Status:
ACTIVE_NOT_RECRUITING
The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency
Lead Sponsor:
Inozyme Pharma
Conditions:
Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency
Autosomal Recessive Hypophosphatemic Rickets
Eligibility:
All Genders
1-12 years
Phase:
PHASE3
Brief Summary
The primary purpose of Study INZ701-106 (The ENERGY 3 Study) is to assess the efficacy and safety of INZ-701 in children with ENPP1 Deficiency.
Detailed Description
INZ-701 is an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy (ERT) in development for the treatment of ENPP1 Deficiency, an ultra-rare genetic disorder with an i...
Eligibility Criteria
Inclusion
- Inclusion Criteria
- Study participants must meet all of the following inclusion criteria:
- Caregiver's written or electronic informed 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)
- Study participant's assent in accordance with local regulations
- A confirmed postnatal 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 regional equivalent
- Males and females ≥1 year and \<13 years of age at Study Day 1
- Open growth plates of the distal femur and proximal tibia in both legs
- Plasma PPi concentration of \<1400 nM at Screening
- 25-hydroxyvitamin D (25\[OH\]D) levels of ≥12 ng/mL at Screening
- Radiographic evidence of skeletal abnormalities based on an RSS ≥2
- Female participants of childbearing potential must have a negative serum pregnancy test at Screening and must not be breastfeeding
- Study participants of childbearing potential who are sexually active must agree to use a highly effective form of contraception in accordance with Clinical Trials Facilitation and Coordination Group (CTFG) guidance and local guidelines for the duration of the study
- In the opinion of the Investigator, able to complete all aspects of the study
- Exclusion Criteria
- Study participants meeting any of the following exclusion criteria will not be eligible to participate in the study:
- In the opinion of the Investigator, has clinically significant disease or laboratory abnormality not associated with ENPP1 Deficiency that will preclude study participation and/or may confound the interpretation of study results
- If receiving any of the following prohibited medications as indicated in the protocol: systemic corticosteroids (\>5 mg prednisone equivalent per day), anti-fibroblast growth factor 23 (FGF23), and oral and/or IV bisphosphonates
- Unable or unwilling to discontinue calcitriol or other active forms of vitamin D3 (or analogs) within 7 days prior to Study Day 1 and/or oral phosphate supplements within 36 hours prior to Study Day 1 if randomized to the INZ-701 arm
- Planned orthopedic surgery that may confound the interpretation of study results during the 52-week Randomized Treatment Period
- Known intolerance to INZ-701 or any of its excipients
- A positive COVID-19 test within 5 days prior to Randomization, only if required as per local regulations or institutional policy
- Previous treatment with INZ-701
- Concurrent participation in another interventional clinical study and/or has received an investigational drug within 5 half-lives of the last dose or within 4 weeks prior to Randomization, whichever is longer, or use of an investigational device
Exclusion
Key Trial Info
Start Date :
November 5 2023
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 1 2026
Estimated Enrollment :
27 Patients enrolled
Trial Details
Trial ID
NCT06046820
Start Date
November 5 2023
End Date
February 1 2026
Last Update
May 1 2025
Active Locations (15)
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1
Children's Hospital of Colorado
Aurora, Colorado, United States, 80045
2
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, United States, 60611
3
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
4
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205