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	NCT04193592 (ClinicalTrials.gov)	December 1, 2019	6/11/2019	Efficacy and Safety of Pirfenidone Treatment in HPS-ILD	Pirfenidone in the Treatment of Hermansky Pudlak Syndrome (HPS) - Related Interstitial Lung Disease (ILD)	Hermansky Pudlak Syndrome;Interstitial Lung Disease	Drug: Pirfenidone	Jesse Roman	Genentech, Inc.	Not yet recruiting	18 Years	75 Years	All	50	Phase 2	United States;Puerto Rico
2	NCT00001596 (ClinicalTrials.gov)	September 2005	3/11/1999	Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome	Therapeutic Clinical Trial of Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome	Albinism;Inborn Errors of Metabolism;Oculocutaneous Albinism;Platelet Storage Pool Deficiency;Pulmonary Fibrosis	Drug: Pirfenidone;Drug: Placebo	William Gahl, M.D.	National Human Genome Research Institute (NHGRI)	Completed	18 Years	N/A	All	35	Phase 2	United States;Puerto Rico

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

NCT04193592
(ClinicalTrials.gov)

December 1, 2019

6/11/2019

Efficacy and Safety of Pirfenidone Treatment in HPS-ILD

Pirfenidone in the Treatment of Hermansky Pudlak Syndrome (HPS) - Related Interstitial Lung Disease (ILD)

Hermansky Pudlak Syndrome;Interstitial Lung Disease

Drug: Pirfenidone

Jesse Roman

Genentech, Inc.

Not yet recruiting

18 Years

75 Years

All

Phase 2

United States;Puerto Rico

NCT00001596
(ClinicalTrials.gov)

September 2005

3/11/1999

Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome

Therapeutic Clinical Trial of Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome

Albinism;Inborn Errors of Metabolism;Oculocutaneous Albinism;Platelet Storage Pool Deficiency;Pulmonary Fibrosis

Drug: Pirfenidone;Drug: Placebo

William Gahl, M.D.

National Human Genome Research Institute (NHGRI)

Completed

18 Years

N/A

All

Phase 2

United States;Puerto Rico