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Sorbent Therapy of the Cutaneous Porphyrias (EPP)

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ClinicalTrials.gov Identifier: NCT01422915
Recruitment Status : Completed
First Posted : August 25, 2011
Results First Posted : April 25, 2017
Last Update Posted : April 25, 2017
Sponsor:
Information provided by (Responsible Party):
Peter Tishler, Brigham and Women's Hospital

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Erythropoietic Protoporphyria
Intervention Drug: Colestipol
Enrollment 4

Recruitment Details Dates: February and March 2011.Location types: suburbs of Massachusetts (3 subjects) and in New Jersey (1).
Pre-assignment Details All 4 subjects were enrolled in the study. Initially, baseline blood protoporphyrin concentrations and answers to sun sensitivity questionnaire were obtained.
Arm/Group Title Colestipol Treatment
Hide Arm/Group Description Colestipol 2 grams twice daily for 5-6 months. Completion of sun sensitivity questionnaire and blood protoporphyrin concentrations. were obtained monthly.
Period Title: Overall Study
Started 4 [1]
Completed 3
Not Completed 1
Reason Not Completed
Adverse Event             1
[1]
1 subject with abnormal liver chemistries removed during the first period.
Arm/Group Title Colestipol Therapy of Protoporphyria
Hide Arm/Group Description 4 subjects were assigned, all between 18-65 years of age.
Overall Number of Baseline Participants 4
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
<=18 years
0
   0.0%
Between 18 and 65 years
4
 100.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 4 participants
43  (15)
Age, Customized   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 4 participants
<=18 years 0
Between 18 and 65 years 4
>=65 years 0
[1]
Measure Description: For baseline in Period 1 and Period 2: ~monthly completion of sun sensitivity questionnaire; donate whole blood for RBC and plasma protoporphyrin
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
Female
4
 100.0%
Male
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 4 participants
4
1.Primary Outcome
Title Photosensitivity, Assessed by Measuring the Number of Minutes of Sun Tolerance
Hide Description Minutes of sun tolerance
Time Frame At 60 days of treatment
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All of the original 4 subjects had bona fide erythropoietic protoporphyria (EPP). One subject was removed during Perdiod 1. The data from the same 3 subjects who completed periods 1 and 2 were analyzed for the study results. Data collected was not tractable for statistical analysis given the range of results
Arm/Group Title Colestipol Treatment
Hide Arm/Group Description:
  1. gram morning and bedtime for 90 days; then
  2. grams morning and bedtime for 90 days. Sun sensitivity questionnaires and blood protoporphyrin concns. were determined monthly.
Overall Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: minutes
65.8  (58.8)
2.Primary Outcome
Title Protoporphyrin Concentration in Blood
Hide Description
  1. erythrocyte protoporphyrin concentration, ug/dl
  2. plasma protoporphyrin concentration, ug/dl
Time Frame Samples collected while on treatment (range 93-208 treatment days)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Colestipol Treatment
Hide Arm/Group Description:
Sun sensitivity and protoporphyrin levels
Overall Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: ug/dl
Erythrocyte Protoporphyrin 1712  (455)
Plasma Protoporphyrin 5.8  (8.3)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title 1st Period - Colestipol Optimal Dosage
Hide Arm/Group Description
  1. gram morning and bedtime for 90 days; then
  2. grams morning and bedtime for 90 days. Sun sensitivity questionnaires and blood protoporphyrin concentrations were determined monthly.
All-Cause Mortality
1st Period - Colestipol Optimal Dosage
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
1st Period - Colestipol Optimal Dosage
Affected / at Risk (%) # Events
Total   1/4 (25.00%)    
Hepatobiliary disorders   
Abnormal liver enzymes  [1]  1/4 (25.00%)  1
Indicates events were collected by systematic assessment
[1]
Abnormal liver enzymes on one occasion.
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
1st Period - Colestipol Optimal Dosage
Affected / at Risk (%) # Events
Total   0/4 (0.00%)    
1 subject was removed during Period 1 because of abnormal liver chemistries.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Dr. Peter V. Tishler
Organization: BrighamHospital
Phone: 617-525-2123
Responsible Party: Peter Tishler, Brigham and Women's Hospital
ClinicalTrials.gov Identifier: NCT01422915     History of Changes
Other Study ID Numbers: 2010P002253
First Submitted: August 23, 2011
First Posted: August 25, 2011
Results First Submitted: August 20, 2013
Results First Posted: April 25, 2017
Last Update Posted: April 25, 2017