Clinical trials: Neuronal Ceroid-Lipofuscinoses
Inflammatory Bowel Diseases, Crohn Disease
N-3 Polyunsaturated Fatty Acids Prevent Postoperative Recurrence of Crohn's Disease
Crohn's disease (CD) is a chronic recurrent intestinal inflammation involving the whole digestive tract, with high disability rate, high surgical rate and high recurrent rate postoperatively.
- Conditions
- Inflammatory Bowel Diseases, Crohn Disease, Polyunsaturated Acid Lipidosis
- Trial name
- NCT04761952
- Testing
- azathioprine, infliximab, N-3 Polyunsaturated Fatty Acids
- Volunteers
- 236 people
- Sponsor
- Sixth Affiliated Hospital, Sun Yat-sen University
MLD, Krabbe Disease
Longitudinal Study of Neurodegenerative Disorders
The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the…
- Conditions
- MLD, Krabbe Disease, ALD, MPS I
- Trial name
- NCT03333200
- Testing
- Palliative Care, Hematopoetic Stem Cell Transplantation
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- University of Pittsburgh
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
Mucopolysaccharidosis I, Mucopolysaccharidosis VI
Human Placental-Derived Stem Cell Transplantation
The purpose of this clinical trial is to investigate the safety of human placental-derived stem cells (HPDSC) given in conjunction with umbilical cord blood (UCB) stem cells in patients with various malignant or nonmalignant…
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease
- Trial name
- HPDSC (NCT01586455)
- Testing
- Human Placental Derived Stem Cell
- Where
- 3 sites in United States
- Volunteers
- 43 people
- Sponsor
- New York Medical College
Psoriasis
Exploratory Study to Investigate Efficacy of LAS41004 in a Psoriasis Plaque Test
The aim of the exploratory study is to compare the dose-related efficacy of LAS41004 in a Psoriasis Plaque Test (PPT).
- Trial name
- NCT01360944
- Testing
- LAS41004, reference
- Where
- Germany
- Volunteers
- 22 people
- Sponsor
- Almirall, S.A.
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.
Juvenile Neuronal Ceroid Lipofuscinosis, Smith-Lemli-Opitz Syndrome
Functional Near-Infrared Spectroscopy (fNIRS) Combined With Diffuse Correlation Spectroscopy (DCS) in Neurocognitive Disease as Compared to Healthy Neurotypical Controls
Background: Neurocognitive disorders affect how the brain uses oxygen. They may affect mental development in children. These disorders can be studied with imaging scans that use radiation; however, these methods are not ideal for…
- Conditions
- Juvenile Neuronal Ceroid Lipofuscinosis, Smith-Lemli-Opitz Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type C1
- Trial name
- NCT05642221
- Where
- United States
- Volunteers
- 73 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Batten Disease, CLN6
Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae
CLN6 is a rare, neurodegenerative disease that causes progressive loss of acquired skills with motor delay, visual loss, seizures and ataxia.
- Trial name
- Batten'sCLN6 (NCT03285425)
- Testing
- Natural history
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Emily de los Reyes
Ceroid Lipofuscinosis, Neuronal 5
A Natural History Study of Neuronal Ceroid Lipofuscinosis Type 5 (CLN5)
CLN5 is a form of Batten Disease, a neurodegenerative disorder in children causing psychomotor regression, seizures, blindness, loss of ambulation and premature death, and has no available treatments.
- Trial name
- NCT03822650
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Neurogene Inc.
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)
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
Neuronal Ceroid Lipofuscinosis CLN5
Gene Therapy Study for Children With CLN5 Batten Disease
This is a prospective, non-randomized, open-label, dose escalation study of a single administration of gene therapy in children who are 3 to 9 years old with Neuronal Ceroid Lipofuscinosis (Batten) Subtype 5 (CLN5) disease.
- Trial name
- CLN5-200 (NCT05228145)
- Testing
- NGN-101
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 6 people
- Sponsor
- Neurogene Inc.
ALS, Amyotrophic Lateral Sclerosis
NYSCF Scientific Discovery Biobank
The New York Stem Cell Foundation (NYSCF) Research Institute is performing this research to accelerate diverse disease research using cells from the body (such as skin or blood cells) to make stem cells and other types of cells…
- Conditions
- ALS, Amyotrophic Lateral Sclerosis, Alzheimer Disease, Alzheimer Disease, Early Onset
- Trial name
- NCT06203106
- Testing
- Biological Sample Collection
- Where
- United States
- Volunteers
- 10,000 people
- Sponsor
- New York Stem Cell Foundation Research Institute
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
CLN3, Batten Disease
Gene Therapy for Children With CLN3 Batten Disease
This is a phase 1/2, open-label, single dose, dose-escalation clinical trial to evaluate the safety and efficacy of CLN-301 (previous NCH Code: scAAV9.P546.CLN3) delivered intrathecally into the lumbar spinal cord region of…
- Trial name
- NCT03770572
- Testing
- Low dose CLN-301, High dose CLN-301
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Neela Therapeutics