Clinical trial NCT02489344: Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease
Official 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Fabry Disease
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2015-07-03
Enrollment: 8
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Primary Objective:

To assess the long-term safety of GZ/SAR402671 in adult male participants with Fabry disease who previously completed study ACT13739 (NCT02489344).

Secondary Objective:

To assess the long-term effect of GZ/SAR402671 on pharmacodynamic and exploratory efficacy endpoints in adult male participants with Fabry disease who previously completed study ACT13739.

Detailed description: The total duration of this extension study (LTS14116) was up to 31 months (30 months of treatment and one month post-treatment follow-up).

Eligibility 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.

Primary outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs)
timeFrame: From baseline of ACT13739 study up to 37 months post-ACT13739 baseline
measure: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematological Parameters
timeFrame: From baseline of ACT13739 study up to 37 months post-ACT13739 baseline
measure: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes
timeFrame: From baseline of ACT13739 study up to 37 months post-ACT13739 baseline
measure: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters
timeFrame: From baseline of ACT13739 study up to 37 months post-ACT13739 baseline
measure: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters
timeFrame: From baseline of ACT13739 study up to 37 months post-ACT13739 baseline
measure: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Function Parameters
timeFrame: From baseline of ACT13739 study up to 37 months post-ACT13739 baseline
measure: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Urinalysis
timeFrame: From baseline of ACT13739 study up to 37 months post-ACT13739 baseline
measure: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities
timeFrame: From baseline of ACT13739 study up to 37 months post-ACT13739 baseline
measure: Number of Participants With Potentially Clinically Significant Electrocardiogram (ECG) Abnormalities
timeFrame: From baseline of ACT13739 study up to 37 months post-ACT13739 baseline
Secondary outcomes:
measure: Change From Baseline in Plasma Globotriaosylceramide (GL-3) Concentration at Weeks 26, 52, 104, and 156
timeFrame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
measure: Change From Baseline in Plasma Lyso Globotriaosylceramide (Lyso GL-3) Concentration at Weeks 26, 52, 104, and 156
timeFrame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
measure: Change From Baseline in Plasma Glucosylceramide (GL-1) Concentration At Weeks 26, 52, 104, and 156
timeFrame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
measure: Change From Baseline in Plasma Monosialodihexosylganglioside (GM3) Concentration At Weeks 26, 52, 104, and 156
timeFrame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
measure: Change From Baseline in Urine GL-3 Concentration At Weeks 26, 52, 104, and 156
timeFrame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
measure: Change From Baseline in High Sensitivity Cardiac Troponin T At Weeks 26, 52, 104, and 156
timeFrame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
measure: Change From Baseline in Podocyturia Counts (Per Milligram of Creatinine) At Weeks 12, 26, and 156
timeFrame: Baseline of ACT13739 study and Weeks 12, 26, and 156 post-ACT13739 baseline
measure: 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
timeFrame: Baseline of ACT13739 study and Weeks 12, 26, 52, and 156 post-ACT13739 baseline
measure: 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
timeFrame: Baseline of ACT13739 study and Weeks 12, 26, 52, and 156 post-ACT13739 baseline
measure: 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
timeFrame: Baseline of ACT13739 study and Weeks 12, 26, 52, and 156 post-ACT13739 baseline
measure: 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
timeFrame: Baseline of ACT13739 study and Weeks 12, 26, 52, and 156 post-ACT13739 baseline
measure: 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
timeFrame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
measure: Gastrointestinal (GI) Symptoms: Number of Participants With Abdominal Pain at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156
timeFrame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline
measure: Gastrointestinal Symptoms: Abdominal Pain Severity Score at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156
timeFrame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline
measure: Gastrointestinal Symptoms: Number of Days With Abdominal Pain Score at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156
timeFrame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline
measure: Gastrointestinal Symptoms: Number of Participants With Abdominal Distension at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156
timeFrame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline
measure: Gastrointestinal Symptoms: Abdominal Distension Severity Score at Baseline and Weeks 2, 4, 8, 12, 18, 26, and 156
timeFrame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, and 156 post-ACT13739 baseline
measure: 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
timeFrame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline
measure: Gastrointestinal Symptoms: Satisfaction Over Bowel Habits at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156
timeFrame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline
measure: 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
timeFrame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline
measure: 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
timeFrame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline
measure: 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
timeFrame: Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline
measure: Gastrointestinal Symptoms: Number of Participants With Stool Consistency Assessment by Bristol Stool Scale Scoring at Baseline and Weeks 26, 52, 104, and 156
timeFrame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
measure: Change From Baseline in Beck Depression Inventory (BDI) Total Score at Weeks 26, 104, and 156
timeFrame: Baseline of ACT13739 study and Weeks 26, 104, and 156 post-ACT13739 baseline
measure: Change From Baseline in Albumin/Creatinine Ratio (ACR) and Protein/Creatinine Ratio (PCR) at Weeks 26, 52, 104, and 156
timeFrame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
measure: Number of Participants in Categories of Echocardiogram (ECHO) Results at Baseline and at Weeks 26, 52, 104, and 156
timeFrame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
measure: Number of Participants in Categories of Brain Magnetic Resonance Imaging (MRI) Results at Baseline and Weeks 26, and 156
timeFrame: Baseline of ACT13739 study and Weeks 26, and 156 post-ACT13739 baseline
measure: Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Weeks 26, 52, 104, and 156
timeFrame: Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
measure: Chitotriosidase (Chit1) Plasma Concentration Levels at Weeks 52, 104,156 and 160 (End of Treatment Follow-up)
timeFrame: Weeks 52, 104, 156 and 160 (End of Treatment Follow-up) post-ACT13739 baseline
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Investigational Site Number 840002
state: Georgia
city: Cincinnati
country: United States
facility: Investigational Site Number 840003
state: Ohio
city: Fairfax
country: United States
facility: Investigational Site Number 840001
state: Virginia
city: Garches
country: France
facility: Investigational Site Number 250001
city: Warszawa
country: Poland
facility: Investigational Site Number 616001
city: Moscow
country: Russian Federation
facility: Investigational Site Number 643002
city: Cambridge
country: United Kingdom
facility: Investigational Site Number 826002
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: