Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
PROPEL - A Prospective Observational Patient Registry to Evaluate ENPP1 and ABCC6 Deficiency
NCT06302439 · View on ClinicalTrials.gov ↗
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.
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 |
Interested in This Trial?
Always speak with your doctor before enrolling in a clinical trial.
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06302439
The ClinicalTrials.gov registry entry for NCT06302439 describes a study currently listed as recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 1,000 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Inozyme Pharma, which has 6 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 2 conditions, with ATP-Binding Cassette Subfamily C Member 6 Deficiency appearing as the primary indexed condition, and to 1 intervention — of which No Intervention for this observational study is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06302439 reports 14 study locations spanning 7 distinct geographic areas — top geographies include Other, Illinois, Massachusetts. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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: ATP-Binding Cassette Subfamily C Member 6 Deficiency, Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Deficiency. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT06302439?
The interventions under investigation include: No Intervention for this observational study (OTHER). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT06302439?
This trial is sponsored by Inozyme Pharma, which has 6 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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.
Learn More About Clinical Trials
How Clinical Trials Work
Understand phases 1-4, trial design, randomization, and the informed consent process.
Patient Rights in Clinical Trials
Your rights as a participant: consent, withdrawal, privacy, and who to contact.
Finding the Right Clinical Trial
A practical guide to searching trials, understanding eligibility, and evaluating options.
All Guides
Browse our complete library of clinical trial educational resources.
Read our methodology — how this data is sourced, computed, and verified.