What side effects are associated with this type of intervention?

Common treatments for Adrenoleukodystrophy (ALD) include dietary management, such as Lorenzo's oil, and medications like corticosteroids for adrenal insufficiency. Dopamine agonists like Pramipexole, used for treating Restless Leg Syndrome in ALD, can cause side effects such as nausea, dizziness, somnolence, and orthostatic hypotension. Other treatments may include hematopoietic stem cell transplantation, which carries risks like graft-versus-host disease and infections.

What prior FDA approvals exist?

The provided context does not mention specific FDA-approved treatments for Adrenoleukodystrophy (ALD) or treatments similar to Pramipexole, a dopamine agonist, for ALD. However, it does note that up to 25% of women with ALD have moderate to severe Restless Leg Syndrome (RLS) and that pramipexole is being studied to improve RLS symptoms, sleep, and gait measures in these patients. For a comprehensive treatment plan, patients should consult their healthcare provider.

What other types of research exist?

For Adrenoleukodystrophy patients considering treatment in 2024, promising novel alternatives to Pramipexole (Dopamine Agonist) include Sodium Phenylbutyrate/Taurursodiol, which has shown efficacy in neurodegenerative conditions like Amyotrophic Lateral Sclerosis (ALS). Additionally, therapies targeting neurotrophic factors and antisense oligonucleotides, such as those used in ALS and Huntington's disease, may offer potential benefits. Consultation with a healthcare provider is essential to tailor treatment to individual patient needs.

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Dopamine Agonist

Pramipexole for Restless Legs Syndrome in ALD

Phase 4

Recruiting

Led By Florian S Eichler, MD

Research Sponsored by Massachusetts General Hospital

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age 18-75 years

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 8 and week 17

Awards & highlights

Study Summary

This trial will study whether a medication called pramipexole can help reduce symptoms of restless leg syndrome (RLS) in women with a rare disorder called X-linked adrenoleukodystrophy (ALD).

Who is the study for?

This trial is for women aged 18-75 with X-linked adrenoleukodystrophy (ALD) who suffer from moderate to severe Restless Leg Syndrome (RLS). Participants must have a confirmed diagnosis of ALD, be able to consent, and comply with study procedures. Exclusions include recent alcohol or drug dependence, pregnancy, prior RLS treatment with dopamine agonists, certain medical conditions like renal disease or inflammatory brain issues, and use of dopaminergic drugs within the last month.Check my eligibility

What is being tested?

The study aims to determine how common RLS is in women with ALD and if pramipexole can improve their symptoms. It will also look at sleep quality and walking ability. Women participating will either receive pramipexole or a placebo without knowing which one they are taking.See study design

What are the potential side effects?

Pramipexole may cause dizziness, nausea, sleepiness during daytime activities; it might also lead to sudden onset of sleep which could be dangerous while driving or doing other tasks that require alertness.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 18 and 75 years old.

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I am a woman with ALD and have severe restless leg syndrome.

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I have been diagnosed with ALD through genetic or metabolic testing.

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I can give my verbal consent.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ pre-intervention

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~pre-intervention

This trial's timeline: 3 weeks for screening, Varies for treatment, and pre-intervention for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change in the International Restless Legs Severity (IRLS) score

Prevalence of Restless Leg Syndrome in Women with X-linked Adrenoleukodystrophy

Secondary outcome measures

Change in quality of sleep and leg movements per hour of sleep measured by Polysomnography

Change in sleep/wake parameters measured by actigraphy

Change in the 13-item Spasticity Screening Tool score

+10 more

Trial Design

2Treatment groups

Active Control

Placebo Group

Group I: PramipexoleActive Control1 Intervention

Participants will be on the pramipexole arm for 2 months and will then cross over to the placebo arm for 2 months

Group II: PlaceboPlacebo Group1 Intervention

Participants will be on the placebo arm for 2 months and will then cross over to the pramipexole arm for 2 months

0 / 4Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Lorenzo's oil works by reducing the levels of very long-chain fatty acids (VLCFAs) in the body, which are elevated in Adrenoleukodystrophy (ALD) and contribute to the disease's progression. By lowering VLCFA levels, Lorenzo's oil aims to prevent or delay the onset of neurological symptoms in asymptomatic boys and slow the progression of adrenomyeloneuropathy in patients without cerebral involvement. Pramipexole, a dopamine agonist, is being studied for its potential to alleviate Restless Leg Syndrome (RLS) symptoms in women with ALD. Dopamine agonists work by stimulating dopamine receptors in the brain, which can help improve motor control and reduce symptoms of RLS. These treatments are important for ALD patients as they target specific symptoms and biochemical abnormalities associated with the disease, potentially improving quality of life and slowing disease progression.

Recent Advancements in the Diagnosis and Treatment of Leukodystrophies."Lorenzo's oil" therapy for X-linked adrenoleukodystrophy: rationale and current assessment of efficacy.