NCT06621043

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

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: Fenix Innovation Group · Industry
• Sex: Female
• Age: 5 Years – 20 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This study is an 18-week open-label trial designed to assess the effectiveness of Full-Spectrum Medicinal Cannabis Plant Extract containing 0.08% THC (NTI164) on the severity of symptoms in children and young people with Rett syndrome (RTT). The main objective is to determine how well NTI164 alleviates the symptoms of RTT over a 16-week treatment period. The study involves multiple phases: 1. \*\*Up-titration Phase (4 weeks):\*\* Treatment begins with an initial daily dose of 5 mg/kg of NTI164. Over the first four weeks, this dose is incrementally increased based on patient tolerance, aiming for a maximum daily dose of 20 mg/kg. 2. \*\*Treatment Phase (8 weeks):\*\* Once the maximum tolerated dose is established, participants continue to receive this dose for the following eight weeks. This phase focuses on maintaining a steady dosage to observe the therapeutic effects on RTT symptoms. 3. \*\*Down-titration Phase (4 weeks):\*\* After completing the treatment phase, the dosage is systematically reduced by 5 mg/kg each week for four weeks until treatment cessation. This gradual reduction helps in assessing any withdrawal effects and the stability of symptom improvement post-treatment. Throughout the study, the efficacy of NTI164 is evaluated using specifically designed questionnaires that track changes in the emotional, behavioral, and cognitive functions of the participants, providing insights into how the treatment impacts various aspects of RTT. Additionally, full blood examinations are conducted at key points during the study to monitor the physiological responses to the treatment and to ensure the safety and tolerability of NTI164. This comprehensive approach allows for a detailed assessment of both the immediate and longer-term effects of the medicinal cannabis extract on Rett syndrome symptoms.

Conditions

• Rett Syndrome

Interventions

Type	Name	Description
DRUG	NTI164	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.

Timeline

• Start date: 2023-09-20
• Primary completion: 2025-12-01
• Completion: 2025-12-01
• First posted: 2024-10-01
• Last updated: 2024-10-01

Locations

1 site across 1 country: Australia