Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT03770572 · ClinicalTrials.gov registry record · Phase 1
Gene Therapy for Children With CLN3 Batten Disease
A Phase 1 study of Batten Disease and CLN3, sponsored by Alcyone Therapeutics.
- Active
- Registry status
- Phase 1
- Development phase
- 7
- Enrollment target
- 1
- Study location
NCT03770572 is a Phase 1 study of Batten Disease and CLN3 that is active but no longer recruiting, run by Alcyone Therapeutics. The registered enrollment target is 7 participants, below the 2,829-participant average among 11 other Batten Disease trials with a reported enrollment target (100% lower). The trial reports 1 study location across 1 state.
The verdict
NCT03770572, a Phase 1 study of Batten Disease and CLN3, is active but no longer recruiting, sponsored by Alcyone Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 7 participants
- Enrollment target
- 1
- Study location
Study Summary
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 subjects with CLN3 Batten disease.
Conditions Studied
Interventions
- GENETIC Low dose CLN-301
- GENETIC High dose CLN-301
Study Locations (1)
Ohio
- Nationwide Children's Hospital - Columbus
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 7 participants |
| Start Date | 2018-11-13 |
| Est. Completion | 2029-09-30 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03770572
The ClinicalTrials.gov registry entry for NCT03770572 describes a study currently listed as active not recruiting, categorized as Phase 1. The registered enrollment target is 7 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 2,829-participant average among 11 other Batten Disease trials with a reported enrollment target (100% lower). The listed sponsor is Alcyone Therapeutics, which has 3 total studies on file at ClinicalTrials.gov.
The record links to 2 conditions, with Batten Disease appearing as the primary indexed condition, and to 2 interventions - of which Low dose CLN-301 is the first listed.
NCT03770572 reports 1 study location spanning 1 distinct geographic area - top geographies include Ohio.
Frequently Asked Questions
What is clinical trial NCT03770572 about?
NCT03770572 is a clinical study titled "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 subjects with CLN3 Batten disease.
What is the current status of trial NCT03770572?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 7 participants. The study started on 2018-11-13. Estimated completion is 2029-09-30.
What conditions does trial NCT03770572 study?
This clinical trial studies the following conditions: Batten Disease, CLN3.
What interventions are being tested in trial NCT03770572?
The interventions under investigation include: Low dose CLN-301 (GENETIC), High dose CLN-301 (GENETIC).
Who is sponsoring clinical trial NCT03770572?
This trial is sponsored by Alcyone Therapeutics, which has 3 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03770572 being conducted?
This trial has 1 study location across Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Batten Disease
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
-
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
RECRUITING · Phase 1
-
Human Placental-Derived Stem Cell Transplantation
COMPLETED · Phase 1
-
Inherited Retinal Degenerative Disease Registry
RECRUITING
-
Clinical and Neuropsychological Investigations in Batten Disease
RECRUITING
-
NYSCF Scientific Discovery Biobank
RECRUITING
-
Investigations of Juvenile Neuronal Ceroid Lipofuscinosis
RECRUITING
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.