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Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome

NCT07257978 · Status: NOT_YET_RECRUITING · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 40

Last updated 2025-12-02

No results posted yet for this study

Summary

The FENRTT2 study will investigate the efficacy and safety of a medicinal cannabis plant extract with extremely low THC (delta-9-tetrahydrocannabinol), NTI164, on Rett syndrome (RTT) in a crossover design. RTT is a devastating rare genetic condition affecting females and involves debilitating physical and intellectual symptoms. NTI164 is an oil which has demonstrated efficacy in reducing symptoms in several paediatric neurological conditions, including RTT, autism spectrum disorder (ASD), and paediatric acute-onset neuropsychicatric syndrome (PANS). A Phase I/II clinical trial of NTI164 in RTT (FENRTT1/NTIRTT1) showed NTI164 is safe in this population and significantly improved overall clinical severity of illness, as well as core RTT symptoms, including anxiety, mental alertness, communication skills, socialisation/eye contact, and attentiveness. The FENRTT2 study will investigate NTI164 in a larger number of patients, and compare NTI164 to a placebo control. Research tests on patient blood will also be included to further investigate how NTI164 works in the body.

Conditions

Interventions

DRUG

NTI164

NTI164 is a full-spectrum medicinal cannabis plant extract with \<0.3% THC.

DRUG

Placebo

Placebo

Sponsors & Collaborators

  • Neurotech International Limited

    collaborator INDUSTRY

  • Fenix Innovation Group

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
4 Years
Max Age
25 Years
Sex
FEMALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-07-01
Primary Completion
2028-10-01
Completion
2028-10-01

Countries

  • Australia

Study Locations

More Related Trials

Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07257978 on ClinicalTrials.gov