DDrare: Database of Drug Development for Rare Diseases

Searched query = "Peroxisomal disease (except Adrenoleukodystrophy)", "Peroxisomal disease", "Peroxisomal disorder", "Peroxisome biogenesis disorder", "PEX gene disord ... show all
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 = ALN-65585; ALN-GO1; CA; CALD HR-D (High-Risk, Regimen C); CALD HR-D (High-Risk, Regimen D); CALD SR-A (Standard-Risk, Regimen A); CALD SR-B (Standard-Risk, Regimen B); CHOLIC ACID; Campath; Campath-1H; Chenodeoxycholic acid; Cholic Acid; Cholic Acids; Cholic acid; Clofarabine; Cyclosporine; Cyclosporine A; DCR-1171X; DCR-L1360; DCR-PH1; DCR-PHXC; Hematopoietic stem cell transplantation; Hydroxychloroquine; IMD; IMD Preparative Regimen; LUMASIRAN; Lumasiran; Melphalan; Mycophenolate; Mycophenolate mofetil; Nedosiran; Osteopetrosis Haploidentical Only Preparative Regimen; Osteopetrosis Only Preparative Regimen; Radiation: Total Body Irradiation with Marrow Boosting; Stem Cell Transplantation; Sterile Normal Saline (0.9% NaCl); Ursodiol

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	NCT01626092 (ClinicalTrials.gov)	July 11, 2012	20/6/2012	Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders	Treatment of High Risk, Inherited Lysosomal and Peroxisomal Disorders by Reduced-Intensity Hematopoietic Cell Transplantation and Low-Dose Total Body Irradiation With Marrow Boosting by Volumetric-Modulated Arc Therapy (VMAT)	Lysosomal Storage Disease;Peroxisomal Disorder	Drug: Campath -1H;Drug: Clofarabine;Drug: Melphalan;Radiation: Total Body Irradiation with Marrow Boosting;Biological: Hematopoietic stem cell transplantation;Drug: Cyclosporine A;Drug: Mycophenolate mofetil	Masonic Cancer Center, University of Minnesota	NULL	Completed	N/A	55 Years	All	3	N/A	United States

No.

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agemin

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NCT01626092
(ClinicalTrials.gov)

July 11, 2012

20/6/2012

Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

Treatment of High Risk, Inherited Lysosomal and Peroxisomal Disorders by Reduced-Intensity Hematopoietic Cell Transplantation and Low-Dose Total Body Irradiation With Marrow Boosting by Volumetric-Modulated Arc Therapy (VMAT)

Lysosomal Storage Disease;Peroxisomal Disorder

Drug: Campath -1H;Drug: Clofarabine;Drug: Melphalan;Radiation: Total Body Irradiation with Marrow Boosting;Biological: Hematopoietic stem cell transplantation;Drug: Cyclosporine A;Drug: Mycophenolate mofetil

Masonic Cancer Center, University of Minnesota

NULL

Completed

N/A

55 Years

All

N/A

United States