Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy: A Key to Improving Sleep and Gait Performance
Who is this study for? Women with x-linked adrenoleukodystrophy
What treatments are being studied? Pramipexole
Status: Recruiting
Location: See all (2) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 4
SUMMARY
The investigators recently observed that up to 25% of women with X-linked adrenoleukodystrophy (ALD) have moderate to severe Restless Leg Syndrome (RLS). In this study, the investigators aim to estimate the prevalence of RLS among women with ALD and to assess whether pramipexole improves RLS symptoms as well as sleep and gait measures in women with ALD.
Eligibility
Participation Requirements
Sex: Female
Minimum Age: 18
Maximum Age: 75
Healthy Volunteers: f
View:
• Women of any ethnic origin.
• Ability to provide verbal consent
• A willingness and ability to comply with study procedures.
• Age 18-75 years
• Metabolically or genetically confirmed diagnosis of ALD
• Participation in Phase 1
• Ability to provide written informed consent
• Women with ALD who have Restless Leg Syndrome (IRLS \> 15)
Locations
United States
Massachusetts
Massachusetts General Hospital
RECRUITING
Boston
Other Locations
Netherlands
University Medical Center of Amsterdam
RECRUITING
Amsterdam
Contact Information
Primary
Riya Saxena
rsaxena2@mgh.harvard.edu
603-674-6743
Time Frame
Start Date: 2023-04-01
Estimated Completion Date: 2025-11-01
Participants
Target number of participants: 24
Treatments
Placebo_comparator: Placebo
Participants will be on the placebo arm for 2 months and will then cross over to the pramipexole arm for 2 months
Active_comparator: Pramipexole
Participants will be on the pramipexole arm for 2 months and will then cross over to the placebo arm for 2 months
Related Therapeutic Areas
Sponsors
Collaborators: European Leukodystrophy Association
Leads: Massachusetts General Hospital