Sponsor study identifier
interventional
6
2 countries
2
Study Start
January 31, 2022
Actual (reported)Primary Completion
November 2028
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
November 2028
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
November 2028
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
December 17, 2021
Actual (reported)January 31, 2022
Actual (reported)February 8, 2022
Actual (reported)November 2028
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
November 2028
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Incidence of Treatment Emergent Adverse Events (TEAEs)
Incidence, type, severity, and frequency of TEAEs
5 years (multiple visits)
Incidence of Serious Adverse Events (SAEs)
Incidence, type, severity, and frequency of SAEs
5 years (multiple visits)
Incidence of clinical laboratory abnormalities
Incidence, type, severity, and frequency of clinical laboratory abnormalities
5 years (multiple visits)
Incidence of new nerve conduction study (NCS) abnormalities
Incidence, type, severity, and frequency of new nerve conduction study (NCS) abnormalities
5 years (multiple visits)
Change in Hamburg Scale, Motor and Language domain scores
5 years (multiple visits)
Change in Spectral Domain-Optical Coherence Tomography (SD-OCT)
5 years (multiple visits)
Change in Unified Batten Diseases Rating Scale (UBDRS)
5 years (multiple visits)
Change in Caregiver global impression of change
5 years (multiple visits)
Change in visual acuity measurements
5 years (multiple visits)
The study treatment is a recombinant serotype 9 adeno-associated virus encoding a codon-optimized human CLN5 transgene (hCLN5opt).
The study treatment is a higher dose of recombinant serotype 9 adeno-associated virus encoding a codon-optimized human CLN5 transgene (hCLN5opt).
The study treatment is a higher dose of recombinant serotype 9 adeno-associated virus encoding a codon- optimized human CLN5 transgene (hCLN5opt).
Participants with confirmed mutations in the CLN5 gene who meet all the inclusion and none of the exclusion criteria will be treated with a single intracerebroventricular (ICV) dose and a single intravitreal (IVT) dose of the study treatment.
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
University of Rochester
Rochester, New York, 14642, United States
Great Ormond Street Hospital for Children
London, WC1N 3JH, United Kingdom
Murray SJ, Wellby MP, Barrell GK, Russell KN, Deane AR, Wynyard JR, Gray SJ, Palmer DN, Mitchell NL. Efficacy of dual intracerebroventricular and intravitreal CLN5 gene therapy in sheep prompts the first clinical trial to treat CLN5 Batten disease. Front Pharmacol. 2023 Oct 24;14:1212235. doi: 10.3389/fphar.2023.1212235. eCollection 2023.
Mitchell NL, Murray SJ, Wellby MP, Barrell GK, Russell KN, Deane AR, Wynyard JR, Palmer MJ, Pulickan A, Prendergast PM, Casy W, Gray SJ, Palmer DN. Long-term safety and dose escalation of intracerebroventricular CLN5 gene therapy in sheep supports clinical translation for CLN5 Batten disease. Front Genet. 2023 Aug 8;14:1212228. doi: 10.3389/fgene.2023.1212228. eCollection 2023.
Effie Albanis, MD
Neurogene Inc.
6 participants (actual enrollment reported by the registry).
First Submitted
December 17, 2021
First Posted
February 8, 2022
Study Start
January 31, 2022
Actual (reported)Incidence of new physical and neurologic exam abnormalities
Incidence, type, severity, and frequency of new physical and neurologic exam abnormalities
5 years (multiple visits)
Contact the study team to confirm eligibility.
Primary Completion
November 2028
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
November 2028
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Last Updated
August 12, 2024
Record last verified: 2024-08
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Source: ClinicalTrials.gov.
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