164. Oculocutaneous albinism Clinical trials / Disease details
Clinical trials : 15 / Drugs : 54 - (DrugBank : 34) / Drug target genes : 35 - Drug target pathways : 141
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04193592 (ClinicalTrials.gov) | December 1, 2019 | 6/11/2019 | Efficacy and Safety of Pirfenidone Treatment in HPS-ILD | Pirfenidone in the Treatment of Hermansky Pudlak Syndrome (HPS) - Related Interstitial Lung Disease (ILD) | Hermansky Pudlak Syndrome;Interstitial Lung Disease | Drug: Pirfenidone | Jesse Roman | Genentech, Inc. | Not yet recruiting | 18 Years | 75 Years | All | 50 | Phase 2 | United States;Puerto Rico |
| 2 | NCT02200263 (ClinicalTrials.gov) | November 2014 | 23/7/2014 | The Effects of Lutein and Zeaxanthin Supplementation on Vision in Patients With Albinism | A Randomized Placebo-controlled Trial to Investigate the Effect of Lutein and Zeaxanthin Supplementation on Macular Pigment and Visual Function in Albinism - LUtein for VIsion in Albinism (LUVIA) | Ocular Albinism (OA);Oculocutaneous Albinism (OCA) | Dietary Supplement: Lutein plus Zeaxanthin;Dietary Supplement: Placebo | Johns Hopkins University | Clark Charitable Foundation Inc. | Completed | 12 Years | N/A | All | 10 | N/A | United States |
| 3 | NCT01917708 (ClinicalTrials.gov) | January 2014 | 24/7/2013 | Bone Marrow Transplant With Abatacept for Non-Malignant Diseases | Abatacept for Post-Transplant Immune Suppression in Children and Adolescents Receiving Allogeneic Hematopoietic Stem Cell Transplants for Non-Malignant Diseases | 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 | Drug: Abatacept | Emory University | NULL | Completed | N/A | 21 Years | All | 10 | Phase 1 | United States |
| 4 | NCT01838655 (ClinicalTrials.gov) | April 16, 2013 | 20/4/2013 | Nitisinone for Type 1B Oculocutaneous Albinism | A Pilot Study of Nitisinone in the Treatment of Oculocutaneous Albinism, Type 1B | Albinism;Vision Loss | Drug: Nitisinone | National Eye Institute (NEI) | National Human Genome Research Institute (NHGRI) | Completed | 18 Years | N/A | All | 5 | Phase 1/Phase 2 | United States |
| 5 | NCT01821781 (ClinicalTrials.gov) | March 2013 | 19/3/2013 | Immune Disorder HSCT Protocol | A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime | 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 | Drug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan | Washington University School of Medicine | NULL | Recruiting | N/A | 21 Years | All | 20 | Phase 2 | United States |
| 6 | NCT01663935 (ClinicalTrials.gov) | October 17, 2012 | 9/8/2012 | Vision Response to Dopamine Replacement | Interventional Study of Levodopa Replacement on Retinal Function in Oculocutaneous Albinism | Albinism;Oculocutaneous Albinism | Drug: Levodopa/carbidopa | University of Wisconsin, Madison | NULL | Terminated | 3 Years | N/A | All | 20 | Phase 2 | United States |
| 7 | NCT01652092 (ClinicalTrials.gov) | September 4, 2012 | 25/7/2012 | Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies | Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies | 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 | Drug: Alemtuzumab 0.3 mg;Drug: Cyclophosphamide;Drug: Busulfan;Biological: Stem Cell Transplantation;Drug: Fludarabine phosphate 40 mg;Drug: Melphalan;Drug: Alemtuzumab 0.2 mg;Drug: Fludarabine phosphate 30 mg;Drug: MESNA | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 50 Years | All | 30 | N/A | United States |
| 8 | NCT01319851 (ClinicalTrials.gov) | September 2010 | 15/9/2010 | Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation | Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation for Children With Non-Malignant Diseases Who Have Been Multiply Transfused: a Pilot Study | Thalassemia;Sickle Cell Disease;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic-granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Schwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Fanconi Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic Anemia | Drug: Alefacept | Emory University | Children's Healthcare of Atlanta | Terminated | N/A | 21 Years | All | 3 | N/A | United States |
