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The ENERGY Study: Evaluation of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Infants with ENPP1 Deficiency or ATP-binding Cassette Sub-family C Member 6 (ABCC6) Deficiency
EU CTIS ID: 2023-507384-20-00
What this study is testing
To assess the safety and tolerability of INZ-701
- Human Pharmacology (Phase I)- Other
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) must provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, following International Council for Harmonisation (ICH) Good Clinical Practice (GCP).
- Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency with biallelic mutations (ie, homozygous or compound heterozygous) performed using an assay that meets CE-marked requirements, or by a College of American Pathologists/Clinical Laboratory Improvement Amendments (CAP/CLIA) certified laboratory or a local equivalent
- Study participant must be male or female from birth to <1 year of age at Baseline (Day 1)
- Study participant must weigh ≥0.5 kg at the time of the first dose of INZ-701 in this study
- In the opinion of the Investigator, the study participant must be able to complete all aspects of the study
- Study participant’s caregiver(s) must agree to provide access to their child’s relevant medical records
You likely can't join if
- In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality (outside of those considered associated with the diagnosis of ENPP1 Deficiency or ABCC6 Deficiency) that precludes study participation or may confound interpretation of study results, including known uncontrolled thyroid disease or unrelated connective tissue, bone, mineral, or muscle disease
- Care has been withdrawn or subject is receiving end of life care or hospice only
- Known malignancy
- Known intolerance to INZ-701 or any of its excipients
- Concurrent participation in another non-Inozyme interventional study
- Receipt of any non-Inozyme investigational new drug within 5 half-lives of the last dose of the other investigational product or within 4 weeks prior to the first dose of INZ-701, whichever is longer, or use of an investigational device through completion of participation in the study
See the full eligibility criteria
Who can join
- Caregiver(s) must provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, following International Council for Harmonisation (ICH) Good Clinical Practice (GCP).
- Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency with biallelic mutations (ie, homozygous or compound heterozygous) performed using an assay that meets CE-marked requirements, or by a College of American Pathologists/Clinical Laboratory Improvement Amendments (CAP/CLIA) certified laboratory or a local equivalent
- Study participant must be male or female from birth to <1 year of age at Baseline (Day 1)
- Study participant must weigh ≥0.5 kg at the time of the first dose of INZ-701 in this study
- In the opinion of the Investigator, the study participant must be able to complete all aspects of the study
- Study participant’s caregiver(s) must agree to provide access to their child’s relevant medical records
- Study participants must have clinical manifestations of GACI or GACI-2, which may include, but are not limited to, pathologic ectopic calcification, heart failure, respiratory distress, edema, cyanosis, hypertension, and cardiomegaly
What rules you out
- In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality (outside of those considered associated with the diagnosis of ENPP1 Deficiency or ABCC6 Deficiency) that precludes study participation or may confound interpretation of study results, including known uncontrolled thyroid disease or unrelated connective tissue, bone, mineral, or muscle disease
- Care has been withdrawn or subject is receiving end of life care or hospice only
- Known malignancy
- Known intolerance to INZ-701 or any of its excipients
- Concurrent participation in another non-Inozyme interventional study
- Receipt of any non-Inozyme investigational new drug within 5 half-lives of the last dose of the other investigational product or within 4 weeks prior to the first dose of INZ-701, whichever is longer, or use of an investigational device through completion of participation in the study
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
- 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; United States. 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.