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
Status: Recruiting
Location: See all (8) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
The primary purpose of ENERGY 2 (Study INZ701-105) is to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.
Eligibility
Participation Requirements
Sex: All
Maximum Age: 1
Healthy Volunteers: f
View:
• Infant aged ≤ 1 year at the time of enrollment.
• Confirmed diagnosis of ENPP1 deficiency, based on genetic testing.
• Clinical features consistent with generalized arterial calcification of infancy (GACI) (e.g., vascular calcification or cardiac involvement).
• Medically stable to participate in a 52-week treatment study.
• Written informed consent provided by a parent or legal guardian.
Locations
Other Locations
Brazil
Hospital Universitario Pedro Ernesto/Rio de Janeiro
RECRUITING
Rio De Janeiro
France
Hopital Necker - Enfants Malades
RECRUITING
Paris
Hungary
Gyermekgyogyaszat, DE
RECRUITING
Debrecen
Italy
Azienda Ospedaliera Universitaria Meyer
RECRUITING
Florence
Saudi Arabia
King Faisal Specialist Hospital and Research Center
RECRUITING
Riyadh
Spain
Hospital Sant Joan de Deu Edificio Consultas Externas. Unitat de Recerca
RECRUITING
Barcelona
Turkey
Umraniye Traiing and Research Hospital
RECRUITING
Istanbul
United Kingdom
Royal Manchester Children's Hospital
RECRUITING
Manchester
Contact Information
Primary
Trial Specialist
medinfo@bmrn.com
+1 800-983-4587
Backup
Medical Director, MD
medinfo@bmrn.com
+1 800-983-4587
Time Frame
Start Date: 2025-03-26
Estimated Completion Date: 2028-11-07
Participants
Target number of participants: 12
Treatments
Experimental: INZ-701
Participants receive INZ-701 (rhENPP1-Fc) administered by subcutaneous injection once weekly at the protocol-specified dose. The visit final volume to administer (mL) is determined using protocol-defined parameters.
Related Therapeutic Areas
Sponsors
Collaborators: BioMarin Pharmaceutical
Leads: Inozyme Pharma