Treating Leg Symptoms in Women with X-linked Adrenoleukodystrophy: A Key to Improving Sleep and Gait Performance

Phase 4

Conditions: restless legs syndrome
x-gebonden adrenoleukodystrofie
10029299
10034606

Registration Number: NL-OMON52216

Lead Sponsor: Academisch Medisch Centrum

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Pending

Sex: Not specified

Target Recruitment: 50

Inclusion Criteria

PHASE 1 (PREVALENCE STUDY)
Inclusion Criteria:
- 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

PHASE 2 (CROSS-OVER STUDY)
Inclusion Criteria:
- Participation in Phase 1
- Ability to provide written informed consent
- 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
10. Patients who are deemed a fall risk as determined by the PI

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Main study parameters/endpoints:<br /><br>IRLS Score - The International Restless Legs Severity Scale (IRLS) is a<br /><br>severity rating scale developed by the International Restless Legs Syndrome<br /><br>Study Group. This scale consists of 10 items that evaluate RLS symptoms, sleep<br /><br>disturbance from RLS, impact of RLS on daily activities, and mood disturbance<br /><br>resulting from RLS symptoms. The scores for the 10 items are summed to produce<br /><br>a total score, which ranges from 0 (no severity) to 40 (most severe). </p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Exploratory Aims (PHASE 2):<br /><br>- To determine whether pramipexole will have improved self-reported and<br /><br>objective sleep metrics (e.g., quality, disturbance, sleep latency and<br /><br>duration, and periodic limb movements of sleep) in women with ALD.<br /><br>- To determine whether pramipexole will improve measures of walking and motor<br /><br>performance (25 feet walk test as well as the timed up and go test).</p><br>