BridgeMD
Tests treatment safety and results for Duchenne Muscular Dystrophy
Official title Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy
ClinicalTrials.gov ID: NCT05933057
What this study is testing
What is Givinostat?
Givinostat is an investigational medicine, given as an once-daily, being studied as a potential treatment for duchenne muscular dystrophy.
Also referred to as ITF2357.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a randomised, double-blind, placebo-controlled, multicentre study to evaluate the efficacy, safety, and tolerability of givinostat in non-ambulant male paediatric (aged 9 to \<18 years) patients with DMD. 138 patients will be randomised 2:1 to givinostat or placebo and will be treated for 18 months.
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 9 to 17, men only
You may be able to join if
- Patients must satisfy all the following criteria:
- Children and adolescent males aged ≥ 9 to \<18 years at screening (patients ≥ 18 years of age at screening will not be enrolled into the study)
- Are able to give informed assent and/or consent in writing signed by the patient and/or parent/legal guardian (according to local regulations)
- A genetic diagnosis of DMD
- Non-ambulant, defined as being wheelchair bound and:
You likely can't join if
- Patients will be excluded from the study if they satisfy any of the following criteria:
- Exposure to another investigational drug within 3 months prior to start of study treatment.
- Have exposure to any dystrophin restoration product (eg, Ataluren, Exon skipping) within 6 months prior to the start of study treatment
- Having received any gene therapy (eg, AAV Micro-dystrophin delivery) prior to start of study treatment
- Use of any pharmacologic treatment or supplement (other than corticosteroids), that might have had an effect on muscle strength or function within 3...
- Use of testosterone, unless used as a replacement therapy for the treatment of delayed puberty. The testosterone dose and regimen should be stable...
See the full eligibility criteria
Who can join
- Patients must satisfy all the following criteria:
- Children and adolescent males aged ≥ 9 to \<18 years at screening (patients ≥ 18 years of age at screening will not be enrolled into the study)
- Are able to give informed assent and/or consent in writing signed by the patient and/or parent/legal guardian (according to local regulations)
- A genetic diagnosis of DMD
- Non-ambulant, defined as being wheelchair bound and:
- Unable to perform the 10-meter walk/run test (10MWT), or
- Unable to complete the 10MWT in 30 seconds or less, without any support or devices
- Performance of the Upper Limb test (PUL version 2.0) entry item scores 3 to 6
- If on medication for DMD-associated cardiomyopathy (eg, ACE inhibitor, β-blocker, diuretics), stable for ≥1 month immediately prior to start of study treatment, if any
- Stable corticosteroids, defined as:
- Receiving systemic corticosteroids for a minimum of 6 months immediately prior to start of study treatment
- No significant change in dose or dosing regimen (except for adjustments due to body weight change) for a minimum of 6 months immediately prior to start of study treatment
- Willing to use adequate contraception. Effective contraceptive methods must be used from randomisation visit through 3 months after the last dose of study drug, and include the following:
- True abstinence (ie, absence of any sexual intercourse), when in line with the preferred and usual lifestyle of the patient. Periodic abstinence (eg, calendar, ovulation, post-ovulation, and symptothermal methods) and...
- Condom with spermicide and the female partner must use an effective method of contraception, such as an oral, transdermal, injectable or implanted hormonal contraceptive; intrauterine device; bilateral tubal occlusion...
What rules you out
- Patients will be excluded from the study if they satisfy any of the following criteria:
- Exposure to another investigational drug within 3 months prior to start of study treatment.
- Have exposure to any dystrophin restoration product (eg, Ataluren, Exon skipping) within 6 months prior to the start of study treatment
- Having received any gene therapy (eg, AAV Micro-dystrophin delivery) prior to start of study treatment
- Use of any pharmacologic treatment or supplement (other than corticosteroids), that might have had an effect on muscle strength or function within 3 months prior to the start of study treatment (eg, growth hormone)...
- Use of testosterone, unless used as a replacement therapy for the treatment of delayed puberty. The testosterone dose and regimen should be stable within 6 months prior to the start of study treatment, and circulating...
- Elbow-flexion contractures \>30° in the dominant arm
- Inability to perform consistent PUL 2.0 measurement within ±2 points without shoulder domain or within ±3 points with shoulder domain during paired testing at screening
- Forced Vital Capacity % of predicted \<40%
- Requirement for daytime ventilator assistance (Note: Night ventilator assistance and use of bi-level positive airway pressure therapy is allowed)
- Episode of respiratory failure within the 8 weeks prior to screening
- Symptomatic cardiomyopathy or heart failure and/or left ventricular ejection fraction \<45%
- Baseline corrected QT interval using Fredericia's formula (QTcF) \>450 msec (as the mean of 3 consecutive readings 5 minutes apart) or history of additional risk factors for torsades de pointes (eg, heart failure...
- Major surgical procedure (including scoliosis surgery) planned within 1 year of the start of study treatment
- Poorly controlled asthma or underlying lung disease such as bronchitis, bronchiectasis, emphysema, recurrent pneumonia that in the opinion of the Investigator might impact respiratory function
- Platelets, white blood cells, and/or haemoglobin \< lower limit of normal (LLN) at screening (Note: for abnormal screening laboratory test results [\<LLN], the platelets count, white blood cell, and haemoglobin will be...
- Fasting triglycerides \>300 mg/dL (3.42 mmol/L) at screening (Note: if the value is \>300 mg/dL, the triglycerides will be repeated once; if the repeated test result is still \>300 mg/dL, the patient should be excluded)
- Current or history of liver disease or impairment, including but not limited to a baseline elevated total bilirubin (ie, \>1.5 × upper limit of normal [ULN]), unless secondary to Gilbert disease or pattern consistent...
- Inadequate renal function, as defined by serum Cystatin C result \>2 × ULN (Note: if the value is \>2 × ULN, the serum Cystatin C will be repeated once; if the repeated test result is still \>2 × ULN, the patient should...
- Positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at screening
- Hypersensitivity to any component of study medication
- Sorbitol intolerance or malabsorption, or have the hereditary form of fructose intolerance
- Diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD, based on Investigator judgement
- Psychiatric illness or social situations rendering the potential patient unable to understand and comply with the muscle function tests and/or with the study protocol procedures, based on Investigator judgement
- Have contraindications to MRI scan (eg, claustrophobia, metal implants, or uncontrolled seizure disorder), based on Investigator's judgement.
The study team makes the final eligibility decision.
Where it's taking place
- Leuven, Belgium
- Vancouver, British Columbia, Canada
- London, Ontario, Canada
- Ottawa, Ontario, Canada
- Toronto, Ontario, Canada
- Prague, Czechia
- Lille, France
- Marseille, France
- Paris, France
- Berlin, Germany
- Essen, Germany
- Freiburg im Breisgau, Germany
- Hamburg, Germany
- Lecco, Italy
- Milan, Italy
- Padova, Italy
- Roma, Italy
- Leiden, Netherlands
- Nijmegen, Netherlands
- Gdansk, Poland
+ 8 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 9 years to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Leuven, Belgium; Vancouver, British Columbia, Canada; London, Ontario, Canada; Ottawa, Ontario, Canada; Toronto, Ontario, Canada; Prague, Czechia and 22 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.