Phase 3
N=67
A Trial of Levodopa in Angelman Syndrome
Angelman Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT01281475 ↗Enrolled (actual)
67
Serious AEs
6.0%
Results posted
Jan 2017
Primary outcome: Primary: Bayley Cognitive Age Equivalent at 1 Year — 19; 19 months — p=0.75
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Levodopa (Drug); Placebo Oral Capsule (Drug)
- Age
- Pediatric · 4+ yrs
- Sex
- All
- Sponsor
- Wen-Hann Tan
- Primary completion
- Jul 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Bayley Cognitive Age Equivalent at 1 Year |
19; 19 | 0.75 |
| SECONDARY Presence of Tremors |
14; 12 | — |
Summary
This study is designed to determine whether levodopa will lead to an improvement in the development and tremor in children with Angelman syndrome (AS).
It has been suggested that levodopa, a medication that is usually used to treat Parkinson disease in adults, may help children with AS in their overall development and reduce the tremor that some of them have.
If levodopa is found to be beneficial for children with AS, this could lead to a new treatment for AS.
Funding Source - FDA-OOPD
Eligibility Criteria
Inclusion Criteria
- Age between 4 years and 12 years (i.e., before the 13th birthday)
- Molecular confirmation of the diagnosis of AS, which may include abnormal methylation studies or UBE3A mutation analyses - only subjects with a molecular diagnosis will be allowed to enroll
- Not on LD, CD, or any dopamine agonists in the 2 weeks prior to participation
Exclusion Criteria
- Co-morbid disorders that may be associated with developmental or cognitive delays
- Poorly controlled seizures - An average of more than 2 clinical seizures per month in the 12 months prior to enrollment.
- Use of medications that may interact with LD/CD including atypical antipsychotics (aripiprazole, asenapine, iloperidone, olanzapine, paliperidone, risperidone, ziprasidone), monoamine oxidase inhibitors (isocarboxazid, phenelzine, selegiline, tranylcypromine), or phenytoin within the last 14 days, or other investigational interventions within the past 3 months
- Presence of cardiovascular disease or instability, respiratory disease, liver disease, peptic ulcer disease, renal impairment, or hematological disorders
- Pregnancy
Data sourced from ClinicalTrials.gov (NCT01281475). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.