Trial Outcomes & Findings for Sorbent Therapy of the Cutaneous Porphyrias (NCT NCT01422915)

NCT ID: NCT01422915

Last Updated: 2017-04-25

Results Overview

Minutes of sun tolerance

• Recruitment status: COMPLETED
• Study phase: PHASE2/PHASE3
• Target enrollment: 4 participants
• Primary outcome timeframe: At 60 days of treatment
• Results posted on: 2017-04-25

Participant Flow

Dates: February and March 2011.Location types: suburbs of Massachusetts (3 subjects) and in New Jersey (1).

All 4 subjects were enrolled in the study. Initially, baseline blood protoporphyrin concentrations and answers to sun sensitivity questionnaire were obtained.

Participant milestones

Measure	Colestipol Treatment Colestipol 2 grams twice daily for 5-6 months. Completion of sun sensitivity questionnaire and blood protoporphyrin concentrations. were obtained monthly.
Overall Study STARTED	4
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	Colestipol Treatment Colestipol 2 grams twice daily for 5-6 months. Completion of sun sensitivity questionnaire and blood protoporphyrin concentrations. were obtained monthly.
Overall Study Adverse Event	1

Baseline Characteristics

Sorbent Therapy of the Cutaneous Porphyrias

Baseline characteristics by cohort

Measure	Colestipol Therapy of Protoporphyria n=4 Participants 4 subjects were assigned, all between 18-65 years of age.
Age, Categorical <=18 years	0 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=93 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants
Age, Continuous	43 years STANDARD_DEVIATION 15 • n=93 Participants
Age, Customized <=18 years	0 participants n=93 Participants
Age, Customized Between 18 and 65 years	4 participants n=93 Participants
Age, Customized >=65 years	0 participants n=93 Participants
Sex: Female, Male Female	4 Participants n=93 Participants
Sex: Female, Male Male	0 Participants n=93 Participants
Region of Enrollment United States	4 participants n=93 Participants

PRIMARY outcome

Timeframe: At 60 days of treatment

Population: 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

Minutes of sun tolerance

Outcome measures

Measure	Colestipol Treatment n=3 Participants 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.
Photosensitivity, Assessed by Measuring the Number of Minutes of Sun Tolerance	65.8 minutes Standard Deviation 58.8

PRIMARY outcome

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

1. erythrocyte protoporphyrin concentration, ug/dl

2. plasma protoporphyrin concentration, ug/dl

Outcome measures

Measure	Colestipol Treatment n=3 Participants 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.
Protoporphyrin Concentration in Blood Erythrocyte Protoporphyrin	1712 ug/dl Standard Deviation 455
Protoporphyrin Concentration in Blood Plasma Protoporphyrin	5.8 ug/dl Standard Deviation 8.3

Adverse Events

1st Period - Colestipol Optimal Dosage

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	1st Period - Colestipol Optimal Dosage n=4 participants at risk 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.
Hepatobiliary disorders Abnormal liver enzymes	25.0% 1/4 • Number of events 1

Other adverse events

Adverse event data not reported

Additional Information

Dr. Peter V. Tishler

BrighamHospital

Phone: 617-525-2123

Email: repvt@channing.harvard.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place