Leukodystrophy, Globoid Cell clinical trials
The registry lists 27 studies for Leukodystrophy, Globoid Cell across more than 10 countries. 7 are recruiting or about to start. Per ClinicalTrials.gov, last updated 2026-09-03.
Status
- Completed
- 8
- Recruiting
- 6
- Active, not recruiting
- 5
- Terminated
- 3
- Withdrawn
- 2
- Not yet recruiting
- 1
Phase
- Phase 2
- 11
- Phase 1
- 6
- Phase 3
- 1
Where
- United States
- 24
- United Kingdom
- 1
- Netherlands
- 1
- Israel
- 1
- Sri Lanka
- 1
- Egypt
- 1
Who runs them
Masonic Cancer Center, University of Minnesota (5) · Forge Biologics, Inc (3) · Paul Szabolcs (2) · University of Pittsburgh (2) · Children's Hospital of Philadelphia (2) · Talaris Therapeutics Inc. (1) · State University of New York at Buffalo (1) · CENTOGENE GmbH Rostock (1)
Studies
Recruiting first, then most recently updated. Showing 27 of 27 — search for more.
- UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like CellsRecruitingNCT02254863Phase 11 country
- Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)Not yet recruitingNCT07740512Phase 2
- Krabbe Disease Global Patient RegistryRecruitingNCT029937961 country
- Longitudinal Study of Neurodegenerative DisordersRecruitingNCT033332001 country
- Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RICRecruitingNCT045283551 country
- Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCTRecruitingNCT01962415Phase 21 country
- The Myelin Disorders Biorepository ProjectRecruitingNCT030473691 country
- Early Check: Expanded Screening in NewbornsActive, not recruitingNCT036552231 country
- Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe DiseaseSuspendedNCT04771416Phase 1/26 countries
- Gene Transfer Clinical Trial for Krabbe DiseaseActive, not recruitingNCT04693598Phase 1/21 country
- Diffusion Tensor Imaging (DTI) in Infants With Krabbe DiseaseActive, not recruitingNCT007878651 country
- MT2013-31: Allo HCT for Metabolic Disorders and Severe OsteopetrosisActive, not recruitingNCT02171104Phase 21 country
- Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCTActive, not recruitingNCT05739643Phase 1/21 country
- LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for LeukodystrophiesCompletedNCT026991901 country
- Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe PatientsTerminatedNCT063087181 country
- Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic DisordersTerminatedNCT01372228Phase 1/21 country
- Biomarker for Krabbe Disease (BioKrabbe)WithdrawnNCT014254894 countries
- Human Placental-Derived Stem Cell TransplantationCompletedNCT01586455Phase 11 country
- An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD)WithdrawnNCT01093105
- Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool ChildrenCompletedNCT019380141 country
- HSCT for High Risk Inherited Inborn ErrorsCompletedNCT00383448Phase 21 country
- The Natural History of Infantile Globoid Cell LeukodystrophyCompletedNCT009838791 country
- Allogeneic Bone Marrow Transplant for Inherited Metabolic DisordersCompletedNCT01043640Phase 21 country
- Stem Cell Transplant for Inborn Errors of MetabolismCompletedNCT00176904Phase 2/31 country
- Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of MetabolismTerminatedNCT00668564Phase 21 country
- Diagnostic and Screening Study of Genetic DisordersCompletedNCT000060571 country
- Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell TransplantationStatus unknownNCT000059001 country