Study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy of givinostat in patients with Becker Muscular Dystrophy

Phase 1

• Conditions: Distrofia Muscolare di Becker (DMB); MedDRA version: 20.0Level: PTClassification code 10059117Term: Becker's muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: EUCTR2017-001629-41-NL
• Lead Sponsor: ITALFARMACO S.P.A.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-05-31
• Last Update Posted: 25 May 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 48

Inclusion Criteria

1. Ambulant male patients aged =18 years to < 65 years at randomization with BMD diagnosis confirmed by genetic testing.
2. Able and willing to give informed consent in writing.
3. Able to perform 6MWT at screening with a minimum distance of 200 m and maximum distance of 450 m.
4. If in treatment with systemic corticosteroids and/or ACE inhibitor, and/or ß or a adrenergic receptor blocker, no significant
change in dosage or dosing regimen (excluding changes related to body weight change) for a minimum of 6 months immediately
prior to start of study treatment.
5. Patients must be willing to use adequate contraception. Contraceptive methods must be used from Randomization through 3
months after the last dose of study treatment.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 48
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Exposure to another investigational drug within 3 months prior to the start of study treatment.
2. Use of any pharmacologic treatment, other than corticosteroids, that might have an effect on muscle strength or function within
3 months prior to the start of study treatment (e.g., growth hormone). Vitamin D, calcium, and any other supplements will be
allowed.
3. Surgery that might affect muscle strength or function within 3 months before study entry or planned surgery at any time during
the study.
4. Presence of other clinically significant disease that in the Investigator’s opinion could adversely affect the safety of the patient,
making it unlikely that the course of treatment or follow-up is completed, or could impair the assessment of study results.
5. A diagnosis of other uncontrolled neurological diseases or presence of relevant somatic disorders not related to BMD that may interfere with the ability to perform the muscle function tests and/or to comply with the study protocol procedures.
6. Platelet count, WBC count and hemoglobin at screening < Lower Limit of Normal (LLN). If laboratory screening results are < LLN,
platelet count, WBC count and hemoglobin are to be repeated once, and if again < LLN become exclusionary.
7. Symptomatic cardiomyopathy or heart failure (New York Heart Association Class III or IV) or left ventricular ejection fraction <
50% at screening or with heart transplant.
8. Current liver disease or impairment, including but not limited to elevated total bilirubin (> 1.5 x ULN), unless secondary to
Gilbert’s disease or pattern consistent with Gilbert's disease.
9. Inadequate renal function, as defined by serum Cystatin C > 2 x the upper limit of normal (ULN). If the value is > 2 x ULN,
serum Cystatin C will be repeated once, and if again > 2 x ULN becomes exclusionary.
10. Positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at screening.
11. Baseline corrected QT interval, Fredericia’s correction (QTcF) > 450 msec, (as the mean of 3 consecutive readings 5 minutes
apart) or history of additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, or family history of long QT
syndrome).
12. Current psychiatric illness/social situations rendering the potential patient unable to understand and comply with the muscle function
tests and/or with the study protocol procedures.
13. Hypersensitivity to the components of study medication.
14. Sorbitol intolerance or sorbitol malabsorption, or the hereditary form of fructose intolerance.
15. Contraindications to muscle biopsy.
16. Contraindications to MRI/MRS (e.g., claustrophobia, metal implants, or seizure disorder).
17. Hypertriglyceridemia (> 1.5 x upper limit of normal [ULN])*
* At screening, patients with hypertriglyceridemia can be enrolled if in stable treatment and with controlled levels of triglycerides (i.e. within normal range) for at least six months.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified