Clinical Trials on Leigh+Syndrome+With+Leukodystrophy

Total 751 results

Sohag University

Not yet recruiting

Characterization and Outcome of Children With Leukodystrophy: An Observational Study at Sohag University Hospital

Children With Leukodystrophy
Washington University School of Medicine

Completed

Aclarubicin for the Treatment of Retinal Vasculopathy With Cerebral Leukodystrophy (RVCL)

Vasculopathy, Retinal, With Cerebral Leukodystrophy | Cerebroretinal Vasculopathy, Hereditary

United States
Horizon Pharma USA, Inc.

Completed

Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease (MITO-001)

Inherited Mitochondrial Disease, Including Leigh Syndrome

United States
PTC Therapeutics
Axio Research. LLC

Completed

Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome

Leigh Syndrome

United States
PTC Therapeutics

Completed

Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome

Leigh Syndrome

United States
Aadi Bioscience, Inc.

Withdrawn

ABI-009 (Nab-sirolimus) in Patients With Genetically-confirmed Leigh or Leigh-like Syndrome

Leigh Syndrome
Taysha Gene Therapies, Inc.

Withdrawn

A Study of the Natural Course of SURF1 Deficiency

Leigh Syndrome

United States
Talaris Therapeutics Inc.
Duke University

Terminated

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Niemann-Pick Disease | Alpha-mannosidosis | Tay Sachs Disease | Sandhoff Disease | Metachromatic Leukodystrophy (MLD) | Hurler-Scheie Syndrome | Hurler Syndrome (MPS I) | Hunter Syndrome (MPS II) | Sanfilippo Syndrome (MPS III) | Krabbe Disease (Globoid Leukodystrophy) | Adrenoleukodystrophy (ALD and AMN) | Pelizaeus...

United States
National Cancer Institute (NCI)

Completed

Lenalidomide in Treating Young Patients With Relapsed or Refractory Solid Tumors or Myelodysplastic Syndromes

Unspecified Childhood Solid Tumor, Protocol Specific | Childhood Myelodysplastic Syndromes | Previously Treated Myelodysplastic Syndromes | Secondary Myelodysplastic Syndromes | de Novo Myelodysplastic Syndromes | Refractory Anemia | Refractory Anemia With Excess Blasts | Refractory Cytopenia With... and other conditions

United States
Central Hospital, Nancy, France
Inserm U954 - N-GERE (Nutrition, Genetics and Exposition to Environmental...

Unknown

High Risk Myelodysplasia Treated by Azacytidine : Genetic and Epigenetic (MYRAGE) (MYRAGE)

High-risk Myelodysplastic Syndromes With Excess Blasts

France
Masonic Cancer Center, University of Minnesota

Completed

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Mucopolysaccharidosis | Hurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Krabbe Disease | Adrenoleukodystrophy (ALD) | Hunter Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Metachromatic Leukodystrophy (MLD) | Maroteaux-Lamy Syndrome

United States
Ruijin Hospital

Recruiting

Evaluate the Efficacy and Safety of Allogeneic Haematopoietic Stem Cell Transplantation With FBM Conditioning for MDS

Myelodysplastic Syndromes | Myelodysplastic Syndrome With Excess Blasts-2 | Myelodysplastic Syndrome With Excess Blasts-1

China
University Hospital, Brest

Recruiting

Haploinsufficiency of the RBM22 and SLU7 Genes in Del(5q) Myelodysplastic Syndromes (SMD-RMB22)

Myelodysplastic Syndromes | Myelodysplastic Syndrome With Isolated Del(5Q) | Myelodysplastic Syndrome With Del(5Q)

France
The University of Texas Health Science Center,...

Recruiting

The International Registry for Leigh Syndrome

Leigh Disease | Leigh Syndrome | Leigh's Necrotizing Encephalopathy | Subacute Necrotizing Encephalomyopathy | Subacute Necrotizing Encephalomyelopathy

United States
Khondrion BV
Drug Research Unit Ghent, Belgium

Completed

A Dose-escalating Clinical Trial With KH176

Mitochondrial Disease | MELAS | Leigh Syndrome | LHON | Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation

Belgium
Children's Hospital of Philadelphia
Eli Lilly and Company; University of Pennsylvania; Takeda; National Institute of... and other collaborators

Recruiting

The Myelin Disorders Biorepository Project (MDBP)

Mucopolysaccharidoses | Leukoencephalopathies | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2... and other conditions

United States
National Cancer Institute (NCI)

Completed

Thalidomide in Treating Patients With Myelodysplastic Syndrome

Chronic Myelomonocytic Leukemia | Previously Treated Myelodysplastic Syndromes | Secondary Myelodysplastic Syndromes | de Novo Myelodysplastic Syndromes | Refractory Anemia | Refractory Anemia With Excess Blasts | Refractory Anemia With Excess Blasts in Transformation | Refractory Anemia With Ringed...

