Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 1706-1617 | Other Identifier | NIH Protocol Registration Number | |
| 2016-003023-30 | EudraCT Number |
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The primary objective of the study is to determine the safety of single doses of DTX401, including the incidence of dose-limiting toxicities (DLTs) at each dose level.
Participants enrolled in the 401GSDIA01 study will be monitored for 52 weeks following DTX401 administration. Participants in Cohorts 1, 2, and 3 will receive reactive oral steroid treatment for possible vector-induced hepatitis following treatment with DTX401. Participants in Cohort 4 will receive prophylactic oral steroid treatment to prevent possible vector-induced hepatitis. After completion of the Week 52 visit or early withdrawal, participants will be offered enrollment into a 4-year extension study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| DTX401 Cohort 1 | Experimental | Dose 1 (2.0 × 10^12 GC/kg) with a reactive steroid regimen (6 weeks, at a starting dose of 40 mg/day, after alanine aminotransferase [ALT] elevation) |
|
| DTX401 Cohort 2 | Experimental | Dose 2 (6.0 × 10^12 GC/kg) with a reactive steroid regimen (6 weeks, at a starting dose of 40 mg/day, after ALT elevation) |
|
| DTX401 Cohort 3 | Experimental | Dose 2 (6.0 × 10^12 GC/kg) with an optimized reactive steroid regimen (7 weeks, at a starting dose of 60 mg/day, after ALT elevation) |
|
| DTX401 Cohort 4 | Experimental | Dose 2 (6.0 × 10^12 GC/kg) with a prophylactic steroid regimen (8 weeks, at a starting dose of 60 mg/day, starting on Day 1) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| DTX401 | Genetic | DTX401 administered as a single peripheral intravenous (IV) infusion |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events (AEs) Treatment-Emergent AEs (TEAEs) Serious TEAEs, Discontinuations Due to TEAEs, and Dose-Limiting Toxicities (DLTs) | An AE is defined as any untoward medical occurrence, regardless of its causal relationship to study product. An SAE is defined as any event that: results in death; is immediately life threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; or an important medical event, in the opinion of the investigator. The relationship to study drug was categorized as unrelated, possible, probable or definite. A DLT is defined as any AE/SAE ≥ Grade 3 that is considered by the Investigator and/or Sponsor to be related to DTX401, based on the Nation Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version 5.0 or later version. Per protocol, SAEs that occurred > 30 days after EOS or Early withdrawal visit, did not need to be reported unless Investigator considered them related to study product. | AEs Prior to Dosing: From signing the informed consent form (ICF) to first dose of study drug. TEAEs: From first dose of study drug through the End of Study (EOS)/Early Withdrawal visit (up to Week 52) plus 30 days. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Time to First Hypoglycemic Event Over Time | The change from baseline in time (in hours) to first hypoglycemic event (defined as glucose < 54 mg/dL [< 3.0 mmol/L]) during a controlled fasting challenge at 12, 24, and 52 weeks after IV administration of DTX401. A positive change from baseline is favorable. | Baseline, Weeks 12, 24, 52 |
Not provided
Key Inclusion Criteria:
Key Exclusion Criteria:
Note additional inclusion/exclusion criteria may apply, per protocol.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Ultragenyx Pharmaceutical Inc | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCONN Health | Farmington | Connecticut | 06030-3213 | United States | ||
| Michigan Medicine University of Michigan |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40064185 | Derived | Weinstein DA, Derks TG, Rodriguez-Buritica DF, Ahmad A, Couce ML, Mitchell JJ, Riba-Wolman R, Mount M, Sallago JB, Ross KM, van der Klauw MM, de Boer F, van der Schaaf C, Saavedra H, Martinez-Olmos M, Atanga E, Hosseini A, Mitragotri D, Crombez E. Safety and Efficacy of DTX401, an AAV8-Mediated Liver-Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a (GSDIa). J Inherit Metab Dis. 2025 Mar;48(2):e70014. doi: 10.1002/jimd.70014. | |
| 32430177 |
Not provided
Not provided
Eligible participants were enrolled sequentially into 4 cohorts of 3 participants each and received a single intravenous (IV) infusion of DTX401, with steroids (prednisone/prednisolone) to manage alanine aminotransferase (ALT) elevation.
