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Activo, no reclutandoFASE2INTERVENCIONISTA

A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy

This research study is investigating a new medication called RO7204239 for adults who have Facioscapulohumeral Muscular Dystrophy (FSHD). The main goal is to understand how this medication works in the body, if it's safe to use, and whether it can help improve the condition. Participants will either receive the new medication or a placebo (a dummy treatment) to compare the effects. Researchers will be checking various aspects, including how the medicine moves through the body, its overall safety, and if it makes any difference to the symptoms of FSHD. This is a Phase 2 study, which means it's still in the earlier stages of testing.

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Estado
Activo, no reclutando
Fase
FASE2
Patrocinador
Hoffmann-La Roche
Objetivo de inscripción
51
Inicio
07 Feb 2023
Estimación de finalización
09 Oct 2026

¿De qué trata este estudio?

This study is an important step in understanding and potentially treating Facioscapulohumeral Muscular Dystrophy, often called FSHD. FSHD is a genetic condition that causes muscles to weaken over time. Researchers are exploring a new medication, known as RO7204239, which is a type of antibody designed to target a specific protein in the body. This protein, called myostatin, can sometimes stop muscles from growing.

The main aim of this study is to learn more about how RO7204239 affects people with FSHD. This includes looking at how the body handles the medication, if it's safe and well-tolerated, and whether it has any positive effects on the condition itself. Participants will be carefully monitored throughout the study to gather this information.

This is a 'Phase 2' study, which means it's an important stage for checking the medication's safety and effectiveness in a smaller group of people. If the results are promising, it could lead to larger studies in the future. The information gained from this research will help scientists understand if RO7204239 could be a helpful treatment option for people living with FSHD.

Puntos clave

  • This study is testing a new medication (RO7204239) for FSHD.
  • It aims to see if the medication is safe, well-tolerated, and effective.
  • You might receive the new medication or a placebo.
  • Participation involves regular clinic visits for checks, tests, and medication.
  • You must be an adult with confirmed FSHD, able to walk unassisted.
  • Puede retirarse del estudio en cualquier momento.

¿Quién puede ser elegible?

To join this study, you must be an adult between 18 and 65 years old and have a confirmed diagnosis of FSHD (either type 1 or type 2), with symptoms that match the condition. You need to be able to walk without help and your FSHD should be within a certain range of severity. It's also important that you agree to keep your physiotherapy, occupational therapy, and other exercise routines the same throughout the study.

There are some reasons why you might not be able to join. For example, if you are pregnant, breastfeeding, or planning to become pregnant within 17 months after the study ends. You also can't have been treated with similar myostatin-targeting medications before, or with any other experimental medications in the last 90 days (or longer, depending on the medication). If you have certain medical conditions like serious heart problems, allergies to the study drug, or some types of cancer, you won't be able to take part.

Additionally, if you have any conditions that would make MRI scans unsafe, or issues with your skin where injections might be given, you wouldn't be eligible. Recent injuries or surgeries that could affect how your movement is assessed would also prevent you from joining, as would any planned surgery that might impact your motor function during the study.

¿Podría este estudio ser adecuado para ti?

Responde estas preguntas rápidas para ver si puedes ser elegible. Esto es solo una guía: el equipo de investigación toma la decisión final.

  1. ¿Tienes entre 18 y 65 años?
  2. Do you have a confirmed diagnosis of FSHD?
  3. Are you able to walk without help?
  4. Are you currently pregnant, breastfeeding, or planning to become pregnant?
  5. Do you have any serious heart conditions or severe allergies?
  6. Have you been treated with similar experimental drugs recently?
Responde cada pregunta para ver tu resultado.

¿En qué consiste la participación?

If you decide to take part in this study, you will either receive the new medication RO7204239 or a placebo (a treatment that looks like the medication but has no active ingredients). This is decided randomly, meaning you won't know which one you're getting, and neither will the study team. You will have regular visits to the study clinic for check-ups, assessments, and to receive your medication.

During these visits, the study team will perform various tests. These include physical examinations, blood tests, assessments of your muscle strength and movement, and potentially MRI scans. They will also ask you about any side effects you might be experiencing. The medication will likely be given as an injection under the skin. You'll need to continue your usual physiotherapy and exercise routines, but it's important not to change the frequency or intensity while you're in the study.

The total duration of your participation in the study, including follow-up appointments after your last dose of medication, will be explained by the study team. It's a commitment to regular visits and assessments over an extended period.

Riesgos y beneficios potenciales

Taking part in a clinical trial may offer potential benefits, such as contributing to medical knowledge about FSHD and potentially receiving a new medication that could help your condition. However, there's no guarantee that you will personally benefit from the study medication. As with any medication, there are potential risks and side effects that will be explained to you in detail. The study team will closely monitor you for any adverse reactions throughout your participation. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (9)

  • University of Irvine Medical Center (UCIMC)
    Código postal verificado
    Orange, Estados Unidos
  • Regents of the University of Colorado
    Código postal verificado
    Aurora, Estados Unidos
  • Centro Médico de la Universidad de Kansas
    Código postal verificado
    Fairway, Estados Unidos
  • Kennedy Krieger Institute
    Código postal verificado
    Baltimore, Estados Unidos
  • Rigshospitalet
    Código postal verificado
    København Ø, Denmark
  • Policlinico Universitario Agostino Gemelli
    Código postal verificado
    Rome, Italy
  • Asst Grande Ospedale Metropolitano Niguarda
    Código postal verificado
    Milan, Italy
  • National Hospital for Neurology and Neurosurgery,
    Código postal verificado
    Londres, Reino Unido
  • Royal Victoria Infirmary
    Código postal verificado
    Newcastle upon Tyne, United Kingdom

Preguntas comunes

What is FSHD?

FSHD stands for Facioscapulohumeral Muscular Dystrophy, a genetic condition that causes muscles to gradually weaken, often starting in the face, shoulders, and upper arms.

¿Qué es un placebo?

A placebo is a 'dummy' treatment that looks exactly like the study medication but contains no active ingredients. It helps researchers understand if the new medication is truly having an effect, or if improvements are due to other factors.

What is a 'humanized monoclonal antibody'?

It's a type of medication that is specially designed to target specific things in your body. In this study, it's designed to interact with a protein called myostatin, which is involved in muscle growth.

Will I know if I'm getting the actual medication or the placebo?

No, this is a 'blinded' study, meaning neither you nor the study team will know whether you are receiving RO7204239 or the placebo. This helps ensure the study results are as accurate as possible.

¿Cuánto tiempo estaré en el estudio?

The full length of your participation, including any follow-up visits after your last dose, will be explained during the consent process. It's an extended commitment that will involve various visits over time.

Cómo obtener más información

Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.

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