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PROPEL - A Prospective Observational Patient Registry to Evaluate ENPP1 and ABCC6 Deficiency

NCT06302439 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 1000

Last updated 2025-12-22

No results posted yet for this 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

  • Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Deficiency
  • ATP-Binding Cassette Subfamily C Member 6 Deficiency

Interventions

OTHER

No Intervention for this observational study

No Intervention for this observational study

Sponsors & Collaborators

  • GACI Global

    collaborator UNKNOWN

  • Inozyme Pharma

    lead INDUSTRY

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-07-25
Primary Completion
2034-05-31
Completion
2034-05-31

Countries

  • United States
  • Canada
  • Germany
  • Italy
  • Japan
  • Oman
  • Spain
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06302439 on ClinicalTrials.gov