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Long-Term Safety and Efficacy Study of Givinostat for Patients with Duchenne Muscular Dystrophy Previously Treated with Givinostat

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What is this study about?

This clinical trial is focused on studying Duchenne Muscular Dystrophy (DMD), a genetic disorder characterized by progressive muscle weakness and degeneration. The treatment being investigated is called GIVINOSTAT, which is administered as an oral suspension. The purpose of the study is to evaluate the long-term safety, tolerability, and effectiveness of GIVINOSTAT in patients with Duchenne Muscular Dystrophy who have previously participated in other studies involving this medication.

During the study, all participants will receive the same investigational drug, GIVINOSTAT. The study will monitor the type, frequency, and severity of any side effects that may occur. For patients who can walk, changes in physical function and muscle strength will be assessed. For those who cannot walk, changes in physical function, daily living activities, and upper limb strength will be evaluated. Additionally, the study will track changes in respiratory function and quality of life for all participants.

The study will continue over several years, with assessments taking place at specific intervals, such as after 48 weeks and then annually. The goal is to gather comprehensive data on how GIVINOSTAT affects patients with Duchenne Muscular Dystrophy over an extended period. This information will help determine the long-term benefits and risks of using GIVINOSTAT as a treatment option for this condition.

1 joining the study

Participation requires previous involvement in a GIVINOSTAT study for Duchenne Muscular Dystrophy (DMD) or screening in study DSC/14/2357/48.

Eligibility includes being at least 6 years old and able to provide written consent or assent.

2 medication administration

The investigational drug, GIVINOSTAT, is administered as an oral suspension.

The dosage and frequency are determined by the study protocol and are consistent throughout the study duration.

3 monitoring and assessments

Regular assessments are conducted to monitor the type, incidence, and severity of any adverse effects.

For ambulant patients, physical function is evaluated using tests like the 6-minute walk test (6MWT) and muscle strength assessments at week 48 and then yearly.

4 non-ambulant patient assessments

Non-ambulant patients undergo evaluations of physical function using the Egen Klassifikation (EK) score and muscle strength assessments.

Changes in daily living activities are measured using the Barthel Index at week 48 and then yearly.

5 general assessments

All patients have their respiratory function and quality of life assessed at week 48 and then yearly.

The study tracks major disease milestones such as age at loss of ambulation and need for respiratory support.

6 study duration

The study is expected to continue until December 3, 2027.

Assessments and monitoring occur regularly throughout the study period to ensure safety and efficacy.

Who Can Join the Study?

  • The study is for patients with Duchenne Muscular Dystrophy (DMD).
  • Participants must have been part of a previous study with the drug GIVINOSTAT for DMD and completed the study visit, or they must have been screened in a specific study and met all the necessary criteria without being excluded.
  • Participants must be at least 6 years old.
  • Participants need to be able to provide written agreement to join the study, which can be signed by them or their parent/legal guardian, depending on local rules.
  • Participants must agree to use reliable birth control methods during the study and for three months after the last dose of the study drug. Acceptable methods include:
    • True abstinence, meaning no sexual intercourse, if it fits the participant’s lifestyle.
    • Using a condom with spermicide, and the female partner must also use a reliable birth control method, such as birth control pills, patches, injections, implants, or a barrier method like a diaphragm with spermicide.

Who Cannot Join the Study?

  • Only males can participate in the study. Females are not eligible.
  • Participants must not have any other serious health conditions that could interfere with the study.
  • Participants must not have any allergies or reactions to the study medication or similar drugs.
  • Participants must not be taking any other medications that could interfere with the study medication.
  • Participants must not have participated in another clinical trial recently.
  • Participants must not have any history of drug or alcohol abuse.
  • Participants must not have any mental health conditions that could affect their ability to participate in the study.
  • Participants must not have any infections or illnesses that could affect the study results.
  • Participants must not have any heart problems that could be worsened by the study medication.
  • Participants must not have any liver or kidney problems that could affect how the body processes the study medication.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy
Hospital Universitario Y Politecnico La Fe Valencia Spain
Associazione La Nostra Famiglia Conegliano Italy
Centre Hospitalier Universitaire De Lille Lille France
Katholieke Universiteit te Leuven Leuven Belgium

Other Sites

Site Name City Country Status
IRCCS Istituto Giannina Gaslini Genoa Italy
Hospital Sant Joan De Deu Barcelona Esplugues De Llobregat Spain
Centre Hospitalier Regional De La Citadelle Liege Belgium
Azienda Ospedaliera di Padova Padua Italy
Virgen del Rocío University Hospital Sevilla Spain
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Assistance Publique Hopitaux De Paris Paris France
Medical Center – University Of Freiburg Freiburg Im Breisgau Germany
Centre Hospitalier Universitaire De Nantes Nantes France
A.O.U. Policlinico G. Martino Di Messina Messina Italy
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Fondazione I.R.C.C.S. Istituto Neurologico Besta Milan Italy
Czlxev Ckpiwmg Neyv Milan Italy
Ukupsplvmz Msykzms Cnxfdn Hkejpztinnibtwzrz Hamburg Germany
Ubagyrmrmzivsaihdagye Epfth Ais Essen Germany
Lovng Uybvspawcnli Mthunoo Cexrvaw (prdwc Leiden The Netherlands
Avybboatbz Pudunptb Hbyxqnxy Db Mmoxshbeo Marseille France
Sdifhhdfr Rnwodjw Uratpjhdrf Meffqgh Cvunbr Nijmegen The Netherlands
Kpacwcwd dzl Umbxengscdab Mnmylqmz Arx Munich Germany
Hasbonhd Vjnc dkuxoaqb Barcelona Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Recruiting
03.07.2017
France France
Recruiting
03.07.2017
Germany Germany
Recruiting
03.07.2017
Italy Italy
Recruiting
03.07.2017
Spain Spain
Recruiting
03.07.2017
The Netherlands The Netherlands
Recruiting
03.07.2017

Trial locations

Investigated drugs:

Givinostat is a medication being studied for its long-term safety, tolerability, and effectiveness in patients with Duchenne Muscular Dystrophy (DMD). This study includes patients who have previously been treated with Givinostat in other studies, as well as new patients who meet specific criteria. The goal is to understand how well patients tolerate the medication over a longer period and to monitor any potential side effects while assessing its benefits in managing DMD symptoms.

Investigated diseases:

Duchenne Muscular Dystrophy – Duchenne Muscular Dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness. It primarily affects boys and is caused by mutations in the dystrophin gene, which is crucial for maintaining muscle cell structure. Symptoms usually begin in early childhood, with affected children experiencing difficulty in walking, running, and jumping. As the disease progresses, muscle weakness spreads to the arms, neck, and other areas, leading to loss of ambulation. Over time, individuals with DMD may also experience respiratory and cardiac complications due to muscle deterioration. The progression of the disease varies, but it generally leads to significant physical disability.

Trial ID:
2023-504520-26-00
Protocol code:
DSC/14/2357/51
Trial Phase:
Therapeutic confirmatory (Phase III)

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