Clinical Trials

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Clinical Trial
  • Status: Not yet recruiting
  • Phase: Phase 1
  • Intervention Type: Genetic
  • Participants: 4
  • Start Date: May 1, 2021
Phase 1 Intrathecal Lumbar Administration of AAV9/CLN7 for Treatment of CLN7 Disease
Clinical Trial
  • Status: Not yet recruiting
  • Participants: 50
  • Start Date: March 31, 2021
A Retrospective, Chart Review Study to Evaluate Ocular Disease Progression in Children With Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
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Clinical Trial
  • Status: Not yet recruiting
  • Participants: 50
  • Start Date: March 31, 2021
A Prospective, Observational Study to Evaluate Ocular Disease Progression in Children With CLN2 Batten Disease
Clinical Trial
  • Status: Not yet recruiting
  • Phase: Phase 2
  • Intervention Type: Drug
  • Participants: 24
  • Start Date: February 1, 2021
A Safety, Tolerability, and Efficacy Study of PLX-200 in Participants With Mild-to-Moderate Juvenile Neuronal Ceroid Lipofuscinosis (CLN3) Disease
Clinical Trial
  • Status: Not yet recruiting
  • Participants: 195
  • Start Date: February 2021
Natural History Study of Batten Disease
Clinical Trial
  • Status: Recruiting
  • Intervention Type: Drug, Device
  • Participants: 35
  • Start Date: August 19, 2020
Cerliponase Alfa Observational Study
Clinical Trial
  • Status: Recruiting
  • Phase: N/A
  • Intervention Type: Genetic
  • Participants: 172
  • Start Date: July 1, 2020
Using Normalization Process Theory to Evaluate Providing Pediatric Palliative Care at End-of-Life as Web-Based Training Intervention for Nurses:
Clinical Trial
  • Status: Recruiting
  • Intervention Type: Other
  • Participants: 500
  • Start Date: April 8, 2020
Natural History and Long Term Clinical Assessments of All Forms of Neuronal Ceroid Lipofuscinoses - Capturing Key Symptoms and Disease Progression as Part of the Independent, International NCL DEM-CHILD Patient Database
Clinical Trial
  • Status: Recruiting
  • Intervention Type: Genetic
  • Participants: 13
  • Start Date: January 24, 2020
Long-Term Follow-Up of AT-GTX-501 scAAV9 Gene Transfer in Subjects With CLN6 Batten Disease
Clinical Trial
  • Status: Recruiting
  • Participants: 5
  • Start Date: November 1, 2019
Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency (Neuronal Ceroid Lipofuscinosis Type 2) Patients
Clinical Trial
  • Status: Recruiting
  • Participants: 30
  • Start Date: March 13, 2019
A Natural History and Outcome Measure Discovery Study of Variant Late Infantile Neuronal Ceroid Lipofuscinosis Type 5 (CLN5) and Variant Late Infantile Neuronal Ceroid Lipofuscinosis Type 7 (CLN7)
Clinical Trial
  • Status: Recruiting
  • Intervention Type: Other
  • Participants: 30
  • Start Date: December 1, 2018
Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease
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