The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency (NCT06046820) | Clinical Trial Compass
Active — Not RecruitingPhase 3
The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency
United States27 participantsStarted 2023-11-05
Plain-language 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.
Who can participate
Age range1 Year – 12 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Exclusion criteria
✕. 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
What they're measuring
1
Change from Baseline in Plasma Inorganic Pyrophosphate (PPi) concentration through Week 52
✕. 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