Clinical trials: CLN2 Disease, open to volunteers
Neuronal Ceroid Lipofuscinosis, Batten Disease
Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database
This is an observational study that aims at assessing the natural history of NCL diseases as part of the international DEM-CHILD Database. 1.
- Conditions
- Neuronal Ceroid Lipofuscinosis, Batten Disease, CLN1 Disease, CLN2 Disease
- Trial name
- NCT04613089
- Testing
- Natural History
- Where
- Germany
- Volunteers
- 500 people
- Sponsor
- Universitätsklinikum Hamburg-Eppendorf
Lysosomal Storage Disorders, Sandhoff Disease
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)
The purpose of this study is to evaluate the safety, tolerability and clinical activity of PLX-200 in pediatric patients with lysosomal storage disorders.
- Conditions
- Lysosomal Storage Disorders, Sandhoff Disease, Krabbe Disease, CLN2
- Trial name
- NCT07740512
- Testing
- PLX-200
- Volunteers
- 24 people
- Sponsor
- Polaryx Therapeutics, Inc.
Neuronal Ceroid Lipofuscinosis Type 2
A Registrational Study of TTX-381 to Treat Vision Loss in CLN2 Disease
This is a registrational, first-in-human, open-label, dose escalation and expansion study evaluating the safety, tolerability and effectiveness of TTX-381, administered by injection under the retina, for the treatment of eye…
- Trial name
- NCT05791864
- Testing
- TTX-381
- Where
- 2 sites in Germany, United Kingdom
- Volunteers
- 26 people
- Sponsor
- Tern Therapeutics, LLC
Neuronal Ceroid Lipofuscinosis, Neuronal Ceroid Lipofuscinosis CLN1
Clinical and Neuropsychological Investigations in Batten Disease
This study aims to assess the natural history of Batten disease (Neuronal Ceroid Lipofuscinosis) by obtaining information about the motor, behavioral, and functional capabilities of individuals with Batten disease.
- Conditions
- Neuronal Ceroid Lipofuscinosis, Neuronal Ceroid Lipofuscinosis CLN1, Neuronal Ceroid Lipofuscinosis CLN2, Neuronal Ceroid Lipofuscinosis CLN3
- Trial name
- NCT01873924
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- University of Rochester
Batten Disease, CLN2
Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease
The investigators propose a study to assess cognitive and developmental outcomes of patients with CLN2 that are untreated and receiving cerliponase alfa.
- Conditions
- Batten Disease, CLN2, Neuronal Ceroid-Lipofuscinoses
- Trial name
- NCT03862274
- Testing
- CLN2 Treatment
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Jessica Scherr