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Tests treatment safety and results for Ectonucleotide Pyrophosphatase/phosphodiesterase1...
Official title ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency
ClinicalTrials.gov ID: NCT07473973
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 ENERGY 2 (Study INZ701-105) is to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.
- Phase 3: a large, late-stage study
- Time commitment: about 52 weeks
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 0 to 1
You may be able to join if
- Participants must meet all of the following:
- Infant aged ≤ 1 year at the time of enrollment.
- Confirmed diagnosis of ENPP1 deficiency, based on genetic testing.
- Clinical features consistent with generalized arterial calcification of infancy (GACI) (e.g., vascular calcification or cardiac involvement).
- Medically stable to participate in a 52-week treatment study.
You likely can't join if
- Participants will not be eligible if any of the following apply:
- Receiving end-of-life or hospice care.
- Prior treatment with INZ-701, unless received through an approved expanded access program.
- Concurrent participation in another treatment clinical trial.
- Planned major surgery during the study period that would interfere with study participation.
See the full eligibility criteria
- Participants must meet all of the following:
- Infant aged ≤ 1 year at the time of enrollment.
- Confirmed diagnosis of ENPP1 deficiency, based on genetic testing.
- Clinical features consistent with generalized arterial calcification of infancy (GACI) (e.g., vascular calcification or cardiac involvement).
- Medically stable to participate in a 52-week treatment study.
- Written informed consent provided by a parent or legal guardian.
- Participants will not be eligible if any of the following apply:
- Receiving end-of-life or hospice care.
- Prior treatment with INZ-701, unless received through an approved expanded access program.
- Concurrent participation in another treatment clinical trial.
- Planned major surgery during the study period that would interfere with study participation.
The study team makes the final eligibility decision.
Where it's taking place
- Rio de Janeiro, Brazil
- Paris, France
- Debrecen, Hungary
- Florence, Italy
- Riyadh, Saudi Arabia
- Barcelona, Esplugues de Llobregat, Spain
- Istanbul, Turkey (Türkiye)
- 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 study runs about 52 weeks per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 0 years to 1 year. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Rio de Janeiro, Brazil; Paris, France; Debrecen, Hungary; Florence, Italy; Riyadh, Saudi Arabia; Barcelona, Esplugues de Llobregat, Spain and 2 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 ClinicalTrials.gov; the study team decides eligibility.