ADAPT
Research type
Research Study
Full title
The ADAPT Study: An Open-label, Long-term Safety Study of INZ-701 in Patients with ENPP1 Deficiency and ABCC6 Deficiency
IRAS ID
1009552
Contact name
Elizabeth Alessi
Contact email
Sponsor organisation
Inozyme Pharma Inc.
Eudract number
2024-512715-42
Research summary
ENPP1 deficiency is a rare, genetic disorder caused by a change in the ENPP1 gene. This gene provides instructions for making the ENPP1 enzyme, a kind of protein. The ENPP1 enzyme is needed to prevent calcium phosphate compound from being deposited in wrong parts of the body including in ligaments and blood vessels. This condition can result in heart attacks, stroke or cardiac or multiorgan failure for children. Children experience rickets, while adults experience hearing loss, arterial calcification and cardiac and/or neurological involvement.
ABCC6 deficiency is caused by mutations in the ABCC6 gene, responsible for making ABCC6 protein. ABCC6 works together with the enzyme ENPP1 to keep inorganic pyrophosphate levels in the blood from falling too low. The deficiency affects the skin, eyes, vascular system.
This study is sponsored by Inozyme Pharma Inc and will take place at one site in UK. The purpose of this study is to assess long-term safety and efficacy of INZ-701 (investigational drug) to treat the mentioned deficiencies.
The participants who completed previous studies in ENPP1/ABCC6 deficiencies may be eligible for participation in this study and may continue until INZ-701 is commercially available in their country or until Inozyme chooses to discontinue development.
Participants will receive an injection of INZ-701 weekly. During the study, participants are expected to visit the research site while having an option to perform some activities remotely, under supervision by the study doctor (as applicable). For 1st month (1m), 5 visits are planned (with 2 being mandatory in clinic), after which the participants will have follow up visits every 3m with a visit to the clinic every 6m until the end of the study.
Additional research or study-specific assessments/procedures, such as blood draws will also occur during the visits, as applicable. After the study completion participants will have a safety follow-up 30d after the last visit.REC name
London - Chelsea Research Ethics Committee
REC reference
24/LO/0369
Date of REC Opinion
15 Jul 2024
REC opinion
Further Information Favourable Opinion