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Histone Deacetylase Inhibitor

Givinostat for Duchenne Muscular Dystrophy (ULYSSES Trial)

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
Must not have
Inadequate renal function
Use of testosterone, unless used as a replacement therapy for the treatment of delayed puberty
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to 18 months
Awards & highlights
Pivotal 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.
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.
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.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My arm and hand abilities score is between 3 to 6.
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I cannot walk and use a wheelchair.
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I am a boy aged 9 to 17.
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I have been genetically diagnosed with Duchenne Muscular Dystrophy (DMD).

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My kidneys are not working well.
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I use testosterone only for delayed puberty treatment.
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I have not received gene therapy before starting this study.
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I have or had liver disease.
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My dominant arm cannot fully straighten, bending more than 30 degrees.
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I cannot tolerate sorbitol or have a hereditary fructose intolerance.
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I need help from a machine to breathe during the day.
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My asthma or lung disease is not well-managed.
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My heart's pumping ability is weak.
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I do not have uncontrolled neurological or unrelated health issues.

Timeline

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

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change of Performance of Upper Limb 2.0 (PUL) total score at 18 months of treatment of givinostat compared to placebo group.
Other study objectives
Pharmacokinetic/Pharmacodynamic Endpoints
Proportion of responders of givinostat compared to placebo group

Side effects data

From 2017 Phase 1 & 2 trial • 48 Patients • NCT01901432
57%
Diarrhoea
46%
Thrombocytopenia
40%
Blood creatinine increased
29%
Nausea
29%
Asthenia
23%
Abdominal pain
17%
Anaemia
14%
Hypocalcaemia
14%
Dyspepsia
11%
Abdominal pain upper
11%
Pyrexia
11%
Decreased appetite
11%
Alopecia
9%
Palpitations
9%
Constipation
9%
Dysgeusia
9%
Cough
9%
Pruritus
6%
Thrombocytosis
6%
Blood bilirubin increased
6%
Bone pain
6%
Iron deficiency
6%
Dizziness
6%
Neutropenia
6%
Flatulence
6%
Fatigue
6%
Oedema peripheral
6%
Urinary tract infection
6%
Weight decreased
6%
Hypoaesthesia
6%
Acute kidney injury
6%
Dyspnoea
6%
Dyspnoea exertional
6%
Thrombosis
6%
Hypertension
3%
Pneumonia
3%
Headache
3%
Oropharyngeal pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
Givinostat at MTD (100 mg b.i.d.) (Part B)
Givinostat DL1 Expanded (100 mg b.i.d.) (Part A)
Givinostat DL6 (100 mg + 50 mg) (Part A)
Givinostat DL0 (50 mg b.i.d.) (Part A)
Givinostat DL1 (100 mg b.i.d.) (Part A)

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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
~150

Find a Location

Who is running the clinical trial?

FortreaIndustry Sponsor
20 Previous Clinical Trials
5,493 Total Patients Enrolled
ItalfarmacoLead Sponsor
34 Previous Clinical Trials
4,385 Total Patients Enrolled
~92 spots leftby Feb 2028
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