Duvyzat® clinical development
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Duvyzat® clinical development programme* in Duchenne muscular dystrophy (DMD)1-5
Duvyzat® clinical development programme
*In patients over 6 years and above
OLE: Open-label Extension
EPIDYS study design and endpoints are shown below. Additional study design descriptions can be found at the bottom of this page
EPIDYS study design
EPIDYS evaluated the efficacy and safety of Duvyzat® in DMD boys ages 6 and above who were ambulant 2,5
- Randomised, double-blind, parallel-group, placebo-controlled study with a total of 179 ambulant boys randomised 2:1 (Duvyzat® : control)
- All participants were on a stable dose of corticosteroid (CS)
Real patient currently on Duvyzat® treatment. Photograph for illustrative purposes only, individual results may vary
EPIDYS and Open-label Extension (OLE) study design
All patients from EPIDYS and the phase II Study 43 were moved to the on-going open-label extension study.
Those patients in the control arm of EPIDYS were moved to Duvyzat® treatment.
Additionally, 30 Duvyzat® naïve patients were also enrolled in the OLE cohort.
Baseline demographics and disease characteristics of intention-to-treat population5
Data are median (IQR), n (%), or mean (SD); range. The group A population was used for the prespecified efficacy analyses; supportive efficacy analyses were conducted post-hoc in the overall population, which was also used for the safety evaluations.
*All patients were also receiving systemic corticosteroids, in a dose and regimen that was to remain unchanged over the follow-up period. †Race was self-reported.
EPIDYS study endpoints
Primary endpoint 2,5
Real patient currently on Duvyzat® treatment. Photograph for illustrative purposes only, individual results may vary
Secondary endpoints 2,5
- NSAA total score
- NSAA cumulative loss of function
- TTR
- 6MWT
- Knee extension
- Elbow flexion
- MRS VLFF
NSAA: North Star Ambulatory Assessment TTR: Time-to-rise 6MWT: 6-minute Walk Test MRS VLFF: Magnetic Resonance Spectroscopy Vastus Lateralis Fat Fraction
Additional study design
Duvyzat® long term safety, tolerability and efficacy are evaluated in an ongoing prospective open label, long-term extension (OLE) study named STUDY 51. Patients who completed the Duvyzat® phase 2 trial (STUDY 43) and patients who completed the Duvyzat® phase 3 trials (EPIDYS) were enrolled in STUDY 51. Additionally, 30 Duvyzat® naïve patients were also enrolled in the OLE cohort. All patients were on a stable dose of corticosteroids before enrolling and continue corticosteroid treatment throughout the study.
ULYSSES study is an ongoing randomised, double-blind, placebo-controlled, multicentre, 18 months study to evaluate the efficacy, safety and tolerability of Duvyzat® in non-ambulant patients with DMD. The primary objective of the study is to demonstrate the efficacy of Duvyzat® in reducing muscle decline in non-ambulant DMD patients, as measured by the Performance of the Upper Limb (PUL) scale, an externally assessed clinical scale, providing an upper extremity functional score, designed for the DMD patients. More information is provided on www.clinicaltrials.gov (NCT05933057), on the DMD hub (https://dmdhub.org) or on the study website (www.ulyssesDMD.study).
- Phase 3 in non-ambulant boys aged ≥ 9 to < 18 years with DMD; duration 18 months
- Estimated N = 138 (2:1 Duvyzat®; control)
- Primary endpoints: PUL
STUDY 43 was an open label phase 2 study in 20 boys aged 7 to < 11 years with a DMD diagnosis. The primary study objective was the evaluation of the histological effects of Duvyzat® at baseline and after ≥ 12 months of treatment treated patients’ muscle biopsies (brachial biceps). Secondary objectives of the study were safety and tolerability, and functional assessments (6MWT, NSAA and upper limb performance).
- Phase 1/2; duration 12 months
- N = 20 ambulant boys aged 7 to < 11 years with DMD
- Primary endpoints: muscle histology
- Secondary endpoints: 6MWT; NSAA; PUL
- Safety, tolerability and PK
Adverse events should be reported. Reporting forms and information can be found at https://yellowcard.mhra.gov.uk/ or search for MHRA Yellow Card in the Google Play or Apple App Store.
Adverse events should also be reported to ITF Pharma Ltd Medical Information on: 0800 098 4040 or UK.Medical.Information@italfarmacogroup.com
Product Quality Complaints should also be reported to ITF Pharma Ltd Medical Information on: 0800 098 4040 or UK.Medical.Information@italfarmacogroup.com
References
- ClinicalTrials.gov. NCT01761292. Updated 7 November 2023. Available at https://clinicaltrials.gov/study/NCT01761292. Accessed December 2023.
- ClinicalTrials.gov. NCT02851797. Updated 2 February 2023. Available at https://clinicaltrials.gov/study/NCT02851797. Accessed December 2023.
- ClinicalTrials.gov. NCT03373968. Updated 7 November 2023. Available at https://clinicaltrials.gov/study/NCT03373968. Accessed December 2023.
- ClinicalTrials.gov. NCT05933057. Updated 17 July 2023. Available at https://clinicaltrials.gov/study/NCT05933057. Accessed December 2023.
- Mercuri E, Vilchez JJ, Boespflug-Tanguy O, et al. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol. 2024; 23: 393-403.
