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Clinical trialNCT01688895CompletedObservational

Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial Impact

SponsorIcahn School of Medicine at Mount SinaiEnrollment150EPPErythropoietic ProtoporphyriaX-Linked Dominant Erythropoietic ProtoporphyriaX-Linked ProtoporphyriaXLDPXLEPP+2 more conditions
View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

The initial objective of this protocol is to assemble a well-documented group of patients with confirmed diagnoses of the erythropoietic protoporphyrias, including autosomal recessive Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP) for clinical, biochemical, and genetic studies. The long-term objectives are (1) to conduct a longitudinal investigation of the natural history, complications, and therapeutic outcomes in people with erythropoietic protoporphyria, (2) to systematically investigate the psychological effects of the erythropoietic protoporphyrias on children and adults, and (3) to investigate the correlation between the identified genotypes and the resulting clinical presentation, also determining the possible interaction of other genetic markers.

Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

6 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United States
  • University of Alabama, Birmingham

    Birmingham, Alabama, United States

  • University of California, San Francisco

    San Francisco, California, United States

  • Icahn School of Medicine at Mount Sinai

    New York, New York, United States

  • Wake Forest University Health Sciences

    Winston-Salem, North Carolina, United States

  • University of Texas Medical Branch

    Galveston, Texas, United States

  • University of Utah

    Salt Lake City, Utah, United States

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

No drug or biological interventions are in our data for this study. It may test a procedure, device or behavioral intervention, or the intervention list isn't in our current snapshot. See the registry record ↗. Why? →

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start
31 Jul 2012
Primary completion
1 Jul 2019
Completion
1 Jul 2019
Last update posted
17 Apr 2020
Registered enrollment
150
Registry id
NCT01688895

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.