Clinical trials: With Ataxia Symptoms
- Condition With Ataxia Symptoms
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Rett Syndrome
A Safety Study of NNZ-2566 in Patients With Rett Syndrome
The purpose of this study is to determine whether NNZ-2566 is safe and well tolerated in the treatment of Rett Syndrome in adolescent and adult females.
- Trial name
- NCT01703533
- Testing
- NNZ-2566
- Where
- 3 sites in United States
- Volunteers
- 67 people
- Sponsor
- Neuren Pharmaceuticals Limited
Glut1 Deficiency Syndrome, De Vivo Disease
Early Diagnosis of the GLUT1 Deficiency Syndrome With a Blood Based Test
The study aims at validating the diagnostic performances of the METAglut1, a blood in vitro diagnostic test, for the simple and early diagnosis of the Glut1 deficiency syndrome (Glut1DS, or De Vivo disease).
- Conditions
- Glut1 Deficiency Syndrome, De Vivo Disease, Seizures, Movement Disorders
- Trial name
- METAglut1 (NCT03722212)
- Testing
- METAglut1
- Where
- 28 sites in France
- Volunteers
- 636 people
- Sponsor
- METAFORA biosystems
Friedreich Ataxia, Cardiomyopathy, Secondary
Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
The purpose of Study LX2006-03, a multicenter, Phase 2, open-label, randomized, controlled study, is to evaluate the efficacy and safety of LX2006 gene therapy in participants with Friedreich ataxia (FA) cardiomyopathy (CM).
- Trial name
- SUNRISE-FA 2 (NCT07721025)
- Testing
- LX2006, Usual Care
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Lexeo Therapeutics
Phosphomannomutase 2 Deficiency, PMM2-CDG
A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG
This study is evaluating the safety, effectiveness, and how the body absorbs, distributes, and eliminates GLM101, for participants with PMM2-CDG, including children, adolescents, and adults.
- Trial name
- NCT06892288
- Testing
- GLM101 (Part A, Double-blind), GLM101 (Part B, Open-label)
- Where
- 15 sites in United States, Belgium and 8 more
- Volunteers
- 50 people
- Sponsor
- Glycomine, Inc.
Gaucher Disease, Type 3
High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3)
Type: Prospective, open-label, single center study Duration: 6 months with an optional 12-month extension phase Participants: 12 pediatric patients diagnosed with type III Gaucher disease (GD3) aged ≥3 to ≤18 years old treatment…
- Trial name
- NCT07285369
- Testing
- Ambroxol
- Where
- Pakistan
- Volunteers
- 12 people
- Sponsor
- Agyany Pharma LTD
Nervous System Disease, Genetic Syndrome
Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Telangiectasia Patients
Objectives: The objective of study was to evaluate the safety and the efficacy of EryDex (Dexamethasone sodium phosphate encapsulated in autologous erythrocytes, using the EryDex System - EDS) at two dose levels (low dose and…
- Trial name
- ATTeST (NCT02770807)
- Testing
- EryDex Low dose DSP, EryDex High dose DSP
- Where
- 22 sites in United States, Australia and 10 more
- Volunteers
- 176 people
- Sponsor
- Quince Therapeutics S.p.A.