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.
Överblick
Vad handlar denna studie om?
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.
Viktiga punkter
- 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.
- Du kan dra dig ur studien när som helst.
Vem kan vara berättigad?
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.
Kan denna studie passa dig?
Svara på dessa snabba frågor för att se om du kan vara berättigad. Detta är endast en vägledning — forskarteamet fattar det slutgiltiga beslutet.
- Är du mellan 18 och 65 år gammal?
- Do you have a confirmed diagnosis of FSHD?
- Are you able to walk without help?
- Are you currently pregnant, breastfeeding, or planning to become pregnant?
- Do you have any serious heart conditions or severe allergies?
- Have you been treated with similar experimental drugs recently?
Vad innebär deltagande?
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.
Potentiella risker och fördelar
Platser (9)
- University of Irvine Medical Center (UCIMC)Verifierat postnummerOrange, USA
- Regents of the University of ColoradoVerifierat postnummerAurora, USA
- University of Kansas Medical CenterVerifierat postnummerFairway, USA
- Kennedy Krieger InstitutetVerifierat postnummerBaltimore, USA
- RigshospitaletVerifierat postnummerKøbenhavn Ø, Denmark
- Policlinico Universitario Agostino GemelliVerifierat postnummerRome, Italy
- Asst Grande Ospedale Metropolitano NiguardaVerifierat postnummerMilano, Italien
- National Hospital for Neurology and Neurosurgery,Verifierat postnummerLondon, Storbritannien
- Royal Victoria InfirmaryVerifierat postnummerNewcastle upon Tyne, Storbritannien
Vanliga frågor
Vad är 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.
Vad är en 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.
Hur länge kommer jag att vara med i studien?
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.
Hur du får reda på mer
Prata alltid med din läkare eller specialist innan du bestämmer dig för att delta i en studie.
Diskussion
Gemenskapsdiskussion
Drivs av vårt forum på community.patient.info. Var vänlig och respektfull — detta är inte medicinsk rådgivning.