Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy

Launched by MASSACHUSETTS GENERAL HOSPITAL · Aug 5, 2021

Keywords

ClinConnect Summary

This clinical trial is looking at how common Restless Leg Syndrome (RLS) is in women who have a condition called X-linked adrenoleukodystrophy (ALD), and whether a medication called pramipexole can help improve their symptoms. RLS is a condition that causes uncomfortable sensations in the legs, often leading to trouble sleeping. The study aims to understand how many women with ALD experience RLS and to see if pramipexole can help with both RLS symptoms and overall sleep quality.

To participate in this trial, women aged 18 to 75 who have been diagnosed with ALD are eligible, provided they can give consent and follow the study rules. Participants will first take part in a study to assess the prevalence of RLS and then, if they qualify, may receive pramipexole to see if it helps their symptoms. It's important to note that women who are pregnant or have certain medical or psychiatric conditions might not be able to join. Overall, this trial hopes to find better ways to manage leg symptoms and improve the quality of life for women living with ALD.

Gender

FEMALE

Eligibility 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