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

ConditionsRETT Syndrome With Proven MECP2 Mutation Rett Syndrome

Trial statusNot yet recruiting

Trial phasePhase 2, Phase 3

Trial typeInterventional

Biological sexFemale

Age4-25

SponsorFenix Innovation Group

About this trial

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.

Eligibility criteria

Qualifiers

Females aged 4-25 years of age

Weight ≥12 kg

Classical/typical RTT as confirmed with a documented pathogenic variant in the MECP2 gene

At least 6 months post-regression at screening (i.e. no loss or degradation in ambulation, hand function, speech, non-verbal communication, or social skills within 6 months of screening)

Disqualifiers

Current clinically significant cardiovascular, endocrine (such as hypo- or hyperthyroidism, type 1 diabetes, or uncontrolled type 2 diabetes), renal, hepatic, respiratory, or gastrointestinal disease (such as coeliac disease or inflammatory bowel disease), or major surgery planned

Known history or symptoms of long QT syndrome

QTcF interval >450 milliseconds, history of risk factor for torsades de pointes or clinically significant QT prolongation deemed to increase risk

Currently receiving treatment with DAYBUE™ (Trofinetide)

Trial design

Treatments tested in this trial

NTI164
Placebo

Treatment groups

40 Participants

are divided into 2 treatment groups

Sponsors and collaborators

Fenix Innovation Group

Lead sponsor

Neurotech International Limited

Collaborator

Source ClinicalTrials.gov