What side effects are associated with this type of intervention?

The most common treatments for Duchenne Muscular Dystrophy (DMD) include glucocorticoids like prednisone and deflazacort, and genetic therapies such as eteplirsen, golodirsen, and ataluren. Glucocorticoids are associated with side effects such as weight gain, decreased linear growth, cushingoid appearance, delayed puberty, bone fractures, and behavioral changes. Genetic therapies like eteplirsen and golodirsen can cause upper respiratory tract infections, balance disorders, vomiting, and hypersensitivity reactions. Ataluren may lead to vomiting, decreased appetite, weight loss, and hypertension. While specific side effects of histone deacetylase inhibitors like GIVINOSTAT are not detailed in the provided context, they generally include gastrointestinal symptoms, fatigue, and hematologic abnormalities.

What prior FDA approvals exist?

The FDA has approved several drugs for the treatment of Duchenne Muscular Dystrophy (DMD), primarily focusing on genetic therapies designed to increase dystrophin production. These include eteplirsen, golodirsen, viltolarsen, and casimersen, which are exon-skipping drugs targeting specific deletions in the DMD gene. Eteplirsen skips exon 51, golodirsen and viltolarsen skip exon 53, and casimersen skips exon 45. These treatments aim to produce a functional dystrophin protein, albeit shorter. While GIVINOSTAT, a Histone Deacetylase Inhibitor, is being studied for its long-term safety and efficacy, it represents a different therapeutic approach compared to the exon-skipping drugs currently approved.

What other types of research exist?

Promising novel alternatives to GIVINOSTAT for Duchenne Muscular Dystrophy patients include: 1. Eteplirsen (Exon Skipping Therapy) - Approved for specific mutations in the DMD gene. 2. Viltolarsen (Exon Skipping Therapy) - Approved for specific mutations in the DMD gene. 3. Casimersen (Exon Skipping Therapy) - Approved for specific mutations in the DMD gene. 4. Ataluren (Translational Read-Through Drug) - For patients with nonsense mutations in the DMD gene. 5. Gene Therapy Approaches - Ongoing research in gene editing and replacement therapies, such as CRISPR/Cas9 and micro-dystrophin gene therapy, showing potential in clinical trials.

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Histone Deacetylase Inhibitor

Givinostat for Duchenne Muscular Dystrophy

Phase 2 & 3

Waitlist Available

Research Sponsored by Italfarmaco

Eligibility Criteria Checklist

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

Must have

Must not have

Use of any current investigational drug other than Givinostat

Inadequate renal function, as defined by serum Cystatin C >2 x the upper limit of normal (ULN) at screening visit

Timeline

Screening 3 weeks

Treatment Varies

Follow Up through study completion, an average of 1 year

Awards & highlights

No Placebo-Only Group

Summary

This trial tests the safety and effectiveness of GIVINOSTAT, a liquid medicine taken by mouth, in patients with Duchenne's muscular dystrophy who have used it before. The medicine aims to improve muscle function and slow down muscle damage.

Who is the study for?

This trial is for boys aged ≥6 with Duchenne Muscular Dystrophy who've been in a previous Givinostat study. They must have specific muscle fat levels, be able to consent, and use contraception if needed. Excluded are those with hypersensitivity to the drug's components, certain intolerances or diseases, heart failure, liver issues, psychiatric conditions affecting compliance, recent non-steroid muscle treatments, other neurological disorders or abnormal blood tests.

What is being tested?

The trial is testing the long-term safety and effectiveness of Givinostat in patients with Duchenne Muscular Dystrophy. It's an open-label study meaning everyone knows they're getting Givinostat and it focuses on those who've previously taken part in related studies.

What are the potential side effects?

While not explicitly listed here, potential side effects may include reactions similar to any known allergies to ingredients in Givinostat. Patients should also monitor for signs of liver impairment or digestive issues due to sorbitol intolerance mentioned as exclusion criteria.

Eligibility Criteria

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am not using any experimental drugs except Givinostat.

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My kidney function is poor, with high Cystatin C levels.

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I do not have liver disease or high bilirubin levels.

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I have severe heart failure.

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I do not have uncontrolled neurological or other serious health issues unrelated to DMD.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ through study completion, an average of 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~through study completion, an average of 1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and through study completion, an average of 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

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

Palpitations

Constipation

Dysgeusia

Cough

Pruritus

Thrombocytosis

Blood bilirubin increased

Bone pain

Iron deficiency

Dizziness

Neutropenia

Flatulence

Fatigue

Oedema peripheral

Urinary tract infection

Weight decreased

Hypoaesthesia

Acute kidney injury

Dyspnoea

Dyspnoea exertional

Thrombosis

Hypertension

Pneumonia

Headache

Oropharyngeal pain

100%

80%

60%

40%

20%

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

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: givinostatExperimental Treatment1 Intervention

Givinostat oral suspension (10 mg/mL) twice daily in a fed state

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Givinostat

2013

Completed Phase 2

~150

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Duchenne Muscular Dystrophy (DMD) include glucocorticoids, exon-skipping therapies, and histone deacetylase inhibitors (HDAC inhibitors) like GIVINOSTAT. Glucocorticoids reduce inflammation and immune response, slowing muscle degeneration. Exon-skipping therapies, such as eteplirsen, skip over faulty parts of the dystrophin gene to produce a functional dystrophin protein. HDAC inhibitors modify gene expression related to muscle repair and regeneration, potentially improving muscle function and slowing disease progression. These treatments are vital for DMD patients as they help preserve muscle function and enhance quality of life.