Clinical trials: Erythropoietic Protoporphyria (EPP)
Erythropoietic Protoporphyria
Light Exposure Patterns and Symptoms Among Patients With Erythropoietic Protoporphyria
Background: Erythropoietic protoporphyria (EPP) is characterized by development of painful skin symptoms upon exposure to visible light dye to accumulation of the photoactive substance protoporphyrin IX (PpIX) in the skin.This…
- Trial name
- NCT03682731
- Where
- 2 sites in Denmark
- Volunteers
- 14 people
- Sponsor
- Bispebjerg Hospital
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, Iron-deficiency
Iron Therapy in Erythropoietic Protoporphyria
Giving oral iron to patients with erythropoietic protoporphyria (EPP) who is iron deficient is thought to be beneficial. This is, however, not well documented.
- Trial name
- NCT05572125
- Where
- Denmark
- Volunteers
- 10 people
- Sponsor
- Bispebjerg Hospital
Erythropoietic Protoporphyria (EPP), X-Linked Porphyria (XLP)
Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)
The purpose of this study is to collect information about the effects of EPP/XLP in adults and adolescents. This is an observational study in which participants will not receive any treatment.
- Trial name
- stEPP (NCT07567131)
- Where
- 19 sites in United States, Belgium and 7 more
- Volunteers
- 50 people
- Sponsor
- Portal Therapeutics, Inc.
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
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 (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.
Protoporphyria
Effect of Bitopertin on the Liver and on Levels of Protoporphyrin IX in Bile, Blood, Liver, and Stool in Patients With Erythropoietic Protoporphyria/X-linked Protoporphyria and Increased Liver Stiffness and/or Liver Enzymes at Baseline
The primary goal of this study is to assess safety and tolerability of bitopertin in subjects with Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP) and evidence of compensated liver disease.
- Trial name
- NCT07123363
- Testing
- bitopertin
- Sponsor
- Wake Forest University Health Sciences
Erythropoietic Protoporphyria
Phase III Study of L-Cysteine in Patients With Erythropoietic Protoporphyria
OBJECTIVES: I. Determine the long-term efficacy and safety of L-cysteine in the prevention photosensitivity in patients with erythropoietic protoporphyria.
- Trial name
- NCT00004940
- Testing
- cysteine hydrochloride
- Volunteers
- 50 people
- Sponsor
- Brigham and Women's Hospital
Erythropoietic Protoporphyria
Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria
OBJECTIVES: I. Determine the efficacy of cysteine hydrochloride in preventing or decreasing photosensitivity in patients with erythropoietic protoporphyria.
- Trial name
- NCT00004831
- Testing
- cysteine hydrochloride
- Volunteers
- 20 people
- Sponsor
- FDA Office of Orphan Products Development
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
Expanded Access Program of Bitopertin For Participants With EPP or XLP
The goal of this expanded access program is to provide bitopertin to patients with EPP and XLP who have no satisfactory treatment options available in the US and to learn if bitopertin is safe to treat EPP and XLP.
- Trial name
- NCT07603401
- Testing
- DISC-1459
- Where
- 7 sites in United States
- Sponsor
- Disc Medicine, Inc
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
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
Study of Bitopertin in Participants With EPP or XLP (APOLLO)
The goal of this clinical trial is to learn if bitopertin works and is safe to treat EPP or XLP in participants 12 years or older.
- Trial name
- APOLLO (NCT06910358)
- Testing
- DISC-1459
- Where
- 27 sites in United States, Australia and 10 more
- Volunteers
- 183 people
- Sponsor
- Disc Medicine, Inc
Erythropoietic Protoporphyria (EPP)
GATEWAY: A Phase 2a Study of PORT-77 in Adults With Erythropoietic Protoporphyria
A Phase 2a study of PORT-77 in adults with erythropoietic protoporphyria (EPP)
- Trial name
- EPP (NCT06971900)
- Testing
- PORT-77
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Portal Therapeutics, Inc.
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
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
EPP, XLP
Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
To evaluate the long-term safety and tolerability of oral dersimelagon.
- Trial name
- NCT05005975
- Testing
- MT-7117
- Where
- 45 sites in United States, Australia and 13 more
- Volunteers
- 286 people
- Sponsor
- Tanabe Pharma America, Inc.
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
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
Protection Against Visible Light by Dihydroxyacetone in Erythropoietic Protoporphyria
What is the visible light protective effect of dihydroxyacetone in individuals with erythropoietic protoporphyria? This will be tested in this randomized blinded placebo controlled study.
- Trial name
- NCT05780840
- Testing
- Dihydroxyacetone
- Where
- Denmark
- Volunteers
- 6 people
- Sponsor
- Bispebjerg Hospital