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Histone Deacetylase Inhibitor

Givinostat for Duchenne Muscular Dystrophy (ULYSSES Trial)

Q: Is the inclusion criteria for this research study open to individuals aged 55 and above?

Patients aged between 9 and 17 years are sought for participation in this study. There are a total of 67 trials available for individuals under 18, while there are 56 options for those above the age of 65.

Q: Do numerous medical facilities in North America participate in this study?

The clinical trial is conducted at Charite-Universitaetsmedizin in Berlin, British Columbia; Universitaetsklinikum Freiburg in Freiburg, Ontario; and Universitaire Ziekenhuizen Leuven in Leuven, Scotland. Furthermore, the trial includes additional sites across 20 other locations.

Q: Is this medical study currently open for enrollment?

Indeed, as per details on clinicaltrials.gov, this study is currently in search of eligible participants. Initially shared on February 19th, 2024, the latest update was made on February 20th, 2024. A total of 138 individuals are sought for enrollment across a network of 20 sites.

Q: What is the total number of subjects being examined in this particular research investigation?

A total of 138 eligible individuals are needed to join this research project. Patients have the opportunity to take part in this study at diverse sites such as Charite-Universitaetsmedizin Berlin in Berlin, British Columbia, and Universitaetsklinikum Freiburg in Freiburg, Ontario.

Q: Has Givinostat received approval from the FDA?

According to our evaluation at Power, Givinostat's safety rating is 3 due to its Phase 3 trial status, indicating a foundation of efficacy data and extensive safety evidence.

Phase 3

Recruiting

Research Sponsored by Italfarmaco

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Performance of the Upper Limb test (PUL version 2.0) entry item scores 3 to 6

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to 18 months

Awards & highlights

ULYSSES Trial Summary

This trial will test the drug givinostat in young male patients with DMD to see if it is effective and safe. 138 patients will be randomly assigned to receive either givin

Who is the study for?

This trial is for non-ambulant male children and teens aged 9 to <18 with Duchenne Muscular Dystrophy (DMD). They must have a genetic diagnosis of DMD, be unable to walk or run 10 meters in under 30 seconds without help, and have been on stable corticosteroid treatment for at least 6 months. Participants need parental consent and agree to use contraception during the study.Check my eligibility

What is being tested?

The trial tests Givinostat's effectiveness, safety, and tolerability against a placebo in patients who can't walk due to DMD. It's randomized and double-blind, meaning neither doctors nor participants know who gets the real drug or placebo. The treatment lasts for 18 months with follow-ups.See study design

What are the potential side effects?

While specific side effects are not listed here, Givinostat could potentially cause adverse reactions ranging from mild discomforts like headaches or nausea to more serious issues depending on how it affects muscle tissue and other organs.

ULYSSES Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My arm and hand abilities score is between 3 to 6.

Select...

I cannot walk and use a wheelchair.

Select...

I am a boy aged 9 to 17.

Select...

I have been genetically diagnosed with Duchenne Muscular Dystrophy (DMD).

ULYSSES Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to 18 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to 18 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to 18 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change of Performance of Upper Limb 2.0 (PUL) total score at 18 months of treatment of givinostat compared to placebo group.

Secondary outcome measures

Change from baseline electrocardiogram and echocardiogram

Change from baseline of Forced Vital Capacity percent predicted (FVC%p) at 18 months of treatment of givinostat compared to placebo group

Change from baseline of Peak Expiratory Flow percent predicted (PEF%p) at 18 months of treatment of givinostat compared to placebo group

+5 more

Other outcome measures

Age at loss of hand-to-mouth function assessed by PUL

Age at loss of turn in bed ability assessed by EK

Change from baseline as measured by Motor Function Measure (MFM) at 18 months of treatment of givinostat compared to placebo group

+11 more

Side effects data

From 2017 Phase 1 & 2 trial • 48 Patients • NCT01901432

67%

Diarrhoea

67%

Dysgeusia

33%

Haemorrhoids

33%

Asthenia

33%

Blood creatinine increased

33%

Abdominal pain

33%

Flatulence

33%

Fatigue

33%

Electrocardiogram qt prolonged

33%

Hyperglycaemia

33%

Hyperkalaemia

33%

Alopecia

33%

Myocardial infarction

33%

Chromaturia

33%

Chronic kidney disease

33%

Flushing

100%

80%

60%

40%

20%

Study treatment Arm

Givinostat DL6 (100 mg + 50 mg) (Part A)

Givinostat at MTD (100 mg b.i.d.) (Part B)

Givinostat DL0 (50 mg b.i.d.) (Part A)

Givinostat DL1 (100 mg b.i.d.) (Part A)

Givinostat DL1 Expanded (100 mg b.i.d.) (Part A)

ULYSSES Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: GivinostatExperimental Treatment1 Intervention

Patients will receive concomitant corticosteroid treatment as part of the standard of care.

Group II: PlaceboPlacebo Group1 Intervention

Patients will receive concomitant corticosteroid treatment as part of the standard of care.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Givinostat

2013

Completed Phase 2

~100

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

ItalfarmacoLead Sponsor

33 Previous Clinical Trials

4,361 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is the inclusion criteria for this research study open to individuals aged 55 and above?

"Patients aged between 9 and 17 years are sought for participation in this study. There are a total of 67 trials available for individuals under 18, while there are 56 options for those above the age of 65."

Answered by AI

Do numerous medical facilities in North America participate in this study?

"The clinical trial is conducted at Charite-Universitaetsmedizin in Berlin, British Columbia; Universitaetsklinikum Freiburg in Freiburg, Ontario; and Universitaire Ziekenhuizen Leuven in Leuven, Scotland. Furthermore, the trial includes additional sites across 20 other locations."

Answered by AI

Is this medical study currently open for enrollment?

"Indeed, as per details on clinicaltrials.gov, this study is currently in search of eligible participants. Initially shared on February 19th, 2024, the latest update was made on February 20th, 2024. A total of 138 individuals are sought for enrollment across a network of 20 sites."

Answered by AI

What is the total number of subjects being examined in this particular research investigation?

"A total of 138 eligible individuals are needed to join this research project. Patients have the opportunity to take part in this study at diverse sites such as Charite-Universitaetsmedizin Berlin in Berlin, British Columbia, and Universitaetsklinikum Freiburg in Freiburg, Ontario."

Answered by AI

Has Givinostat received approval from the FDA?

"According to our evaluation at Power, Givinostat's safety rating is 3 due to its Phase 3 trial status, indicating a foundation of efficacy data and extensive safety evidence."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy

2024. "Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05933057.

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