Evaluating the safety of INZ-701 in infants with specific genetic deficiencies
The ENERGY Study: An Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Infants With Ectonucleotide Pyrophosphatase/ Phosphodiesterase 1 (ENPP1) Deficiency or ATP-binding Cassette Sub-family C Member 6 (ABCC6) Deficiency
This study is testing a new enzyme replacement therapy called INZ-701 to see if it is safe for infants with specific genetic deficiencies.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 16 (estimated) |
| Ages | N/A to 1 Year |
| Sex | All |
| Sponsor | Inozyme Pharma Industry-sponsored |
| Locations | 7 sites (San Diego, California and 6 other locations) |
| Trial ID | NCT05734196 on ClinicalTrials.gov |
What this trial studies
The ENERGY study is a Phase 1b, open-label clinical trial designed to assess the safety and tolerability of INZ-701, an enzyme replacement therapy, in infants diagnosed with ENPP1 Deficiency or ABCC6 Deficiency. The study includes a 60-day screening period followed by a 52-week treatment phase where participants will receive INZ-701. Participants will be monitored for pharmacokinetics and pharmacodynamics, and an extension period may allow continued treatment until the therapy is commercially available. Follow-up will occur quarterly for survival outcomes after treatment discontinuation.
Who should consider this trial
Good fit: Ideal candidates for this study are infants under one year of age with a confirmed genetic diagnosis of ENPP1 or ABCC6 Deficiency.
Not a fit: Patients who do not have a confirmed diagnosis of ENPP1 or ABCC6 Deficiency or are older than one year will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new treatment option for infants suffering from rare genetic disorders associated with severe health complications.
How similar studies have performed: While this approach is novel for these specific deficiencies, similar enzyme replacement therapies have shown success in treating other genetic disorders.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Infant aged ≤ 1 year at the time of enrollment 2. Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency 3. Study participants must have clinical manifestations of generalized arterial calcification of infancy (GACI) or GACI-2, which must include at least one of the following: ectopic calcification, heart failure, respiratory distress, edema, cyanosis, hypertension, and cardiomegaly. 4. Study participant must weigh ≥0.5 kg at the time of the first dose of INZ-701 in this study 5. Written informed consent provided by a parent or legal guardian Exclusion Criteria: 1. In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality that precludes study participation or may confound interpretation of study result 2. Receiving end of life or hospice care 3. Known malignancy 4. Concurrent participation in another non-Inozyme interventional study 5. Treatment with any non-Inozyme product or investigational device during study participation
Where this trial is running
San Diego, California and 6 other locations
- Rady Children's Hospital — San Diego, California, United States (Recruiting)
- Boston Children's Hospital — Boston, Massachusetts, United States (Withdrawn)
- Nationwide Children's Hospital — Columbus, Ohio, United States (Recruiting)
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (Recruiting)
- The University of Utah — Salt Lake City, Utah, United States (Withdrawn)
- Hospital Sant Joan de Déu — Barcelona, Spain (Terminated)
- Royal Manchester Children's Hospital — Manchester, United Kingdom (Terminated)
Study contacts
- Study coordinator: Trial Specialist
- Email: medinfo@bmrn.com
- Phone: +1 800-983-4587
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.