Trial record 12 of 13 for:    "Protoporphyria"

Sorbent Therapy of the Cutaneous Porphyrias (EPP)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01422915
Recruitment Status : Completed
First Posted : August 25, 2011
Results First Posted : April 25, 2017
Last Update Posted : April 25, 2017
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

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Dates: February and March 2011.Location types: suburbs of Massachusetts (3 subjects) and in New Jersey (1).

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
All 4 subjects were enrolled in the study. Initially, baseline blood protoporphyrin concentrations and answers to sun sensitivity questionnaire were obtained.

Reporting Groups
Colestipol Treatment Colestipol 2 grams twice daily for 5-6 months. Completion of sun sensitivity questionnaire and blood protoporphyrin concentrations. were obtained monthly.

Participant Flow:   Overall Study
    Colestipol Treatment
STARTED   4 [1] 
Adverse Event                1 
[1] 1 subject with abnormal liver chemistries removed during the first period.

  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
Colestipol Therapy of Protoporphyria 4 subjects were assigned, all between 18-65 years of age.

Baseline Measures
   Colestipol Therapy of Protoporphyria 
Overall Participants Analyzed 
[Units: Participants]
[Units: Participants]
Count of Participants
<=18 years      0   0.0% 
Between 18 and 65 years      4 100.0% 
>=65 years      0   0.0% 
[Units: Years]
Mean (Standard Deviation)
 43  (15) 
Age, Customized [1] 
[Units: Participants]
<=18 years   0 
Between 18 and 65 years   4 
>=65 years   0 
[1] 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 
[Units: Participants]
Count of Participants
Female      4 100.0% 
Male      0   0.0% 
Region of Enrollment 
[Units: Participants]
United States   4 

  Outcome Measures

1.  Primary:   Photosensitivity, Assessed by Measuring the Number of Minutes of Sun Tolerance   [ Time Frame: At 60 days of treatment ]

2.  Primary:   Protoporphyrin Concentration in Blood   [ Time Frame: Samples collected while on treatment (range 93-208 treatment days) ]

  Serious Adverse Events

  Other Adverse Events

  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
1 subject was removed during Period 1 because of abnormal liver chemistries.

  More Information

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 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