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Tests treatment safety and results for Ectonucleotide Pyrophosphatase/phosphodiesterase1...
Official title The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
ClinicalTrials.gov ID: NCT05734196
What this study is testing
What is INZ-701?
INZ-701 is an investigational medicine, being studied as a potential treatment for ectonucleotide pyrophosphatase/phosphodiesterase1 deficiency.
Also referred to as (rhENPP1-Fc)..
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 Deficiency.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 1
You may be able to join if
- Infant aged ≤ 1 year at the time of enrollment
- Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency
- Study participants must have clinical manifestations of generalized arterial calcification of infancy (GACI) or GACI-2, which must include at least...
- Study participant must weigh ≥0.5 kg at the time of the first dose of INZ-701 in this study
- Written informed consent provided by a parent or legal guardian
You likely can't join if
- In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality that precludes study participation or...
- Receiving end of life or hospice care
- Known malignancy
- Concurrent participation in another non-Inozyme treatment study
- Treatment with any non-Inozyme product or investigational device during study participation
The study team makes the final eligibility decision.
Where it's taking place
- San Diego, California, United States
- Boston, Massachusetts, United States
- Columbus, Ohio, United States
- Philadelphia, Pennsylvania, United States
- Salt Lake City, Utah, United States
- Barcelona, Spain
- Manchester, 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, up to 1 year. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include San Diego, California, United States; Boston, Massachusetts, United States; Columbus, Ohio, United States; Philadelphia, Pennsylvania, United States; Salt Lake City, Utah, United States; Barcelona, Spain and 1 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.