| 9 | NCT00514982 (ClinicalTrials.gov) | August 7, 2007 | 9/8/2007 | Medical Treatment of Colitis in Patients With Hermansky-Pudlak Syndrome | An Observational Study of the Immunopathogenesis of and Response to Step-Up Inflammatory Bowel Disease Therapy for Hermansky-Pudlak Syndrome-Associated Colitis | Hermanski-Pudlak Syndrome;Colitis;Cytokines;Lymphocytes;Drug Evaluation | Drug: Mesalamine;Drug: Infliximab;Drug: Corticosteroids;Drug: 6-Mercaptopurine;Drug: Tacrolimus;Drug: Adalimumab | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Withdrawn | 18 Years | N/A | All | 0 | Phase 2 | United States |
| 10 | NCT00467831 (ClinicalTrials.gov) | April 2007 | 28/4/2007 | Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome | Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome | Hermansky-Pudlak Syndrome (HPS);Pulmonary Fibrosis;Oculocutaneous Albinism;Platelet Storage Pool Deficiency;Metabolic Disease | Drug: Losartan;Drug: Zileuton;Drug: N-Acetylcysteine;Drug: Pravastatin;Drug: Erythromycin | National Human Genome Research Institute (NHGRI) | NULL | Terminated | 18 Years | 70 Years | All | 3 | Phase 1/Phase 2 | United States |
| 11 | NCT00001596 (ClinicalTrials.gov) | September 2005 | 3/11/1999 | Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome | Therapeutic Clinical Trial of Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome | Albinism;Inborn Errors of Metabolism;Oculocutaneous Albinism;Platelet Storage Pool Deficiency;Pulmonary Fibrosis | Drug: Pirfenidone;Drug: Placebo | William Gahl, M.D. | National Human Genome Research Institute (NHGRI) | Completed | 18 Years | N/A | All | 35 | Phase 2 | United States;Puerto Rico |
| 12 | NCT00176865 (ClinicalTrials.gov) | August 2002 | 12/9/2005 | Stem Cell Transplant for Immunologic or Histiocytic Disorders | Allogeneic Hematopoietic Stem Cell Transplant for Patients With Immunologic or Histiocytic Disorders Using a Non-Myeloablative Preparative Regimen to Achieve Stable Mixed Chimerism | Hemophagocytic Lymphohistiocytosis;X-Linked Lymphoproliferative Disorders;Chediak-Higashi Syndrome;Griscelli Syndrome;Immunologic Deficiency Syndromes;Langerhans-Cell Histiocytosis | Procedure: Stem Cell Transplant;Drug: Fludarabine;Drug: Melphalan;Drug: Anti-thymocyte globulin (ATG);Drug: Campath 1H;Drug: Cyclosporin A;Drug: Mycophenolate mofetil;Drug: Intravenous immunoglobulin (IVIG) | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | 35 Years | All | 19 | Phase 2 | United States |
| 13 | NCT00176826 (ClinicalTrials.gov) | September 2000 | 12/9/2005 | T-Cell Depletion and Stem Cell Transplant for Immune Deficiencies and Histiocytic Disorders | In-vivo T-cell Depletion and Hematopoietic Stem Cell Transplantation for Life-Threatening Immune Deficiencies and Histiocytic Disorders | Hemophagocytic Lymphohistiocytosis;X-Linked Lymphoproliferative Disorders;Chediak-Higashi Syndrome;Griscelli Syndrome;Immunologic Diseases;Langerhans-Cell Histiocytosis;Hematologic Diseases | Procedure: Stem Cell Transplant;Drug: Myeloablative conditioning regimen | Masonic Cancer Center, University of Minnesota | NULL | Unknown status | N/A | 55 Years | All | 22 | Phase 2/Phase 3 | United States |
| 14 | NCT00006056 (ClinicalTrials.gov) | March 2000 | 5/7/2000 | Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders | Chediak-Higashi Syndrome;Graft Versus Host Disease;X-Linked Lymphoproliferative Syndrome;Familial Erythrophagocytic Lymphohistiocytosis;Hemophagocytic Lymphohistiocytosis;Virus-Associated Hemophagocytic Syndrome | Drug: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: cyclosporine;Drug: etoposide;Drug: filgrastim;Drug: methotrexate;Procedure: allogeneic hematopoietic stem cell transplantation | Fairview University Medical Center | NULL | Active, not recruiting | N/A | 55 Years | Both | 40 | N/A | United States | |
| 15 | NCT00006054 (ClinicalTrials.gov) | March 2000 | 5/7/2000 | Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies | 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 | Drug: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: cyclosporine;Drug: etoposide;Drug: methotrexate;Drug: methylprednisolone;Drug: prednisone;Procedure: Allogeneic Bone Marrow Transplantation | Fairview University Medical Center | NULL | Terminated | N/A | 35 Years | Both | N/A | United States |