juvenile Sandhoff
Showing 1 - 25 of 508
GM1 and GM2 Gangliosidosis PROspective Neurological Disease
Recruiting
- GM1 Gangliosidosis
- +2 more
-
Oakland, California
- +12 more
Nov 16, 2022
GM2 Gangliosidosis, Supportive Care Trial in Kashan, Mashhad, Tehran (Miglustat)
Recruiting
- GM2 Gangliosidosis
- Supportive Care
-
Kashan, Isfahan, Iran, Islamic Republic of
- +2 more
Oct 23, 2022
Tay-Sachs Disease Sandhoff Disease Trial in Worldwide (venglustat GZ402671, )
Active, not recruiting
- Tay-Sachs Disease Sandhoff Disease
- venglustat GZ402671
- placebo
-
Los Angeles, California
- +23 more
Jan 31, 2022
A Natural History Study of the Gangliosidoses
Recruiting
- Tay-Sachs Disease
- +4 more
-
Minneapolis, MinnesotaUniversity of Minnesota - Pediatric Genetics and Metabolism
Feb 28, 2022
Glycosphingolipid and Glycoprotein Lysosomal Storage Disorders
Recruiting
- Neurological Regression
- +3 more
-
Bethesda, MarylandNational Institutes of Health Clinical Center
Jan 14, 2023
GM1 Gangliosidoses, GM2 Gangliosidoses, Tay-Sachs Disease Trial in Minneapolis (miglustat, Ketogenic Diet)
Terminated
- GM1 Gangliosidoses
- +3 more
- miglustat
- Ketogenic Diet
-
Minneapolis, MinnesotaUniversity of Minnesota
Mar 18, 2021
Tay-Sachs Disease, Sandhoff Disease Trial in Boston, Worcester (AXO-AAV-GM2 Starting Dose, AXO-AAV-GM2 Low Dose, AXO-AAV-GM2
Recruiting
- Tay-Sachs Disease
- Sandhoff Disease
- AXO-AAV-GM2 Starting Dose
- +3 more
-
Boston, Massachusetts
- +1 more
Jan 26, 2021
Biomarker for Gangliosidosis: BioGM1/BioGM2 (BioGM1/GM2)
Active, not recruiting
- Hepato-splenomegaly
- +3 more
-
Cairo, Egypt
- +4 more
May 12, 2021
Juvenile Systemic Lupus Erythematosus
Recruiting
- Systemic Lupus Erythematosus
- lipid profile
-
Assiut, EgyptAssiut university
Nov 21, 2023
Severity in Juvenile and Adult-onset Dermatomyositis
Not yet recruiting
- Dermatomyositis
- evaluation of clinical severity
- (no location specified)
Aug 16, 2023
Fibrinogen to Albumin Ratio and C-reactive Protein to Albumin
Not yet recruiting
- Juvenile Idiopathic Arthritis
- fibrinogen to albumin ratio (AFR) and C-reactive protein to albumin ratio (CAR)
- (no location specified)
Aug 29, 2023
Active Systemic Juvenile Idiopathic Arthritis Trial in China (GenaKumab)
Not yet recruiting
- Active Systemic Juvenile Idiopathic Arthritis
-
Beijing, Benjing, China
- +9 more
Jul 5, 2023
Juvenile Idiopathic Arthritis Trial in Al Kharj (Accommodating variable-resistance training, Standard physical therapy)
Completed
- Juvenile Idiopathic Arthritis
- Accommodating variable-resistance training
- Standard physical therapy
-
Al Kharj, Riyadh, Saudi ArabiaRagab K. Elnaggar
Mar 28, 2023
Adenosine Deaminase and Immunoglobulin E in Chronic Spontaneous
Not yet recruiting
- Chronic Spontaneous Urticaria
- Adenosine deaminase serum level
- IgE antibodies serum level
- (no location specified)
Aug 7, 2023
Still's Disease, Juvenile Onset, Still's Disease, Adult-Onset Trial in Japan (Anakinra, Placebo)
Recruiting
- Still's Disease, Juvenile Onset
- Still's Disease, Adult-Onset
- Anakinra
- Placebo
-
Chiba-shi, Chiba, Japan
- +17 more
Apr 3, 2023
Plexin D1 as a Potential Biomarker inPM/DM
Not yet recruiting
- Polymyositis
- Dermatomyositis
- plexinD1
- (no location specified)
Nov 25, 2022
Juvenile Recurrent Respiratory Papillomatosis: Establishment
Recruiting
- Juvenile Recurrent Respiratory Papillomatosis
-
Paris, Francehôpital Necker Enfants malades
Aug 17, 2023
Neurodevelopmental Disorders in Youth With Criminal Behaviors
Completed
- Attention Deficit Disorder With Hyperactivity
- +2 more
- collect data
-
Taipei, TaiwanNational Taiwan University Hospital
May 23, 2023
Active Juvenile Psoriatic Arthritis Trial in Worldwide (Apremilast, Placebo)
Recruiting
- Active Juvenile Psoriatic Arthritis
- Apremilast
- Placebo
-
Bregenz, Austria
- +36 more
Feb 1, 2023
Adult and Juvenile Myositis
Recruiting
- Dermatomyositis
- +2 more
-
Bethesda, Maryland
- +1 more
Jan 20, 2023
Leukemia, Juvenile Myelomonocytic, JMML, JCML Trial (Trametinib, Azacitidine, Fludarabine)
Not yet recruiting
- Leukemia, Juvenile Myelomonocytic
- +4 more
- Trametinib
- +3 more
- (no location specified)
May 4, 2023
Childhood Absence Epilepsy, Juvenile Absence Epilepsy Trial in Worldwide (Brivaracetam Film-coated tablet, Brivaracetam oral
Enrolling by invitation
- Childhood Absence Epilepsy
- Juvenile Absence Epilepsy
- Brivaracetam Film-coated tablet
- Brivaracetam oral solution
-
Birmingham, Alabama
- +21 more
Jan 5, 2023
Spondylarthropathies Trial in Nîmes, Montpellier (Stool collection at the patient's home., Blood test)
Recruiting
- Spondylarthropathies
- Stool collection at the patient's home.
- Blood test
-
Nîmes, Gard, France
- +1 more
Jul 27, 2022