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NCT03333200
- Conditions
- ALD, Alpha-Mannosidosis, Batten Disease, +78 more
- Locations
-
- UPMC Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
NCT04528355
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +119 more
- Locations
-
- UPMC Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
NCT01962415
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +125 more
- Locations
-
- UPMC Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
NCT01019876
- Conditions
- Anemia, Anemia, Aplastic, Anemia, Hypoplastic, Congenital, +23 more
- Locations
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- Columbia University Medical Center New York, United States
Completed
ACTIMMUNE in Intermediate Osteopetrosis
NCT02666768
- Conditions
- Bone Diseases, Bone Diseases, Developmental, Musculoskeletal Diseases, +3 more
- Locations
-
- Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center Torrance, California, United States
- University of Minnesota Minneapolis, Minnesota, United States
Completed
Use of ACTIMMUNE in Patients With ADO2
NCT02584608
- Conditions
- Autosomal Dominant Osteopetrosis Type 2, Bone Diseases, Bone Diseases, Developmental, +4 more
- Locations
-
- Indiana University School of Medicine Indianapolis, Indiana, United States
NCT00775931
- Conditions
- Bone Diseases, Bone Diseases, Developmental, Musculoskeletal Diseases, +4 more
- Locations
-
- University of MInnesota, Fairview Minneapolis, Minnesota, United States
Completed
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
NCT00730314
- Conditions
- Agranulocytosis, Albinism, Anemia, +77 more
- Locations
-
- Children Hospital Los Angeles Los Angeles, California, United States
Completed
Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant Osteopetrosis
NCT00043329
- Conditions
- Bone Diseases, Bone Diseases, Developmental, Musculoskeletal Diseases, +3 more
- Locations
-
- InterMune, Inc. Brisbane, California, United States
Active, Not Recruiting
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
NCT02171104
- Conditions
- Abnormalities, Multiple, Acyl-CoA Oxidase Deficiency, Adrenoleukodystrophy With Cerebral Involvement, +85 more
- Locations
-
- Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States
Active, Not Recruiting
Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant
NCT03301168
- Conditions
- Acute Lymphoblastic Leukemia, Anemia, Anemia, Aplastic, +34 more
- Locations
-
- Children's Hospital Los Angeles Los Angeles, California, United States
- Stanford University - Division of Pediatric Stem Cell Transplant & Regenerative Medicine Palo Alto, California, United States
- Children's National Medical Center Washington D.C., District of Columbia, United States
- Children's Healthcare of Atlanta Atlanta, Georgia, United States