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| ID | Title | Description |
|---|---|---|
| FG000 | DTX401 Cohort 1 | DTX401 Dose 1 (2.0 × 10^12 GC/kg) with a reactive steroid regimen (6 weeks, at a starting dose of 40 mg/day, after ALT elevation) |
| FG001 | DTX401 Cohort 2 | DTX401 Dose 2 (6.0 × 10^12 GC/kg) with a reactive steroid regimen (6 weeks, at a starting dose of 40 mg/day, after ALT elevation) |
| FG002 | DTX401 Cohort 3 | DTX401 Dose 2 (6.0 × 10^12 GC/kg) with an optimized reactive steroid regimen (7 weeks, at a starting dose of 60 mg/day, after ALT elevation) |
| FG003 | DTX401 Cohort 4 | DTX401 Dose 2 (6.0 × 10^12 GC/kg) with a prophylactic steroid regimen (8 weeks, at a starting dose of 60 mg/day, starting on Day 1) |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | DTX401 Cohort 1 | DTX401 Dose 1 (2.0 × 10^12 GC/kg) with a reactive steroid regimen (6 weeks, at a starting dose of 40 mg/day, after ALT elevation) |
| BG001 | DTX401 Cohort 2 | DTX401 Dose 2 (6.0 × 10^12 GC/kg) with a reactive steroid regimen (6 weeks, at a starting dose of 40 mg/day, after ALT elevation) |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 Adverse Events (AEs) Treatment-Emergent AEs (TEAEs) Serious TEAEs, Discontinuations Due to TEAEs, and Dose-Limiting Toxicities (DLTs) | An AE is defined as any untoward medical occurrence, regardless of its causal relationship to study product. An SAE is defined as any event that: results in death; is immediately life threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; or an important medical event, in the opinion of the investigator. The relationship to study drug was categorized as unrelated, possible, probable or definite. A DLT is defined as any AE/SAE ≥ Grade 3 that is considered by the Investigator and/or Sponsor to be related to DTX401, based on the Nation Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version 5.0 or later version. Per protocol, SAEs that occurred > 30 days after EOS or Early withdrawal visit, did not need to be reported unless Investigator considered them related to study product. | Posted | Count of Participants | Participants | No | AEs Prior to Dosing: From signing the informed consent form (ICF) to first dose of study drug. TEAEs: From first dose of study drug through the End of Study (EOS)/Early Withdrawal visit (up to Week 52) plus 30 days. |
From enrollment (all-cause mortality) or first dose of study drug (treatment emergent adverse events) through the End of Study/Early Withdrawal visit (up to Week 52) plus 30 days.
Per protocol, SAEs that occurred > 30 days after EOS or Early withdrawal visit, did not need to be reported unless Investigator considered them related to study product.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | DTX401 Cohort 1 | DTX401 Dose 1 (2.0 × 10^12 GC/kg) with a reactive steroid regimen (6 weeks, at a starting dose of 40 mg/day, after ALT elevation) |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cellulitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Iron Deficiency Anaemia | Blood and lymphatic system disorders | MedDRA 23.0 | Systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Information | Ultragenyx Pharmaceutical Inc | 1-888-756-8657 | medinfo@ultragenyx.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Feb 16, 2021 | Oct 4, 2022 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 4, 2022 | Oct 4, 2022 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D005953 | Glycogen Storage Disease Type I |
| D044882 | Glucose Metabolism Disorders |
| ID | Term |
|---|---|
| D006008 | Glycogen Storage Disease |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
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|
| steroid regimen | Drug | prednisone or prednisolone to manage alanine aminotransferase (ALT) elevation |
|
| Ann Arbor |
| Michigan |
| 48109 |
| United States |
| UT Health - McGovern Medical School | Houston | Texas | 77030 | United States |
| Montreal Children Hospital, McGill University Health Centre | Montreal | Quebec | H4A3J1 | Canada |
| University Medical Center Groningen | Groningen | 9700RB | Netherlands |
| Complejo Hospitalario Universitario de Santiago | Santiago de Compostela | A Coruna | 15706 | Spain |
| Derived |
| Zhang L, Lee C, Arnaoutova I, Anduaga J, Starost MF, Mansfield BC, Chou JY. Gene therapy using a novel G6PC-S298C variant enhances the long-term efficacy for treating glycogen storage disease type Ia. Biochem Biophys Res Commun. 2020 Jun 30;527(3):824-830. doi: 10.1016/j.bbrc.2020.04.124. Epub 2020 May 16. |
