Actively Recruiting
ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency
Led by Inozyme Pharma · Updated on 2026-03-18
12
Participants Needed
8
Research Sites
188 weeks
Total Duration
On this page
Sponsors
I
Inozyme Pharma
Lead Sponsor
B
BioMarin Pharmaceutical
Collaborating Sponsor
AI-Summary
What this Trial Is About
The primary purpose of ENERGY 2 (Study INZ701-105) is to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.
CONDITIONS
Official Title
ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Infant aged 60; 1 year at the time of enrollment.
- Confirmed diagnosis of ENPP1 deficiency through genetic testing.
- Clinical features consistent with generalized arterial calcification of infancy (GACI), such as 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.
You will not qualify if you...
- Receiving end-of-life or hospice care.
- Prior treatment with INZ-701, unless received through an approved expanded access program.
- Concurrent participation in another interventional clinical trial.
- Planned major surgery during the study period that would interfere with study participation.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 8 locations
1
Hospital Universitario Pedro Ernesto/Rio de Janeiro
Rio de Janeiro, Brazil, 20551-030
Actively Recruiting
2
Hopital Necker - Enfants Malades
Paris, France, 75015
Actively Recruiting
3
Gyermekgyogyaszat, DE
Debrecen, Hungary
Actively Recruiting
4
Azienda Ospedaliera Universitaria Meyer
Florence, Italy, 50139
Actively Recruiting
5
King Faisal Specialist Hospital and Research Center
Riyadh, Saudi Arabia, 12713
Actively Recruiting
6
Hospital Sant Joan de Deu Edificio Consultas Externas. Unitat de Recerca
Barcelona, Esplugues de Llobregat, Spain, 08950
Actively Recruiting
7
Umraniye Traiing and Research Hospital
Istanbul, Turkey (Türkiye)
Actively Recruiting
8
Royal Manchester Children's Hospital
Manchester, United Kingdom, M13 9WL
Actively Recruiting
Research Team
T
Trial Specialist
CONTACT
M
Medical Director, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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