Pegtibatinase as a Treatment for Participants With Classical Homocystinuria (HCU) Receiving Standard of Care Treatment
This study is investigating a new medication called pegtibatinase for individuals aged 12 to 65 who have a rare genetic condition called Homocystinuria (HCU). HCU means the body struggles to break down certain proteins. The study will compare pegtibatinase to a placebo (a dummy drug) to see how well it works and if it's safe. Participants will continue their usual HCU treatments throughout the study. The main goal is to find out if pegtibatinase can help manage HCU more effectively. This is a "Phase 3" study, meaning it's one of the final steps before a medicine might be approved for wider use.
Auf einen Blick
Worum geht es in dieser Studie?
This research is looking into a new treatment called pegtibatinase for a condition called Homocystinuria (HCU). HCU is a rare inherited condition where your body has trouble processing a building block of protein called methionine. This can lead to a harmful substance called total homocysteine (tHcy) building up in your blood, which can cause serious health problems if not managed.
The main aim of this study is to see if pegtibatinase is effective and safe for people with HCU. It will compare the new drug with a placebo, which looks just like the study drug but doesn't contain any active medicine. This is a common way to test new treatments, as it helps researchers understand if any changes are truly due to the new drug. All participants in the study will continue to receive their standard HCU care, such as special diets and other medications, alongside the study treatment.
This study is a 'Phase 3' trial, which means it's a large, important step in testing a new medicine. If the results are positive, it could lead to pegtibatinase becoming a new treatment option for people with HCU. The study is international and will involve around 70 people to get a good understanding of how the drug works.
Wichtige Erkenntnisse
- This study is testing a new drug, pegtibatinase, for Homocystinuria (HCU).
- It aims to find out if the drug is both safe and works better than a dummy treatment (placebo).
- Participants will continue their usual HCU diet and treatments.
- The study lasts up to 38 weeks and involves regular check-ups.
- You will not know if you're getting the active drug or placebo during the main treatment period.
Wer könnte berechtigt sein?
To join this study, you need to be between 12 and 65 years old and have a confirmed diagnosis of Homocystinuria (HCU). Your blood test for total homocysteine (tHcy) needs to show levels of 50 µM (micromoles per litre) or higher. It's important that you're willing to stick to a stable diet and continue your current HCU treatments (like betaine or special medical foods) throughout the study, unless your doctor says otherwise for your health.
There are also some reasons why you might not be able to join. For example, if you have certain other medical conditions (like Marfan syndrome or recent serious blood clots), or if you've had a bad reaction to similar drugs in the past. If you weigh more than 160 kg, or if you've previously taken pegtibatinase or similar study drugs, you wouldn't be able to participate. If you could become pregnant, you'll need a negative pregnancy test and to use effective contraception.
Könnte diese Studie für Sie geeignet sein?
Beantworten Sie diese kurzen Fragen, um zu sehen, ob Sie möglicherweise berechtigt sind. Dies ist nur ein Leitfaden – das Forschungsteam trifft die endgültige Entscheidung.
- Are you between 12 and 65 years old?
- Do you have a confirmed diagnosis of Homocystinuria (HCU)?
- Is your homocysteine level typically 50 µM or higher?
- Are you willing to stick to a stable diet and your current HCU treatments during the study?
- Have you NOT had a serious blood clot in the last 6 months?
- Have you NOT taken pegtibatinase or similar study drugs before?
Was beinhaltet die Teilnahme?
If you decide to take part, the study will last up to 38 weeks (about 9 months). It starts with a screening period of up to 10 weeks to check if you're suitable. This includes an initial check-up and a period of up to 6 weeks where your diet will be carefully monitored to make sure it's consistent. During this time, your regular HCU diet and treatments will be recorded.
After screening, you'll enter the 'blinded treatment period' for 24 weeks. This means you won't know if you're receiving pegtibatinase or the placebo, and neither will your study doctor. This helps to ensure the results are accurate. You'll have regular study visits, some at home and some possibly online, where your diet and HCU treatments will continue to be monitored. After your last dose of the study medicine, there will be a 4-week safety check-up, unless you choose to join a long-term follow-up study.
Potenzielle Risiken und Vorteile
Locations (52)
- Phoenix KinderkrankenhausVerifizierte PostleitzahlPhoenix, Vereinigte Staaten· Rekrutierung
- Yale University School of MedicineVerifizierte PostleitzahlNew Haven, Vereinigte Staaten· Rekrutierung
- Emory UniversityVerifizierte PostleitzahlAtlanta, Vereinigte Staaten· Rekrutierung
- Ann & Robert H. Lurie Kinderkrankenhaus von ChicagoVerifizierte PostleitzahlChicago, Vereinigte Staaten· Rekrutierung
- Maine Health - Maine Medical CenterVerifizierte PostleitzahlPortland, Vereinigte Staaten· Rekrutierung
- Uncommon CuresVerifizierte PostleitzahlChevy Chase, United States· Rekrutierung
- The Mount Sinai HospitalVerifizierte PostleitzahlNew York, Vereinigte Staaten· Rekrutierung
- Science 37 - Virtual SiteVerifizierte PostleitzahlMorrisville, United States· Rekrutierung
- Oregon Gesundheits- und WissenschaftsuniversitätVerifizierte PostleitzahlPortland, Vereinigte Staaten· Noch nicht rekrutiert
- Children's Hospital of PhiladelphiaVerifizierte PostleitzahlPhiladelphia, Vereinigte Staaten· Rekrutierung
- University of Pittsburgh Medical Center - Children's Hospital of PittsburghVerifizierte PostleitzahlPittsburgh, United States· Rekrutierung
- University of Texas Southwestern Medical CenterVerifizierte PostleitzahlDallas, Vereinigte Staaten· Rekrutierung
Häufige Fragen
What is Homocystinuria (HCU)?
HCU is a rare genetic condition where your body has trouble processing certain proteins, leading to a build-up of a harmful substance called homocysteine in your blood.
Was ist ein Placebo?
A placebo is a 'dummy' drug that looks exactly like the real medicine but doesn't contain any active ingredients. It's used to compare the new drug's effects accurately.
Will I know if I'm getting the real drug or the placebo?
No, during the 24-week treatment period, neither you nor your study doctor will know if you're receiving pegtibatinase or the placebo. This is called 'blinded' and helps ensure fair results.
Wie lange werde ich an der Studie teilnehmen?
The total study duration is up to 38 weeks, which includes screening, diet standardisation, and 24 weeks of active treatment.
Can I continue my usual HCU treatments during the study?
Yes, you will continue your standard HCU care, including your special diet and any other HCU medications, throughout the study.
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