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Understanding the Spectrum of ENPP1 Deficiency and Acute ABCC6 Deficiency

NCT04372446 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 38

Last updated 2021-04-01

No results posted yet for this study

Summary

To date, the investigators lack characterization of, the burden of, and the systemic progression of disease in ENPP1 deficiency and ABCC6 deficiency from a patient and/or parent perspective. This study aims to document this characterization, progression as well as the burden of disease. Link to the study registration-

https://www.engagehealth.com/survey/TakeSurvey.aspx?SurveyID=8252n62

Conditions

  • Generalized Arterial Calcification in Infancy
  • Autosomal Recessive Hypophosphatemic Rickets Type 2

Sponsors & Collaborators

  • GACI Global

    collaborator UNKNOWN

  • Engage Health Inc.

    collaborator INDUSTRY

  • Inozyme Pharma

    lead INDUSTRY

Principal Investigators

  • Pedro Huertas, MD · Inozyme Pharma

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-05-06
Primary Completion
2020-09-30
Completion
2020-09-30

Countries

  • United States

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 NCT04372446 on ClinicalTrials.gov