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ClinicalTrials.gov public records Last synced Oct 18, 2026, 8:19 AM EDT

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Showing 1–24 of 30 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: XLP Clear all
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
7
States / cities
Miami, Florida • Boston, Massachusetts • Detroit, Michigan + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 18, 2026, 8:19 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 18, 2026, 8:19 AM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Comparative Autoantibody and Immunologic Cell Marker Study

NCT02422875
View directory record Official record
Conditions
Autoimmune Diseases, Lupus Erythematosus, Systemic, Communicable Diseases
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
18 Years and older
Enrollment
2,500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2028
U.S. locations
3
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 18, 2026, 8:19 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 11, 2020 · Synced Oct 18, 2026, 8:19 AM EDT
Not listed Not applicable Interventional

Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders

NCT00006056
View directory record Official record
Conditions
Chediak-Higashi Syndrome, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome, Familial Erythrophagocytic Lymphohistiocytosis, Hemophagocytic Lymphohistiocytosis, Virus-Associated Hemophagocytic Syndrome
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, etoposide, filgrastim, methotrexate, allogeneic hematopoietic stem cell transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years to 55 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 8:19 AM EDT
Recruiting Phase 2 Interventional

Study of the ITK Inhibitor Soquelitinib to Reduce Lymphoproliferation and Improve Cytopenias in Autoimmune Lymphoproliferative Syndrome (ALPS)-FAS Patients

NCT06730126
View directory record Official record
Conditions
Autoimmune Lymphoproliferative Syndrome
Interventions
Soquelitinib
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
16 Years to 120 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
3
States / cities
Bethesda, Maryland • Philadelphia, Pennsylvania • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 18, 2026, 8:19 AM EDT
Active, not recruiting Phase 1 Interventional

CASPALLO: Allodepleted T Cells Transduced With Inducible Caspase 9 Suicide Gene

NCT00710892
View directory record Official record
Conditions
Acute Lymphoblastic Leukemia, Non-Hodgkin's Lymphoma, Myelodysplastic Syndrome, Chronic Myeloid Leukemia
Interventions
Allodepleted T Cells
Biological
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Up to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2027
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Oct 18, 2026, 8:19 AM EDT
Terminated Not applicable Interventional

Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies

NCT00006054
View directory record Official record
Conditions
Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome, Common Variable Immunodeficiency, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome, Familial Erythrophagocytic Lymphohistiocytosis, Hemophagocytic Lymphohistiocytosis, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, X-linked Hyper IgM Syndrome, Severe Combined Immunodeficiency, Leukocyte Adhesion Deficiency Syndrome, Virus-Associated Hemophagocytic Syndrome
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, etoposide, methotrexate, methylprednisolone, prednisone, Allogeneic Bone Marrow Transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
Up to 35 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2002
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 15, 2009 · Synced Oct 18, 2026, 8:19 AM EDT
Completed Phase 3 Interventional

Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency As Open Label Extension

NCT03512314
View directory record Official record
Conditions
XIAP Deficiency, NLRC4-MAS
Interventions
Tadekinig alfa
Drug
Lead sponsor
AB2 Bio Ltd.
Industry
Eligibility
Not listed
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
8
States / cities
La Jolla, California • Gainsville, Florida • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Mar 17, 2025 · Synced Oct 18, 2026, 8:19 AM EDT
Active, not recruiting Phase 2 Interventional

Immune Disorder HSCT Protocol

NCT01821781
View directory record Official record
Conditions
Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Hyper-IgM, DiGeorge Syndrome, Chediak-Higashi Syndrome, Common Variable Immune Deficiency, Immune Dysregulatory Disorders, Hemophagocytic Lymphohistiocytosis, IPEX, Autoimmune Lymphoproliferative Syndrome, X-linked Lymphoproliferative Syndrome
Interventions
Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
Up to 21 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Feb 19, 2026 · Synced Oct 18, 2026, 8:19 AM EDT
Recruiting No phase listed Observational

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

NCT04528355
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 18, 2026, 8:19 AM EDT
Completed Phase 1 Interventional

Pyrimethamine and Sulfadoxine for Treatment of Autoimmune Lymphoproliferative Syndrome

