A Phase I/II study to assess the safety and efficacy of full-spectrum medicinal cannabis plant extract 0.08% THC (NTI164) in the treatment of Rett syndrome (RTT)

Phase 1

Recruiting

• Conditions: Rett Syndrome; Neurological - Neurodegenerative diseases

• Registration Number: ACTRN12623000563662
• Lead Sponsor: Fenix Innovation Group

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2023-05-25
• Last Update Posted: 6 November 2023

Recruitment & Eligibility

• Status: Recruiting
• Sex: Female
• Target Recruitment: 14

Inclusion Criteria

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.

Exclusion Criteria

- 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.

Study & Design

• Study Type: Interventional
• Study Design: Not specified