United States
National Cancer Institute (NCI)

Completed

Tipifarnib in Treating Patients With Myelodysplastic Syndromes

Chronic Myelomonocytic Leukemia | Previously Treated Myelodysplastic Syndromes | de Novo Myelodysplastic Syndromes | Refractory Anemia | Refractory Anemia With Excess Blasts | Refractory Cytopenia With Multilineage Dysplasia | Refractory Anemia With Excess Blasts in Transformation | Refractory Anemia...

United States
Masonic Cancer Center, University of Minnesota

Recruiting

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Hurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Metachromatic Leukodystrophy | Alpha-Mannosidosis | Hunter Syndrome | Mucopolysaccharidosis Disorders | Maroteaux Lamy Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Glycoprotein Metabolic Disorders | Recessive Leukodystrophies | Globoid... and other conditions

United States
Children's Hospital of Philadelphia
Illumina, Inc.

Active, not recruiting

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

Mucopolysaccharidoses | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2 Gangliosidosis | Zellweger... and other conditions

United States
Fred Hutchinson Cancer Center
Jazz Pharmaceuticals

Terminated

CPX-351 Versus Immediate Stem Cell Transplantation for the Treatment of High-Grade Myeloid Cancers With Measurable Residual Disease

Acute Myeloid Leukemia | Myelodysplastic Syndrome With Excess Blasts-2 | Myeloid Neoplasm

United States
Eric Stephen Winer, MD

Recruiting

BXCL701 Phase 1 R/R Acute Myeloid Leukemia or Myelodysplastic Syndrome

Acute Myeloid Leukemia | Myelodysplastic Syndrome With Excess Blasts-2

United States
University of Ulm
Sunesis Pharmaceuticals

Completed

Study of Vosaroxin With Azacitidine in Patients With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome With Excess Blasts-2

Acute Myeloid Leukemia | Myelodysplastic Syndrome With Excess Blasts-2 (MDS-EB-2)

Germany
Stichting Hemato-Oncologie voor Volwassenen Nederland
Deutsch-Österreichische Studiengruppe Akute Myeloische Leukämie (AMLSG)

Recruiting

A Study of Ivosidenib or Enasidenib in Combination With Induction Therapy and Consolidation Therapy, Followed by Maintenance Therapy in Patients With Newly Diagnosed Acute Myeloid Leukemia or Myedysplastic Syndrome EB2, With an IDH1 or IDH2 Mutation, Respectively, Eligible for Intensive Chemotherapy (HOVON150AML)

Acute Myeloid Leukemia | Myelodysplastic Syndrome With Excess Blasts-2

Australia, Austria, Belgium, Estonia, Finland, France, Germany, Ireland, Lithuania, Luxembourg, Netherlands, Norway, Spain, Sweden, Switzerland
Stichting Hemato-Oncologie voor Volwassenen Nederland
Astellas Pharma Global Development, Inc.; Deutsch-Österreichische Studiengruppe...

Active, not recruiting

A Study of Gilteritinib Versus Midostaurin in Combination With Induction and Consolidation Therapy Followed by One-year Maintenance in Patients With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndromes With Excess Blasts-2 With FLT3 Mutations Eligible for Intensive Chemotherapy (HOVON 156 AML)

Acute Myeloid Leukemia | Myelodysplastic Syndrome With Excess Blasts-2

Netherlands, Belgium, Germany, Switzerland, Australia, Finland, Norway, Sweden, Austria, France, Ireland, Lithuania, Luxembourg, Spain
Sidney Kimmel Cancer Center at Thomas Jefferson...

Active, not recruiting

Sirolimus and Azacitidine in Treating Patients With High Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia That is Recurrent or Not Eligible for Intensive Chemotherapy

Recurrent Adult Acute Myeloid Leukemia | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Del(5q) | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) | Adult Acute Myeloid Leukemia With t(8... and other conditions

United States
Case Comprehensive Cancer Center

Completed

Differentiation Therapy With Decitabine in Treating Patients With Myelodysplastic Syndrome

Myelodysplastic Syndromes | Thrombocytopenia | Chronic Myelomonocytic Leukemia | de Novo Myelodysplastic Syndromes | Refractory Anemia | Refractory Anemia With Excess Blasts | Refractory Cytopenia With Multilineage Dysplasia | Refractory Anemia With Ringed Sideroblasts

United States
Onconova Therapeutics, Inc.

