INZ-701 for infants with ENPP1 deficiency and GACI
The ENERGY 2 Study: An Open-Label Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Infants With Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency
This study will try the drug INZ-701 in infants under 1 year with ENPP1 deficiency to see if it safely reduces blood vessel calcification and related complications.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 0 Years to 1 Year |
| Sex | All |
| Sponsor | Inozyme Pharma Industry-sponsored |
| Locations | 8 sites (Rio de Janeiro and 7 other locations) |
| Trial ID | NCT07473973 on ClinicalTrials.gov |
What this trial studies
ENERGY 2 is a multicenter, single-arm, open-label Phase 3 trial administering INZ-701 to infants with genetically confirmed ENPP1 deficiency and clinical features of generalized arterial calcification of infancy. After up to a 60-day screening period, enrolled infants receive 52 weeks of treatment with the option for a 52-week extension, and an end-of-treatment visit 30 days after the last dose. The protocol focuses on measures of vascular calcification, cardiac outcomes, safety, and developmental status, with regular site visits for dosing and monitoring. Sites include pediatric centers in Brazil, France, and Hungary and the sponsor collaborators include Inozyme Pharma and BioMarin Pharmaceutical.
Who should consider this trial
Good fit: Infants aged one year or younger with a genetically confirmed ENPP1 deficiency and clinical signs of GACI who are medically stable and whose parent or guardian can give informed consent are ideal candidates.
Not a fit: Infants receiving end-of-life or hospice care, those who previously received INZ-701 outside approved access, those enrolled in another interventional trial, or those planning major surgery during the treatment period are not eligible and would not benefit from participation.
Why it matters
Potential benefit: If successful, INZ-701 could reduce vascular calcification, lower the risk of cardiac complications, and improve growth and long-term outcomes for affected infants.
How similar studies have performed: Early-phase trials and compassionate-use reports of ENPP1 replacement approaches have shown promising biological activity and case-level improvements in calcification, but robust Phase 3 evidence is still limited.
Eligibility criteria
Show full inclusion / exclusion criteria
Participants must meet all of the following: Inclusion Criteria: 1. Infant aged ≤ 1 year at the time of enrollment. 2. Confirmed diagnosis of ENPP1 deficiency, based on genetic testing. 3. Clinical features consistent with generalized arterial calcification of infancy (GACI) (e.g., vascular calcification or cardiac involvement). 4. Medically stable to participate in a 52-week treatment study. 5. Written informed consent provided by a parent or legal guardian. Exclusion Criteria Participants will not be eligible if any of the following apply: 1. Receiving end-of-life or hospice care. 2. Prior treatment with INZ-701, unless received through an approved expanded access program. 3. Concurrent participation in another interventional clinical trial. 4. Planned major surgery during the study period that would interfere with study participation.
Where this trial is running
Rio de Janeiro and 7 other locations
- Hospital Universitario Pedro Ernesto/Rio de Janeiro — Rio de Janeiro, Brazil (Recruiting)
- Hopital Necker - Enfants Malades — Paris, France (Recruiting)
- Gyermekgyogyaszat, DE — Debrecen, Hungary (Recruiting)
- Azienda Ospedaliera Universitaria Meyer — Florence, Italy (Recruiting)
- King Faisal Specialist Hospital and Research Center — Riyadh, Saudi Arabia (Recruiting)
- Hospital Sant Joan de Deu Edificio Consultas Externas. Unitat de Recerca — Barcelona, Esplugues de Llobregat, Spain (Recruiting)
- Umraniye Traiing and Research Hospital — Istanbul, Turkey (Türkiye) (Recruiting)
- Royal Manchester Children's Hospital — Manchester, United Kingdom (Recruiting)
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.