AN OPEN-LABEL, MULTICENTER STUDY IN MALE PEDIATRIC PATIENTS WITH CEREBRAL X-LINKED ADRENOLEUKODYSTROPHY (CALD) TO ASSESS THE EFFECTS OF MIN-102 TREATMENT ON DISEASE PROGRESSION PRIOR TO HUMAN STEM CELL TRANSPLANT (HSCT)

Conditions

CEREBRAL X-LINKED ADRENOLEUKODYSTROPHY (CALD)

Brief summary

The primary efficacy endpoint will be the number of patients meeting "arrested disease" criteria at Visit 12.

Detailed description

Sustained change from Baseline in the score composed of NFS items 1 (hearing/auditory processing), 2 (aphasia/apraxia), 4 (vision impairment), 10 (spastic gait) and 13 (incontinence)., Sustained change from Baseline in total NFS score “Sustained change” is defined as the same total score for NFS items 1, 2, 4, 10, and 13 and no change >1 in NFS total score observed in two the consecutive Visits 6–8, and Visits 11-12, respectively. If “sustained change” definition is not met, the average of the two scores of Visit 6 and 8, and Visit 11 and 12, respectively, will be used. If NFS total score differs by 1 point between V6-8, or V11-12, the higher of the two scores is used., Change from baseline in Loes MRI severity score., Change from baseline in Gadolinium Intensity Score (GIS)., Overall survival of patients who have not undergone HSCT., Number of patients meeting HSCT criteria

Related papers

No related papers found yet.

Interventions

None listed

Eligibility

Sex/Gender

Male

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
The primary efficacy endpoint will be the number of patients meeting "arrested disease" criteria at Visit 12.	—

Secondary

Measure	Time frame
Sustained change from Baseline in the score composed of NFS items 1 (hearing/auditory processing), 2 (aphasia/apraxia), 4 (vision impairment), 10 (spastic gait) and 13 (incontinence)., Sustained change from Baseline in total NFS score “Sustained change” is defined as the same total score for NFS items 1, 2, 4, 10, and 13 and no change >1 in NFS total score observed in two the consecutive Visits 6–8, and Visits 11-12, respectively. If “sustained change” definition is not met, the average of the two scores of Visit 6 and 8, and Visit 11 and 12, respectively, will be used. If NFS total score differs by 1 point between V6-8, or V11-12, the higher of the two scores is used., Change from baseline in Loes MRI severity score., Change from baseline in Gadolinium Intensity Score (GIS)., Overall survival of patients who have not undergone HSCT., Number of patients meeting HSCT criteria	—

Measure

Time frame

Sustained change from Baseline in the score composed of NFS items 1 (hearing/auditory processing), 2 (aphasia/apraxia), 4 (vision impairment), 10 (spastic gait) and 13 (incontinence)., Sustained change from Baseline in total NFS score “Sustained change” is defined as the same total score for NFS items 1, 2, 4, 10, and 13 and no change >1 in NFS total score observed in two the consecutive Visits 6–8, and Visits 11-12, respectively. If “sustained change” definition is not met, the average of the two scores of Visit 6 and 8, and Visit 11 and 12, respectively, will be used. If NFS total score differs by 1 point between V6-8, or V11-12, the higher of the two scores is used., Change from baseline in Loes MRI severity score., Change from baseline in Gadolinium Intensity Score (GIS)., Overall survival of patients who have not undergone HSCT., Number of patients meeting HSCT criteria

—

Countries

France, Germany, Spain

Outcome results