Clinical trials: Erythropoietic Protoporphyria
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
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
Does Exercise and Heat Increase the Lightsensibility in Patients With Erythropoietic Protoporphyria
The purpose of the study is to evaluate the effect of exercise and heat on the light sensibility of patients with erythropoietic protoporphyria
- Trial name
- NCT00206869
- Testing
- exercise, heat, red light illumination
- Where
- Denmark
- Sponsor
- Bispebjerg Hospital
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
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
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
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 (EPP), X-Linked Protoporphyria (XLP)
A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP)
PATHWAY is a Phase 2b/3 clinical study with an Open-Label Extension (OLE). It tests PORT-77 in patients with EPP, EPP2, and XLP.
- Conditions
- Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP), EPP2
- Trial name
- PATHWAY (NCT07820085)
- Testing
- PORT-77
- Where
- 2 sites in United States
- Volunteers
- 225 people
- Sponsor
- Portal Therapeutics, Inc.
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
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 (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