DDrare: Database of Drug Development for Rare Diseases

164. 眼皮膚白皮症
［臨床試験数：15，薬物数：57（DrugBank：34），標的遺伝子数：34，標的パスウェイ数：136］

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	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
2	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

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

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

Phase 2

United States

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

Phase 2;Phase 3

United States