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	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

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

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

N/A

United States