Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02489344
• Date of registration: 30/06/2015
• Prospective Registration: Yes
• Primary sponsor: Genzyme, a Sanofi Company
• Public title: Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease
• Scientific title: An Open-label, Multicenter, Multinational Extension Study of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Adult Male Patients Diagnosed With Fabry Disease
• Date of first enrolment: July 7, 2015
• Target sample size: 8
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02489344
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

France, Poland, Russian Federation, United Kingdom, United States

Contacts

• Name: Clinical Sciences & Operations
• Affiliation: Sanofi

Key inclusion & exclusion criteria

Inclusion criteria :

- Male participant with Fabry disease who previously completed study ACT13739.

- Participants, willing and able to provide signed informed consent.

- Sexually active participants, willing to practice true abstinence in line with their
preferred and usual lifestyle or using two acceptable effective methods of
contraception.

Exclusion criteria :

-Participants, in the opinion of the Investigator, unable to adhere to the requirements of
the study.

The above information was not intended to contain all considerations relevant to a
participant's potential participation in a clinical trial.



Age minimum: 18 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Drug: GZ/SAR402671

Primary Outcome(s)

Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters [Time Frame: From baseline of ACT13739 study up to 37 months post-ACT13739 baseline]

Number of Participants With Potentially Clinically Significant Electrocardiogram (ECG) Abnormalities [Time Frame: From baseline of ACT13739 study up to 37 months post-ACT13739 baseline]

Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes [Time Frame: From baseline of ACT13739 study up to 37 months post-ACT13739 baseline]

Number of Participants With Treatment Emergent Adverse Events (TEAEs) [Time Frame: From baseline of ACT13739 study up to 37 months post-ACT13739 baseline]

Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities [Time Frame: From baseline of ACT13739 study up to 37 months post-ACT13739 baseline]

Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Function Parameters [Time Frame: From baseline of ACT13739 study up to 37 months post-ACT13739 baseline]

Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Urinalysis [Time Frame: From baseline of ACT13739 study up to 37 months post-ACT13739 baseline]

Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematological Parameters [Time Frame: From baseline of ACT13739 study up to 37 months post-ACT13739 baseline]

Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters [Time Frame: From baseline of ACT13739 study up to 37 months post-ACT13739 baseline]

Secondary Outcome(s)

Gastrointestinal Symptoms: Abdominal Distension Severity Score at Baseline and Weeks 2, 4, 8, 12, 18, 26, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, and 156 post-ACT13739 baseline]

Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Weeks 26, 52, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline]

Change From Baseline in Albumin/Creatinine Ratio (ACR) and Protein/Creatinine Ratio (PCR) at Weeks 26, 52, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline]

Change From Baseline in High Sensitivity Cardiac Troponin T At Weeks 26, 52, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline]

Change From Baseline in Plasma Globotriaosylceramide (GL-3) Concentration at Weeks 26, 52, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline]

Change From Baseline in Podocyturia Counts (Per Milligram of Creatinine) At Weeks 12, 26, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 12, 26, and 156 post-ACT13739 baseline]

Change From Baseline in Urine GL-3 Concentration At Weeks 26, 52, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline]

Gastrointestinal Symptoms: Frequency of Bowel Movements - Least Number of Times Bowel Movement Per Day at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline]

Gastrointestinal Symptoms: Frequency of Bowel Movements - Most Number of Times Bowel Movement Per Day at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline]

Gastrointestinal (GI) Symptoms: Number of Participants With Abdominal Pain at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline]

Summary of Shifts From Baseline in Skin GL-3 Score in Superficial Capillary Endothelial Cells Over Time: Number of Participants in Categories of Shift in GL-3 Score [Time Frame: Baseline of ACT13739 study and Weeks 12, 26, 52, and 156 post-ACT13739 baseline]

Number of Participants in Categories of Brain Magnetic Resonance Imaging (MRI) Results at Baseline and Weeks 26, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 26, and 156 post-ACT13739 baseline]

Gastrointestinal Symptoms: Number of Participants With Abdominal Distension at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline]

Number of Participants in Categories of Echocardiogram (ECHO) Results at Baseline and at Weeks 26, 52, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline]

Change From Baseline in Beck Depression Inventory (BDI) Total Score at Weeks 26, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 26, 104, and 156 post-ACT13739 baseline]

Change From Baseline in Plasma Lyso Globotriaosylceramide (Lyso GL-3) Concentration at Weeks 26, 52, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline]

Gastrointestinal Symptoms: Number of Participants in Categories of Response Regarding Eating Less Due to Abdominal Pain/Bloating at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline]

Change From Baseline in Plasma Glucosylceramide (GL-1) Concentration At Weeks 26, 52, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline]

Gastrointestinal Symptoms: Abdominal Pain Severity Score at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline]

Gastrointestinal Symptoms: Number of Days With Abdominal Pain Score at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline]

Summary of Shifts From Baseline in Skin GL-3 Score in Deep Vessels Smooth Muscle Cells Over Time: Number of Participants in Categories of Shift in GL-3 Score [Time Frame: Baseline of ACT13739 study and Weeks 12, 26, 52, and 156 post-ACT13739 baseline]

Change From Baseline in Mental Component Summary and Physical Component Summary of the Short Form-36 (SF-36) Health Survey at Weeks 26, 52, 104 and 156 [Time Frame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline]

Change From Baseline in Plasma Monosialodihexosylganglioside (GM3) Concentration At Weeks 26, 52, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline]

Gastrointestinal Symptoms: Influence of GI Symptoms of Fabry Disease on Life at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline]

Gastrointestinal Symptoms: Number of Participants With Stool Consistency Assessment by Bristol Stool Scale Scoring at Baseline and Weeks 26, 52, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline]

Gastrointestinal Symptoms: Satisfaction Over Bowel Habits at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 [Time Frame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline]

Summary of Shifts From Baseline in Skin GL-3 Score in Deep Vessels Endothelial Cells Over Time: Number of Participants in Categories of Shift in GL-3 Score [Time Frame: Baseline of ACT13739 study and Weeks 12, 26, 52, and 156 post-ACT13739 baseline]

Summary of Shifts From Baseline in Skin GL-3 Score in Perineurium Cells Over Time: Number of Participants in Categories of Shift in GL-3 Score [Time Frame: Baseline of ACT13739 study and Weeks 12, 26, 52, and 156 post-ACT13739 baseline]

Secondary ID(s)

U1111-1165-9049

LTS14116

2014-004995-49

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 09/12/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02489344