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Showing 1–24 of 40 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Albinism, Ocular Clear all
Completed Phase 2 Interventional Results available

A Study of the Efficacy and Safety of Imatinib Mesylate in Patients With Unresectable or Metastatic Gastrointestinal Stromal Tumors Expressing C-kit Gene

NCT00237185
View directory record Official record
Conditions
Unresectable or Metastatic Malignant Gastrointestinal Stromal Tumor (GIST)
Interventions
Imatinib mesylate
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
148 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2013
U.S. locations
3
States / cities
Boston, Massachusetts • Portland, Oregon • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 18, 2014 · Synced Jul 23, 2026, 2:58 PM EDT
Not listed Phase 2 Interventional

Efficacy and Safety of Pirfenidone Treatment in HPS-ILD

NCT04193592
View directory record Official record
Conditions
Hermansky Pudlak Syndrome, Interstitial Lung Disease
Interventions
Pirfenidone
Drug
Lead sponsor
Jesse Roman
Other
Eligibility
18 Years to 75 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 9, 2019 · Synced Jul 23, 2026, 2:58 PM EDT
Completed Phase 4 Interventional Results available

Study to Allow Access to Nilotinib for Patients Who Are on Nilotinib Treatment in a Novartis-sponsored Study

NCT01735955
View directory record Official record
Conditions
Chronic Myelogenous Leukemia (CML), Metastatic Gastrointestinal Stromal Tumors (GIST), Acute Lymphoblastic Leukemia (ALL), Receptor Tyrosine Kinase (KIT) Mutated Melanoma
Interventions
Nilotinib
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
2
States / cities
Albany, New York • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 7, 2024 · Synced Jul 23, 2026, 2:58 PM EDT
Completed Phase 2 Interventional Results available

Stem Cell Transplant for Immunologic or Histiocytic Disorders

NCT00176865
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 27, 2017 · Synced Jul 23, 2026, 2:58 PM EDT
Completed Phase 3 Interventional

Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis

NCT00814073
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 2, 2019 · Synced Jul 23, 2026, 2:58 PM EDT
Recruiting Phase 1 Interventional

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

NCT06805825
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 22, 2025 · Synced Jul 23, 2026, 2:58 PM EDT
Completed No phase listed Observational

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

NCT00005933
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 23, 2026, 2:58 PM EDT
Recruiting No phase listed Observational

Clinical and Basic Investigations Into Hermansky-Pudlak Syndrome

NCT00001456
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Jul 23, 2026, 2:58 PM 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 14, 2009 · Synced Jul 23, 2026, 2:58 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Allergy and Immunology Natural History Study

NCT05584488
View directory record Official record
Conditions
Immune Deficiency, Immune Dysregulation Disorder, Allergic Inflammation
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Up to 99 Years
Enrollment
10,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 3, 2026 · Synced Jul 23, 2026, 2:58 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

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

NCT00467831
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 1, 2013 · Synced Jul 23, 2026, 2:58 PM EDT
Active, not recruiting Not applicable Interventional

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

NCT01652092
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Jul 23, 2026, 2:58 PM EDT
Completed No phase listed Observational

A Longitudinal Study of Hermansky-Pudlak Syndrome Pulmonary Fibrosis

NCT02368340
View directory record Official record
Conditions
Hermansky Pudlak Syndrome
Interventions
Pulmonary function test, Chest CT, Sample collection
Other
Lead sponsor
Vanderbilt University
Other
Eligibility
12 Years to 90 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
5
States / cities
Maywood, Illinois • Boston, Massachusetts • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2020 · Synced Jul 23, 2026, 2:58 PM EDT
Completed No phase listed Observational

Clinical, Cellular, and Molecular Investigation Into Oculocutaneous Albinism

NCT00808106
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 5, 2020 · Synced Jul 23, 2026, 2:58 PM EDT
Completed No phase listed Observational

Visual Function and Ocular Pigmentation in Albinism

NCT00001153
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 23, 2026, 2:58 PM 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 18, 2026 · Synced Jul 23, 2026, 2:58 PM EDT
Completed Phase 2 Interventional Results available

Trial of L-DOPA as a Treatment to Improve Vision in Albinism

NCT01176435
View directory record Official record
Conditions
Albinism
Interventions
Levodopa, Placebo
Drug
Lead sponsor
University of Minnesota
Other
Eligibility
3 Years to 60 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated May 1, 2018 · Synced Jul 23, 2026, 2:58 PM EDT
Recruiting No phase listed Observational

Study of Chediak-Higashi Syndrome

NCT00005917
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 15, 2026 · Synced Jul 23, 2026, 2:58 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Korean Translation and Validation of the K-HEPI in NYC

NCT05544578
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 14, 2024 · Synced Jul 23, 2026, 2:58 PM EDT
Terminated Phase 2 Interventional Results available

Vision Response to Dopamine Replacement

NCT01663935
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 10, 2019 · Synced Jul 23, 2026, 2:58 PM EDT
Completed Phase 1Phase 2 Interventional

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

NCT00730314
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions
Hematopoietic stem cell transplantation
Procedure
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 22, 2016 · Synced Jul 23, 2026, 2:58 PM EDT
Completed Phase 1 Interventional

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

NCT01917708
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 25, 2019 · Synced Jul 23, 2026, 2:58 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Nurses Taking On Readiness Measures - Mercer County, New Jersey

NCT05455580
View directory record Official record
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
31 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 4, 2024 · Synced Jul 23, 2026, 2:58 PM 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 14, 2025 · Synced Jul 23, 2026, 2:58 PM EDT