Actively Recruiting
The ADAPT Study: An Open-label, Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
Led by Inozyme Pharma · Updated on 2024-11-29
200
Participants Needed
5
Research Sites
4 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the long-term safety of INZ-701, an investigational protein treatment for patients with ENPP1 Deficiency or ABCC6 Deficiency. These rare genetic conditions are linked to gene mutations causing arterial calcification and other complications. The study focuses on patients who have previously received INZ-701 in earlier trials and are continuing treatment to assess ongoing safety. INZ-701 is given as a once-weekly subcutaneous injection. Children aged 1 to under 13 years receive a dose based on their weight (2.4 mg/kg), while participants 13 years and older receive either a 1.8 mg/kg dose or a flat 150 mg dose. The study includes a 30-day screening period followed by an open-label treatment phase. Participants will continue treatment until the drug becomes commercially available or the sponsor stops development, with a safety follow-up visit about 30 days after their last treatment. Participants will be monitored closely throughout the study with safety assessments including tracking treatment-emergent adverse events and the presence of anti-drug antibodies over six years. Additional measures include blood tests for drug concentration and plasma markers related to the condition. The study ensures long-term observation of participants receiving INZ-701 to gather important safety data while supporting their ongoing care.
CONDITIONS
Brief Title
ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Provide written or electronic informed consent before any study procedures
- Provide assent if under 18 years old, according to local rules
- Male or female, age greater than 1 year
- Completed required safety and efficacy periods of a prior INZ-701 clinical study for ENPP1 or ABCC6 Deficiency
- Sexually active females of childbearing potential must use highly effective contraception during and shortly after treatment
- Sexually active males must use condoms during and shortly after treatment
- Able to complete all study procedures as judged by the Investigator
You will not qualify if you...
- Any significant disease or lab abnormality unrelated to ENPP1 or ABCC6 Deficiency that may affect participation or results
- Known intolerance to INZ-701 or its components
- Currently in another interventional clinical study or recently received investigational drugs/devices other than INZ-701
- Pregnant, trying to become pregnant, or breastfeeding
- Male participants trying to father a child
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 30 days
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to approximately 6 years
Participants receive once-weekly subcutaneous doses of INZ-701 and continue treatment until the drug is commercially available in their country or the study is discontinued.
Weekly visits for dosing and safety assessments
Duration - Approximately 30 days
Participants complete a safety follow-up visit approximately 30 days after their last study visit to monitor for any treatment-related effects.
1 visit (in-person)
Trial Site Locations
Total: 5 locations
1
Mayo Clinic
Rochester, Minnesota, United States, 55905
Actively Recruiting
2
Clinilabs Drug Development Corporation
Eatontown, New Jersey, United States, 07724
Actively Recruiting
3
Necker-Enfants Malades Hospital
Paris, France
Actively Recruiting
4
Universitätsklinikum Hamburg-Eppendorf (UKE)
Hamburg, Germany
Actively Recruiting
5
VCTC
Oxford, United Kingdom
Actively Recruiting
Research Team
I
Inozyme Clinical Trial Information
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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