Clinical trials: Erythropoietic Protoporphyria
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
Acute Porphyrias, Cutaneous Porphyrias
Longitudinal Study of the Porphyrias
The objective of this protocol is to conduct a longitudinal multidisciplinary investigation of the human porphyrias including the natural history, morbidity, pregnancy outcomes, and mortality in people with these disorders.
- Trial name
- NCT01561157
- Where
- 16 sites in United States
- Volunteers
- 1,500 people
- Sponsor
- The American Porphyrias Expert Collaborative
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
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
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
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
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 (EPP), X-Linked Protoporphyria (XLP)
INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP
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 adolescents with EPP or XLP.
- Trial name
- INSPIRE (NCT06144840)
- Testing
- Dersimelagon
- Where
- 35 sites in United States, Australia and 9 more
- Volunteers
- 165 people
- Sponsor
- Tanabe Pharma America, 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
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 (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 (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 (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
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 (EPP)
Mitoferrin-1 Expression in Erythropoietic Protoporphyria (Porphyria Rare Disease Clinical Research Consortium (RDCRC))
The purpose of this study is to identify the biochemical/genetic defects in erythropoietic protoporphyria (EPP). People with EPP have skin sensitivity to sunlight and occasionally develop liver disease.
- Trial name
- NCT01880983
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- University of Alabama at Birmingham
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
HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP
This is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.
- Trial name
- NCT05883748
- Testing
- DISC-1459
- Where
- 26 sites in United States, Australia and 9 more
- Volunteers
- 230 people
- Sponsor
- Disc Medicine, 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