Active — Not RecruitingPhase 1/2

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

Australia14 participantsStarted 2023-09-20

Plain-language 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.

Who can participate

Age range

5 Years – 20 Years

Sex

FEMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Rett Syndrome: Symptom Index Score

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

Trial details

NCT IDNCT06621043

SponsorFenix Innovation Group

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-12

View on ClinicalTrials.gov More Rett Syndrome trials