Phase 3
Completed N=179
Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Source: ClinicalTrials.gov NCT02851797 ↗
Enrolled (actual)
179
Serious AEs
5.6%
Results posted
Feb 2023
Primary outcomePrimary: Mean Change From Baseline in 4 Standard Stairs (4SC) Climb After 18 Months of Treatment — 1.27; 1.48 seconds — p==0.0345
◆ Published Evidence
Highly cited
115citations · ~58 / year
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Summary
Primary Objective
The primary objective of the study was to establish the effects of givinostat versus placebo administered chronically over 18 months to slow disease progression in ambulant DMD subjects.
Secondary Objectives
The secondary objectives of this study were:
* To assess the safety and tolerability of givinostat versus placebo administered chronically in DMD subjects
* To evaluate the PK profile of givinostat administered chronically in DMD subjects
* To evaluate the impact on quality of life (QoL) and activities of daily living of givinostat versus placebo administered chronically.
Linked Publications
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Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Mean Change From Baseline in 4 Standard Stairs (4SC) Climb After 18 Months of Treatment |
1.27; 1.48 | =0.0345 sig |
| SECONDARY Mean Change From Baseline in Time to Rise From Floor After 18 Months of Treatment |
9.33; 12.61 | =0.3044 |
| SECONDARY Mean Change From Baseline in the Six-minute Walking Test (6MWT) After 18 Months of Treatment |
-38.43; -48.38 | =0.3723 |
| SECONDARY Mean Change From Baseline in Total North Star Ambulatory Assessment (NSAA) Score After 18 Months of Treatment |
-2.66; -4.58 | =0.0209 sig |
| SECONDARY Cumulative Loss of Function on the NSAA |
3.42; 5.56 | =0.0202 sig |
| SECONDARY Mean Change From Baseline of Muscle Strength Normalized Overtime |
-0.32; -0.50; -0.10; -0.19 | =0.0902 |
| SECONDARY Mean Change From Baseline in Vastus Lateralis Muscle Fat Fraction (VL MFF) at 18 Months |
7.63; 10.56 | =0.0354 sig |
| SECONDARY Number of Subjects Experiencing Treatment-emergent AEs (TEAEs), Serious AEs (SAEs), Mild TEAE Moderate TEAE, Severe TEAE |
112; 57; 8; 2; 69; 39 | — |
| SECONDARY Evaluation of Acceptability/Palatability of the Oral Suspension |
31; 11; 21; 17; 30; 16 | — |
Eligibility Criteria
Inclusion Criteria
- Are an ambulant male aged ≥6 years at randomisation with DMD characteristic clinical symptoms or signs (e.g., proximal muscle weakness, Gowers' maneuver, elevated serum creatinine kinase level) already present at screening;
- Have DMD diagnosis confirmed by genetic testing;
- Are able to give informed assent and/or consent in writing signed by the subject and/or parent/legal guardian (according to local regulations);
- Are able to complete 2 Four Stairs Climb test (4SC) screening assessments; the results of these tests must be within ±1 second of each other;
- Have the mean of 2 screening 4SC assessments ≤8 seconds;
- Have time to rise from floor between ≥3 and 2 x the upper limit of normal (ULN);
- Have Triglycerides > 300 mg/dL (3.42 mmol/L) in fasting condition at screening visit;
- Have a positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at screening15.
- Have a baseline QTcF >450 msec, or history of additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, or family history of long QT syndrome);
- Have a psychiatric illness/social situations rendering the potential subject unable to understand and comply with the muscle function tests and/or with the study protocol procedures;
- Have any hypersensitivity to the components of study medication;
- Have a sorbitol intolerance or sorbitol malabsorption, or have the hereditary form of fructose intolerance.
- Have contraindications to MRI or MRS (e.g., claustrophobia, metal implants, or seizure disorder).
At the discretion of the Investigator, subjects not meeting inclusion/exclusion criteria may be re-screened twice with an interval of at least 3 months between assessments.
Data sourced from ClinicalTrials.gov (NCT02851797) and the linked publication. 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. Informational only — not medical advice.