NCT00013689
View directory record Official record
Conditions
Autoimmune Disease, Lymphoproliferative Disorder
Interventions
Fansidar (pyrimethamine and sulfadoxine)
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 18, 2026, 8:19 AM EDT
Active, not recruiting Phase 2 Interventional

Study to Evaluate the Efficacy, Safety and Tolerability of MAS825 in Patients With Monogenic IL-18 Driven Autoinflammatory Diseases, Including NLRC4-GOF, XIAP Deficiency, or CDC42 Mutations

NCT04641442
View directory record Official record
Conditions
NLRC4-GOF, AIFEC (Autoinflammation With Infantile Enterocolitis), XIAP Deficiency, CDC42 Mutations
Interventions
MAS825, Placebo
Biological
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
0 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2032
U.S. locations
4
States / cities
Cincinnati, Ohio • Philadelphia, Pennsylvania • Houston, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 9, 2026 · Synced Oct 18, 2026, 8:19 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 9, 2026 · Synced Oct 18, 2026, 8:19 AM EDT
Recruiting Phase 2 Interventional

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

NCT01962415
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions
Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 18, 2026, 8:19 AM 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 17, 2020 · Synced Oct 18, 2026, 8:19 AM EDT
Active, not recruiting Phase 3 Interventional

Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

NCT05005975
View directory record Official record
Conditions
EPP, XLP
Interventions
MT-7117
Drug
Lead sponsor
Tanabe Pharma America, Inc.
Industry
Eligibility
12 Years to 75 Years
Enrollment
286 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
13
States / cities
Huntington Beach, California • San Francisco, California • Miami, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 18, 2026, 8:19 AM 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 15, 2026 · Synced Oct 18, 2026, 8:19 AM EDT
Recruiting No phase listed Observational

Study of Autoimmune Lymphoproliferative Syndrome (ALPS)

NCT00001350
View directory record Official record
Conditions
Benign Lymphoproliferative Disorder
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Up to 99 Years
Enrollment
1,200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1993
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 18, 2026, 8:19 AM EDT
Completed Phase 2 Interventional Results available

T Cell Depletion for Recipients of HLA Haploidentical Related Donor Stem Cell Grafts

NCT00368355
View directory record Official record
Conditions
Acute Lymphoblastic Leukemia, Non Hodgkins Lymphoma, Myelodysplastic Syndrome, Acute Myeloid Leukemia, Chronic Myelogenous Leukemia, Hemophagocytic Lymphohistiocytosis (HLH), Familial Hemophagocytic Lymphohistiocytosis (FLH), Viral-associated Hemophagocytic Syndrome (VAHS), X-linked Lymphoproliferative Disease (XLP)
Interventions
Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation, Stem Cell Infusion
Drug · Biological · Radiation + 1 more
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Up to 55 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2016
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 21, 2020 · Synced Oct 18, 2026, 8:19 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 18, 2026, 8:19 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Sirolimus for Autoimmune Disease of Blood Cells

NCT00392951
View directory record Official record
Conditions
Autoimmune Pancytopenia, Autoimmune Lymphoproliferative Syndrome (ALPS), Evans Syndrome, Idiopathic Thrombocytopenic Purpura, Anemia, Hemolytic, Autoimmune, Autoimmune Neutropenia, Lupus Erythematosus, Systemic, Inflammatory Bowel Disease, Rheumatoid Arthritis
Interventions
sirolimus
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
1 Year to 30 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2016
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 19, 2019 · Synced Oct 18, 2026, 8:19 AM EDT
Completed Phase 1 Interventional

Pyrimethamine to Treat Autoimmune Lymphoproliferative Syndrome

NCT00065390
View directory record Official record
Conditions
Autoimmune Disease, Lymphatic Disease, Lymphoproliferative Disorder
Interventions
Pyrimethamine
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 18, 2026, 8:19 AM EDT
Recruiting Phase 1 Interventional

Quercetin in Patients With XIAP (X-linked Inhibitor of Apoptosis) Deficiency

NCT07433621
View directory record Official record
Conditions
XIAP Deficiency
Interventions
Quercetin
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
2 Years and older · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 27, 2026 · Synced Oct 18, 2026, 8:19 AM EDT