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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: Active Phase 1 study of Batten Disease and CLN3, sponsored by Alcyone Therapeutics.
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. According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
The DLT is defined as any unanticipated AE that is considered related to CLN-301 and is Common Terminology Criteria for Adverse Events Grade 3 or higher.
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 |
What the registry record for NCT03770572 still lists
NCT03770572 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 7 participants, a relatively small participant target, below the 2,829-participant average among 11 other Batten Disease trials with a reported enrollment target (100% lower).
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 a single indexed study location in 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
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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.
Where NCT03770572's enrollment target sits among peer trials
7 12th of 11 the lowest of 11 other Batten Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Batten Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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