Clinical trials: X-Linked Protoporphyria
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), 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 (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, 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), 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 (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.
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
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
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 (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