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Results for “GM1 Gangliosidosis, Type 2”

10 trials

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Showing 10 of 10 results

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 applicableStudy completedNCT04310163
What this trial is testing

Interviews and Video Capture in Patients With GM1 Gangliosidosis

Who this might be right for
GM1 Gangliosidosis
LYSOGENE 25
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
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
Post-approval studies (Phase 4)Ended earlyNCT02030015
What this trial is testing

Synergistic Enteral Regimen for Treatment of the Gangliosidoses

Who this might be right for
GM1 GangliosidosesGM2 GangliosidosesTay-Sachs Disease+1 more
University of Minnesota 16
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
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
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
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
Not applicableStudy completedNCT04470713
What this trial is testing

Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2

Who this might be right for
GM1 GangliosidosisGM2 GangliosidosisGaucher Disease, Type 2+3 more
Idorsia Pharmaceuticals Ltd. 226