With the Human Research Ethics Committee (HREC) approval and Clinical Trial Notification (CTN) scheme clearance by the Therapeutic Goods Administration (TGA) under its wing, Neurotech will investigate the use of NTI164 in female Rett Syndrome patients.

Rett Syndrome is the second leading cause of intellectual disability in girls, with an urgent medical need to develop safe and effective therapies to treat this progressive neurological disease.

Neurotech (ASX:NTI) executive director Dr Thomas Duthy says with its positive effects on neuroinflammation, NTI164 could represent an effective intervention in Rett Syndrome, where mutations in the MECP2 gene are known to exacerbate neuroinflammation.

The Phase 2 clinical trial will examine the effects of daily oral treatment of NTI164 on 14 Rett Syndrome patients initially and will be conducted across three centres in Australia.

And if successful, the company will follow with a 14-week double-blind, randomised, placebo-controlled Phase 2 in 34 participants to determine further efficacy and safety, which will require a separate HREC submission and approval.

Trial to assess safety, tolerability and effectiveness of daily oral NTI164

Associate professor Carolyn Ellaway says girls who suffer from Rett Syndrome require continued supportive care, with the disease negatively impacting their quality of life and activities of daily living.

“Despite recent advances in the field, as a clinician who has witnessed first-hand the impact of this progressive disorder on my patients, I am very excited to be involved with this clinical trial which will assess safety, tolerability and effectiveness of daily oral NTI164 in these girls,” she explains.

“We certainly look forward to the results of the study following 12 weeks of initial treatment.”

What’s next?

The proposed primary endpoint at 12 weeks of treatment is an improvement from baseline in the Clinical Global Impression Scale – Improvement (CGII).

Patient recruitment is expected to begin in early Q3 CY2023 and the preliminary (top-line) results of the trial are anticipated in Q1 CY2024.

This article was developed in collaboration with Neurotech, a Stockhead advertiser at the time of publishing.  

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