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	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 Deficiency;Metabolic Disease	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

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

NCT00467831
(ClinicalTrials.gov)

April 2007

28/4/2007

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 Deficiency;Metabolic Disease

Drug: Losartan;Drug: Zileuton;Drug: N-Acetylcysteine;Drug: Pravastatin;Drug: Erythromycin

National Human Genome Research Institute (NHGRI)

NULL

Terminated

18 Years

70 Years

All

Phase 1;Phase 2

United States