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 164. 眼皮膚白皮症　［臨床試験数：14，薬物数：54（DrugBank：36），標的遺伝子数：34，標的パスウェイ数：132］ 

Searched query = "Oculocutaneous albinism", "Hermansky Pudlak syndrome", "Chediak Higashi syndrome", "Griscelli syndrome"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollement		Last_Refreshed_
on		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT02200263November 20148 January 2018The Effects of Lutein and Zeaxanthin Supplementation on Vision in Patients With AlbinismA 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: PlaceboJohns Hopkins UniversityClark Charitable Foundation Inc.Recruiting12 YearsN/AAll28N/AUnited States
2NCT01917708January 201419 November 2018BMT Abatacept for Non-Malignant DiseasesAbatacept for Post-Transplant Immune Suppression in Children and Adolescents Receiving Allogeneic Hematopoietic Stem Cell Transplants for Non-Malignant DiseasesHurler 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 DiseaseDrug: AbataceptEmory UniversityNot recruitingN/A21 YearsAll10Phase 1United States
3NCT01838655April 16, 201316 December 2017Nitisinone for Type 1B Oculocutaneous AlbinismA Pilot Study of Nitisinone in the Treatment of Oculocutaneous Albinism, Type 1BAlbinism;Vision LossDrug: NitisinoneNational Eye Institute (NEI)National Human Genome Research Institute (NHGRI)Not recruiting18 YearsN/AAll5Phase 1/Phase 2United States
4NCT01821781March 20138 April 2019Immune Disorder HSCT ProtocolA Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory RegimeImmune 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 SyndromeDrug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalanWashington University School of MedicineRecruitingN/A21 YearsAll20Phase 2United States
5NCT01652092September 4, 201228 January 2019Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune DeficienciesAllogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune DeficienciesSCID;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 HistiocytosisDrug: Fludarabine phosphate 30 mg;Drug: MESNA;Drug: Alemtuzumab 0.3 mg;Drug: Cyclophosphamide;Drug: Busulfan;Biological: Stem Cell Transplantation;Drug: Fludarabine phosphate 40 mg;Drug: Melphalan;Drug: Alemtuzumab 0.2 mgMasonic Cancer Center, University of MinnesotaRecruitingN/A50 YearsAll30N/AUnited States
No.TrialIDDate_
enrollement		Last_Refreshed_
on		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
6NCT01663935August 201216 December 2017Vision Response to Dopamine ReplacementInterventional Study of Levodopa Replacement on Retinal Function in Oculocutaneous AlbinismAlbinism;Oculocutaneous AlbinismDrug: Levodopa/carbidopaUniversity of Wisconsin, MadisonRecruiting3 YearsN/AAll50Phase 2United States
7NCT01319851September 201016 December 2017Alefacept and Allogeneic Hematopoietic Stem Cell TransplantationAlefacept and Allogeneic Hematopoietic Stem Cell Transplantation for Children With Non-Malignant Diseases Who Have Been Multiply Transfused: a Pilot StudyThalassemia;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 AnemiaDrug: AlefaceptEmory UniversityChildren's Healthcare of AtlantaNot recruitingN/A21 YearsAll3N/AUnited States
8NCT00514982August 7, 200716 December 2017Medical Treatment of Colitis in Patients With Hermansky-Pudlak SyndromeAn Observational Study of the Immunopathogenesis of and Response to Step-Up Inflammatory Bowel Disease Therapy for Hermansky-Pudlak Syndrome-Associated ColitisHermanski-Pudlak Syndrome;Colitis;Cytokines;Lymphocytes;Drug EvaluationDrug: Mesalamine;Drug: Infliximab;Drug: Corticosteroids;Drug: 6-Mercaptopurine;Drug: Tacrolimus;Drug: AdalimumabNational Institute of Allergy and Infectious Diseases (NIAID)Not recruiting18 YearsN/AAll0Phase 2United States
9NCT00467831April 200719 October 2017Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak SyndromePilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak SyndromeHermansky-Pudlak Syndrome (HPS);Pulmonary Fibrosis;Oculocutaneous Albinism;Platelet Storage Pool Deficiency;Metabolic DiseaseDrug: Losartan;Drug: Zileuton;Drug: N-Acetylcysteine;Drug: Pravastatin;Drug: ErythromycinNational Human Genome Research Institute (NHGRI)Not recruiting18 Years70 YearsAll3Phase 1/Phase 2United States
10NCT00001596September 200516 December 2017Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak SyndromeTherapeutic Clinical Trial of Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak SyndromeAlbinism;Inborn Errors of Metabolism;Oculocutaneous Albinism;Platelet Storage Pool Deficiency;Pulmonary FibrosisDrug: Pirfenidone;Drug: PlaceboWilliam Gahl, M.D.National Human Genome Research Institute (NHGRI)Not recruiting18 YearsN/AAll35Phase 2United States;Puerto Rico
No.TrialIDDate_
enrollement		Last_Refreshed_
on		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
11NCT00176865August 200216 December 2017Stem Cell Transplant for Immunologic or Histiocytic DisordersAllogeneic Hematopoietic Stem Cell Transplant for Patients With Immunologic or Histiocytic Disorders Using a Non-Myeloablative Preparative Regimen to Achieve Stable Mixed ChimerismHemophagocytic Lymphohistiocytosis;X-Linked Lymphoproliferative Disorders;Chediak-Higashi Syndrome;Griscelli Syndrome;Immunologic Deficiency Syndromes;Langerhans-Cell HistiocytosisProcedure: 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 MinnesotaNot recruitingN/A35 YearsAll19Phase 2United States
12NCT00176826September 200019 October 2017T-Cell Depletion and Stem Cell Transplant for Immune Deficiencies and Histiocytic DisordersIn-vivo T-cell Depletion and Hematopoietic Stem Cell Transplantation for Life-Threatening Immune Deficiencies and Histiocytic DisordersHemophagocytic Lymphohistiocytosis;X-Linked Lymphoproliferative Disorders;Chediak-Higashi Syndrome;Griscelli Syndrome;Immunologic Diseases;Langerhans-Cell Histiocytosis;Hematologic DiseasesProcedure: Stem Cell Transplant;Drug: Myeloablative conditioning regimenMasonic Cancer Center, University of MinnesotaNot recruitingN/A55 YearsAll22Phase 2/Phase 3United States
13NCT00006054March 200019 February 2015Allogeneic Bone Marrow Transplantation in Patients With Primary ImmunodeficienciesImmunologic 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 SyndromeDrug: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: cyclosporine;Drug: etoposide;Drug: methotrexate;Drug: methylprednisolone;Drug: prednisone;Procedure: Allogeneic Bone Marrow TransplantationFairview University Medical CenterNot recruitingN/A35 YearsBothN/AUnited States
14NCT00006056March 200019 February 2015Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic DisordersChediak-Higashi Syndrome;Graft Versus Host Disease;X-Linked Lymphoproliferative Syndrome;Familial Erythrophagocytic Lymphohistiocytosis;Hemophagocytic Lymphohistiocytosis;Virus-Associated Hemophagocytic SyndromeDrug: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: cyclosporine;Drug: etoposide;Drug: filgrastim;Drug: methotrexate;Procedure: allogeneic hematopoietic stem cell transplantationFairview University Medical CenterNot recruitingN/A55 YearsBoth40N/AUnited States

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