Search the Studies

21 Studies found from all possible combinations of your search terms. Select a protocol that you wish to review.
Protocol Number Title Protocol Status Min-Max Age Institute Keywords
02-CH-0311Investigations into Inborn Errors of Cholesterol Synthesis and Related DisordersParticipants currently recruited/enrolled0-125 YearsNICHDDysplasia X-Linked
000798-DA Phase 2 Study of Burosumab for Fibroblast Growth Factor-23 Mediated Hypophosphatemia in Fibrous DysplasiaParticipants currently recruited/enrolled1-125 YearsNIDCRDysplasia
000780-DA Phase 2 Study of Denosumab for Prevention of Skeletal Disease Progression in Children with Fibrous DysplasiaParticipants currently recruited/enrolled4-14 YearsNIDCRDysplasia
000661-CComprehensive Molecular and Clinical Evaluation of Pediatric and Adult Adult Myelodysplastic Syndromes (MDS)Participants currently recruited/enrolled0-125 YearsNCI Dysplasia
000404-CA Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects with VEXAS (Vacuoles, E1 enzyme, X-linked, Autoinflammatory, Somatic) SyndromeParticipants currently recruited/enrolled18-75 YearsNCI X-Linked
000213-HGClinical and Laboratory Study of Rare Skeletal DisordersParticipants currently recruited/enrolled0-125 YearsNHGRIDysplasia
000186-IPart B- Phase I/II, Non-randomized, Multicenter, Open-label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients with X-Linked Chronic Granulomatous DiseaseClinical hold/Recruitment or enrollment suspended3-12 YearsNIAIDX-Linked
18-N-0083ASPIRO: A Phase 1/2/3, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) PatientsNo longer recruiting/follow-up only3-5 YearsNINDSX-Linked
18-D-0114An Open Label Pilot Study of Denosumab Treatment for Fibrous DysplasiaNo longer recruiting/follow-up only18-125 YearsNIDCRDysplasia
15-I-0008A Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients with X-linked Chronic Granulomatous Disease (CGD)Completed Study; data analyses ongoing3-65 YearsNIAIDX-Linked
15-EI-0038A Phase I/IIa Study of RS1 Ocular Gene Transfer for X-linked RetinoschisisNo longer recruiting/follow-up only18-125 YearsNEI X-Linked
14-I-0020Hematopoietic Stem Cell Mobilization in Idiopathic CD4 Lymphocytopenia Patients and Healthy Controls for the Study of T Cell Maturation and Trafficking in Murine ModelsParticipants currently recruited/enrolled18-65 YearsNIAIDDysplasia
11-I-0007Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age with X-Linked Severe Combined ImmunodeficiencyClinical hold/Recruitment or enrollment suspended2-40 YearsNIAIDX-Linked
05-I-0213Screening and Baseline Assessment of Patients with Abnormalities of Immune FunctionParticipants currently recruited/enrolled0-125 YearsNIAIDX-Linked
03-EI-0033X-Linked Juvenile Retinoschisis - Clinical and Molecular StudiesParticipants currently recruited/enrolled0-125 YearsNEI X-Linked
01-D-0184A Natural History Study of Bone and Mineral DisordersParticipants currently recruited/enrolled0-125 YearsNIDCRDysplasia
00-N-0089Genetic Analysis of the Chiari I MalformationCompleted Study; data analyses ongoing1-125 YearsNINDSDysplasia
00-HG-0209Molecular and Clinical Studies of Primary Immunodeficiency DiseasesNo longer recruiting/follow-up only3-125 YearsNHGRIX-Linked
98-D-0145Screening and Natural History of Patients with Polyostotic Fibrous Dysplasia and the McCune-Albright SyndromeParticipants currently recruited/enrolled0-125 YearsNIDCRDysplasia
97-H-0041Screening Protocol for Subjects Being Evaluated for National Heart, Lung and Blood Institute (NHLBI) ProtocolsEnrolling by Invitation2-125 YearsNHLBIDysplasia
94-I-0073Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and GranulocytesParticipants currently recruited/enrolled18-70 YearsNIAIDX-Linked