NCT03770572

Gene Therapy for Children With CLN3 Batten Disease

Active, enrolled Phase 1, PHASE2 Last updated 7 August 2025

What this trial tests

Phase 1, PHASE2 trial testing Low dose CLN-301 in CLN3 in 7 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

13 November 2018

Primary endpoint
30 September 2029

30 September 2029

Quick facts

Lead sponsor	Alcyone Therapeutics, Inc
Phase	Phase 1, PHASE2
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	non randomized
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	7
Start date	13 November 2018
Primary completion	30 September 2029
Estimated completion	30 September 2029
Sites	1 location across United States

Drugs / interventions tested

• Low dose CLN-301
• High dose CLN-301

Conditions studied

• CLN3 — all drugs for CLN3 →
• Batten Disease — all drugs for Batten Disease →

Sponsor

Alcyone Therapeutics, Inc — full company profile →

Who can join

Adults 3 to 10, any sex, with CLN3 or Batten Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Current Clinical Applications of In Vivo Gene Therapy with AAVs.
   Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, et al · · 2021 · cited 544× · PMID 33309881 · DOI 10.1016/j.ymthe.2020.12.007
2. Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality.
   Hudry E, Vandenberghe LH. · · 2019 · cited 263× · PMID 30844402 · DOI 10.1016/j.neuron.2019.02.017
3. Viral vector-based gene therapies in the clinic.
   Zhao Z, Anselmo AC, Mitragotri S. · · 2022 · cited 171× · PMID 35079633 · DOI 10.1002/btm2.10258
4. Management of Neuroinflammatory Responses to AAV-Mediated Gene Therapies for Neurodegenerative Diseases.
   Perez BA, Shutterly A, Chan YK, Byrne BJ, et al · · 2020 · cited 88× · PMID 32098339 · DOI 10.3390/brainsci10020119
5. Current and Emerging Treatment Strategies for Neuronal Ceroid Lipofuscinoses.
   Kohlschütter A, Schulz A, Bartsch U, Storch S. · · 2019 · cited 85× · PMID 30877620 · DOI 10.1007/s40263-019-00620-8
6. Comparison of high-dose intracisterna magna and lumbar puncture intrathecal delivery of AAV9 in mice to treat neuropathies.
   Bailey RM, Rozenberg A, Gray SJ. · · 2020 · cited 61× · PMID 32289279 · DOI 10.1016/j.brainres.2020.146832
7. Impaired autophagy: The collateral damage of lysosomal storage disorders.
   Myerowitz R, Puertollano R, Raben N. · · 2021 · cited 58× · PMID 33341443 · DOI 10.1016/j.ebiom.2020.103166
8. Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord.
   Jensen TL, Gøtzsche CR, Woldbye DPD. · · 2021 · cited 53× · PMID 34690692 · DOI 10.3389/fnmol.2021.695937