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	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 Disease Therapy for Hermansky-Pudlak Syndrome-Associated Colitis	Hermanski-Pudlak Syndrome;Colitis;Cytokines;Lymphocytes;Drug Evaluation	Drug: Mesalamine;Drug: Infliximab;Drug: Corticosteroids;Drug: 6-Mercaptopurine;Drug: Tacrolimus;Drug: Adalimumab	National Institute of Allergy and Infectious Diseases (NIAID)	NULL	Withdrawn	18 Years	N/A	All	0	Phase 2	United States

No.

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Date_
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registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

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Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

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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 Disease Therapy for Hermansky-Pudlak Syndrome-Associated Colitis

Hermanski-Pudlak Syndrome;Colitis;Cytokines;Lymphocytes;Drug Evaluation

Drug: Mesalamine;Drug: Infliximab;Drug: Corticosteroids;Drug: 6-Mercaptopurine;Drug: Tacrolimus;Drug: Adalimumab

National Institute of Allergy and Infectious Diseases (NIAID)

NULL

Withdrawn

18 Years

N/A

All

Phase 2

United States