants received ISIS 443139, 90 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.

Other Pre-specified

Time to Maximum Plasma Concentration (Tmax) for ISIS 443139

PK population included all participants who were randomized to ISIS 443139, received at least one dose and had sufficient sampling to permit PK evaluation.

Posted

Median

Full Range

hour (h)

Days 1 and 85

ID	Title	Description
OG000	ISIS 443139 10 mg	Participants received ISIS 443139, 10 milligrams (mg), by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG001	ISIS 443139 30 mg	Participants received ISIS 443139, 30 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG002	ISIS 443139 60 mg	Participants received ISIS 443139, 60 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG003	ISIS 443139 90 mg	Participants received ISIS 443139, 90 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.

Other Pre-specified

Change From Baseline in CSF Mutant Huntingtin (fM) Protein Concentration

Baseline was defined as the last non-missing measure prior to the first dose.

Per protocol set included all participants who were randomized and received all doses of the protocol-specified study drug.

Posted

Mean

Standard Deviation

ng/mL

Baseline to Final Assessment (Day 85 or 113)

ID	Title	Description
OG000	Placebo	Participants received placebo, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG001	ISIS 443139 10 mg	Participants received ISIS 443139, 10 milligrams (mg), by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG002	ISIS 443139 30 mg	Participants received ISIS 443139, 30 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG003	ISIS 443139 60 mg	Participants received ISIS 443139, 60 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.

Other Pre-specified

Change From Baseline in CSF Neurofilament Light Chain Concentration

Baseline was defined as the last non-missing measure prior to the first dose.

Per protocol set included all participants who were randomized and received all doses of the protocol-specified study drug.

Posted

Mean

Standard Deviation

nanograms per liter (ng/L)

Baseline to Final Assessment (Day 85 or 113)

ID	Title	Description
OG000	Placebo	Participants received placebo, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG001	ISIS 443139 10 mg	Participants received ISIS 443139, 10 milligrams (mg), by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG002	ISIS 443139 30 mg	Participants received ISIS 443139, 30 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG003	ISIS 443139 60 mg	Participants received ISIS 443139, 60 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.

Other Pre-specified

Ventricular Volume as Assessed by Structural Magnetic Resonance Imaging (MRI)

Per protocol set included all participants who were randomized and received all doses of the protocol-specified study drug.

Posted

Mean

Standard Deviation

Screening, Days 113, and 197

ID	Title	Description
OG000	Placebo	Participants received placebo, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG001	ISIS 443139 10 mg	Participants received ISIS 443139, 10 milligrams (mg), by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG002	ISIS 443139 30 mg	Participants received ISIS 443139, 30 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG003	ISIS 443139 60 mg	Participants received ISIS 443139, 60 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.

Other Pre-specified

Huntington's Disease (HD) Cognitive Assessment Battery Composite Score

The HD Cognitive Battery was developed as a means of measuring cognitive dysfunction in late premanifest and early manifest HD patients. The 6 tests that comprise the battery were selected based on test sensitivity, practice effects, reliability, domain coverage, feasibility for use in clinical trials, and tolerability. A composite cognitive score was calculated by the average z-score of the 6 individual tests. A positive change from baseline indicated improvement in cognitive function; a negative change indicated worsening. Baseline was defined as the last non-missing measure prior to the first dose.

Per protocol set included all participants who were randomized and received all doses of the protocol-specified study drug.

Posted

Mean

Standard Deviation

score on a scale

Baseline to Days 84, 141, and 197

ID	Title	Description
OG000	Placebo	Participants received placebo, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG001	ISIS 443139 10 mg	Participants received ISIS 443139, 10 milligrams (mg), by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.
OG002	ISIS 443139 30 mg

Arm/Groups

ID	Title	Description	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Placebo	Participants received placebo, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.	12	1	12	12	12
EG001	ISIS 443139 10 mg	Participants received ISIS 443139, 10 milligrams (mg), by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.	3	0	3	3	3
EG002	ISIS 443139 30 mg	Participants received ISIS 443139, 30 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.	6	0	6	6	6
EG003	ISIS 443139 60 mg	Participants received ISIS 443139, 60 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.	6	0	6	6	6
EG004	ISIS 443139 90 mg	Participants received ISIS 443139, 90 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.	9	0	9	9	9
EG005	ISIS 443139 120 mg	Participants received ISIS 443139, 120 mg, by intrathecal injection, at 4 week intervals over the course of a 13 week treatment period.	10	0	10	9	10

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Notes	Statistical Information
Post lumbar puncture syndrome	Injury, poisoning and procedural complications	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG0030 affected6 at risk EG004

