Clinical trials: Batten's Disease
Batten Disease, Late Infantile Neuronal Ceroid Lipofuscinosis
Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis
The primary aim of the study is to assess the genotype - phenotype correlations of the CNS manifestations of late infantile neuronal ceroid lipofuscinosis (LINCL), a fatal, rare, recessive disorder of the CNS in children.
- Trial name
- NCT01035424
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Weill Medical College of Cornell University
Cancer, Cardiac Anomaly
Using NPT to Evaluate Providing PPC as ELNEC-PPC WBT for Nurses
The purpose of this study is to explain the provision of palliative care at the end of life by the implementation of the ELNEC course, as WBT Program using the Normalization Process Theory, that focus attention on how complex…
- Conditions
- Cancer, Cardiac Anomaly, Cystic Fibrosis, Muscular Dystrophy
- Trial name
- ELNEC-PPC (NCT04461561)
- Testing
- end-of-life nursing education consortium-pediatric palliative care as web based-training plus usual care
- Where
- Iraq
- Volunteers
- 172 people, healthy volunteers welcome
- Sponsor
- Altoosi University College
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
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
Adrenoleukodystrophy, Metachromatic Leukodystrophy
Stem Cell Transplant for Inborn Errors of Metabolism
The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for an inherited metabolic…
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease
- Trial name
- NCT00176904
- Testing
- Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
- Where
- United States
- Volunteers
- 135 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Batten Disease, Late-Infantile Neuronal Ceroid Lipfuscinosis
Genotype - Phenotype Correlations of LINCL
In a separate protocol the Department of Genetic Medicine is proposing to carry out a study using gene transfer to treat the central nervous system (CNS) manifestations of late infantile neuronal ceroid lipofuscinosis (LINCL), a…
- Trial name
- NCT00151268
- Volunteers
- 18 people
- Sponsor
- Weill Medical College of Cornell University
Neuronal Ceroid Lipofuscinosis, Batten's Disease
Neuronal Ceroid Lipofuscinosis and Associated Sleep Abnormalities
Neuronal Ceroid Lipofuscinosis (NCL) also known at Batten's disease is the most common neurodegenerative disorder in children. Families often report the patient has a sleep disturbance.
- Trial name
- NCT01966757
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- Nationwide Children's Hospital
Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease
Collection of Cerebrospinal Fluid in Healthy Children
The purpose of this study is to obtain cerebrospinal fluid from healthy children already undergoing a lumbar puncture procedure at New York Presbyterian Hospital, to act as healthy controls when compared to children with late…
- Trial name
- NCT01698229
- Testing
- Group 1
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Weill Medical College of Cornell University