Overview

The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency

Status:
Not yet recruiting

Trial end date:
2025-12-01

Target enrollment:
0

Participant gender:
All

Summary

The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency.

Phase:

Phase 1

Accepts Healthy Volunteers?

Details

Lead Sponsor:

Inozyme Pharma

Criteria

Inclusion Criteria:

1. Caregiver(s) must provide written or electronic consent after the nature of the study
has been explained, and prior to any research-related procedures, per International
Conference on Harmonisation (ICH) Good Clinical Practice (GCP).

2. Subject must have a post-natal confirmed molecular genetic diagnosis of ENPP1
Deficiency with biallelic mutations (ie, homozygous or compound heterozygous)
performed by a College of American Pathologists/Clinical Laboratory Improvement
Amendments (CAP/CLIA) certified laboratory or equivalent.

3. Subject must be male or female ages ≥ 1 month to <1 year of age at Baseline

4. Subject must weigh ≥ 0.5 kg at the time of the first dose of INZ-701

5. In the opinion of the Investigator, the subject must be able to complete all aspects
of the study

6. Subject's caregiver(s) must agree to provide access to their child's relevant medical
records

Exclusion Criteria:

1. In the opinion of the Investigator, presence of any clinically significant disease or
laboratory abnormality (outside of those considered associated with the diagnosis of
ENPP1 Deficiency) that precludes study participation or may confound interpretation of
study results, including known uncontrolled thyroid disease or unrelated connective
tissue, bone, mineral, or muscle disease

2. Care has been withdrawn or subject is receiving end of life care or hospice only

3. Known malignancy

4. Known intolerance to INZ-701 or any of its excipients

5. Concurrent participation in another non-Inozyme interventional study and/or receipt of
any other investigational new drug within 5 half-lives of the last dose of the other
investigational product or from 4 weeks prior to the first dose of INZ-701, whichever
is longer, or use of an investigational device, through completion of participation in
the study