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Tests treatment safety and results for Duchenne Muscular Dystrophy
Official title Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old
ClinicalTrials.gov ID: NCT06769633
What this study is testing
What is Givinostat Hydrochloride?
Givinostat Hydrochloride is an investigational medicine, being studied as a potential treatment for duchenne muscular dystrophy.
Also referred to as Cohort 1.
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 Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in younger DMD Patients. Planned screening duration: approximately 4 weeks Planned Core Treatment duration: approximately 48 weeks Planned Extension Treatment duration: approximately 96 weeks Planned Follow Up duration: approximately 4 weeks (± 7 days) Total duration of study participation: up to 151 weeks (ie, 37-38 months)
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 6, men only
You may be able to join if
- Core Phase:
- Male children aged ≥2 to \<6 years at screening (people ≥6 years of age at screening will not be enrolled into the study)
- Written consent provided by parent/legal guardian and subject written assent, if applicable (according to local regulation)
- A genetic diagnosis of DMD
- Corticosteroid treatment considerations:
You likely can't join if
- Core Phase
- Exposure to another investigational drug within 3 months prior to the start of the study drug
- Exposure to any dystrophin restoration product (eg, Ataluren, Exon skipping) within 6 months prior to the start of study drug
- Received any gene therapy (eg, AAV Micro-dystrophin delivery) within 12 months prior to start of study drug
- Use of any pharmacologic treatment, other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to...
- Have had surgery that might have an effect on muscle strength or function within 3 months prior to start of the study drug or planned surgery at any...
See the full eligibility criteria
Who can join
- Core Phase:
- Male children aged ≥2 to \<6 years at screening (people ≥6 years of age at screening will not be enrolled into the study)
- Written consent provided by parent/legal guardian and subject written assent, if applicable (according to local regulation)
- A genetic diagnosis of DMD
- Corticosteroid treatment considerations:
- For subject receiving a stable dose or oral systemic corticosteroids: No significant change in dose or dosing regimen (except for adjustments due to body weight change) for a minimum of 3 months immediately prior to the...
- For people without current corticosteroid treatment: Must not start corticosteroids in the Core Phase of the study (ie, first 48 weeks).
- Extension Phase:
- Must have participated in the Core Phase study (48 weeks) and have attended the End of Treatment Visit
- Give informed consent and /or assent in writing signed by the parent/legal guardian and/or subject (according to local regulation)
- In stable oral systemic corticosteroids treatment with no significant change in dose or dosing regimen (except for adjustments due to body weight change). For people without corticosteroids during the Core Phase, the...
What rules you out
- Core Phase
- Exposure to another investigational drug within 3 months prior to the start of the study drug
- Exposure to any dystrophin restoration product (eg, Ataluren, Exon skipping) within 6 months prior to the start of study drug
- Received any gene therapy (eg, AAV Micro-dystrophin delivery) within 12 months prior to start of study drug
- Use of any pharmacologic treatment, other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to the start of the study drug (eg, growth hormone). Note: Vitamin D...
- Have had surgery that might have an effect on muscle strength or function within 3 months prior to start of the study drug or planned surgery at any time during the study
- The presence of other clinically significant disease, which, in the Investigator's opinion, could adversely affect subject's safety, making it unlikely to complete the study or to be compliant with study-specific...
- Diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD, based on Investigator clinical medical judgement
- Platelet count, white blood cells, and/or haemoglobin counts \< lower limit of normal (LLN) at screening (Note: for abnormal screening laboratory test results [\<LLN], the platelet count, white blood cell, and...
- 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 subject will...
- 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 in fasting, the subject should...
- Positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at screening
- Baseline corrected QT interval using Fridericia's formula (QTcF) \>450 msec (as the mean of 3 consecutive readings taken 5 minutes apart) or history of additional risk factors for torsades de pointes (ie, heart failure...
- Psychiatric illness or social situations rendering the potential subject unable to understand and comply with the muscle function tests and/or with the study protocol procedures, based on the Investigator's clinical...
- Hypersensitivity to any component of study drug
- Sorbitol intolerance or malabsorption or have the hereditary form of fructose intolerance.
- Body weight \<10 kg at screening. Exclusion Criteria - Extension Phase
- Platelet count, white blood cells, and/or haemoglobin \<LLN at EOT/V12 (Note: for abnormal laboratory test results [\<LLN], the platelet count, white blood cell, and haemoglobin will be repeated once; if the repeat test...
- Current liver disease or impairment, including but not limited to an elevated total bilirubin (ie, \>1.5 × ULN), unless secondary to Gilbert disease or pattern consistent with Gilbert disease
- 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 subject will...
- Fasting triglycerides \>300 mg/dL (3.42 mmol/L; Note: if the value is \>300 mg/dL, the triglycerides will be repeated once; if the repeated test result is still \>300 mg/dL in fasting condition, the subject should be...
- Have presence of other clinically significant disease, which, in the Investigator's opinion, could adversely affect the safety of the subject, making it unlikely that the course of treatment or follow-up would be...
- Evidence of psychiatric illness or social situations rendering the potential subject unable to understand and comply with the muscle function tests and/or with the study protocol procedures, based on the Investigator's...
The study team makes the final eligibility decision.
Where it's taking place
- Brussels, Belgium
- Milan, Italy
- Roma, Italy
- Leiden, Netherlands
- Leeds, England, United Kingdom
- London, England, United Kingdom
- Newcastle upon Tyne, England, United Kingdom
- Oxford, England, United Kingdom
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, 2 years to 6 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Brussels, Belgium; Milan, Italy; Roma, Italy; Leiden, Netherlands; Leeds, England, United Kingdom; London, England, United Kingdom and 2 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.