Clinical trials: EPP
Erythropoietic Protoporphyria
Sorbent Therapy of the Cutaneous Porphyrias
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…
- Trial name
- EPP (NCT01422915)
- Testing
- Colestipol
- Where
- United States
- Volunteers
- 4 people, healthy volunteers welcome
- Sponsor
- Brigham and Women's Hospital
Erythropoietic Protoporphyria
Pharmacokinetics of Afamelanotide in Erythropoietic Protoporphyria Patients
The purpose of this study is to evaluate the concentration of afamelanotide in serum after the administration of afamelanotide in adolescent and adult EPP patients.
- Trial name
- NCT06388642
- Testing
- Afamelanotide 16mg implant
- Where
- 2 sites in Belgium, Netherlands
- Volunteers
- 28 people
- Sponsor
- Clinuvel Europe Limited
Erythropoietic Protoporphyria
Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)
This is a randomized placebo-controlled study to be conducted in two parallel study arms for a six month period (three doses). Approximately 10 eligible patients per center will be enrolled and will receive afamelanotide (16 mg…
- Trial name
- NCT01097044
- Testing
- Afamelanotide
- Where
- 6 sites in United States
- Volunteers
- 77 people
- Sponsor
- Clinuvel Pharmaceuticals Limited
Erythropoietic Protoporphyria, EPP
Oral Iron for Erythropoietic Protoporphyrias
In the medical literature there are conflicting reports on whether iron improves symptoms in patients with EPP and XLP. Giving iron to people who are iron deficient is thought to improve EPP symptoms.
- Conditions
- Erythropoietic Protoporphyria, EPP, X-linked Protoporphyria, XLP
- Trial name
- EPP (NCT02979249)
- Testing
- Oral Iron
- Where
- 6 sites in United States
- Volunteers
- 16 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Erythropoietic Protoporphyria
Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP
This is a Phase 2, multi-center, double-blind, placebo-controlled, parallel group study of bitopertin to evaluate the safety, tolerability, efficacy, and PPIX concentration change in participants with EPP.
- Trial name
- NCT05308472
- Testing
- DISC-1459
- Where
- 9 sites in United States
- Volunteers
- 75 people
- Sponsor
- Disc Medicine, Inc
Erythropoietic Protoporphyria (EPP)
Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria
The purpose of this study is to investigate the efficacy and safety of MT-7117 on sunlight exposure duration without symptoms and tolerance in subjects with EPP.
- Trial name
- NCT03520036
- Testing
- MT-7117 low dose, MT-7117 high dose
- Where
- 9 sites in United States
- Volunteers
- 102 people
- Sponsor
- Tanabe Pharma America, Inc.
Erythropoietic Protoporphyria
Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)
Afamelanotide is a man-made drug being studied for use as a preventative medication for EPP sufferers. It is a synthetically produced analogue of human alpha melanocyte stimulating hormone (alpha-MSH) and is not yet available on…
- Trial name
- NCT00979745
- Testing
- Afamelanotide
- Where
- 8 sites in Finland, France and 4 more
- Volunteers
- 74 people
- Sponsor
- Clinuvel Pharmaceuticals Limited
Erythropoietic Protoporphyria (EPP), X Linked Erythropoietic Protoporphyria
Effect of Isoniazid on Protoporphyrin Levels in Erythropoietic Protoporphyria
In erythropoietic protoporphyria there is an accumulation of protoporphyrin IX (PPIX) in the plasma and liver. The reason it builds up is either the last step to make heme, insertion of iron into PPIX, is rate limiting or there…
- Trial name
- INHEPP (NCT01550705)
- Testing
- Isoniazid
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- University of Utah
Erythropoietic Protoporphyria, X-linked Protoporphyria
Effect of Oral Cimetidine in the Protoporphyrias
Erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) result from genetic defects of heme biosynthesis that cause life-long, painful cutaneous sensitivity to light.
- Trial name
- NCT05020184
- Testing
- Cimetidine
- Where
- 3 sites in United States
- Volunteers
- 26 people
- Sponsor
- Amy K. Dickey, M.D.
Erythropoietic Protoporphyria, EPP
Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial Impact
The initial objective of this protocol is to assemble a well-documented group of patients with confirmed diagnoses of the erythropoietic protoporphyrias, including autosomal recessive Erythropoietic Protoporphyria (EPP) and…
- Conditions
- Erythropoietic Protoporphyria, EPP, X-Linked Protoporphyria, XLP
- Trial name
- NCT01688895
- Where
- 6 sites in United States
- Volunteers
- 150 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Erythropoietic Protoporphyria
Multicentre Phase III Erythropoietic Protoporphyria Study
This was a phase III, multicentre, randomised, double-blind, placebo-controlled study, to evaluate the safety and efficacy of subcutaneous bioresorbable afamelanotide implants in patients with Erythropoietic Protoporphyria (EPP).
- Trial name
- NCT04053270
- Testing
- Afamelanotide
- Volunteers
- 100 people
- Sponsor
- Clinuvel Pharmaceuticals Limited
EPP, XLP
Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria or X-Linked Protoporphyria
The primary objective of this study is to investigate the efficacy of MT-7117 on time to onset and severity of first prodromal symptoms (burning, tingling, itching, or stinging) associated with sunlight exposure in adults and…
- Trial name
- NCT04402489
- Testing
- MT-7117 Low Dose, MT-7117 High Dose
- Where
- 31 sites in United States, Australia and 8 more
- Volunteers
- 184 people
- Sponsor
- Tanabe Pharma America, Inc.
Erythropoietic Protoporphyria
A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)
The main objectives of the study were to evaluate the safety and tolerability of afamelanotide by measuring treatment emergent adverse events (AEs) and to determine whether afamelanotide can improve the quality of life of EPP…
- Trial name
- NCT04578496
- Testing
- Afamelanotide
- Volunteers
- 16 people
- Sponsor
- Clinuvel Pharmaceuticals Limited
Erythropoietic Protoporphyria
Phase III Confirmatory Study in Erythropoietic Protoporphyria
This is a randomized placebo-controlled study to be conducted in two parallel study arms for a six month period (three doses). Between 75 and 100 eligible patients will be enrolled.
- Trial name
- NCT01605136
- Testing
- Afamelanotide
- Where
- 7 sites in United States
- Volunteers
- 93 people
- Sponsor
- Clinuvel Pharmaceuticals Limited