164. 眼皮膚白皮症 Oculocutaneous albinism Clinical trials / Disease details

臨床試験数 : 15 / 薬物数 : 54 - (DrugBank : 34) / 標的遺伝子数 : 35 - 標的パスウェイ数 : 141

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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 ...	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 Supplementa ...	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 He ...	Hurler Syndrome;Fanconi Anemia;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic Granulomato ...	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 Reduc ...	Immune Deficiency Disorders;Severe Combined Immunodeficiency;Chronic Granulomatous Disease;X-linked ...	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 ...	Drug: Alemtuzumab 0.3 mg;Drug: Cyclophosphamide;Drug: Busulfan;Biological: Stem Cell Transplantation; ...	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 Dis ...	Thalassemia;Sickle Cell Disease;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic-granulomat ...	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 Disea ...	Hermanski-Pudlak Syndrome;Colitis;Cytokines;Lymphocytes;Drug Evaluation	Drug: Mesalamine;Drug: Infliximab;Drug: Corticosteroids;Drug: 6-Mercaptopurine;Drug: Tacrolimus;Drug ...	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 Defi ...	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

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