Leukodystrophy, Globoid Cell: Clinical Trials

4 completed, 2 active not recruiting, 2 terminated, 1 not yet recruiting. 11 Phase 2, 3 Phase 1, 1 Phase 3.

By phase and status

Phase	Trials	Status
Phase 2	11	Completed 4, Active not recruiting 2, Terminated 2, Not yet recruiting 1, Recruiting 1, Suspended 1
Phase 1	3	Active not recruiting 1, Suspended 1, Terminated 1
Phase 3	1	Completed 1

Follow Leukodystrophy, Globoid Cell: new trials by email Literature Review of Leukodystrophy, Globoid Cell trials Research Report on Leukodystrophy, Globoid Cell

Trials (filtered) newest first · largest first

All 11 trials

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

Trial	Phase	Status	Sponsor	Enrollment	Start	Updated
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol) PLX-200 - Lysosomal Storage Disorders, Sandhoff Disease	Phase 2	Not yet recruiting	Polaryx Therapeutics, Inc.	24	2026-12-01	2026-07-31
Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT FBX-101 - Krabbe Disease	Phase 1 / Phase 2	Active not recruiting	Duke University	9	2023-02-03	2026-11-04
Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease PBKR03 - Leukodystrophy, Globoid Cell	Phase 1 / Phase 2	Suspended	Gemma Biotherapeutics	24	2022-02-24	2026-06-30
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT) MGTA-456 - Inherited Metabolic Disorders (IMD)	Phase 2	Completed	Magenta Therapeutics, Inc.	8	2018-02-09	2021-11-03
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen - Mucopolysaccharidosis Disorders, Hurler Syndrome	Phase 2	Active not recruiting	Masonic Cancer Center, University of Minnesota	149	2014-07-10	2026-11-03
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT Hydroxyurea, Alemtuzumab, Fludarabine - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes	Phase 2	Recruiting	Paul Szabolcs	100	2014-02-04	2025-12-15
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders hematopoietic stem cell infusion - Hurler Syndrome (MPS I), Hurler-Scheie Syndrome	Phase 1 / Phase 2	Terminated	Talaris Therapeutics Inc.	3	2011-04-01	2023-04-12
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Campath-1H, Cyclophosphamide, Busulfan - Mucopolysaccharidosis, Hurler Syndrome	Phase 2	Completed	Masonic Cancer Center, University of Minnesota	46	2009-12-01	2018-02-05
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Stem Cell Transplantation, Cyclophosphamide, Campath-1H - Hurler's Syndrome, Maroteaux-Lamy Syndrome	Phase 2	Terminated	Masonic Cancer Center, University of Minnesota	18	2008-03-01	2017-12-28
HSCT for High Risk Inherited Inborn Errors Clofarabine, Total body Irradiation, Melphalan - Adrenoleukodystrophy, Metachromatic Leukodystrophy	Phase 2	Completed	Masonic Cancer Center, University of Minnesota	38	2006-09-01	2019-07-11
Stem Cell Transplant for Inborn Errors of Metabolism Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin - Adrenoleukodystrophy, Metachromatic Leukodystrophy	Phase 2 / Phase 3	Completed	Masonic Cancer Center, University of Minnesota	135	1995-01-01	2017-12-28

Narrower conditions

Leukodystrophy, Metachromatic (8)  ·  Mucopolysaccharidosis I (6)  ·  Adrenoleukodystrophy (5)  ·  alpha-Mannosidosis (5)  ·  Fucosidosis (4)  ·  Mucopolysaccharidosis II (4)  ·  Sandhoff Disease (4)  ·  Sphingolipidoses (4)  ·  Aspartylglucosaminuria (3)  ·  Lysosomal Storage Diseases (3)  ·  Metabolism, Inborn Errors (3)  ·  Mucopolysaccharidosis VI (3)  ·  Mucopolysaccharidosis VII (3)  ·  Peroxisomal Disorders (3)

Broader conditions

Congenital, Hereditary, and Neonatal Diseases and Abnormalities (11)  ·  Nutritional and Metabolic Diseases (11)  ·  Brain Diseases (10)  ·  Brain Diseases, Metabolic (10)  ·  Brain Diseases, Metabolic, Inborn (10)  ·  Central Nervous System Diseases (10)  ·  Demyelinating Diseases (10)  ·  Genetic Diseases, Inborn (10)  ·  Hereditary Central Nervous System Demyelinating Diseases (10)  ·  Leukoencephalopathies (10)  ·  Lipid Metabolism Disorders (10)  ·  Lipid Metabolism, Inborn Errors (10)  ·  Lipidoses (10)  ·  Lysosomal Storage Diseases, Nervous System (10)  ·  Metabolic Diseases (10)

Interventions

Stem Cell Transplantation (5)  ·  Alemtuzumab (4)  ·  Busulfan (3)  ·  Cyclophosphamide (3)  ·  Bone Marrow Transplantation (2)  ·  Hydroxyurea (2)  ·  Melphalan (2)  ·  Antilymphocyte Serum (1)  ·  Clofarabine (1)  ·  Cyclosporine (1)  ·  Gemfibrozil (1)  ·  Hematopoietic Stem Cell Transplantation (1)  ·  Mycophenolic Acid (1)  ·  Regimen B (1)  ·  Thiotepa (1)

Sponsors

Masonic Cancer Center, University of Minnesota (5)  ·  Duke University (1)  ·  Gemma Biotherapeutics (1)  ·  Magenta Therapeutics, Inc. (1)  ·  Paul Szabolcs (1)  ·  Polaryx Therapeutics, Inc. (1)  ·  Talaris Therapeutics Inc. (1)

Countries

United States (10)  ·  Brazil (1)  ·  Canada (1)  ·  Israel (1)  ·  Netherlands (1)  ·  United Kingdom (1)

Top investigators

Paul Orchard, MD (4)  ·  Joanne Kurtzberg, MD (1)  ·  May Orfali, MD (1)  ·  Paul Orchard, M.D. (1)  ·  Paul Szabolcs, MD (1)  ·  Suzanne T Ildstad, MD (1)

Results publications

PMID 21586746, PMID 36196048, PMID 32634238, PMID 32750127, PMID 35333110, PMID 39499628