NCT01422915

Brief Summary

The investigators demonstrated that cholestyramine is an effective binding agent in vitro for porphyrins. A few isolated case reports of treatment of individuals with a cutaneous porphyria suggest that cholestyramine and colestipol effectively remove porphyrins. Hypothesis: orally administered colestipol will effectively reduce sun sensitivity and lower erythrocyte porphyrin concentrations in subjects with erythropoietic protoporphyria (EPP).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
4

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started May 2011

Shorter than P25 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 2011

Completed
4 months until next milestone

First Submitted

Initial submission to the registry

August 23, 2011

Completed
2 days until next milestone

First Posted

Study publicly available on registry

August 25, 2011

Completed
6 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2012

Completed
5.2 years until next milestone

Results Posted

Study results publicly available

April 25, 2017

Completed
Last Updated

April 25, 2017

Status Verified

March 1, 2017

Enrollment Period

10 months

First QC Date

August 23, 2011

Results QC Date

August 20, 2013

Last Update Submit

March 14, 2017

Conditions

Erythropoietic Protoporphyria

Keywords

SorbentResinEPPCutaneous porphyriaAdultover age 21without intercurrent illnessnot pregnantwilling to participate

Outcome Measures

Primary Outcomes (2)

  • Photosensitivity, Assessed by Measuring the Number of Minutes of Sun Tolerance

    Minutes of sun tolerance

    At 60 days of treatment

  • Protoporphyrin Concentration in Blood

    1. erythrocyte protoporphyrin concentration, ug/dl 2. plasma protoporphyrin concentration, ug/dl

    Samples collected while on treatment (range 93-208 treatment days)

Study Arms (1)

colestipol treatment

EXPERIMENTAL

2 grams morning and bedtime for 180 days

Drug: Colestipol

Interventions

ColestipolDRUG

2 grams morning and bedtime for 90 days.

colestipol treatment

Eligibility Criteria

Age22 Years - 60 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64)

You may qualify if:

  • Adult over age 21
  • healthy

You may not qualify if:

  • Intercurrent illness
  • pregnancy

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Brigham & Women's Hospital

Boston, Massachusetts, 02115, United States

Location

MeSH Terms

Conditions

Protoporphyria, Erythropoietic

Interventions

Colestipol

Condition Hierarchy (Ancestors)

Porphyrias, HepaticLiver DiseasesDigestive System DiseasesSkin Diseases, GeneticGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSkin DiseasesSkin and Connective Tissue DiseasesPorphyriasMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

PolyaminesAminesOrganic ChemicalsPolymersMacromolecular SubstancesBiomedical and Dental MaterialsManufactured MaterialsTechnology, Industry, and Agriculture

Limitations and Caveats

1 subject was removed during Period 1 because of abnormal liver chemistries.

Results Point of Contact

Title
Dr. Peter V. Tishler
Organization
BrighamHospital
repvt@channing.harvard.edu
617-525-2123

Publication Agreements

PI is Sponsor Employee
Yes
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
physician

Study Record Dates

First Submitted

August 23, 2011

First Posted

August 25, 2011

Study Start

May 1, 2011

Primary Completion

March 1, 2012

Study Completion

March 1, 2012

Last Updated

April 25, 2017

Results First Posted

April 25, 2017

Record last verified: 2017-03

Locations

US(1)