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

Sponsor
Fenix Innovation Group
Study ID
NCT07257978
Phase
PHASE2/PHASE3
Status
Not Yet Recruiting

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Conditions

Eligibility Criteria

Sex
FEMALE
Age
4 Years - 25 Years
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date
Jul 1, 2026
Status verified
Jul 2025
Primary completion
Oct 1, 2028
Completion
Oct 1, 2028

Study Design

Enrollment
40 participants (estimated)
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT

Arms

  • Placebo Comparator: Placebo
    Placebo
  • Active Comparator: NTI164 active
    NTI164

Primary Outcome Measure

Rett Syndrome Behaviour Questionnaire (RSBQ) [ Time Frame: Baseline, Week 12, and Week 28 ]

Central Contacts

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