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NCT04686175 · ClinicalTrials.gov registry record · Phase 1
Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency
A Phase 1 study of Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency and Autosomal Recessive Hypophosphatemic Rickets, sponsored by Inozyme Pharma.
- Completed
- Registry status
- Phase 1
- Development phase
- 9
- Enrollment target
- 7
- Study locations
NCT04686175: Completed Phase 1 study of Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency and Autosomal Recessive Hypophosphatemic Rickets, sponsored by Inozyme Pharma.
NCT04686175 is a Phase 1 study of Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency and Autosomal Recessive Hypophosphatemic Rickets that has completed, run by Inozyme Pharma. The registered enrollment target is 9 participants, below the 311-participant average among 4 other Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency trials with a reported enrollment target (97% lower). The trial reports 7 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04686175, a Phase 1 study of Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency and Autosomal Recessive Hypophosphatemic Rickets, has completed, sponsored by Inozyme Pharma.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 9 participants
- Enrollment target
- 7
- Study locations
Study Summary
The purpose of this study is to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of multiple ascending doses of INZ-701, an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy, for the treatment of ENPP1 Deficiency. The goal of the study is to identify a dose regimen for further clinical development in the treatment of ENPP1 Deficiency.
Primary Outcome
Treatment-emergent AEs are defined as any AE occurring from the first dose of INZ-701 through 30 days after the last dose of INZ-701.
Conditions Studied
Interventions
- DRUG INZ-701
Study Locations (7)
Other
- Necker University Hospital-Sick Children - Paris
- Parexel International GmbH - Berlin
- University of Hamburg (Universitatklinikum Hamburg-Eppendorf) - Hamburg
Minnesota
- Mayo Clinic - Rochester
New Jersey
- Clinilabs Drug Development Corporation - Eatontown
Saskatchewan
- University of Saskatchewan - Saskatoon
London Bridge
- Richmond Pharmacology (RPL) - London
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 9 participants |
| Start Date | 2021-11-21 |
| Est. Completion | 2024-12-13 |
| Phase | Phase 1 |
What the finished NCT04686175 record still lists
NCT04686175 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 9 participants, a relatively small participant target, below the 311-participant average among 4 other Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency trials with a reported enrollment target (97% lower).
The record links to 3 conditions, with Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency appearing as the primary indexed condition, and to 1 intervention - of which INZ-701 is the first listed.
NCT04686175 lists 7 locations in 5 states (Other, Minnesota, New Jersey).
Frequently Asked Questions
What is clinical trial NCT04686175 about?
NCT04686175 is a clinical study titled "Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency". The purpose of this study is to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of multiple ascending doses of INZ-701, an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy, for the treatment of ENPP1 Deficiency. The goal of the s...
What is the current status of trial NCT04686175?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 9 participants. The study started on 2021-11-21. Estimated completion is 2024-12-13.
What conditions does trial NCT04686175 study?
This clinical trial studies the following conditions: Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy.
What interventions are being tested in trial NCT04686175?
The interventions under investigation include: INZ-701 (DRUG).
Who is sponsoring clinical trial NCT04686175?
This trial is sponsored by Inozyme Pharma, which has 9 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04686175 being conducted?
This trial has 7 study locations across Minnesota, New Jersey, Saskatchewan, London Bridge. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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