Clinical trials: CLN3
CLN3-related Disorders, Non-CLN3 Related Low Vision
Optic-to-Audio Device in a Pediatric Cohort With CLN3-related Conditions or Low Vision
Background: CLN3 involves vision loss observed around the preschool years, with eventual progression to blindness within 1-3 years.
- Trial name
- NCT04974307
- Testing
- OrCam MyEye 2
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
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
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
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
Juvenile Neuronal Ceroid Lipofuscinosis
Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis
The primary objective of this trial is to establish the safety and tolerability of short-term (8 weeks) administration of mycophenolate mofetil in ambulatory children with JNCL.
- Trial name
- JUMP (NCT01399047)
- Testing
- Mycophenolate mofetil
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- University of Rochester
Variant Late-Infantile Neuronal Ceroid Lipofuscinosis
Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease
This is a phase 1/2, open-label, single dose study to evaluate the safety and efficacy of AT-GTX-501 delivered intrathecally into the lumbar spinal cord region of participants with mild to moderate variant late infantile neuronal…
- Trial name
- NCT02725580
- Testing
- AT-GTX-501
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Emily de los Reyes
Batten Disease
An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease
This is an open label study in approximately 6 subjects in 2 centers to assess the safety, PK, and efficacy of the maximum tolerable dose (MTD) of oral miglustat (100 mg once daily [QD] to 200 mg 3 times daily [TID]) in subjects…
- Trial name
- NCT05174039
- Testing
- Miglustat 100 milligrams (mg) Oral Capsule
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Beyond Batten Disease Foundation
Infantile Neronal Ceroid Lipofuscinosis
Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis
This study will examine the effectiveness of a drug called Cystagon in treating infantile neuronal ceroid lipofuscinosis (INCL), a progressive neurological disease affecting children.
- Trial name
- NCT00028262
- Testing
- Cystagon
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)