Study of ARO-DUX4 in Adult and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1
This research study is investigating a new treatment, ARO-DUX4, for adults and teenagers living with facioscapulohumeral muscular dystrophy type 1 (FSHD1). The main goals are to find out if the medicine is safe, how well people tolerate it, and how it affects the body. Participants will either receive the study medicine or a placebo (a dummy medicine) through injections. The study has two parts: in Part 1, participants receive one dose, and in Part 2, they receive four doses. People who finish Part 1 might be able to join Part 2. This study also involves taking small samples of muscle (biopsies) and having MRI scans to see how the medicine is working. After completing the main study, there might be options to continue receiving the medicine.
Em resumo
Sobre o que é este estudo?
This study is investigating a new investigational medicine called ARO-DUX4 for a condition known as facioscapulohumeral muscular dystrophy type 1, often shortened to FSHD1. FSHD1 is a genetic condition that causes muscles to become weak, typically starting in the face, shoulders, and upper arms, but it can affect other muscles over time. The purpose of this research is to understand if ARO-DUX4 is safe for people to take, how well their bodies tolerate it, and how it works inside the body.
Researchers will be looking at things like how the medicine moves through the body (pharmacokinetics) and what effects it has on the muscle (pharmacodynamics). This information is really important because it helps scientists understand if the medicine could be a helpful treatment in the future. It's a 'Phase 1/Phase 2' study, which means it's one of the early steps in testing a new medicine in people, focusing on safety and initial effectiveness before it can be studied in larger groups.
The study is divided into two main parts. Some participants will receive a single dose of ARO-DUX4, while others will receive a placebo – a substance that looks exactly like the study medicine but contains no active drug. This is a common way to compare the effects of the active medicine. Another group will receive four doses of either ARO-DUX4 or the placebo. Participants will have close medical supervision throughout the study, and there will be opportunities to continue receiving the medicine in a follow-up study if the initial results are promising.
Pontos principais
- This study is testing a new medicine (ARO-DUX4) for FSHD1.
- It aims to check the safety and how the medicine affects the body.
- You might receive the active medicine or a placebo (dummy medicine).
- Participation involves injections, scans, and muscle biopsies.
- You can be from 16 to 70 years old with confirmed FSHD1.
- There's a chance to continue treatment after the main study if it's successful.
Quem pode ser elegível?
To join this study, you must be between 16 and 70 years old. You need to have FSHD1 that has been confirmed by a genetic test or clear hospital records. Your muscle weakness should fall within a certain range (a clinical severity score between 3 and 8 out of 10).
You'll also need to have a suitable leg muscle for a small biopsy, which will be checked using an MRI scan. Your heart health will be checked with an ECG to make sure it's safe for you to take part. If you or your partner can have children, you'll need to use effective birth control during and for some time after the study. Men should also not donate sperm during this period.
There are also some reasons why you wouldn't be able to join. These include having certain infections like HIV, hepatitis B, or hepatitis C, or having uncontrolled high blood pressure or severe heart problems. You also can't take part if you have a history of blood clots, very low platelet counts, or certain blood disorders. If you can't walk, or if having a muscle biopsy or MRI scan is not safe for you, you also won't be able to participate.
Este estudo pode ser adequado para você?
Responda a estas perguntas rápidas para ver se você pode ser elegível. Este é apenas um guia — a decisão final é da equipe de pesquisa.
- Are you between 16 and 70 years old?
- Do you have FSHD1 that has been confirmed by a genetic test or medical records?
- Do you have muscle weakness that falls within a specific range (clinical severity score of 3-8)?
- Are you generally healthy, without severe heart conditions, certain infections (like HIV, Hepatitis B/C), or problems with blood clotting?
- Are you able to have a small muscle sample taken and an MRI scan?
- If you or your partner can have children, are you able to use effective birth control during and after the study?
O que envolve a participação?
If you decide to join this study, you will either receive the new study medicine, ARO-DUX4, or a placebo (a dummy medicine without any active ingredients). Both the medicine and the placebo will be given as injections. The study has two parts: in Part 1, you would receive just one dose. In Part 2, you would receive four doses. If you complete Part 1, you might have the option to join Part 2.
Throughout the study, you will have regular visits to the clinic for check-ups and tests. These tests include physical examinations, blood tests, and MRI scans of your muscles. You will also need to have a small muscle sample taken (called a biopsy) before and after receiving the medicine, so the researchers can see how it's affecting your muscles. If you join Part 2, you'll have an additional biopsy. The total duration of your participation will depend on which part of the study you join and if you move into any optional extension studies.
Riscos e benefícios potenciais
Localizações (17)
- Research Site 2Código postal verificadoLiverpool, Australia· Recrutamento
- Research Site 3Código postal verificadoAuchenflower, Austrália· Recrutamento
- Research Site 1Código postal verificadoBirtinya, Australia· Recrutamento
- Research Site 4Código postal verificadoMelbourne, Austrália· Recrutamento
- Research Site 2Código postal verificadoCalgary, Canadá· Ainda não recrutando
- Research Site 3Código postal verificadoEdmonton, Canada· Recrutamento
- Research Site 1Código postal verificadoMontreal, Canadá· Recrutamento
- Research Site 2Código postal verificadoMunique, Alemanha· Recrutamento
- Research Site 1Código postal verificadoUlm, Germany· Recrutamento
- Research Site 1Código postal verificadoMilão, Itália· Recrutamento
- Research Site 2Código postal verificadoRoma, Itália· Recrutamento
- Research Site 1Código postal verificadoLeiden, Países Baixos· Recrutamento
Perguntas frequentes
What is FSHD1?
FSHD1 is a genetic condition that causes muscles to gradually weaken, often starting in the face, shoulders, and arms.
What is ARO-DUX4?
ARO-DUX4 is a new, investigational medicine being tested in this study to see if it can help people with FSHD1.
Eu definitivamente receberei o medicamento ativo?
You might receive the active medicine ARO-DUX4 or a placebo (a dummy medicine), as this helps researchers compare its effects.
What is a muscle biopsy?
A muscle biopsy is a small procedure where a tiny sample of muscle is taken for examination, usually from your leg, to understand how the medicine is working.
Quanto tempo estarei no estudo?
The length of your participation depends on which part of the study you join, but there may be options to continue in further follow-up studies.
Como saber mais
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