| 30714174 | Derived | Zhang L, Cho JH, Arnaoutova I, Mansfield BC, Chou JY. An evolutionary approach to optimizing glucose-6-phosphatase-alpha enzymatic activity for gene therapy of glycogen storage disease type Ia. J Inherit Metab Dis. 2019 May;42(3):470-479. doi: 10.1002/jimd.12069. Epub 2019 Feb 22. |
| BG002 | DTX401 Cohort 3 | DTX401 Dose 2 (6.0 × 10^12 GC/kg) with an optimized reactive steroid regimen (7 weeks, at a starting dose of 60 mg/day, after ALT elevation) |
| BG003 | DTX401 Cohort 4 | DTX401 Dose 2 (6.0 × 10^12 GC/kg) with a prophylactic steroid regimen (8 weeks, at a starting dose of 60 mg/day, starting on Day 1) |
| BG004 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Controlled Fasting Challenge: Time to First Hypoglycemic Event | Time (in hours) to first hypoglycemic event (defined as glucose < 54 mg/dL [< 3.0 mmol/L]) during a controlled fasting challenge. | Mean | Standard Deviation | hours |
|
| ID | Title | Description |
|---|---|---|
| OG000 | DTX401 Cohort 1 | DTX401 Dose 1 (2.0 × 10^12 GC/kg) with a reactive steroid regimen (6 weeks, at a starting dose of 40 mg/day, after ALT elevation) |
| OG001 | DTX401 Cohort 2 | DTX401 Dose 2 (6.0 × 10^12 GC/kg) with a reactive steroid regimen (6 weeks, at a starting dose of 40 mg/day, after ALT elevation) |
| OG002 | DTX401 Cohort 3 | DTX401 Dose 2 (6.0 × 10^12 GC/kg) with an optimized reactive steroid regimen (7 weeks, at a starting dose of 60 mg/day, after ALT elevation) |
| OG003 | DTX401 Cohort 4 | DTX401 Dose 2 (6.0 × 10^12 GC/kg) with a prophylactic steroid regimen (8 weeks, at a starting dose of 60 mg/day, starting on Day 1) |
|
|
| Secondary | Change From Baseline in Time to First Hypoglycemic Event Over Time | The change from baseline in time (in hours) to first hypoglycemic event (defined as glucose < 54 mg/dL [< 3.0 mmol/L]) during a controlled fasting challenge at 12, 24, and 52 weeks after IV administration of DTX401. A positive change from baseline is favorable. | Participants with an assessment at given time point | Posted | Mean | Standard Deviation | hours | Baseline, Weeks 12, 24, 52 |
|
|
|
| 0 |
| 3 |
| 2 |
| 3 |
| 3 |
| 3 |
| EG001 | DTX401 Cohort 2 | DTX401 Dose 2 (6.0 × 10^12 GC/kg) with a reactive steroid regimen (6 weeks, at a starting dose of 40 mg/day, after ALT elevation) | 0 | 3 | 1 | 3 | 3 | 3 |
| EG002 | DTX401 Cohort 3 | DTX401 Dose 2 (6.0 × 10^12 GC/kg) with an optimized reactive steroid regimen (7 weeks, at a starting dose of 60 mg/day, after ALT elevation) | 0 | 3 | 1 | 3 | 3 | 3 |
| EG003 | DTX401 Cohort 4 | DTX401 Dose 2 (6.0 × 10^12 GC/kg) with a prophylactic steroid regimen (8 weeks, at a starting dose of 60 mg/day, starting on Day 1) | 0 | 3 | 0 | 3 | 3 | 3 |
| EG004 | Total | DTX401 Dose 1 (2.0 × 10^12 GC/kg) or DTX401 Dose 2 (6.0 × 10^12 GC/kg) with a reactive steroid regimen (6 weeks, at a starting dose of 40 mg/day, after ALT elevation), an optimized reactive steroid regimen (7 weeks, at a starting dose of 60 mg/day, after ALT elevation), or a prophylactic steroid regimen (8 weeks, at a starting dose of 60 mg/day, starting on Day 1) | 0 | 12 | 4 | 12 | 12 | 12 |
| Lactic Acidosis | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
|
| Metabolic Disorder | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
|
| Migraine | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
|
| Nephrolithiasis | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
|
| Polycythaemia | Blood and lymphatic system disorders | MedDRA 23.0 | Systematic Assessment |
|
| Tachycardia | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
|
| Adrenal Insufficiency | Endocrine disorders | MedDRA 23.0 | Systematic Assessment |
|
| Adrenal Suppression | Endocrine disorders | MedDRA 23.0 | Systematic Assessment |
|
| Glucocorticoid Deficiency | Endocrine disorders | MedDRA 23.0 | Systematic Assessment |
|
| Abdominal Distension | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Abdominal Pain | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Dyspepsia | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Gastrointestinal Disorder | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Malabsorption | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Mouth Ulceration | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Toothache | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Asthenia | General disorders | MedDRA 23.0 | Systematic Assessment |
|
| Chills | General disorders | MedDRA 23.0 | Systematic Assessment |
|
| Face Oedema | General disorders | MedDRA 23.0 | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA 23.0 | Systematic Assessment |
|
| Hunger | General disorders | MedDRA 23.0 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 23.0 | Systematic Assessment |
|
