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Conditions: SCID, Omenn's Syndrome, Reticular Dysgenesis, Wiskott-Aldrich Syndrome, Bare Lymphocyte Syndrome, Common Variable Immunodeficiency, Chronic Granulomatous Disease, CD40 Ligand Deficiency, Hyper IgM Syndrome, X-linked Lymphoproliferative Disease, Hemophagocytic Lymphohistiocytosis, Griscelli Syndrome, Chediak-Higashi Syndrome, Langerhan's Cell Histiocytosis
Interventions: Alemtuzumab 0.3 mg, Cyclophosphamide, Busulfan, Stem Cell Transplantation, Fludarabine phosphate 40 mg, Melphalan, Alemtuzumab 0.2 mg, Fludarabine phosphate 30 mg, MESNA; Drug · Biological
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Up to 50 Years
Enrollment: 57 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2026
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Cannabinoid Hyperemesis Syndrome
Interventions: Haloperidol; Drug
Lead sponsor: University of Illinois at Chicago; Other
Eligibility: 18 Years and older
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2026 – 2028
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: Idiopathic Pulmonary Fibrosis
Interventions: No intervention; Other
Lead sponsor: 23andMe, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 690 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2023
U.S. locations: 1
States / cities: Sunnyvale, California

Conditions: Hermansky-Pudlak Syndrome (HPS), Pulmonary Fibrosis, Oculocutaneous Albinism, Platelet Storage Pool Deficiency, Metabolic Disease
Interventions: Losartan, Zileuton, N-Acetylcysteine, Pravastatin, Erythromycin; Drug
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 18 Years to 70 Years
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2012
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Albinism, Oculocutaneous Albinism
Interventions: Levodopa/carbidopa; Drug
Lead sponsor: University of Wisconsin, Madison; Other
Eligibility: 3 Years and older
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2018
U.S. locations: 1
States / cities: Madison, Wisconsin

Conditions: Ocular Albinism (OA), Oculocutaneous Albinism (OCA)
Interventions: Lutein plus Zeaxanthin, Placebo; Dietary Supplement
Lead sponsor: Johns Hopkins University; Other
Eligibility: 12 Years and older
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2018
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders, Chediak-Higashi Syndrome, Griscelli Syndrome, Immunologic Diseases, Langerhans-Cell Histiocytosis, Hematologic Diseases
Interventions: Stem Cell Transplant, Myeloablative conditioning regimen; Procedure · Drug
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Up to 55 Years
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2015
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease
Interventions: Abatacept; Drug
Lead sponsor: Emory University; Other
Eligibility: Up to 21 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2019
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Oculocutaneous Albinism
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 1 Year to 80 Years
Enrollment: 206 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2019
U.S. locations: 1
States / cities: Bethesda, Maryland

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

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: Hermansky-Pudlak Syndrome (HPS)
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 1 Month to 115 Years
Enrollment: 600 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 1995
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Chediak Higashi Syndrome, Chronic Granulomatous Disease, Job's Syndrome, Leukocyte Disorder
Interventions: Not listed
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: Not listed
Enrollment: 150 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2005
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Disaster, Vulnerable Populations
Interventions: Nurses Taking On Readiness Measures (N- TORM); Behavioral
Lead sponsor: Hunter College of City University of New York; Other
Eligibility: 18 Years and older
Enrollment: 400 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2024
U.S. locations: 1
States / cities: New York, New York

Conditions: Indolent Systemic Mastocytosis
Interventions: Masitinib, Placebo, Best Supportive Care; Drug · Other
Lead sponsor: AB Science; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 135 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2015
U.S. locations: 2
States / cities: Sacramento, California • Houston, Texas

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: Albinism, Albinism, Ocular, Albinism, Oculocutaneous
Interventions: Not listed
Lead sponsor: National Eye Institute (NEI); NIH
Eligibility: Not listed
Enrollment: 130 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1976 – 2000
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders, Chediak-Higashi Syndrome, Griscelli Syndrome, Immunologic Deficiency Syndromes, Langerhans-Cell Histiocytosis
Interventions: Stem Cell Transplant, Fludarabine, Melphalan, Anti-thymocyte globulin (ATG), Campath 1H, Cyclosporin A, Mycophenolate mofetil, Intravenous immunoglobulin (IVIG); Procedure · Drug
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Up to 35 Years
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2014
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Hermanski-Pudlak Syndrome, Colitis, Cytokines, Lymphocytes, Drug Evaluation
Interventions: Mesalamine, Infliximab, Corticosteroids, 6-Mercaptopurine, Tacrolimus, Adalimumab; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2011
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Gastrointestinal Stromal Tumors, Small-cell Lung Cancer, Adenoid Cystic Carcinoma, Uveal Melanoma, Neuroendocrine Tumors, Chromophobe Renal Cell Carcinoma, Clear Cell Renal Cell Carcinoma
Interventions: NN3201; Drug
Lead sponsor: Novelty Nobility, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 67 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 5
States / cities: Ann Arbor, Michigan • Cleveland, Ohio • Portland, Oregon + 2 more

Conditions: Chediak-Higashi Syndrome
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 1 Month to 70 Years
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2002
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Mastocytosis
Interventions: Collection of blood and bone marrow; Other
Lead sponsor: University of Michigan; Other
Eligibility: Not listed
Enrollment: 136 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2009
U.S. locations: 1
States / cities: Ann Arbor, Michigan

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

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

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Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Haloperidol For Outpatient Symptom Management in Patients Diagnosed With Cannabinoid Hyperemesis Syndrome in the Emergrency Department

23andMe IPF Research Study

Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome

Vision Response to Dopamine Replacement

The Effects of Lutein and Zeaxanthin Supplementation on Vision in Patients With Albinism

T-Cell Depletion and Stem Cell Transplant for Immune Deficiencies and Histiocytic Disorders

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

Clinical, Cellular, and Molecular Investigation Into Oculocutaneous Albinism

Immune Disorder HSCT Protocol

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

Clinical and Basic Investigations Into Hermansky-Pudlak Syndrome

Learning and Behavior Problems in Children With Chronic Granulomatous Disease and Related Disorders

Korean Translation and Validation of the K-HEPI in NYC

Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis

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

Visual Function and Ocular Pigmentation in Albinism

Stem Cell Transplant for Immunologic or Histiocytic Disorders

Medical Treatment of Colitis in Patients With Hermansky-Pudlak Syndrome

A Study of the c-Kit Specific Antibody-Drug Conjugate NN3201 for Advanced and/or Metastatic Solid Tumors Known to Express c-Kit

Study of Chediak-Higashi Syndrome

Molecular Mechanisms and Diagnosis of Mastocytosis

Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies

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