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ClinicalTrials.gov public records Last synced Oct 5, 2026, 7:26 AM EDT

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Showing 25–41 of 41 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Porphyria, Erythropoietic Clear all
Completed Phase 2Phase 3 Interventional

Porphozym in the Treatment of Acute Attacks in AIP

NCT00418795
View directory record Official record
Conditions
Acute Intermittent Porphyria
Interventions
recombinant human porphobilinogen deaminase (Porphozym)
Drug
Lead sponsor
Zymenex A/S
Industry
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 9, 2018 · Synced Oct 5, 2026, 7:26 AM EDT
Recruiting No phase listed Observational

Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)

NCT07567131
View directory record Official record
Conditions
Erythropoietic Protoporphyria (EPP), X-Linked Porphyria (XLP)
Interventions
Not listed
Lead sponsor
Portal Therapeutics, Inc.
Industry
Eligibility
12 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
5
States / cities
Boston, Massachusetts • New York, New York • Wake Forest, North Carolina + 2 more
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Oct 5, 2026, 7:26 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 3, 2026 · Synced Oct 5, 2026, 7:26 AM EDT
Not listed No phase listed Observational

Studies in Porphyria I: Characterization of Enzyme Defects

NCT00004331
View directory record Official record
Conditions
Porphyria
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1992
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 5, 2026, 7:26 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Investigate the Improvement of Photosensitivity in Terms of Skin Lesions Associated With CEP Following Administration of Oral ATL-001 (Ciclopirox Oral Solution) in Participants Aged >18 Years of Age With CEP

NCT07024316
View directory record Official record
Conditions
Congenital Erythropoietic Porphyria (CEP)
Interventions
ATL-001 (Ciclopirox oral solution)
Drug
Lead sponsor
Atlas Molecular Pharma
Industry
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced Oct 5, 2026, 7:26 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 28, 2021 · Synced Oct 5, 2026, 7:26 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 Oct 1, 2026 · Synced Oct 5, 2026, 7:26 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 30, 2026 · Synced Oct 5, 2026, 7:26 AM EDT
Completed Phase 2 Interventional

Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute Porphyrias

NCT00004396
View directory record Official record
Conditions
Porphyria
Interventions
heme arginate, tin mesoporphyrin
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1997
U.S. locations
2
States / cities
New York, New York • Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 5, 2026, 7:26 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 4, 2026 · Synced Oct 5, 2026, 7:26 AM EDT
Completed No phase listed Observational

Mitoferrin-1 Expression in Erythropoietic Protoporphyria (Porphyria Rare Disease Clinical Research Consortium (RDCRC))

NCT01880983
View directory record Official record
Conditions
Erythropoietic Protoporphyria (EPP)
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
7 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2020
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Jun 24, 2021 · Synced Oct 5, 2026, 7:26 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 16, 2019 · Synced Oct 5, 2026, 7:26 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 24, 2005 · Synced Oct 5, 2026, 7:26 AM EDT
Completed Not applicable Interventional

Studies in Porphyria IV: Gonadotropin-Releasing Hormone (GnRH) Analogues for Prevention of Cyclic Attacks

NCT00004330
View directory record Official record
Conditions
Porphyria
Interventions
luteinizing hormone-releasing factor
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 55 Years · Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1987
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 5, 2026, 7:26 AM EDT
Completed Phase 3 Interventional Results available

Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

NCT00599326
View directory record Official record
Conditions
Porphyria Cutanea Tarda
Interventions
Deferasirox
Drug
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Feb 10, 2014 · Synced Oct 5, 2026, 7:26 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 2, 2026 · Synced Oct 5, 2026, 7:26 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 4, 2026 · Synced Oct 5, 2026, 7:26 AM EDT