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The ENERGY 2 Study: A study to investigate if study drug INZ-701 increases survival in infants with ENPP1 Deficiency by preventing heart dysfunction and calcification of arteries
EU CTIS ID: 2024-512991-36-00
What this study is testing
To determine if INZ-701 increases inorganic pyrophosphate (PPi) levels To determine if INZ-701 increases overall survival compared to the Natural History Database
- 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 or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, per International Council on Harmonisation (ICH) Good Clinical Practice (GCP)
- A confirmed postnatal molecular genetic diagnosis of ENPP1 Deficiency with biallelic mutations (ie, homozygous or compound heterozygous) performed using assays that meet CE-marked requirements, or by a College of American Pathologists/Clinical Laboratory Improvement Amendments (CAP/CLIA) certified laboratory, or a regional equivalent
- Clinical manifestations of GACI, which must include at least one of the following: ectopic calcification, heart failure, respiratory distress, edema, cyanosis, hypertension, and cardiomegaly. Heart failure is defined as a structural and/or functional abnormality that produces raised intracardiac pressures and/or inadequate cardiac output resulting in characteristic signs and symptoms including edema and respiratory distress. Cardiomegaly is defined as cardiothoracic ratio exceeding 0.5 by chest X-ray or increased left ventricular mass on echocardiography or cross-sectional imaging relative to normal based on Investigator judgment.
- Males and females from birth to <1 year of age at Study Day 1
- Plasma PPi concentration of <1400 nM: a. at study Screening if study participant has not participated in the EAP. b. at the documented baseline prior to treatment with INZ-701 in the EAP
- Body weight ≥0.5 kg at the time of the first dose of INZ-701
You likely can't join if
- In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality not associated with ENPP1 Deficiency that will preclude study participation and/or may confound interpretation of study results
- Care has been withdrawn or infant is receiving end of life or hospice care
- Planned surgery that may confound the interpretation of study results during the 52-week Treatment Period in the opinion of the Investigator
- Known intolerance to INZ-701 or any of its excipients
- Previous treatment with INZ-701 unless prior treatment was part of the EAP, in which case they may participate upon Sponsor approval
- Concurrent participation in another interventional clinical study and/or has received an investigational drug within 5 half-lives or within 4 weeks prior to the first dose of INZ-701, whichever is longer, or use of an investigational device
See the full eligibility criteria
Who can join
- Caregiver(s) written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, per International Council on Harmonisation (ICH) Good Clinical Practice (GCP)
- A confirmed postnatal molecular genetic diagnosis of ENPP1 Deficiency with biallelic mutations (ie, homozygous or compound heterozygous) performed using assays that meet CE-marked requirements, or by a College of American Pathologists/Clinical Laboratory Improvement Amendments (CAP/CLIA) certified laboratory, or a regional equivalent
- Clinical manifestations of GACI, which must include at least one of the following: ectopic calcification, heart failure, respiratory distress, edema, cyanosis, hypertension, and cardiomegaly. Heart failure is defined as a structural and/or functional abnormality that produces raised intracardiac pressures and/or inadequate cardiac output resulting in characteristic signs and symptoms including edema and respiratory distress. Cardiomegaly is defined as cardiothoracic ratio exceeding 0.5 by chest X-ray or increased left ventricular mass on echocardiography or cross-sectional imaging relative to normal based on Investigator judgment.
- Males and females from birth to <1 year of age at Study Day 1
- Plasma PPi concentration of <1400 nM: a. at study Screening if study participant has not participated in the EAP. b. at the documented baseline prior to treatment with INZ-701 in the EAP
- Body weight ≥0.5 kg at the time of the first dose of INZ-701
- Be able to complete all aspects of the study in the opinion of the Investigator
- Caregiver(s) agree to provide access to their infant’s relevant medical records
What rules you out
- In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality not associated with ENPP1 Deficiency that will preclude study participation and/or may confound interpretation of study results
- Care has been withdrawn or infant is receiving end of life or hospice care
- Planned surgery that may confound the interpretation of study results during the 52-week Treatment Period in the opinion of the Investigator
- Known intolerance to INZ-701 or any of its excipients
- Previous treatment with INZ-701 unless prior treatment was part of the EAP, in which case they may participate upon Sponsor approval
- Concurrent participation in another interventional clinical study and/or has received an investigational drug within 5 half-lives 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
- Canada
- Saudi Arabia
- Turkey
- United Kingdom
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 Canada; Saudi Arabia; Turkey; United Kingdom. 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.