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Trial · NCT03864523

Registered outcomes56

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

ListOutcomeTime frameDescription
Primary2 Minute Walk Test (2MWT)24 monthsThe score at this test corresponds to the distance traveled by the patient during 2 minutes, on a flat surface
SecondaryTimed Up and Go (TUG) test24 monthsIt consists in standing up, walking 3 meters, turning around, walk back to the chair and sitting back down, at regular pace
SecondaryTime to walk 25 Feet (TW25)24 monthsIn this test the patient should walk 7.62 meters (25 feet) as quickly, but safely, as possible without running
Secondary6 Minute Walk Test (6MWT)24 monthsIt measures the distance an individual is able to walk over a total of six minutes on a hard, flat surface
SecondarySensory disturbances: tactile24 monthsFor the evaluation the Total Neuropathy Score (TNS) will be used (0-4)
SecondarySensory disturbances: painful24 monthsFor the evaluation the Total Neuropathy Score (TNS) will be used (0-4)
SecondarySensory disturbances: vibratory24 monthsFor the evaluation the Total Neuropathy Score (TNS) will be used (0-4)
SecondaryExpanded disability status scale (EDSS)24 monthsThis scale measures motor function, ranging from 0 (normal neurological examination) to 10 (death)
SecondaryDynamometer test (optional)24 monthsIt measures the muscle strength
SecondaryAshworth scale24 months
The Modified Ashworth Scale measures spasticity in patients who have lesions of the CNS or neurological disorders. The modified Ashworth scale ranges from 0 (no increase in tone) to 4 (Affected part(s) rigid in flexion or extension)
SecondarySF-Qualiveen24 monthsIt measures the impact of urinary disorders in patients with neurological conditions
SecondaryRevised Faecal Incontinence Scale (RFIS)24 months
The RFIS is a short, reliable and valid five item scale used to asses faecal incontinence and to monitor patient outcomes following treatment. Response options are framed as 5-point Likert-type scales, with 0 indicating no impact of faecal incontinence problems on health-related quality of life and 4 indicating a high adverse impact. The RFIS total score is calculated by adding a person's score for each question. Adding the score for each of the five questions results in a possible score range of 0-20
SecondaryConventional MRI24 months
FLAIR and T2 sequences may show subtle anomalies evaluated using the Loes scoring system. This MRI severity scale has been designed specifically for X-linked adrenoleukodystrophy and has been shown to correlate with severity of neurologic deficits and to be predictive of disease progression. Different brain regions are considered in the MRI severity score. Each area is scored as 0 if normal, 0.5 if unilateral involvement, and 1 if the lesion or atrophy is bilateral. The maximum severity score is 34; a score of 1 is considered abnormal.
SecondaryDiffusion tensor Imaging (DTI)24 monthsApparent diffusion coefficient (ADC) and fractional anisotropy (FA) will be measured
SecondaryBrain MRI spectroscopy (MRS)24 monthsNAA/creatine and choline/creatine ratios will be measured
SecondaryNerve conduction studies: conduction velocity in the peroneal nerve24 monthsm/s
SecondaryNerve conduction studies: amplitude of the signal in the peroneal motor nerve24 months(mV)
SecondaryNerve conduction studies: conduction velocity in the sura sensitive nerve24 months(m/s)
SecondaryNerve conduction studies: amplitude of the signal in the sura sensitive nerve24 months(μV)
SecondaryMotor Evoked Potentials (MEP): F wave24 months(ms) in right and left upper limb and right and left lower limb

Source ClinicalTrials.govtrials-2026-11-12-0.5.0

Sources 3

  • ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
  • ClinicalTrials.govNo license stated
  • NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
  • ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
  • ClinicalTrials.govnone stated
  • NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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