| Thirst | General disorders | MedDRA 23.0 | Systematic Assessment |
|
| Hepatic Pain | Hepatobiliary disorders | MedDRA 23.0 | Systematic Assessment |
|
| Hypertransaminasaemia | Hepatobiliary disorders | MedDRA 23.0 | Systematic Assessment |
|
| Food Allergy | Immune system disorders | MedDRA 23.0 | Systematic Assessment |
|
| Hypersensitivity | Immune system disorders | MedDRA 23.0 | Systematic Assessment |
|
| Abdominal Wall Infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Covid-19 | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Folliculitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Gastrointestinal Infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Gastrointestinal Viral Infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Hordeolum | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Oral Candidiasis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Sinobronchitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Tinea Pedis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Urinary Tract Infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Viral Infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Arthropod Bite | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
|
| Foot Fracture | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
|
| Muscle Strain | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
|
| Stoma Site Discomfort | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
|
| Alanine Aminotransferase Increased | Investigations | MedDRA 23.0 | Systematic Assessment |
|
| Aspartate Aminotransferase Increased | Investigations | MedDRA 23.0 | Systematic Assessment |
|
| Blood Creatine Phosphokinase Increased | Investigations | MedDRA 23.0 | Systematic Assessment |
|
| Blood Creatinine Increased | Investigations | MedDRA 23.0 | Systematic Assessment |
|
| Blood Glucose Fluctuation | Investigations | MedDRA 23.0 | Systematic Assessment |
|
| Blood Uric Acid Increased | Investigations | MedDRA 23.0 | Systematic Assessment |
|
| Heart Rate Increased | Investigations | MedDRA 23.0 | Systematic Assessment |
|
| Hepatic Enzyme Increased | Investigations | MedDRA 23.0 | Systematic Assessment |
|
| Liver Function Test Increased | Investigations | MedDRA 23.0 | Systematic Assessment |
|
| Weight Decreased | Investigations | MedDRA 23.0 | Systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
|
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
|
| Hyperlipidaemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
|
| Hypertriglyceridaemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
|
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
|
| Lactic Acidosis | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
|
| Vitamin D Deficiency | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
| Back Pain | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
| Gouty Arthritis | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
| Joint Noise | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
| Muscle Spasms | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
| Pain In Extremity | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
| Plantar Fasciitis | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
|
| Hypoaesthesia | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
|
| Migraine | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
|
| Tremor | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
|
| Agitation | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
|
| Anxiety | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
|
| Libido Increased | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
|
| Nephrolithiasis | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
|
| Proteinuria | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
|
| Amenorrhoea | Reproductive system and breast disorders | MedDRA 23.0 | Systematic Assessment |
|
| Prostatitis | Reproductive system and breast disorders | MedDRA 23.0 | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Oropharyngeal Pain | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Upper Respiratory Tract Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Acne | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
| Dermatitis Acneiform | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
| Hair Growth Abnormal | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
| Skin Striae | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
| Skin Ulcer | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
| Hypertension | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
|
The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
|
| Change at Week 24 |
|
|
| Change at Week 52 |
|
|