SMART-ALD – A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD)

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Brief Title

SMART-ALD - A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD)

Official Title

SMART-ALD - A New Lifestyle Intervention to Improve Physical and Mental Well-being and Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD)

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 acids (VLCFA) for peroxisomal degradation, thus causing their toxic
      accumulation in body fluids and tissues. The clinical spectrum ranges from adrenal
      insufficiency without neurological symptoms to a rapidly progressive, fatal cerebral
      demyelinating disease that may occur in childhood as well as later in life. The most frequent
      phenotype in adulthood is adrenomyeloneuropathy (AMN), a slowly progressive myelopathy and
      peripheral neuropathy, which may also be prevalent in up to 80% of females carrying the X-ALD
      gene defect. Since signs and symptoms in females are usually milder and with a later onset,
      they are frequently underestimated, overlooked or misinterpreted, e.g. as Multiple Sclerosis.
      Consequently, many women with X-ALD do not receive adequate treatment.

      Against this background, the development of new therapeutic interventions with the help of
      eHealth technology (e.g., counselling and treatment via digital communication tools) is of
      particular relevance, as it provides cost-effective, regular care even for patients who live
      remote from Leukodystrophy clinics.

      The aims of this study is to evaluate the effectiveness of a multi-approach intervention
      ("SMART-ALD") on physical and mental well-being and quality of life in n=30 X-ALD symptomatic
      heterozygous females compared to a waiting list control group (n=30) using electronic health
      (ehealth) technology.
    



Study Type

Interventional


Primary Outcome

Changes in self-reported quality of life (assessed via Short Form Health Survey, SF-36; range: 0-100, with higher scores indicating better quality of life) 6 months after randomization (=after 6 months SMART-ALD) in the EG compared to waiting list CG

Secondary Outcome

 Changes in objectively measured neurological symptoms (Adult Adrenoleukodystrophy Clinical Score, AACS; range: 0-24, with higher scores indicating greater impairment) 6 and 12 months after randomization in the EG compared to waiting list CG

Condition

X-linked Adrenoleukodystrophy

Intervention

SMART-ALD

Study Arms / Comparison Groups

 SMART-ALD - Experimental group (EG)
Description:  Participants will receive 12 months of SMART-ALD intervention

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Behavioral

Estimated Enrollment

60

Start Date

June 2021

Completion Date

May 2023

Primary Completion Date

May 2023

Eligibility Criteria

        Inclusion Criteria:

          -  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

               1. Elevated VLCFA values, or

               2. Mutation in ABCD1 gene

          -  Neurological symptoms as defined by Adult ALD Clinical Score (AACS) ≥ 2

        Exclusion Criteria:

          -  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
      

Gender

Female

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Wolfgang Köhler, MD, +49-341-9720086, [email protected]

Location Countries

Germany

Location Countries

Germany

Administrative Informations


NCT ID

NCT04687007

Organization ID

ELA FOA2020-002


Responsible Party

Sponsor

Study Sponsor

Leipzig University Medical Center

Collaborators

 European Leukodystrophy Association

Study Sponsor

Wolfgang Köhler, MD, Principal Investigator, Leipzig University Medical Center, Leukodystrophy Outpatient Clinic, Department of Neurology, Leipzig, Germany


Verification Date

December 2020