DDrare: Database of Drug Development for Rare Diseases

Searched query = "Oculocutaneous albinism", "Hermansky-Pudlak syndrome", "Chediak-Higashi syndrome", "Griscelli syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = 6-Mercaptopurine; Abatacept; Acetylcysteine; Adalimumab; Alefacept; Alemtuzumab; Alemtuzumab 0.2 mg; Alemtuzumab 0.3 mg; Anti-thymocyte globulin; Anti-thymocyte globulin (ATG); Busulfan; Campath; Campath 1H; Carbidopa; Corticosteroids; Cyclophosphamide; Cyclosporin; Cyclosporin A; Cyclosporine; Erythromycin; Etoposide; Filgrastim; Fludarabine; Fludarabine phosphate 30 mg; Fludarabine phosphate 40 mg; Infliximab; Intravenous immunoglobulin (IVIG); Levodopa; Levodopa/carbidopa; Losartan; Lutein; Lutein plus Zeaxanthin; MESNA; Melphalan; Mercaptopurine; Mesalamine; Mesna; Methotrexate; Methylprednisolone; Mycophenolate; Mycophenolate mofetil; Myeloablative conditioning regimen; N-Acetylcysteine; Nitisinone; Phosphate; Pirfenidone; Pravastatin; Prednisone; Procedure: Allogeneic Bone Marrow Transplantation; Procedure: Stem Cell Transplant; Procedure: allogeneic hematopoietic stem cell transplantation; Stem Cell Transplantation; Tacrolimus; Thiotepa; Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan; Zeaxanthin; Zileuton

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	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

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NCT01652092
(ClinicalTrials.gov)

September 4, 2012

25/7/2012

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

N/A

United States