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Showing 1–24 of 36 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Hepatic Porphyrias Clear all
Completed Phase 2 Interventional

GATEWAY: A Phase 2a Study of PORT-77 in Adults With Erythropoietic Protoporphyria

NCT06971900
View directory record Official record
Conditions
Erythropoietic Protoporphyria (EPP)
Interventions
PORT-77, Placebo
Drug
Lead sponsor
Portal Therapeutics, Inc.
Industry
Eligibility
18 Years to 80 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025
U.S. locations
1
States / cities
Tempe, Arizona
Source: ClinicalTrials.gov public record
Updated Jul 28, 2026 · Synced Sep 13, 2026, 9:18 AM EDT
Completed Phase 2 Interventional Results available

Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP

NCT05308472
View directory record Official record
Conditions
Erythropoietic Protoporphyria
Interventions
DISC-1459, Placebo
Drug
Lead sponsor
Disc Medicine, Inc
Industry
Eligibility
18 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
9
States / cities
Birmingham, Alabama • San Francisco, California • Miami, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Jan 7, 2026 · Synced Sep 13, 2026, 9:18 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)

NCT02949830
View directory record Official record
Conditions
Acute Intermittent Porphyria
Interventions
Givosiran
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
3
States / cities
San Francisco, California • New York, New York • Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 11, 2024 · Synced Sep 13, 2026, 9:18 AM EDT
Completed Phase 3 Interventional

INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP

NCT06144840
View directory record Official record
Conditions
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
Interventions
Dersimelagon, Placebo
Drug
Lead sponsor
Tanabe Pharma America, Inc.
Industry
Eligibility
12 Years to 75 Years
Enrollment
165 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
13
States / cities
Huntington Beach, California • San Francisco, California • Miami, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Sep 13, 2026, 9:18 AM EDT
Recruiting No phase listed Observational

ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)

NCT04883905
View directory record Official record
Conditions
Acute Hepatic Porphyria
Interventions
Not listed
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2030
U.S. locations
13
States / cities
Birmingham, Alabama • Gilbert, Arizona • San Francisco, California + 10 more
Source: ClinicalTrials.gov public record
Updated Sep 7, 2026 · Synced Sep 13, 2026, 9:18 AM EDT
Terminated No phase listed Observational

INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP

NCT03547297
View directory record Official record
Conditions
Acute Hepatic Porphyria, Hepatic Porphyrias, Acute Intermittent Porphyria (AIP), Hereditary Coproporphyria (HCP), Variegate Porphyria (VP), ALA Dehydratase Deficient Porphyria (ADP), Porphyria Acute
Interventions
Not listed
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
16 Years and older
Enrollment
212 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
12
States / cities
Pasadena, California • Jacksonville, Florida • Margate, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Jan 24, 2019 · Synced Sep 13, 2026, 9:18 AM EDT
Completed Not applicable Interventional Results available

Oral Iron for Erythropoietic Protoporphyrias

NCT02979249
View directory record Official record
Conditions
Erythropoietic Protoporphyria, EPP, X-linked Protoporphyria, XLP
Interventions
Oral Iron
Drug
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
6
States / cities
Birmingham, Alabama • San Francisco, California • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 10, 2020 · Synced Sep 13, 2026, 9:18 AM EDT
Completed Phase 2 Interventional Results available

Effect of Oral Cimetidine in the Protoporphyrias

NCT05020184
View directory record Official record
Conditions
Erythropoietic Protoporphyria, X-linked Protoporphyria
Interventions
Cimetidine, Placebo
Drug
Lead sponsor
Amy K. Dickey, M.D.
Other
Eligibility
15 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
3
States / cities
Boston, Massachusetts • Winston-Salem, North Carolina • Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Sep 13, 2026, 9:18 AM EDT
Completed Phase 1 Interventional

A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)

NCT02452372
View directory record Official record
Conditions
Acute Intermittent Porphyria
Interventions
givosiran (ALN-AS1), Sterile Normal Saline (0.9% NaCl)
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 65 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
4
States / cities
Birmingham, Alabama • San Francisco, California • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 13, 2018 · Synced Sep 13, 2026, 9:18 AM EDT
Enrolling by invitation Phase 2Phase 3 Interventional

HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP

NCT05883748
View directory record Official record
Conditions
Erythropoietic Protoporphyria
Interventions
DISC-1459
Drug
Lead sponsor
Disc Medicine, Inc
Industry
Eligibility
12 Years and older
Enrollment
230 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
11
States / cities
Birmingham, Alabama • San Francisco, California • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Sep 13, 2026, 9:18 AM EDT
Active, not recruiting No phase listed Observational

Evaluating the Prevalence of Acute Hepatic Porphyria in Postural Tachycardia Syndrome

NCT05344599
View directory record Official record
Conditions
Postural Orthostatic Tachycardia Syndrome, Acute Hepatic Porphyria
Interventions
Autonomic Function Testing, Genetic Testing, Urine Testing, Blood laboratory Testing
Diagnostic Test
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years to 65 Years
Enrollment
70 participants
Timeline
2022 – 2026
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Sep 13, 2026, 9:18 AM EDT
Completed No phase listed Observational

