A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Dyskinesia Due to Cerebral Palsy
This research is a major study (Phase 3) investigating a new potential medicine called Valbenazine for adults who experience involuntary movements due to cerebral palsy. These uncontrolled movements are known as dyskinesia. The study wants to understand if Valbenazine can help reduce these movements and if it is safe and well-tolerated. It's designed as a 'double-blind' study, meaning neither the patient nor their doctor will know if they are receiving Valbenazine or a 'dummy' medicine (placebo). This helps ensure the results are as unbiased as possible. Researchers will carefully measure changes in movement over several weeks to see how effective the medicine is.
At a glance
What is this study about?
This clinical trial is exploring a new medicine named Valbenazine for adults who have dyskinesia, which are uncontrolled and often jerky movements, caused by cerebral palsy. Cerebral palsy affects movement and muscle coordination, and in some people, it can lead to these involuntary movements.
The main goal of this study is to see how well Valbenazine works at reducing these uncontrolled movements and to make sure it's safe for people to use. It's a 'Phase 3' study, which means it’s one of the final steps before a new medicine might be approved for wider use. Researchers are comparing Valbenazine to a 'placebo,' which is a dummy pill that looks just like the study medicine but contains no active drug. This comparison helps them understand if any improvements are truly due to Valbenazine or if they might happen naturally or from other factors.
After several weeks, doctors will assess how participants' movements have changed using special scales and tools. They will also collect information on how participants, their caregivers, and doctors feel about any improvements. This detailed approach helps the researchers get a very clear picture of the medicine's potential benefits and side effects.
Key takeaways
- A study for adults with involuntary movements (dyskinesia) due to cerebral palsy.
- Testing a new medicine called Valbenazine against a dummy medicine (placebo).
- Aims to see if Valbenazine reduces uncontrolled movements and is safe.
- Participation involves regular clinic visits and assessments over about 14 weeks.
- Results will help understand if this new medicine could be a future treatment option.
Who may be eligible?
This study is looking for adults aged 18 and over who have uncontrolled movements (dyskinesia) because of cerebral palsy. Both men and women are welcome to participate.
To join, you would need to have specific types of involuntary movements that can be measured by the study doctors. There might be some other health conditions or medicines that would prevent you from taking part, to ensure your safety and the accuracy of the trial results. Your doctor will be able to check all the specific requirements.
It's important that you and your doctor feel this study is a good fit for you. Clinical trials follow very strict rules about who can take part to make sure the study is as safe and helpful as possible.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you 18 years old or older?
- Do you have uncontrolled movements (dyskinesia) caused by cerebral palsy?
- Are you able to attend regular clinic appointments?
- Are you willing to take study medication as directed?
- Are you comfortable with the possibility of receiving a dummy medicine?
What does participation involve?
If you join this study, you would receive either the study medicine, Valbenazine, or a matching dummy medicine (placebo) for a set period. Neither you nor your study doctor would know which one you are taking. You would have several visits to the clinic for assessments, which might include answering questions about your condition, having physical examinations, and possibly completing questionnaires about your movements and general well-being.
These visits would track your progress over approximately 14 weeks. During this time, your movements would be carefully assessed at different points to see if there are any changes. You would receive regular check-ups to monitor your health and any side effects. You are always free to leave the study at any time, for any reason.
Potential risks and benefits
Locations (5)
- —UnverifiedPortugal
- —UnverifiedBelgium
- —UnverifiedSpain
- —UnverifiedItaly
- —UnverifiedPoland
Common questions
What is dyskinesia?
Dyskinesia refers to uncontrolled, involuntary movements that can affect different parts of the body.
What is cerebral palsy?
Cerebral palsy is a condition that affects movement and muscle tone or posture, caused by damage to the developing brain.
What does 'double-blind' mean?
It means neither you nor your doctor will know if you're taking the study medicine or the dummy medicine (placebo).
How long will the study last?
The main part of the study involves assessments up to about 14 weeks.
Will I get the new medicine for sure?
No, because it’s a blind study, you might receive the new medicine or a dummy medicine called a placebo.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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