Investigation into the use of colestyramine as a therapy for patients with erythropoietic protoporphyria - EPP001

Status

Active, not recruiting

Phases

Phase 4

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2007-002863-28-GB

Enrollment

Registered

2007-08-15

Start date

2007-09-26

Completion date

Unknown

Last updated

2018-11-26

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Erythropoietic protoporphyria (EPP) MedDRA version: 9.1 Level: LLT Classification code 10015289 Term: Erythropoietic protoporphyria

Interventions

Trade Name: Questran Product Name: Questran Pharmaceutical Form: Powder for oral suspension INN or Proposed INN: COLESTYRAMINE CAS Number: 11041126 Concentration unit: g gram(s) Concentration type: eq

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Patients who regularly attend the Photodermatoses clinic at St Thomas’ Hospital and meet the inclusion criteria below will be invited to participate in this trial: - •Patients with a diagnosis of EPP. •Patients aged 18 years or over. •Patients who are willing and able to give written informed consent to participate in the trial. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: •Patients under 18 years of age •Patients who do not adequately understand verbal explanations or written information given in English, or who have special communication needs and unable to give informed consent. •Patients who are pregnant or breast feeding. •Patients who are unwilling or unable to comply with the study protocol •Patients with complete biliary obstruction •Patients who have diabetes mellitus •Patients with epilepsy •Patients prescribed thyroxine sodium for hypothyroidism

Design outcomes

Primary

Measure	Time frame
Main Objective: Erythropoietic protoporphyria (EPP) is an inherited disease in which protoporphyrin accumulates in red blood cells and causes 2-3 day long bouts of severe pain following exposure of the skin to sunlight. It is a lifelong, severe and disabling condition for which there is no effective treatment. Colestyramine is an anion-exchange resin. It has been shown to bind to protoporphyrin (Stathers, 1966) in the gut and to increase its faecal excretion (McCullough et al., 1988; Bloomer, 1979) (by interrupting the porphyrin's enterohepatic recirculation). The main objective of this trial is to assess whether oral colestyramine is likely to be a useful therapy in EPP, by seeing whether it reduces the plasma and red cell protoporphyrin concentration in EPP patients when given over a three month period. ;Secondary Objective: None;Primary end point(s): Does colestyramine decrease the plasma and red cell protoporphyrin concentration by at least 15% in EPP (15% chosen because it is the level below which a fall in plasma and red cell protoporphyrin is not likely to be clinically significant in terms of symptom control).	—

Measure

Time frame

Main Objective: Erythropoietic protoporphyria (EPP) is an inherited disease in which protoporphyrin accumulates in red blood cells and causes 2-3 day long bouts of severe pain following exposure of the skin to sunlight. It is a lifelong, severe and disabling condition for which there is no effective treatment. Colestyramine is an anion-exchange resin. It has been shown to bind to protoporphyrin (Stathers, 1966) in the gut and to increase its faecal excretion (McCullough et al., 1988; Bloomer, 1979) (by interrupting the porphyrin's enterohepatic recirculation). The main objective of this trial is to assess whether oral colestyramine is likely to be a useful therapy in EPP, by seeing whether it reduces the plasma and red cell protoporphyrin concentration in EPP patients when given over a three month period. ;Secondary Objective: None;Primary end point(s): Does colestyramine decrease the plasma and red cell protoporphyrin concentration by at least 15% in EPP (15% chosen because it is the level below which a fall in plasma and red cell protoporphyrin is not likely to be clinically significant in terms of symptom control).

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Countries

United Kingdom

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026