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Conditions: Lymphoproliferative Disorders, Lymphoma
Interventions: Not listed
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 8 Years and older
Enrollment: 77 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2012
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: NLRC4-MAS, XIAP Deficiency
Interventions: Tadekinig alfa, 0.9% sodium chloride; Drug · Other
Lead sponsor: AB2 Bio Ltd.; Industry
Eligibility: Not listed
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2023
U.S. locations: 6
States / cities: La Jolla, California • Atlanta, Georgia • Boston, Massachusetts + 3 more

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

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: 2
States / cities: Miami, Florida • Boston, Massachusetts

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

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

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

Conditions: NLRC4-GOF, AIFEC (Autoinflammation With Infantile Enterocolitis), XIAP Deficiency, CDC42 Mutations
Interventions: MAS825, Placebo; Biological
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 0 Years to 100 Years
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

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

Conditions: EPP, XLP
Interventions: Placebo, MT-7117 Low Dose, MT-7117 High Dose; Drug
Lead sponsor: Tanabe Pharma America, Inc.; Industry
Eligibility: 12 Years to 75 Years
Enrollment: 184 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2022
U.S. locations: 10
States / cities: Huntington Beach, California • Miami, Florida • Brighton, Massachusetts + 7 more

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 – 2026
U.S. locations: 2
States / cities: Houston, Texas

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

Conditions: EPP, XLP
Interventions: MT-7117; Drug
Lead sponsor: Tanabe Pharma America, Inc.; Industry
Eligibility: 12 Years to 75 Years
Enrollment: 301 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 15
States / cities: Huntington Beach, California • San Francisco, California • Miami, Florida + 11 more

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

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

Conditions: ALPS, Hypersplenism, Lymphadenopathy
Interventions: Valproic Acid, CT Scan, Blood Sample; Drug · Procedure
Lead sponsor: Koneti Rao; NIH
Eligibility: 2 Years to 70 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2011
U.S. locations: 1
States / cities: Bethesda, Maryland

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

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

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

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

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

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

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

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

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Fluorodeoxyglucose-Positron Emission Tomography (FDG-PET) to Evaluate Autoimmune Lymphoproliferative Syndrome (ALPS) and ALPS-associated Lymphoma

Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency

Sirolimus for Autoimmune Disease of Blood Cells

Expanded Access Program of Bitopertin For Participants With EPP or XLP

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

Pyrimethamine to Treat Autoimmune Lymphoproliferative Syndrome

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

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

Oral Iron for Erythropoietic Protoporphyrias

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

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

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

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

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

Longitudinal Study of the Porphyrias

Valproic Acid (Depakote[Registered Trademark]) to Treat Autoimmune Lymphoproliferative Syndrome (ALPS)

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

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

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

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

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

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

Pyrimethamine and Sulfadoxine for Treatment of Autoimmune Lymphoproliferative Syndrome

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