Safety, Tolerability, PK, and PD Study of INZ-701 in adults with PXE
Research type
Research Study
Full title
A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 Followed by an Open-Label Long-Term Extension Period in Adults with ABCC6 Deficiency Manifesting as Pseudoxanthoma elasticum (PXE)
IRAS ID
301575
Contact name
Jorg Taubel
Contact email
Sponsor organisation
Inozyme Pharma, Inc
Eudract number
2020-004000-33
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
US IND Number, 156761
Duration of Study in the UK
0 years, 4 months, 16 days
Research summary
Richmond Pharmacology are to conduct a clinical trial with an experimental (not yet marketed) study drug called INZ-701. The proposed study is a Phase 1/2, open-label (no placebo), first-in-human (FIH), first-in-patient (FIP), multiple ascending dose (MAD), dose-finding study in adult subjects (18 to <65 years of age) with ABCC6 Deficiency.
ABCC6 deficiency is a rare, genetic disorder manifesting as Pseudoxanthoma Elasticum (PXE). PXE causes accumulation of calcium in abnormal elastic fibers in the skin, blood vessels, eyes, and heart, resulting in skin lesions, cardiovascular complications, blindness and reduced mobility. PXE is caused by mutation in the ABCC6 gene that produces a dysfunctional ABCC6 enzyme, leading to low pyrophosphate levels in the blood. INZ701 is designed to increase the amount of ENPP1, an enzyme involved in pyrophosphate upregulation. By restoring normal enzymatic activity, it is hoped that this will overcome the low pyrophosphate levels found in patients with ABCC6 deficiency and relieve their symptoms.
The aim of the trial is to assess the safety, tolerability, immunogenicity (ability to cause an immune response), pharmacokinetics (PK; means the effects of drugs and how they interact in the body) and pharmacodynamics (the effects of the drug) of multiple subcutaneous doses of INZ-701 in patients with ABCC6 Deficiency. This is to be a multicenter, multinational trial to be conducted in the USA and UK, and possibly France and Netherlands. Approximately 9 patients with ABCC6 deficiency will be enrolled in this study; however, the study may be expanded to a total of 24 patients.
This study is different from the regular medical care. The purpose of regular medical care is to improve or manage health; however, the purpose of research is to gather information to advance science and medicine and possibly develop a treatment for ABCC6 Deficiency, and does not replace the regular medical care.
REC name
South Central - Berkshire B Research Ethics Committee
REC reference
21/SC/0305
Date of REC Opinion
17 Dec 2021
REC opinion
Further Information Favourable Opinion