Status:
COMPLETED
SMART-ALD - A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD)
Lead Sponsor:
Leipzig University Medical Center
Collaborating Sponsors:
European Leukodystrophy Association
Conditions:
X-linked Adrenoleukodystrophy
Eligibility:
FEMALE
18+ years
Phase:
NA
Brief Summary
X-linked Adrenoleukodystrophy (X-ALD) is one of the most frequent inborn error of metabolism caused by mutations in the ABCD1 gene, which codes for the transporter of saturated very long-chain fatty a...
Eligibility Criteria
Inclusion
- Informed consent obtained from the patient or a competent guardian with legal capacity to execute a local Institutional Review Board/Independent Ethics Committee approved consent.
- Females ≥18 years at the time of consent, with proven X-ALD as defined by
- Elevated VLCFA values, or
- Mutation in ABCD1 gene
- Neurological symptoms as defined by Adult ALD Clinical Score (AACS) ≥ 2
Exclusion
- No informed consent and assent
- Any medical condition that may interfere with the study, e.g. severe liver, kidney, active infections or major heart diseases (\>New York Heart Association class II)
- Any clinically significant condition with an estimated life-expectancy of \<6 months
- Current pregnancy
Key Trial Info
Start Date :
May 20 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 1 2024
Estimated Enrollment :
68 Patients enrolled
Trial Details
Trial ID
NCT04687007
Start Date
May 20 2022
End Date
February 1 2024
Last Update
March 13 2024
Active Locations (2)
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1
Leipzig University Medical Center, Leukodystrophy Outpatient Clinic, Department of Neurology, Leipzig, Germany
Leipzig, Saxony, Germany, 04103
2
Leukodystrophy Outpatient Clinic
Leipzig, Germany, 04275