BridgeMD
The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency
EU CTIS ID: 2023-507382-26-00
What this study is testing
To determine if INZ-701 increases PPi levels To determine if INZ-701 improves skeletal abnormalities as measured by the RGI-C
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Caregiver’s written 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)
- Males who are sexually active must agree to use condoms from the period following first dose of INZ-701 through 30 days after the last dose of INZ-701
- WOCBP and partners of fertile males who are WOCBP must be using or must agree to use a highly effective form of contraception (as per CTCG) from at least 1 month before the first dose of INZ-701 through 30 days after the last dose of INZ-701 (greater than 5 half-lives of INZ-701)
- In the opinion of the Investigator, able to complete all aspects of the study
- 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
You likely can't join if
- 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-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 or other procedures that may confound the interpretation of study results during the 52-week RTP
- 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
See the full eligibility criteria
Who can join
- Caregiver’s written 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)
- Males who are sexually active must agree to use condoms from the period following first dose of INZ-701 through 30 days after the last dose of INZ-701
- WOCBP and partners of fertile males who are WOCBP must be using or must agree to use a highly effective form of contraception (as per CTCG) from at least 1 month before the first dose of INZ-701 through 30 days after the last dose of INZ-701 (greater than 5 half-lives of INZ-701)
- In the opinion of the Investigator, able to complete all aspects of the study
- 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(OH)D levels of ≥12 ng/mL at Screening
- Radiographic evidence of skeletal abnormalities based on an RSS ≥2
- Women of childbearing potential (WOCBP, as defined in Clinical Trials Coordination Group [CTCG 2024]) must have a negative serum pregnancy test at Screening and must not be breastfeeding
What rules you out
- 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-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 or other procedures that may confound the interpretation of study results during the 52-week RTP
- 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 the first dose of INZ-701, whichever is longer, or use of an investigational device
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
- Saudi Arabia
- United Arab Emirates
- Canada
- Australia
- Turkey
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom; Saudi Arabia; United Arab Emirates; Canada; Australia; Turkey and 1 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.