NV1205 in Pediatric Male Subjects with CCALD
Research type
Research Study
Full title
Phase 1, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics, and Pharmacodynamics of NV1205 in Pediatric Male Subjects with Childhood Cerebral Adrenoleukodystrophy (CCALD)
IRAS ID
235852
Contact name
James Davison
Contact email
Sponsor organisation
NeuroVia Inc.
Eudract number
2017-001684-21
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 0 months, 30 days
Research summary
Childhood Cerebral Adrenoleukodystrophy (CCALD) is the most severe form of X-Linked Adrenoleukodystrophy (X-ALD). X-ALD is a genetic disorder caused by a change in a gene called ABCD1. ABCD1 allows the body to handle the blood fat levels normally by making a substance called ALDP. The change in this gene means that certain fatty substances build-up in the blood. These fatty substances are known as very long chain fatty acids (VLCFA). These VLCFA can damage the nervous system and adrenal glands.
There is another gene called ABCD2, which works in a similar way to ABCD1. It makes a substance called ALDRP, which works like ALDP. The study drug NV1205 works by increasing the amount of ALDRP in the body. This helps the body to manage the blood fat levels properly, which might lower the levels of VLCFA in certain tissues in the body such as the brain.
Eligible subjects are males between 4 and less than 18 years of age who have a confirmed diagnosis of CCALD.
A minimum of 3 subjects will be enrolled in each Cohort. Three initial cohorts will be enrolled with a total of 9 subjects. Additional cohorts up to a total 25 subjects may be enrolled as recommended by the DSMB.
Initially in the UK the study will be performed at 2 specialist paediatric hospitals.
The study is broken down into two parts. Part 1 lasts for three months and involves seven visits to the research centre and one phone call. Part 2 continues for another 21 months and involves nine visits. There are also dose adjustment visits which may be done at any time if required. In total, each participant will be in the study for 24 months (2 years), and will visit the hospital 16 times.
REC name
London - Brent Research Ethics Committee
REC reference
18/LO/0068
Date of REC Opinion
20 Apr 2018
REC opinion
Further Information Favourable Opinion