Clinical trials: Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease
AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis
The investigators propose to assess the safety and efficacy of a new administration method to deliver a biologic to children with a form of Batten disease using an experimental gene transfer procedure.
- Trial name
- NCT01414985
- Testing
- AAVrh.10CUCLN2
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Weill Medical College of Cornell University
Jansky-Bielschowsky Disease, Batten Disease
An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
The Phase 1/2 study (190-201) evaluated the efficacy and safety of a 300 mg dose of BMN 190 administered every other week (qow) to patients with CLN2.
- Conditions
- Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease
- Trial name
- NCT02485899
- Testing
- BMN 190, Intracerebroventricular (ICV) access device
- Where
- 4 sites in United States, Germany and 2 more
- Volunteers
- 23 people
- Sponsor
- BioMarin Pharmaceutical
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
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
Cerliponase Alfa Observational Study in the US
This is a multicenter, observational study for patients with a confirmed diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as TPP1 deficiency, who intend to be or are currently being treated with…
- Trial name
- NCT04476862
- Testing
- Cerliponase Alfa, Administration Kit
- Where
- 16 sites in United States
- Volunteers
- 35 people
- Sponsor
- BioMarin Pharmaceutical
Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
A Retrospective, Natural History Study in Children With CLN2
This is a multi center, retrospective, chart review study to document the evolution of ocular disease progression in pediatric patients with CLN2.
- Trial name
- NCT04480476
- Sponsor
- REGENXBIO Inc.
Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis
Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)
This is a proposed follow up study on the investigators previous gene transfer human clinical trial entitled "Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Expressing the Human CLN2 cDNA to…
- Trial name
- NCT01161576
- Testing
- AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies, AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Weill Medical College of Cornell University
Jansky-Bielschowsky Disease, Batten Disease
A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease
The purpose of this study is to determine whether BMN 190 is safe and effective in the treatment of patients with Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) disease.
- Conditions
- Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease
- Trial name
- NCT01907087
- Testing
- BMN 190
- Where
- 5 sites in United States, Germany and 2 more
- Volunteers
- 24 people
- Sponsor
- BioMarin Pharmaceutical
Jansky-Bielschowsky Disease, Batten Disease
A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
This Phase 2 open-label, multicenter study will evaluate the safety, tolerability, and efficacy of BMN 190 intracerebroventricular (ICV) administration every other week (qow) for a period of 144 weeks, in patients with CLN2.
- Conditions
- Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease
- Trial name
- NCT02678689
- Testing
- BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1), Intracerebroventricular access device
- Where
- 4 sites in United States, Germany and 2 more
- Volunteers
- 14 people
- Sponsor
- BioMarin Pharmaceutical
Neuronal Ceroid-Lipofuscinoses, Neuronal Ceroid Lipofuscinosis CLN2
Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients
The purpose of this study is to gather information on the possible symptoms that patients with atypical neuronal ceroid lipofuscinosis type 2 (also known as aTPP1 or atypical tripeptidyl peptidase deficiency) have and how they…
- Conditions
- Neuronal Ceroid-Lipofuscinoses, Neuronal Ceroid Lipofuscinosis CLN2, Spinocerebellar Ataxia, Autosomal Recessive 7
- Trial name
- NCT04098211
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Children's Hospital of Orange County
Neuronal Ceroid Lipofuscinosis
Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)
Patients with infantile or late infantile NCL have either a reduced amount of, or are missing, the palmitoyl protein thioesterase 1 (PPT1) enzyme or the tripeptidyl peptidase 1 (TPP-I) enzyme.
- Trial name
- NCT00337636
- Testing
- Surgery to implant human CNS stem cells (HuCNS-SC), Medication to suppress the immune system
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- StemCells, Inc.
Neuronal Ceroid Lipofuscinosis Type 2
Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2
This is a phase I/II randomized, masked, clinical trial to determine the safety and efficacy of intravitreal administration of cerliponase alfa.
- Trial name
- NCT05152914
- Testing
- Cerliponase Alfa
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- David L Rogers, MD
Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
An Observational Study in Children With CLN2 Batten Disease
This is a prospective, longitudinal natural history study to document the progression of ocular manifestations of CLN2 disease among a community-dwelling population of pediatric participants affected by this disease.
- Trial name
- NCT04462692
- Sponsor
- REGENXBIO Inc.
Batten Disease, Late Infantile Neuronal Ceroid Lipofuscinosis
Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis
The aim of this study is to treat the signs and symptoms of late infantile neuronal ceroid lipofuscinosis (LINCL), a fatal inherited disease in the brain.
- Trial name
- NCT00151216
- Testing
- AAV2CUhCLN2 (3x10^12 particle units)
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Weill Medical College of Cornell University