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NCT06302439 · ClinicalTrials.gov registry record
PROPEL - A Prospective Observational Patient Registry to Evaluate ENPP1 and ABCC6 Deficiency
A clinical trial of Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency and ATP-Binding Cassette Subfamily C Member 6 Deficiency, sponsored by Inozyme Pharma.
- Recruiting
- Registry status
- 1,000
- Enrollment target
- 14
- Study locations
NCT06302439: Recruiting study of Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency and ATP-Binding Cassette Subfamily C Member 6 Deficiency, sponsored by Inozyme Pharma.
NCT06302439 is a study of Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency and ATP-Binding Cassette Subfamily C Member 6 Deficiency that is actively recruiting participants, run by Inozyme Pharma. The registered enrollment target is 1,000 participants, above the 63-participant average among 4 other Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency trials with a reported enrollment target (1487% higher). The trial reports 14 study locations across 7 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06302439, a study of Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency and ATP-Binding Cassette Subfamily C Member 6 Deficiency, is actively recruiting participants, sponsored by Inozyme Pharma.
- RECRUITING
- Registry status
- 1,000 participants
- Enrollment target
- 14
- Study locations
Study Summary
The purpose of this prospective registry is to characterize the natural history of ectonucleotide pyrophosphatase/phosphodiesterase1(ENPP1) Deficiency and the infantile-onset form of adenosine triphosphate (ATP) binding cassette transporter protein subfamily C member 6 (ABCC6) Deficiency longitudinally. The registry will prospectively gather information about the genetic, biochemical, physiological, anatomic, radiographic, and functional manifestations (including patient reported outcomes \[PROs\]) of each disease during routine, standard-of-care visits, with the aim of developing a comprehensive understanding of the burden of illness and progressive nature of the disease.
Primary Outcome
Assessments will be collected during each subject's routine visit. The assessments will be done per local standard of care.
Conditions Studied
Interventions
- OTHER No Intervention for this observational study
Study Locations (14)
Other
- Universitätsklinikum Hamburg-Eppendorf - Hamburg
- IRCCS San Raffaele Hospital - Main - Milan
- The University of Tokyo Hospital - Tokyo
- Royal Hospital Muscat - Muscat
- EU Hub - VCTC - Barcelona
- Hospital Sant Joan de Deu - Barcelona
- Umraniye Training and Research Hospital - Istanbul
- VCTC - Derby
Illinois
- Ann and Robert H. Lurie Children's Hospital - Chicago
Massachusetts
- Boston Children's Hospital - Boston
Minnesota
- Mayo Clinic - Rochester
New Jersey
- CLINILABS Drug Development Corp - Eatontown
Pennsylvania
- The Children's Hospital of Philadelphia (CHOP) - Philadelphia
Quebec
- CHU Sainte-Justine Research Centre - Montreal
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1,000 participants |
| Start Date | 2024-07-25 |
| Est. Completion | 2034-05 |
What NCT06302439 shows while recruiting
NCT06302439 is an observational study that tracks outcomes without assigning an intervention. Its 1,000 participants enrollment target places it among the larger protocols in the corpus, above the 63-participant average among 4 other Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency trials with a reported enrollment target (1487% higher).
The record links to 2 conditions, with Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency appearing as the primary indexed condition, and to 1 intervention - of which No Intervention for this observational study is the first listed.
NCT06302439 lists 14 locations in 7 states (Other, Illinois, Massachusetts).
Frequently Asked Questions
What is clinical trial NCT06302439 about?
NCT06302439 is a clinical study titled "PROPEL - A Prospective Observational Patient Registry to Evaluate ENPP1 and ABCC6 Deficiency". The purpose of this prospective registry is to characterize the natural history of ectonucleotide pyrophosphatase/phosphodiesterase1(ENPP1) Deficiency and the infantile-onset form of adenosine triphosphate (ATP) binding cassette transporter protein subfamily C member 6 (ABCC6) Deficiency longitudina...
What is the current status of trial NCT06302439?
This trial is currently recruiting. The enrollment target is 1,000 participants. The study started on 2024-07-25. Estimated completion is 2034-05.
What conditions does trial NCT06302439 study?
This clinical trial studies the following conditions: Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, ATP-Binding Cassette Subfamily C Member 6 Deficiency.
What interventions are being tested in trial NCT06302439?
The interventions under investigation include: No Intervention for this observational study (OTHER).
Who is sponsoring clinical trial NCT06302439?
This trial is sponsored by Inozyme Pharma, which has 9 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06302439 being conducted?
This trial has 14 study locations across Illinois, Massachusetts, Minnesota, New Jersey, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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