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Showing 1–24 of 41 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Porphyrias Clear all
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 8, 2026, 5:37 PM 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 4, 2026 · Synced Sep 8, 2026, 5:37 PM 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 8, 2026, 5:37 PM EDT
Recruiting No phase listed Observational

Longitudinal Study of the Porphyrias

NCT01561157
View directory record Official record
Conditions
Acute Porphyrias, Cutaneous Porphyrias
Interventions
Not listed
Lead sponsor
The American Porphyrias Expert Collaborative
Other
Eligibility
1 Minute and older
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2030
U.S. locations
16
States / cities
Birmingham, Alabama • Los Angeles, California • San Francisco, California + 13 more
Source: ClinicalTrials.gov public record
Updated May 14, 2026 · Synced Sep 8, 2026, 5:37 PM EDT
Completed No phase listed Observational Results available

Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial Impact

NCT01688895
View directory record Official record
Conditions
Erythropoietic Protoporphyria, EPP, X-Linked Protoporphyria, XLP, XLPP, X-Linked Dominant Erythropoietic Protoporphyria, XLEPP, XLDP
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
Not listed
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
6
States / cities
Birmingham, Alabama • San Francisco, California • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 16, 2020 · Synced Sep 8, 2026, 5:37 PM 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 8, 2026, 5:37 PM EDT
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 8, 2026, 5:37 PM 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 8, 2026, 5:37 PM 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 8, 2026, 5:37 PM 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 23, 2005 · Synced Sep 8, 2026, 5:37 PM EDT
Recruiting No phase listed Observational

Identification of Acute Intermittent Porphyria Modifying Genes

NCT05502133
View directory record Official record
Conditions
Acute Intermittent Porphyria (AIP)
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
12 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 27, 2025 · Synced Sep 8, 2026, 5:37 PM 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 8, 2026, 5:37 PM 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 8, 2026, 5:37 PM 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 8, 2026, 5:37 PM 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 8, 2026, 5:37 PM EDT
Completed Phase 2Phase 3 Interventional Accepts healthy volunteers Results available

Sorbent Therapy of the Cutaneous Porphyrias

NCT01422915
View directory record Official record
Conditions
Erythropoietic Protoporphyria
Interventions
Colestipol
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
22 Years to 60 Years
Enrollment
4 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2012
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 24, 2017 · Synced Sep 8, 2026, 5:37 PM 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 8, 2026, 5:37 PM EDT
Completed Phase 3 Interventional Results available

ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)

NCT03338816
View directory record Official record
Conditions
Acute Hepatic Porphyria, Acute Intermittent Porphyria, Porphyria, Acute Intermittent, Acute Porphyria, Hereditary Coproporphyria (HCP), Variegate Porphyria (VP), ALA Dehydratase Deficient Porphyria (ADP)
Interventions
Givosiran, Placebo
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
10
States / cities
Little Rock, Arkansas • San Francisco, California • Boston, Massachusetts + 7 more
Source: ClinicalTrials.gov public record
Updated Apr 21, 2024 · Synced Sep 8, 2026, 5:37 PM 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 8, 2026, 5:37 PM 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 23, 2026 · Synced Sep 8, 2026, 5:37 PM EDT
Terminated Not applicable Interventional

The Effect of Low Intensity Exercise on Quality of Life and Hemodynamics in Patients With Permanent Atrial Fibrillation

NCT00680290
View directory record Official record
Conditions
Atrial Fibrillation
Interventions
Exercise
Procedure
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 15, 2019 · Synced Sep 8, 2026, 5:37 PM EDT
Terminated Not applicable Interventional Results available

Effect of Isoniazid on Protoporphyrin Levels in Erythropoietic Protoporphyria

NCT01550705
View directory record Official record
Conditions
Erythropoietic Protoporphyria (EPP), X Linked Erythropoietic Protoporphyria
Interventions
Isoniazid
Drug
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Jan 15, 2017 · Synced Sep 8, 2026, 5:37 PM EDT
Completed Phase 2 Interventional Results available

Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)

NCT01097044
View directory record Official record
Conditions
Erythropoietic Protoporphyria
Interventions
Afamelanotide, Placebo
Drug
Lead sponsor
Clinuvel Pharmaceuticals Limited
Industry
Eligibility
18 Years and older
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
6
States / cities
Birmingham, Alabama • San Francisco, California • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 27, 2019 · Synced Sep 8, 2026, 5:37 PM 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 8, 2026, 5:37 PM EDT