Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy

• Status: Not yet recruiting
• Trial end date: 2022-04-01
• Target enrollment: 0
• Participant gender: Female

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.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Massachusetts General Hospital
• Collaborator: European Leukodystrophy Association
• Treatments: Pramipexole

Criteria

PHASE 1 (PREVALENCE STUDY)

Inclusion Criteria:

1. Women of any ethnic origin.

2. Ability to provide verbal consent

3. A willingness and ability to comply with study procedures.

4. Age 18-75 years

5. Metabolically or genetically confirmed diagnosis of ALD

Exclusion Criteria:

1. Inflammatory brain demyelination

PHASE 2 (CROSS-OVER STUDY)

Inclusion Criteria:

1. Participation in Phase 1

2. Ability to provide written informed consent

3. Women with ALD who have Restless Leg Syndrome (IRLS > 15)

Exclusion Criteria:

1. Pregnant. Research staff perform pregnancy tests upon visit to center.

2. Participants with active or unstable major psychiatric disorder other than ALD, who, in the investigators' judgement, require further treatment

3. Use of dopaminergic agonists or antagonists within the last 30 days

4. Alcohol use disorder within the last 30 days

5. History of being treated for restless legs syndrome, specifically with dopamine agonist medications

6. Methamphetamine or benzodiazepine dependence in the last 30 days

7. 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

8. Medical instability considered to interfere with study procedures

9. Renal disease judged to interfere with drug metabolism and excretion