Pioglitazone 15 mg tablets 2/day during 2 years

Adrenomyeloneuropathy

X-linked Adrenoleukodystrophy

Pioglitazone

Bellvitge University Hospital

Donostia University Hospital

Effect of Pioglitazone Administered to Patients With Adrenomyeloneuropathy

Effect of Pioglitazone Administered to Patients With Adrenomyeloneuropathy: a Phase II, Single-arm, Multicentric Clinical Trial

The score at this test corresponds to the distance traveled by the patient during 2 minutes, on a flat surface

It consists in standing up, walking 3 meters, turning around, walk back to the chair and sitting back down, at regular pace

In this test the patient should walk 7.62 meters (25 feet) as quickly, but safely, as possible without running

It measures the distance an individual is able to walk over a total of six minutes on a hard, flat surface

For the evaluation the Total Neuropathy Score (TNS) will be used (0-4)

This scale measures motor function, ranging from 0 (normal neurological examination) to 10 (death)

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)

It measures the impact of urinary disorders in patients with neurological conditions

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

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.

Apparent diffusion coefficient (ADC) and fractional anisotropy (FA) will be measured

NAA/creatine and choline/creatine ratios will be measured

(ms) in right and left upper limb and right and left lower limb

(μV) in right and left upper limb and right and left lower limb

Glutamic semialdehyde (GSA) will be measured in plasma. Results will be expressed in μmol/mol lysine

Carboxyethyl-lysine (CEL) will be measured in plasma. Results will be expressed in μmol/mol lysine

N2-malondialdehyde-lysine (MDAL) will be measured in plasma. Results will be expressed in μmol/mol lysine

N2-carboxymethyl-lysine (CML) will be measured in plasma. Results will be expressed in μmol/mol lysine

7,8-dihydro-8-oxo-2-deoxyguanosine (8-oxoDG) will be measured in urine. Results will be expressed in ng/mg creatine

HGF will be measured in plasma. Results will be expressed in pg/ml

IL6 will be measured in plasma. Results will be expressed in pg/ml

IL8 will be measured in plasma. Results will be expressed in pg/ml

MCP-1 will be measured in plasma. Results will be expressed in pg/ml

NGF will be measured in plasma. Results will be expressed in pg/ml

TNF will be measured in plasma. Results will be expressed in pg/ml

Adiponectin will be measured in plasma. Results will be expressed in μg/ml

CCR3 will be measured in RNA from peripheral mononuclear cells. Results will be expressed as relative gene expression

CXCL5 will be measured in RNA from peripheral mononuclear cells. Results will be expressed as relative gene expression

CXCL9 will be measured in RNA from peripheral mononuclear cells. Results will be expressed as relative gene expression

IL9R will be measured in RNA from peripheral mononuclear cells. Results will be expressed as relative gene expression

PPARd will be measured in RNA from peripheral mononuclear cells. Results will be expressed as relative gene expression

GPX4 will be measured in RNA from peripheral mononuclear cells. Results will be expressed as relative gene expression

STAT1 will be measured in RNA from peripheral mononuclear cells. Results will be expressed as relative gene expression

pyruvate kinase/atrophy

Effect of Pioglitazone Administered to Patients With Adrenomyeloneuropathy

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