Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT)

NCT06621043 | Active Not Recruiting Phase 1

• 1 other identifier: NTIRTT1
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

Brief Summary

This 18-week open-label study examines the effectiveness of Full-Spectrum Medicinal Cannabis Plant Extract containing 0.08% THC (NTI164) in treating Rett syndrome (RTT) in children and young people. The study aims to determine the impact of NTI164 on RTT symptoms over a 16-week treatment period. Participants will start with a daily dose of 5 mg/kg of NTI164, which will be gradually increased over four weeks until they reach either the maximum tolerated dose or 20 mg/kg per day. They will then maintain this dose for eight weeks. Following this treatment phase, the dosage will be reduced by 5 mg/kg each week for four weeks until treatment concludes. The effectiveness of the treatment will be assessed using tailored questionnaires that measure changes in the patients\' conditions. Additionally, full blood examinations will be conducted at multiple points throughout the study to monitor the effects of the treatment.

Conditions

Rett Syndrome

Keywords

RTT

Outcome Measures

Primary Outcomes (1)

• Rett Syndrome: Symptom Index Score

  The Rett Syndrome: Symptom Index Score (RTT: SIS) measures the severity of various symptoms in individuals with Rett syndrome. Individual scores are from 0 to 7 with lower scores reflecting improvement or milder symptoms.

  Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment

Secondary Outcomes (7)

• Clinical Global Impression - Improvement (CGI-I)

  Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment

• Rett Syndrome Behaviour Questionnaire

  Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment.

• RTT-Clinician Domain Specific Concerns - Visual Analog Scale

  Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment.

• Communication and Symbolic Behaviour Scales Developmental Profile™ Infant-Toddler Checklist

  Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment.

• Impact of Childhood Neurological Disability Scale

  Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment.

Study Arms (1)

Active NTI164 Arm

EXPERIMENTAL

Participants in this arm receive Full-Spectrum Medicinal Cannabis Plant Extract containing 0.08% THC (NTI164), targeted at treating symptoms of Rett syndrome (RTT) in children and young people. The intervention starts with a daily initial dose of 5 mg/kg, which is increased gradually over a four-week up-titration phase to a maximum of 20 mg/kg per day, depending on individual tolerance. Participants then continue at their maximum tolerated dose for eight weeks during the treatment phase. The study concludes with a four-week down-titration phase, where the dosage is reduced by 5 mg/kg each week. The effectiveness of the treatment is monitored through specialized questionnaires and full blood examinations throughout the study duration.

Drug: NTI164

Interventions

NTI164 DRUG

Full-Spectrum Medicinal Cannabis Plant Extract containing 0.08% THC, administered orally starting with a dose of 5 mg/kg, titrated up to 20 mg/kg based on tolerance, followed by a maintenance phase at the maximum tolerated dose, and concluding with a gradual dose reduction.

Active NTI164 Arm

Eligibility Criteria

• Age: 5 Years - 20 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Girls and women, aged 5-20 years
• Weight greater than or equal to 12kg
• Classical/typical RTT
• Documented disease-causing mutation in MECP2 gene
• At least 6 months post regression at screening (ie. no loss or degradation in ambulation, hand function, speech, nonverbal communicative or social skills within 6 months of screening) Rett Syndrome Clinical Severity Scale rating of 10-36
• CGI score of 4 or higher.
• Stable pattern of seizures, or has had no seizures, within 8 weeks of screening.

You may not qualify if:

• 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 celiac disease or inflammatory bowel disease), or major surgery planned during the study.
• Known history or symptoms of long QT syndrome.
• QTcF interval \>450 ms, history of risk factor for torsades de pointes or clinically significant QT prolongation deemed to increase risk.
• Treatment with insulin, IGF-1, or growth hormone within 12 weeks of baseline.
• Currently receiving treatment with DAYBUETM (trofinetide).
• Currently using other unregistered drugs for the treatment of Rett syndrome such as Anavex.
• Currently using or has used recreational or medicinal cannabis, cannabinoid-based medications (including Sativex®, or Epidiolex®) within the 12 weeks prior to screening and is unwilling to abstain for the duration of the trial.
• Participant has any known or suspected hypersensitivity to cannabinoids or any of the excipients.

Sponsors & Collaborators

• Fenix Innovation Group: lead
• Neurotech International Limited: collaborator

Study Sites (1)

The Childrens Hospital at Westmead

Sydney, New South Wales, 2145, Australia

Location

MeSH Terms

Conditions

Rett Syndrome

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 11, 2024
• First Posted: October 1, 2024
• Study Start: September 20, 2023
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: October 1, 2024

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1)