RECRUITING

ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency

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Conditions

Gene MutationsPseudoxanthoma ElasticumArterial CalcificationEctonucleotide Pyrophosphatase/phosphodiesterase1 DeficiencyAutosomal Recessive Hypophosphatemic Rickets Type 2ABCC6ATP-Binding Cassette Subfamily C Member 6 DeficiencyPXEGACIGeneralized Arterial Calcification of InfancyARHR2ENPP1hypopyrophosphatemia

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

The ADAPT Study: An Open-label, Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency

Quick Facts

Study Start:2024-06-19
Study Completion:2030-12
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06462547

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:1 Year
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. 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)
  2. 2. Provide assent in accordance with local regulations, if \<18 years of age
  3. 3. Male or female, greater than 1 year of age
  4. 4. 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
  5. 5. 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
  6. 6. 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
  7. 7. In the opinion of the Investigator, able to complete all aspects of the study
  8. 1. 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
  9. 2. Known intolerance to INZ-701 or any of its excipients
  10. 3. 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
  11. 4. Pregnant, trying to become pregnant, or breastfeeding
  12. 5. Male participants trying to father a child
  1. Pregnancy or breastfeeding
  2. Severe psychiatric disorders
  3. Active substance abuse
  4. Unstable medical conditions
  5. Inability to comply with study requirements

Contacts and Locations

Study Contact

Inozyme Clinical Trial Information
CONTACT
+1 857 330 4340
clinicaltrials@inozyme.com

Principal Investigator

Kurt Gunter, MD
STUDY_DIRECTOR
Inozyme Pharma, Inc.

Study Locations (Sites)

Mayo Clinic
Rochester, Minnesota, 55905
United States
Clinilabs Drug Development Corporation
Eatontown, New Jersey, 07724
United States

Collaborators and Investigators

Sponsor: Inozyme Pharma

  • Kurt Gunter, MD, STUDY_DIRECTOR, Inozyme Pharma, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-06-19
Study Completion Date2030-12

Study Record Updates

Study Start Date2024-06-19
Study Completion Date2030-12

Terms related to this study

Keywords Provided by Researchers

  • ABCC6
  • ATP-Binding Cassette Subfamily C Member 6 Deficiency
  • PXE
  • Pseudoxanthoma elasticum
  • GACI
  • Generalized Arterial Calcification of Infancy
  • ARHR2
  • Autosomal Recessive Hypophosphatemic Rickets Type 2
  • ENPP1
  • ectonucleotide pyrophosphatase/phosphodiesterase1 deficiency
  • hypopyrophosphatemia

Additional Relevant MeSH Terms

  • Gene Mutations
  • Pseudoxanthoma Elasticum
  • Arterial Calcification
  • Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
  • Autosomal Recessive Hypophosphatemic Rickets Type 2