Active, not recruitingPhase 4
Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy
Key facts
- Who runs it
- Massachusetts General Hospital
- Where
- Boston, United StatesSee the siteAlso in 1 other country, 1 site
- Trial code
- Status
- Active, not recruiting
- Phase
- Phase 4
- People taking part
- 24 people, planned
- Started
- 1 Apr 2023
- Ended
- 1 Sept 2026
- Type of study
- Interventional, 2 groupsOne group gets an existing treatment, for comparison.
- Treatments tested
- Pramipexole
- Placebo
- Record updated
- 1 July 2026
Who can take part
- Age
- 18 years to 75 years
- Sex
- Women only
- Healthy volunteers
- Cannot take part
The registry criteria, word for word
Show all 19 criteriaShow less
Who can take part
- 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)
Who cannot take part
- Participants with active or unstable major psychiatric disorder other than ALD, who, in the investigators' judgement, require further treatment
- Alcohol use disorder within the last 30 days
- History of being treated for restless legs syndrome, specifically with dopamine agonist medications
- Neurological disorder or cardiovascular disease raising safety concerns about use of pramipexole and/or judged to interfere with ability to assess efficacy of the treatment
- Renal disease judged to interfere with drug metabolism and excretion
- Inflammatory brain demyelination; PHASE 2 (CROSS-OVER STUDY)
- Pregnant. Research staff perform pregnancy tests upon visit to center
- Use of dopaminergic agonists or antagonists within the last 30 days
- Methamphetamine or benzodiazepine dependence in the last 30 days
- Medical instability considered to interfere with study procedures
Other criteria
- PHASE 1 (PREVALENCE STUDY)
Where in United States
Also in 1 other country, 1 site
- Massachusetts General HospitalBoston, United StatesNo longer taking part
About the trial
What the trial wants to find out
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.
The 2 groups in the trial
- PlaceboPlacebo, for comparison
Participants will be on the placebo arm for 2 months and will then cross over to the pramipexole arm for 2 months
- PramipexoleExisting treatment, for comparison
Participants will be on the pramipexole arm for 2 months and will then cross over to the placebo arm for 2 months
Data and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official title
- Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy: A Key to Improving Sleep and Gait Performance
- Record updated
- 1 July 2026