Terminated

Controlled Study of Rigosertib Versus Physician's Choice of Treatment in MDS Patients After Failure of an HMA (INSPIRE)

Myelodysplastic Syndrome | MDS | Refractory Anemia With Excess Blasts | RAEB

United States, Spain, Canada, France, Australia, Switzerland, Israel, Japan, Belgium, India, Ireland, United Kingdom, Croatia, Czechia, Poland, Estonia, Germany, Italy, Austria, Hungary, Russian Federation, Sweden
M.D. Anderson Cancer Center
National Cancer Institute (NCI)

Recruiting

Fludarabine Phosphate, Cytarabine, Filgrastim-sndz, Gemtuzumab Ozogamicin, and Idarubicin Hydrochloride in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

Untreated Adult Acute Myeloid Leukemia | Myelodysplastic Syndrome With Excess Blasts | High Risk Myelodysplastic Syndrome | de Novo Myelodysplastic Syndrome | Acute Myeloid Leukemia With t(8;21); (q22; q22.1); RUNX1-RUNX1T1 | Acute Myeloid Leukemia With Inv(16)(p13.1q22); CBFB-MYH11 | Acute Myeloid... and other conditions

United States
Fred Hutchinson Cancer Center
National Cancer Institute (NCI)

Completed

Clofarabine and Cytarabine in Treating Older Patients With AML or High-Risk MDS

Recurrent Adult Acute Myeloid Leukemia | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Del(5q) | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) | Adult Acute Myeloid Leukemia With t(8... and other conditions

United States
Fred Hutchinson Cancer Center
National Cancer Institute (NCI)

Completed

Bendamustine and Idarubicin in Treating Older Patients With Previously Untreated AML or MDS

Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Del(5q) | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) | Adult Acute Myeloid Leukemia With t(8;21)(q22;q22) | Untreated Adult Acute Myeloid... and other conditions

United States
University Hospital, Brest

Recruiting

Biocollection in MyeloDysplastic Syndrome (P-MDS) (P-MDS)

Myelodysplastic Syndromes | Chromosome Abnormality | Acute Myeloid Leukemia With Multilineage Dysplasia | Myelodysplastic Syndrome With Ring Sideroblasts | Myelodysplastic Anemia | Myelodysplastic Syndrome With Isolated Del(5Q)

France
University of Wisconsin, Madison
National Cancer Institute (NCI)

Completed

Decitabine and Total-Body Irradiation Followed By Donor Bone Marrow Transplant and Cyclophosphamide in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

Recurrent Adult Acute Myeloid Leukemia | Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Del(5q) | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult... and other conditions

United States
Fred Hutchinson Cancer Center
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute...

Completed

Low-Dose or High-Dose Conditioning Followed by Peripheral Blood Stem Cell Transplant in Treating Patients With Myelodysplastic Syndrome or Acute Myelogenous Leukemia

Recurrent Adult Acute Myeloid Leukemia | Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Del(5q) | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult... and other conditions

United States, Germany
PTC Therapeutics

Completed

A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy (MIT-E)

Mitochondrial Diseases | Drug Resistant Epilepsy | Leigh Disease | Leigh Syndrome | Mitochondrial Encephalopathy (MELAS) | Pontocerebellar Hypoplasia Type 6 (PCH6) | Alpers Disease | Alpers Syndrome

United States, Spain, United Kingdom, Canada, France, Italy, Poland, Sweden, Japan
National Cancer Institute (NCI)

Active, not recruiting

Azacitidine With or Without Lenalidomide or Vorinostat in Treating Patients With Higher-Risk Myelodysplastic Syndromes or Chronic Myelomonocytic Leukemia

Chronic Myelomonocytic Leukemia | Myelodysplastic Syndrome | Myelodysplastic Syndrome With Excess Blasts | Chronic Myelomonocytic Leukemia-1 | Chronic Myelomonocytic Leukemia-2 | Myelodysplastic Syndrome With Excess Blasts-1 | Myelodysplastic Syndrome/Acute Myeloid Leukemia

Canada, United States
Jazz Pharmaceuticals

Recruiting

Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures

Seizure in Participants With Tuberous Sclerosis Complex | Seizure in Participants With Dravet Syndrome | Seizure in Participants With Lennox-Gastaut Syndrome

United States
LMU Klinikum
University of Pisa; German Federal Ministry of Education and Research; European...

Recruiting

Global Registry and Natural History Study for Mitochondrial Disorders

Mitochondrial Diseases | MDS | Mitochondrial Myopathies | MELAS Syndrome | MIDD | Kearns-Sayre Syndrome | MERRF Syndrome | Barth Syndrome | Leigh Syndrome | MNGIE | LHON | Pearson Syndrome | NARP Syndrome | Coenzyme Q10 Deficiency | SANDO | SCAE | MIRAS | CPEO

Austria, Germany, Italy
Hospices Civils de Lyon

Unknown

Autoimmunity in Patients With GAD-Ab and Their Relatives (FamilyGAD)

Neurological Syndromes With GAD-Ab | Organ-specific Autoimmune Diseases
Brian Jonas
Pharmacyclics LLC.