Clinical Diagnosis of Acute Porphyria

NCT01568554
View directory record Official record
Conditions
Hereditary Coproporphyria (HCP), Acute Intermittent Porphyria (AIP), Variegate Porphyria (VP)
Interventions
Not listed
Lead sponsor
University of California, San Francisco
Other
Eligibility
15 Years and older
Enrollment
148 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2018
U.S. locations
6
States / cities
Birmingham, Alabama • San Francisco, California • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 27, 2021 · Synced Sep 13, 2026, 9:18 AM EDT
Completed No phase listed Observational

Evidence-based Assessment of Medication Sensitivity in Acute Hepatic Porphyria

NCT03906214
View directory record Official record
Conditions
Porphyrias
Interventions
Web-based Survey
Other
Lead sponsor
University of South Florida
Other
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Oct 15, 2019 · Synced Sep 13, 2026, 9:18 AM EDT
Terminated Phase 2 Interventional Results available

Panhematin for Prevention of Acute Attacks of Porphyria

NCT02922413
View directory record Official record
Conditions
Acute Intermittent Porphyria, Hereditary Coproporphyria, Variegate Porphyria
Interventions
Hemin for injection, Placebo
Biological · Other
Lead sponsor
The University of Texas Medical Branch, Galveston
Other
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2023
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 29, 2026 · Synced Sep 13, 2026, 9:18 AM EDT
Completed No phase listed Observational

Study of the Pathogenesis of Porphyria Cutanea Tarda

NCT00005103
View directory record Official record
Conditions
Porphyria Cutanea Tarda
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Sep 13, 2026, 9:18 AM EDT
Active, not recruiting Phase 3 Interventional

Study of Bitopertin in Participants With EPP or XLP (APOLLO)

NCT06910358
View directory record Official record
Conditions
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
Interventions
Placebo, DISC-1459
Drug
Lead sponsor
Disc Medicine, Inc
Industry
Eligibility
12 Years and older
Enrollment
183 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
11
States / cities
Huntington Beach, California • San Francisco, California • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Sep 13, 2026, 9:18 AM EDT
Completed Phase 2 Interventional Results available

Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria

NCT03520036
View directory record Official record
Conditions
Erythropoietic Protoporphyria (EPP)
Interventions
MT-7117 low dose, MT-7117 high dose, Placebo
Drug
Lead sponsor
Tanabe Pharma America, Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
102 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
9
States / cities
Los Angeles, California • San Francisco, California • Miami, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Dec 30, 2025 · Synced Sep 13, 2026, 9:18 AM EDT
Completed Phase 2 Interventional Results available

Controlled Trial of Panhematin in Treatment of Acute Attacks of Porphyria

NCT02180412
View directory record Official record
Conditions
Acute Porphyrias
Interventions
Panhematin, Glucose
Biological · Other
Lead sponsor
The University of Texas Medical Branch, Galveston
Other
Eligibility
18 Years to 100 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2022
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 10, 2025 · Synced Sep 13, 2026, 9:18 AM EDT
Available No phase listed Expanded access

Expanded Access Program of Bitopertin For Participants With EPP or XLP

NCT07603401
View directory record Official record
Conditions
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
Interventions
DISC-1459
Drug
Lead sponsor
Disc Medicine, Inc
Industry
Eligibility
12 Years and older
U.S. locations
5
States / cities
Miami, Florida • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 2, 2026 · Synced Sep 13, 2026, 9:18 AM EDT
Completed No phase listed Observational

EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)

NCT02240784
View directory record Official record
Conditions
Acute Hepatic Porphyria
Interventions
Not listed
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
136 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
8
States / cities
San Francisco, California • Miami, Florida • Ann Arbor, Michigan + 5 more
Source: ClinicalTrials.gov public record
Updated May 16, 2021 · Synced Sep 13, 2026, 9:18 AM EDT
Active, not recruiting No phase listed Observational

Acute Porphyria Biomarkers for Disease Activity

NCT02935400
View directory record Official record
Conditions
Acute Intermittent Porphyria, Hereditary Coproporphyria, Variegate Porphyria
Interventions
Hemin
Drug
Lead sponsor
The University of Texas Medical Branch, Galveston
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2026
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced Sep 13, 2026, 9:18 AM EDT
Completed Phase 3 Interventional Results available

Phase III Confirmatory Study in Erythropoietic Protoporphyria

NCT01605136
View directory record Official record
Conditions
Erythropoietic Protoporphyria
Interventions
Afamelanotide, Placebo
Drug
Lead sponsor
Clinuvel Pharmaceuticals Limited
Industry
Eligibility
18 Years and older
Enrollment
93 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
7
States / cities
Birmingham, Alabama • San Francisco, California • Detroit, Michigan + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 18, 2019 · Synced Sep 13, 2026, 9:18 AM EDT
Completed Phase 2 Interventional Results available

Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda

NCT01573754
View directory record Official record
Conditions
Porphyria Cutanea Tarda
Interventions
Hydroxychloroquine, Phlebotomy
Drug · Procedure
Lead sponsor
The University of Texas Medical Branch, Galveston
Other
Eligibility
18 Years to 100 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2021
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 16, 2023 · Synced Sep 13, 2026, 9:18 AM EDT
Completed Phase 2 Interventional Results available

Harvoni Treatment Porphyria Cutanea Tarda

NCT03118674
View directory record Official record
Conditions
Porphyria Cutanea Tarda, Hepatitis C
Interventions
Harvoni
Drug
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
18 Years and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
3
States / cities
San Francisco, California • Winston-Salem, North Carolina • Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 2, 2023 · Synced Sep 13, 2026, 9:18 AM EDT