Phase 2 N=11 Treatment

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

Fabry Disease

Bottom Line

View on ClinicalTrials.gov: NCT02489344 ↗

Enrolled (actual)

Serious AEs

27.3%

Results posted

Dec 2019

Primary outcome: Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) — 9 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: GZ/SAR402671 (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: Male
Sponsor: Genzyme, a Sanofi Company
Primary completion: Nov 2018

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Treatment Emergent Adverse Events (TEAEs)	9	—
PRIMARY Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematological Parameters	1; 0; 0; 6; 0; 0	—
PRIMARY Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes	0; 0; 0; 0; 0; 0	—
PRIMARY Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters	0; 0; 0; 0; 0; 0	—
PRIMARY Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters	3; 1; 0; 0; 2	—
PRIMARY Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Function Parameters	0; 2; 0; 0; 0; 5	—
PRIMARY Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Urinalysis	0; 0	—
PRIMARY Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities	0; 0; 0; 0; 1; 0	—
PRIMARY Number of Participants With Potentially Clinically Significant Electrocardiogram (ECG) Abnormalities	0; 0; 0; 0; 1; 0	—
SECONDARY Change From Baseline in Plasma Globotriaosylceramide (GL-3) Concentration at Weeks 26, 52, 104, and 156	-3.62; -5.06; -6.32; -6.97	—
SECONDARY Change From Baseline in Plasma Lyso Globotriaosylceramide (Lyso GL-3) Concentration at Weeks 26, 52, 104, and 156	-30.99; -37.10; -39.84; -48.13	—
SECONDARY Change From Baseline in Plasma Glucosylceramide (GL-1) Concentration At Weeks 26, 52, 104, and 156	-3.26; -3.58; -3.70; -3.23	—
SECONDARY Change From Baseline in Plasma Monosialodihexosylganglioside (GM3) Concentration At Weeks 26, 52, 104, and 156	-10.77; -8.84; -9.92; -8.12	—
SECONDARY Change From Baseline in Urine GL-3 Concentration At Weeks 26, 52, 104, and 156	-0.25; -0.20; -0.18; -0.18	—
SECONDARY Change From Baseline in High Sensitivity Cardiac Troponin T At Weeks 26, 52, 104, and 156	0.0000; 0.0105; -0.0015; 0.0006	—
SECONDARY Change From Baseline in Podocyturia Counts (Per Milligram of Creatinine) At Weeks 12, 26, and 156	-1.40; -1.65; -2.73	—
SECONDARY 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	4; 1; 3; 1; 4; 1	—
SECONDARY 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	1; 2; 6; 1; 2; 6	—
SECONDARY 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	2; 7; 2; 7; 1; 4	—
SECONDARY 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	1; 8; 1; 8; 1; 1	—
SECONDARY 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	8.39; 7.70; 9.72; 5.30; -3.31; 1.69	—
SECONDARY Gastrointestinal (GI) Symptoms: Number of Participants With Abdominal Pain at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156	5; 6; 6; 5; 7; 4	—
SECONDARY Gastrointestinal Symptoms: Abdominal Pain Severity Score at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156	52.50; 35.60; 29.75; 29.00; 40.00; 43.00	—
SECONDARY Gastrointestinal Symptoms: Number of Days With Abdominal Pain Score at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156	38.33; 26.00; 25.00; 25.00; 35.00; 35.00	—
SECONDARY Gastrointestinal Symptoms: Number of Participants With Abdominal Distension at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156	9; 2; 8; 3; 7; 4	—
SECONDARY Gastrointestinal Symptoms: Abdominal Distension Severity Score at Baseline and Weeks 2, 4, 8, 12, 18, 26, and 156	45.33; 38.67; 21.00; 35.00; 72.00; 69.00	—
SECONDARY 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	4; 5; 2; 6; 3; 2	—
SECONDARY Gastrointestinal Symptoms: Satisfaction Over Bowel Habits at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156	26.55; 30.82; 36.09; 34.27; 21.50; 23.89	—
SECONDARY 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	2.36; 2.00; 2.00; 1.74; 2.04; 2.17	—
SECONDARY 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	0.78; 0.66; 0.65; 0.62; 0.67; 0.73	—
SECONDARY 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	34.36; 26.73; 21.27; 22.09; 17.50; 24.89	—
SECONDARY Gastrointestinal Symptoms: Number of Participants With Stool Consistency Assessment by Bristol Stool Scale Scoring at Baseline and Weeks 26, 52, 104, and 156	11; 0; 0; 8; 1; 0	—
SECONDARY Change From Baseline in Beck Depression Inventory (BDI) Total Score at Weeks 26, 104, and 156	-0.89; 2.00; -3.43	—
SECONDARY Change From Baseline in Albumin/Creatinine Ratio (ACR) and Protein/Creatinine Ratio (PCR) at Weeks 26, 52, 104, and 156	-26.22; 1.00; 12.21; -1.14; -38.33; 0.33	—
SECONDARY Number of Participants in Categories of Echocardiogram (ECHO) Results at Baseline and at Weeks 26, 52, 104, and 156	6; 2; 0; 6; 3; 0	—
SECONDARY Number of Participants in Categories of Brain Magnetic Resonance Imaging (MRI) Results at Baseline and Weeks 26, and 156	6; 4; 7; 2; 4; 3	—
SECONDARY Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Weeks 26, 52, 104, and 156	-3.43; -5.57; -5.71; -4.83	—
SECONDARY Chitotriosidase (Chit1) Plasma Concentration Levels at Weeks 52, 104,156 and 160 (End of Treatment Follow-up)	35.965; 12.785; 0.000; 6.483	—

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.

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.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02489344). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.