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

Recruiting Observational Study
Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Deficiency ATP-Binding Cassette Subfamily C Member 6 Deficiency
No Study Drug Researchers observe your health over time — no experimental treatment is given.
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At a Glance
Sex
Any
Study type
Observational
Participants needed
1,000 (estimated)
Sponsor
Inozyme Pharma · Industry
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About This Trial
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, physiologica…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: Individuals eligible to participate must meet all the following inclusion criteria: 1. Must provide written or electronic consent after the nature of the registry has been explained, and prior to any research-related procedures, per International Council for Harmonisation (ICH)…
Contacts

Jelena Garafalo, Ph.D

(857) 330-4340

jelena.garafalo@inozyme.com

CONTACT