Completed

Ibrutinib and Azacitidine for Treatment of Higher Risk Myelodysplastic Syndrome

Chronic Myelomonocytic Leukemia | Previously Treated Myelodysplastic Syndrome | Secondary Myelodysplastic Syndrome | de Novo Myelodysplastic Syndrome | Refractory Anemia With Excess Blasts in Transformation

United States
University of Alabama at Birmingham

Completed

Efficacy of Cyclosporine Ophthalmic Emulsion in the Treatment of Dry Eye Syndrome in Contact Lens Wearers

Contact Lens Wearers With Dry Eye Syndrome

United States
National Cancer Institute (NCI)

Completed

A Phase II Study Of The Farnesyltransferase Inhibitor ZANESTRA (R115777, NSC #702818, IND #58,359) In Complete Remission Following Induction And/Or Consolidation Chemotherapy In Adults With Poor-Risk Acute Myelogenous Leukemia (AML) And High-Risk Myelodysplasia (MDS)

Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Del(5q) | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) | Adult Acute Myeloid Leukemia With t(8;21)(q22;q22) | Adult Acute Myeloid Leukemia... and other conditions

United States
Fred Hutchinson Cancer Center
National Cancer Institute (NCI)

Completed

Tosedostat in Combination With Cytarabine or Decitabine in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Del(5q) | Secondary Acute Myeloid Leukemia | Untreated Adult Acute Myeloid Leukemia | Previously Treated Myelodysplastic Syndromes | Secondary Myelodysplastic Syndromes | Adult Acute Myeloid Leukemia... and other conditions

United States
Fred Hutchinson Cancer Center
National Cancer Institute (NCI)

Completed

Radiolabeled Monoclonal Antibody Therapy, Fludarabine Phosphate, and Low-Dose Total-Body Irradiation Followed by Donor Stem Cell Transplant and Immunosuppression Therapy in Treating Older Patients With Advanced Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndromes

Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Del(5q) | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) | Adult... and other conditions

United States
Fred Hutchinson Cancer Center
National Cancer Institute (NCI)

Terminated

Iodine I 131 Monoclonal Antibody BC8, Fludarabine Phosphate, Total Body Irradiation, and Donor Stem Cell Transplant Followed by Cyclosporine and Mycophenolate Mofetil in Treating Patients With Advanced Acute Myeloid Leukemia or Myelodysplastic Syndrome

Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Del(5q) | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) | Adult... and other conditions

United States
David Iberri

Terminated

Decitabine and Midostaurin in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia

Acute Myeloid Leukemia (AML) With Multilineage Dysplasia Following Myelodysplastic Syndrome, in Adults | AML (Adult) With 11q23 (MLL) Abnormalities | AML (Adult) With Del (5q) | AML (Adult) With Inv (16) (p13; q22) | AML (Adult) With t (16;16) (p13; q22) | AML (Adult) With t (8; 21) (q22; q22) and other conditions

United States
National Cancer Institute (NCI)

Recruiting

Venetoclax in Combination With ASTX727 for the Treatment of Chronic Myelomonocytic Leukemia and Other Myelodysplastic Syndrome/Myeloproliferative Neoplasm

Chronic Myelomonocytic Leukemia | Myelodysplastic Syndrome | Myelodysplastic Syndrome With Excess Blasts | Myeloproliferative Neoplasm

United States, Canada
National Cancer Institute (NCI)

Completed

Cediranib Maleate in Treating Patients With Relapsed, Refractory, or Untreated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

Recurrent Adult Acute Myeloid Leukemia | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) | Adult Acute Myeloid Leukemia With t(8;21)(q22;q22) | Secondary Acute Myeloid Leukemia and other conditions

United States
Apollo Therapeutics Ltd

Not yet recruiting

A Study to Investigate APL-4098 Alone and/or in Combination With Azacitidine in R/R AML and High-Risk MDS

Myelodysplastic Syndrome With Excess Blasts | Acute Myeloid Leukemia, in Relapse | Acute Myeloid Leukemia Refractory | Myelodysplastic Syndrome Acute Myeloid Leukemia
Boston Children's Hospital

Completed

Pilot of Abatacept-based Immunosuppression for Prevention of Acute GvHD During Unrelated Donor HCT

AML | ALL | Biphenotypic Leukemia | Refractory Anemia | Refractory Cytopenia With Multilineage Dysplasia | Refractory Anemia With Ringed Sideroblasts | Undifferentiated Leukemia | Ref. Cytopenia w Multilineage Dysplasia & Ringed Sideroblasts | Refractory Anemia With Excess Blasts-1 (5-10% Blasts) | Refractory... and other conditions

United States

Clinical Trials on Leigh+Syndrome+With+Leukodystrophy

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