Protoporphyria, Erythropoietic clinical trials
The registry lists 29 studies for Protoporphyria, Erythropoietic across more than 10 countries. 5 are recruiting or about to start. 28 of them are mainly about Protoporphyria, Erythropoietic (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-05.
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Who runs them
Clinuvel Pharmaceuticals Limited (5) · Bispebjerg Hospital (4) · Disc Medicine, Inc (4) · Portal Therapeutics, Inc. (3) · Tanabe Pharma America, Inc. (2) · Icahn School of Medicine at Mount Sinai (2) · Brigham and Women's Hospital (2) · FDA Office of Orphan Products Development (1)
What is being tested here
Drugs under test in Protoporphyria, Erythropoietic studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
The study
- Mar 2027Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP) · Portal Therapeutics, Inc.
Who these studies take
Of the 3 Protoporphyria, Erythropoietic studies recruiting now, 3 (100%) set no upper age limit and 0 (0%) stop at 75 or younger. 3 (100%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 0 (0%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 1 (33%) | 1 (33%) |
| 18 | 3 (100%) | 3 (100%) |
| 30 | 3 (100%) | 3 (100%) |
| 40 | 3 (100%) | 3 (100%) |
| 50 | 3 (100%) | 3 (100%) |
| 65 | 3 (100%) | 3 (100%) |
| 75 | 3 (100%) | 3 (100%) |
| 85 | 3 (100%) | 3 (100%) |
Across all 29 studies, recruiting or not: 38% stop at 75 or younger and 10% at 65; 48% take children; 0% are for women only and 0% for men only; 3% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 29 of 29. search them.
- Expanded Access Program of Bitopertin For Participants With EPP or XLPAvailableNCT076034011 country
- HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLPEnrolling by invitationNCT05883748Phase 2/311 countries
- A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP)RecruitingNCT07820085Phase 2/31 country
- Longitudinal Study of the PorphyriasRecruitingNCT015611571 country
- Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)RecruitingNCT075671319 countries
- Study of Bitopertin in Participants With EPP or XLP (APOLLO)CompletedNCT06910358Phase 312 countries
- Effect of Oral Cimetidine in the ProtoporphyriasCompletedNCT05020184Phase 21 country
- GATEWAY: A Phase 2a Study of PORT-77 in Adults With Erythropoietic ProtoporphyriaCompletedNCT06971900Phase 21 country
- Pharmacokinetics of Afamelanotide in Erythropoietic Protoporphyria PatientsCompletedNCT06388642Phase 1/22 countries
- INcreased Sun Exposure Without Pain In Research Participants With EPP or XLPCompletedNCT06144840Phase 311 countries
- 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 BaselineWithdrawnNCT07123363Phase 1
- Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPPCompletedNCT05308472Phase 21 country
- Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic ProtoporphyriaCompletedNCT03520036Phase 21 country
- Protection Against Visible Light by Dihydroxyacetone in Erythropoietic ProtoporphyriaStatus unknownNCT05780840Not applicable1 country
- Iron Therapy in Erythropoietic ProtoporphyriaCompletedNCT055721251 country
- Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)CompletedNCT00979745Phase 36 countries
- Mitoferrin-1 Expression in Erythropoietic Protoporphyria (Porphyria Rare Disease Clinical Research Consortium (RDCRC))CompletedNCT018809831 country
- A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)CompletedNCT04578496Phase 3
- Oral Iron for Erythropoietic ProtoporphyriasCompletedNCT02979249Not applicable1 country
- Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial ImpactCompletedNCT016888951 country
- Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)CompletedNCT01097044Phase 21 country
- Multicentre Phase III Erythropoietic Protoporphyria StudyCompletedNCT04053270Phase 3
- Phase III Confirmatory Study in Erythropoietic ProtoporphyriaCompletedNCT01605136Phase 31 country
- Light Exposure Patterns and Symptoms Among Patients With Erythropoietic ProtoporphyriaCompletedNCT036827311 country
- Sorbent Therapy of the Cutaneous PorphyriasCompletedNCT01422915Phase 2/31 country
- Effect of Isoniazid on Protoporphyrin Levels in Erythropoietic ProtoporphyriaTerminatedNCT01550705Not applicable1 country
- Phase III Study of L-Cysteine in Patients With Erythropoietic ProtoporphyriaCompletedNCT00004940Phase 3
- Study of Cysteine Hydrochloride for Erythropoietic ProtoporphyriaCompletedNCT00004831Not applicable
- Does Exercise and Heat Increase the Lightsensibility in Patients With Erythropoietic ProtoporphyriaStatus unknownNCT00206869Not applicable1 country