Gene Therapy Study for Children With CLN5 Batten Disease (CLN5-200)

• Research type

  Research Study

• Full title

  A Phase 1/2 Intracerebroventricular and Intravitreal Administration of NGN-101 for Treatment of Neuronal Ceroid Lipofuscinosis Subtype 5 (CLN5) Disease

• IRAS ID

  1004529

• Contact name

  Leslie Jacobsen

• Contact email

  Leslie.Jacobsen@Neurogene.com

• Sponsor organisation

  Neurogene Inc.

• Eudract number

  2022-000091-20

• Clinicaltrials.gov Identifier

  NCT05228145

• Research summary

  This study is a first in human (FIH) open-label study designed to assess the safety and efficacy of administration of an adeno-associated viral vector serotype 9 (AAV9) carrying the gene encoding human ceroid-lipofuscinosis neuronal protein 5 (CLN5) in patients with CLN5 Batten disease.

  Approximately 3 children, aged 3 to 8 years will participate in US and UK.

  The trial is divided into 4 periods
  • Pre-screening – which can be several weeks long to check eligibility
  • Screening – up to 45 days
  • Treatment - The study treatment will be delivered via intracerebroventricular (ICV) and intravitreal (IVT) injection on the same day
  • Follow-up - Each patient will be followed for safety and efficacy for 5 years after treatment

  Participation in the study will be just over 5 years. Participation is voluntary.

  Participants will be expected to attend clinic visits and will have video/telephone calls for efficacy assessments in this study which will evaluate motor, language, visual and cognitive function.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  22/LO/0383

• Date of REC Opinion

  4 Aug 2022

• REC opinion

  Further Information Favourable Opinion