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Notes	Statistical Information
Procedural pain	Injury, poisoning and procedural complications	MedDRA (18.0)	Systematic Assessment		EG0006 affected12 at risk EG0011 affected3 at risk EG0021 affected6 at risk EG0033 affected6 at risk EG004
Post lumbar puncture syndrome	Injury, poisoning and procedural complications	MedDRA (18.0)	Systematic Assessment		EG0004 affected12 at risk EG0011 affected3 at risk EG0022 affected6 at risk EG003
Fall	Injury, poisoning and procedural complications	MedDRA (18.0)	Systematic Assessment		EG0003 affected12 at risk EG0010 affected3 at risk EG0022 affected6 at risk EG003
Skin abrasion	Injury, poisoning and procedural complications	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Contusion	Injury, poisoning and procedural complications	MedDRA (18.0)	Systematic Assessment		EG0002 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Joint injury	Injury, poisoning and procedural complications	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Laceration	Injury, poisoning and procedural complications	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Head injury	Injury, poisoning and procedural complications	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Ligament sprain	Injury, poisoning and procedural complications	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Muscle injury	Injury, poisoning and procedural complications	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Muscle strain	Injury, poisoning and procedural complications	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Tooth injury	Injury, poisoning and procedural complications	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Upper limb fracture	Injury, poisoning and procedural complications	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Nasopharyngitis	Infections and infestations	MedDRA (18.0)	Systematic Assessment		EG0002 affected12 at risk EG0011 affected3 at risk EG0020 affected6 at risk EG003
Upper respiratory tract infection	Infections and infestations	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Rhinitis	Infections and infestations	MedDRA (18.0)	Systematic Assessment		EG0002 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Bronchitis	Infections and infestations	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Influenza	Infections and infestations	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0022 affected6 at risk EG003
Rhinovirus infection	Infections and infestations	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0012 affected3 at risk EG0021 affected6 at risk EG003
Sinusitis	Infections and infestations	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Urinary tract infection	Infections and infestations	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Bronchopneumonia	Infections and infestations	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Conjunctivitis	Infections and infestations	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Gastrointestinal infection	Infections and infestations	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Respiratory tract infection	Infections and infestations	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Viral infection	Infections and infestations	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Vulvovaginal candidiasis	Infections and infestations	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Headache	Nervous system disorders	MedDRA (18.0)	Systematic Assessment		EG0006 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Hypoaesthesia	Nervous system disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Dizziness	Nervous system disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Paraesthesia	Nervous system disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Tension headache	Nervous system disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Head discomfort	Nervous system disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Neuropathy peripheral	Nervous system disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Pleocytosis	Nervous system disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Syncope	Nervous system disorders	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA (18.0)	Systematic Assessment		EG0002 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Back pain	Musculoskeletal and connective tissue disorders	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0022 affected6 at risk EG003
Musculoskeletal pain	Musculoskeletal and connective tissue disorders	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Coccydynia	Musculoskeletal and connective tissue disorders	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Muscle spasms	Musculoskeletal and connective tissue disorders	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Osteopenia	Musculoskeletal and connective tissue disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Synovial cyst	Musculoskeletal and connective tissue disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Fatigue	General disorders	MedDRA (18.0)	Systematic Assessment	General disorders and administration site conditions	EG0000 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Asthenia	General disorders	MedDRA (18.0)	Systematic Assessment	General disorders and administration site conditions	EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Chills	General disorders	MedDRA (18.0)	Systematic Assessment	General disorders and administration site conditions	EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Discomfort	General disorders	MedDRA (18.0)	Systematic Assessment	General disorders and administration site conditions	EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Malaise	General disorders	MedDRA (18.0)	Systematic Assessment	General disorders and administration site conditions	EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Peripheral swelling	General disorders	MedDRA (18.0)	Systematic Assessment	General disorders and administration site conditions	EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Diarrhoea	Gastrointestinal disorders	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Toothache	Gastrointestinal disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0022 affected6 at risk EG003
Abdominal pain upper	Gastrointestinal disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Dental caries	Gastrointestinal disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Dyspepsia	Gastrointestinal disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Gastritis	Gastrointestinal disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Nausea	Gastrointestinal disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Vomiting	Gastrointestinal disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Alopecia	Skin and subcutaneous tissue disorders	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Dermatitis allergic	Skin and subcutaneous tissue disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Dry skin	Skin and subcutaneous tissue disorders	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Rash	Skin and subcutaneous tissue disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Rash erythematous	Skin and subcutaneous tissue disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Scab	Skin and subcutaneous tissue disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Skin reaction	Skin and subcutaneous tissue disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Blood creatine phosphokinase increased	Investigations	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Blood thyroid stimulating hormone increased	Investigations	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Body temperature increased	Investigations	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
C-reactive protein increased	Investigations	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Gamma-glutamyltransferase increased	Investigations	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Thyroxine increased	Investigations	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Urine analysis abnormal	Investigations	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Epistaxis	Respiratory, thoracic and mediastinal disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Throat irritation	Respiratory, thoracic and mediastinal disorders	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Anxiety	Psychiatric disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Insomnia	Psychiatric disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Mood altered	Psychiatric disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Haematoma	Vascular disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0022 affected6 at risk EG003
Bundle branch block right	Cardiac disorders	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Ventricular extrasystoles	Cardiac disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Ocular discomfort	Eye disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0021 affected6 at risk EG003
Vision blurred	Eye disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Lymphadenopathy	Blood and lymphatic system disorders	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Ear pain	Ear and labyrinth disorders	MedDRA (18.0)	Systematic Assessment		EG0001 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003
Erectile dysfunction	Reproductive system and breast disorders	MedDRA (18.0)	Systematic Assessment		EG0000 affected12 at risk EG0010 affected3 at risk EG0020 affected6 at risk EG003

There are no limitations or caveats for this study.

Certain Agreements

ID	Term
D006816	Huntington Disease

Ancestor Terms

ID	Term
D001480	Basal Ganglia Diseases
D001927	Brain Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases
D003704	Dementia
D002819	Chorea
D020820	Dyskinesias
D009069	Movement Disorders
D020271	Heredodegenerative Disorders, Nervous System
D019636	Neurodegenerative Diseases
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D003072	Cognition Disorders
D019965	Neurocognitive Disorders
D001523	Mental Disorders

ID	Term
C000632194	HTT(RX)

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