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Results for “Gangliosidosis, GM1”

31 trials

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Showing 20 of 31 results

Testing effectiveness (Phase 2)Study completedNCT02789332
What this trial is testing

Assessing the Efficacy of Paclitaxel and Olaparib in Comparison to Paclitaxel / Carboplatin Followed by Epirubicin/Cyclophosphamide as Neoadjuvant Chemotherapy in Patients With HER2-negative Early Breast Cancer and Homologous Recombination Deficiency

Who this might be right for
Breast CancerTriple Negative Breast NeoplasmsHRpos Breast Neoplasms+1 more
GBG Forschungs GmbH 107
Not applicableUnknownNCT04320329
What this trial is testing

Natural History of Morquio B and Late-Onset of GM1 Gangliosidosis

Who this might be right for
Morquio B DiseaseGM1 Gangliosidosis Type III
University of British Columbia 30
Testing effectiveness (Phase 2)UnknownNCT05732129
What this trial is testing

The Efficacy and Safety of Fluzoparib Plus Irinotecan as Second-line Treatment in Patients With Homologous Recombination Deficiency (HRD) Metastatic Colorectal Cancer.

Who this might be right for
Homologous Recombination Deficiency Alterations Metastatic Colorectal Cancer
Fudan University 29
Not applicableEnrolling By InvitationNCT05368038
What this trial is testing

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

Who this might be right for
Acid Sphingomyelinase DeficiencyCeroid Lipofuscinosis, Neuronal, 2Cerebrotendinous Xanthomatosis+11 more
Albert Einstein College of Medicine 100,000
Large-scale testing (Phase 3)Study completedNCT00176904
What this trial is testing

Stem Cell Transplant for Inborn Errors of Metabolism

Who this might be right for
AdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+8 more
Masonic Cancer Center, University of Minnesota 135
Large-scale testing (Phase 3)Looking for participantsNCT07054515
What this trial is testing

Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

Who this might be right for
Niemann-Pick Type C DiseaseGM1 GangliosidosisGM2 Gangliosidosis
Azafaros A.G. 147
Testing effectiveness (Phase 2)Looking for participantsNCT03952637
What this trial is testing

Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis

Who this might be right for
Lysosomal DiseasesGangliosidosisGM1
National Human Genome Research Institute (NHGRI) 54
Early research (Phase 1)Looking for participantsNCT06458712
What this trial is testing

Study to Assess Safety, Tolerability and Activity of DSB2455 in Participants With Advanced Malignancies

Who this might be right for
Advanced Malignancies With Homologous Recombination Deficiency (HRD) (Breast, Ovarian, mCRPC, Brain Metastases)
Duke Street Bio Ltd 90
Testing effectiveness (Phase 2)Active Not RecruitingNCT04713475
What this trial is testing

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

Who this might be right for
GM1 GangliosidosisGM1 Gangliosidosis, Type IGM1 Gangliosidosis, Type 2+1 more
Gemma Biotherapeutics 26
Testing effectiveness (Phase 2)Study completedNCT00383448
What this trial is testing

HSCT for High Risk Inherited Inborn Errors

Who this might be right for
AdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+6 more
Masonic Cancer Center, University of Minnesota 38
Not applicableStudy completedNCT05109793
What this trial is testing

GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)

Who this might be right for
GM1 GangliosidosisSandhoff DiseaseTay-Sachs Disease
Azafaros A.G. 31
Early research (Phase 1)Looking for participantsNCT07156253
What this trial is testing

Study of SYN818 With Olaparib for the Treatment of Locally Advanced or Metastatic Solid Tumors

Who this might be right for
Metastatic Solid TumorOvarian CancerBreast Cancer+2 more
Hangzhou SynRx Therapeutics Biomedical Technology Co., Ltd 110
Not applicableLooking for participantsNCT00029965
What this trial is testing

Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders

Who this might be right for
Neurological RegressionMyoclonusCherry Red Spot+1 more
National Human Genome Research Institute (NHGRI) 200
Not applicableLooking for participantsNCT06539169
What this trial is testing

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Who this might be right for
Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more
xCures 1,000
Large-scale testing (Phase 3)Looking for participantsNCT07082543
What this trial is testing

Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

Who this might be right for
Gangliosidoses, GM2Gangliosidosis, GM1
Azafaros A.G. 75
Early research (Phase 1)Not Yet RecruitingNCT07479953
What this trial is testing

Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial

Who this might be right for
GM1 GangliosidosesGM1 Gangliosidosis, Type IGM1 Gangliosidosis, Type 2
Tippi Mackenzie 5
Not applicableLooking for participantsNCT05574673
What this trial is testing

Characterization of Epithelial Ovarian Cancer Patients in Terms of Homologous Recombination Phenotype

Who this might be right for
Ovarian CancerPrimary Peritoneal CancerFallopian Tube Cancer
Nordic Society of Gynaecological Oncology - Clinical Trials Unit 1,000
Not applicableStudy completedNCT04041102
What this trial is testing

Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

Who this might be right for
GM1 Gangliosidosis
University of Pennsylvania 31
Not applicableUnknownNCT04624789
What this trial is testing

Registry Gangliosidoses

Who this might be right for
GangliosidosesGM1 GangliosidosisMorquio B Disease+6 more
SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) 40
Large-scale testing (Phase 3)Ended earlyNCT04395339
What this trial is testing

GM1 Prophylaxis for WBRT Related Cognitive Dysfunction

Who this might be right for
Brain MetastasesRadiotherapy Side EffectCognitive Impairment+2 more
Sun Yat-sen University 51
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