ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: INZ-701.
- Who it may be relevant to
- Registry conditions: Gene Mutations, Pseudoxanthoma Elasticum, Arterial Calcification, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency. Basic parameters: from 1 year · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, France, Germany, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
The ADAPT Study: An Open-label, Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
Overview
The purpose of this study (Study INZ701-304 \[ADAPT\]) is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition.
Detailed description
The investigational product INZ-701 is being developed as a therapeutic protein for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) Deficiency and adenosine triphosphate (ATP)-binding cassette subfamily C member 6 (ABCC6) Deficiency. INZ-701 is a recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin (Ig) G1 antibody.
The ADAPT Study (INZ701-304) is an open-label study to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition.
The study will consist of a 30-day Screening Period, followed by an open-label Treatment Period during which all participants will receive once-weekly subcutaneous (SC) doses of INZ-701 and continue with treatment until INZ-701 is commercially available in the country/region of the participant's residence or until Inozyme chooses to discontinue development of INZ-701. Participants will complete an End of Study (EOS) safety follow-up visit approximately 30 days after their last designated study visit assigned by the Investigator and/or Sponsor.
Interventions
- Drug INZ-701
INZ-701 is a recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody (rhENPP1-Fc).
Primary outcome measures
- Number of Treatment Emergent Adverse Events (TEAEs) [Time frame: 6 years (long term safety assessment)]
- Incidence of Anti-Drug Antibodies (ADA) [Time frame: 6 years (long term safety assessment)]
Secondary outcome measures (3)
- Time to maximum serum concentration (Tmax) [Time frame: 6 years (long term safety assessment)]
- Maximum Plasma Concentration (Cmax) of INZ-701 [Time frame: 6 years (long term safety assessment)]
- Mean Change from Baseline in Plasma PPi Concentration [Time frame: 6 years (long term safety assessment)]
Eligibility criteria
Individuals eligible to participate must meet all of the following inclusion criteria:
- Provide written or electronic informed consent after the nature of the study has been explained, and prior to any research-related procedures, per International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP)
- Provide assent in accordance with local regulations, if <18 years of age
- Male or female, greater than 1 year of age
- Must have completed the protocol-required safety and PK/PD and/or efficacy period(s) of a previous INZ-701 clinical study in ENPP1 or ABCC6 Deficiency, as confirmed by the Sponsor
- Female participants of childbearing potential who are sexually active must be using or agree to use 1 highly effective form of contraception (per CTFG 2020) from at least 1 month before the first dose of INZ-701 through 30 days after last dose of INZ-701 (greater than 5 half-lives of INZ-701); participants must agree to not donate ova from the period following the first dose of INZ-701 through 30 days after the last dose of INZ-701
- Male participants who are sexually active must agree to use condoms from the period following the first dose of INZ-701 through 30 days after the last dose of INZ-701; participants must agree to not donate sperm from the period following the first dose of INZ-701 through 30 days after last dose of INZ-701
- In the opinion of the Investigator, able to complete all aspects of the study
Individuals who meet any of the following exclusion criteria will not be eligible to participate:
- In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality not associated with ENPP1 Deficiency or ABCC6 Deficiency, that will preclude study participation and/or may confound interpretation of study results
- Known intolerance to INZ-701 or any of its excipients
- Concurrent participation in another interventional clinical study and/or has received an investigational drug other than INZ-701 within 5 half-lives or within 4 weeks prior to the first dose of INZ-701 in this study, whichever is longer, or use of an investigational device
- Pregnant, trying to become pregnant, or breastfeeding
- Male participants trying to father a child
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 2 centers
- Mayo Clinic — Rochester
- Clinilabs Drug Development Corporation — Eatontown
France · 1 center
- Necker-Enfants Malades Hospital — Paris
Germany · 1 center
- Universitätsklinikum Hamburg-Eppendorf (UKE) — Hamburg
United Kingdom · 1 center
- VCTC — Oxford
Identifiers
NCT: NCT06462547 · INZ701-304