Clinical trials: GM1 Gangliosidosis, active
GM1 Gangliosidosis, GM1 Gangliosidosis, Type I
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis
PBGM01 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the brain and peripheral tissues.
- Conditions
- GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
- Trial name
- Imagine-1 (NCT04713475)
- Testing
- PBGM01
- Where
- 7 sites in United States, Brazil and 2 more
- Volunteers
- 26 people
- Sponsor
- Gemma Biotherapeutics
Neurological Regression, Myoclonus
Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
Study description: This is a natural history study that will evaluate any patient with enzyme or DNA confirmed GM1 or GM2 gangliosidosis, sialidosis or galactosialidosis.
- Conditions
- Neurological Regression, Myoclonus, Cherry Red Spot, Brain Atrophy
- Trial name
- NCT00029965
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Advanced Solid Tumors, Metastatic Solid Tumors
Master Protocol for Basket Clinical Trials of Targeted Therapies Based on Proteome and Whole Exome Sequencing Screening in Patients With Advanced Solid Tumors
Study Overview This study evaluates a proteogenomic screening platform to identify precise therapeutic targets for patients with advanced solid tumors.
- Conditions
- Advanced Solid Tumors, Metastatic Solid Tumors, Locally Advanced Solid Tumors, Recurrent Solid Tumors
- Trial name
- K-PRECISION (NCT07891169)
- Testing
- Homologous Recombination Deficiency (HRD), ALK/ROS1/MET, standard care treatment
- Where
- 2 sites in South Korea
- Volunteers
- 2,800 people
- Sponsor
- National Cancer Center, Korea
MLD, Krabbe Disease
Longitudinal Study of Neurodegenerative Disorders
The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the…
- Conditions
- MLD, Krabbe Disease, ALD, MPS I
- Trial name
- NCT03333200
- Testing
- Palliative Care, Hematopoetic Stem Cell Transplantation
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- University of Pittsburgh
Niemann-Pick Disease Type C, GM2 Gangliosidosis
Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal Disorders
This is an open-label extension trial to evaluate the long-term safety and tolerability of nizubaglustat (AZ-3102) in participants with rare lysosomal disorders who have participated in a previous nizubaglustat trial and wish to…
- Conditions
- Niemann-Pick Disease Type C, GM2 Gangliosidosis, GM1 Gangliosidosis, Gaucher Disease Type 3
- Trial name
- HARBOR (NCT07885917)
- Testing
- AZ-3102
- Volunteers
- 218 people
- Sponsor
- Azafaros B.V.
Ovarian Cancer, Primary Peritoneal Cancer
Characterization of Epithelial Ovarian Cancer Patients in Terms of Homologous Recombination Phenotype
This is a prospective observational multi-country, multi-center study of a large real-world cohort of first line (1L) epithelial ovarian cancer patients, exposed to standard of care (SOC) treatment stratified according to BRCA1/2…
- Conditions
- Ovarian Cancer, Primary Peritoneal Cancer, Fallopian Tube Cancer
- Trial name
- HERO (NCT05574673)
- Testing
- Standard of care
- Where
- Denmark
- Volunteers
- 1,000 people
- Sponsor
- Nordic Society of Gynaecological Oncology - Clinical Trials Unit
Metastatic Solid Tumor, Ovarian Cancer
Study of SYN818 With Olaparib for the Treatment of Locally Advanced or Metastatic Solid Tumors
This interventional study will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of SYN818 with Olaparib in adult patients with locally advanced or metastatic solid tumors
- Conditions
- Metastatic Solid Tumor, Ovarian Cancer, Breast Cancer, BRCA 1 /2 and / or HRD
- Trial name
- NCT07156253
- Testing
- SYN818 and Olaparib will be administered
- Where
- 2 sites in China
- Volunteers
- 110 people
- Sponsor
- Hangzhou SynRx Therapeutics Biomedical Technology Co., Ltd
Ovarian Cancer
HRD Tests for Ovarian cancER
The study concerns patients with Invasive epithelial ovarian cancer, primary Fallopian tube carcinoma, ovarian-type peritoneal carcinoma, and with an indication of a first-line platinum-based chemotherapy.
- Trial name
- HERO (NCT06152731)
- Testing
- tests to determine HRD status
- Where
- 4 sites in France
- Volunteers
- 88 people
- Sponsor
- Centre Francois Baclesse
Tay-Sachs Disease, Sandhoff Disease
A Natural History Study of the Gangliosidoses
Hypothesis: To characterize and describe disease progression and heterogeneity of the gangliosidosis diseases. This research study seeks to develop a quantitative method to delineate disease progression for the gangliosidosis…
- Conditions
- Tay-Sachs Disease, Sandhoff Disease, Late Onset Tay-Sachs Disease, GM1 Gangliosidosis
- Trial name
- NCT00668187
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- University of Minnesota
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
ScreenPlus is a consented, multi-disorder pilot newborn screening program implemented in conjunction with the New York State Newborn Screening Program that provides families the option to have their newborn(s) screened for a…
- Conditions
- Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2, Cerebrotendinous Xanthomatosis, Fabry Disease
- Trial name
- ScreenPlus (NCT05368038)
- Testing
- Confirmatory Testing
- Where
- 9 sites in United States
- Volunteers
- 100,000 people, healthy volunteers welcome
- Sponsor
- Albert Einstein College of Medicine
Niemann-Pick Type C Disease, GM1 Gangliosidosis
A Study to 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
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in…
- Conditions
- Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
- Trial name
- NCT07054515
- Testing
- AZ-3102
- Where
- 39 sites in United States, Argentina and 15 more
- Volunteers
- 147 people
- Sponsor
- Azafaros B.V.
Gangliosidoses, GM2, Gangliosidosis, GM1
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of GM1 gangliosidosis or GM2…
- Trial name
- NCT07082543
- Testing
- AZ-3102
- Where
- 27 sites in United States, Argentina and 12 more
- Volunteers
- 75 people
- Sponsor
- Azafaros B.V.
Niemann-Pick Type C Disease
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease
- Trial name
- NCT07082725
- Testing
- Nizubaglustat
- Where
- 34 sites in United States, Argentina and 13 more
- Volunteers
- 72 people
- Sponsor
- Azafaros B.V.
GM1 Gangliosidoses, GM1 Gangliosidosis, Type I
Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial
This is a study for the administration of in utero AAV9 transfer in prenatally diagnosed Type I or Type II GM1.
- Conditions
- GM1 Gangliosidoses, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2
- Trial name
- NCT07479953
- Testing
- Gene Transfer with an AAV9 Vector Expressing Human ß-galactosidase
- Volunteers
- 5 people
- Sponsor
- Tippi Mackenzie
Lysosomal Diseases, Gangliosidosis
A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis
Background: GM1 gangliosidosis is a disorder that destroys nerve cells. It is fatal. There is no treatment. People with GM1 are deficient in a certain enzyme. A gene therapy may help the body make this enzyme.
- Conditions
- Lysosomal Diseases, Gangliosidosis, GM1
- Trial name
- NCT03952637
- Testing
- AAV9-GLB1, Abdominal ultrasound, Rituximab
- Where
- United States
- Volunteers
- 54 people
- Sponsor
- National Human Genome Research Institute (NHGRI)