Porphyrias clinical trials
The registry lists 33 studies for Porphyrias across more than 10 countries. 3 are recruiting or about to start. 9 of them are mainly about Porphyrias (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-05. Counting its 3 narrower conditions too, such as Protoporphyria, Erythropoietic: 61 studies, 7 open.
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Who runs them
National Center for Research Resources (NCRR) (8) · Alnylam Pharmaceuticals (6) · Disc Medicine, Inc (4) · The University of Texas Medical Branch, Galveston (3) · Digna Biotech S.L. (2) · Icahn School of Medicine at Mount Sinai (2) · The American Porphyrias Expert Collaborative (1) · Brigham and Women's Hospital (1)
What is being tested here
Drugs under test in Porphyrias 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
- Dec 2026Acute Porphyria Biomarkers for Disease Activity · The University of Texas Medical Branch, Galveston
Who these studies take
Of the 1 Porphyrias studies recruiting now, 1 (100%) set no upper age limit and 0 (0%) stop at 75 or younger. 1 (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 (100%) | 1 (100%) |
| 18 | 1 (100%) | 1 (100%) |
| 30 | 1 (100%) | 1 (100%) |
| 40 | 1 (100%) | 1 (100%) |
| 50 | 1 (100%) | 1 (100%) |
| 65 | 1 (100%) | 1 (100%) |
| 75 | 1 (100%) | 1 (100%) |
| 85 | 1 (100%) | 1 (100%) |
Across all 33 studies, recruiting or not: 21% stop at 75 or younger and 21% at 65; 42% take children; 3% 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 33 of 33. 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
- Longitudinal Study of the PorphyriasRecruitingNCT015611571 country
- Study of Bitopertin in Participants With EPP or XLP (APOLLO)CompletedNCT06910358Phase 312 countries
- Acute Porphyria Biomarkers for Disease ActivityActive, not recruitingNCT029354001 country
- Panhematin for Prevention of Acute Attacks of PorphyriaTerminatedNCT02922413Phase 21 country
- Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPPCompletedNCT05308472Phase 21 country
- Expanded Access Protocol of Givosiran for Patients With Acute Hepatic PorphyriaApproved for marketingNCT04056481
- Dental Health, Diet, Inflammation and Biomarkers in Patients With Acute Intermittent Porphyria(AIP)Active, not recruitingNCT016176421 country
- ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)CompletedNCT03338816Phase 318 countries
- A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)CompletedNCT02949830Phase 1/23 countries
- Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea TardaCompletedNCT01573754Phase 21 country
- Colombia National Porphyria RegistryStatus unknownNCT054969331 country
- Mitoferrin-1 Expression in Erythropoietic Protoporphyria (Porphyria Rare Disease Clinical Research Consortium (RDCRC))CompletedNCT018809831 country
- EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)CompletedNCT0224078420 countries
- Oral Iron for Erythropoietic ProtoporphyriasCompletedNCT02979249Not applicable1 country
- Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial ImpactCompletedNCT016888951 country
- Evidence-based Assessment of Medication Sensitivity in Acute Hepatic PorphyriaCompletedNCT039062141 country
- A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP)CompletedNCT03505853Phase 11 country
- A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)CompletedNCT02452372Phase 13 countries
- 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
- Phase I Gene Therapy Clinical Trial Using the Vector rAAV2/5-PBGD for the Treatment of Acute Intermittent PorphyriaCompletedNCT02082860Phase 11 country
- Observational Study of Acute Intermittent Porphyria PatientsCompletedNCT020767631 country
- Phase I/II Study of Heme Arginate and Tin Mesoporphyrin for Acute PorphyriaCompletedNCT00004789Phase 1/2
- Studies in Porphyria IV: Gonadotropin-Releasing Hormone (GnRH) Analogues for Prevention of Cyclic AttacksCompletedNCT00004330Not applicable1 country
- Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute PorphyriasCompletedNCT00004396Phase 21 country
- Study of the Pathogenesis of Porphyria Cutanea TardaCompletedNCT000051031 country
- Study of Nutritional Factors in PorphyriaCompletedNCT00004788
- Phase I Study of Tin Mesoporphyrin in Patients on Long Term Heme Therapy for Prevention of Acute Attacks of PorphyriaCompletedNCT00004397Phase 1
- Phase I Study of Heme Arginate With or Without Tin Mesoporphyrin in Patients With Acute Attacks of PorphyriaCompletedNCT00004398Phase 1
- Studies in Porphyria I: Characterization of Enzyme DefectsStatus unknownNCT000043311 country