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Low-THC cannabis extract tested against rett syndrome symptoms

NCT ID NCT06621043

Symptom relief Sponsor: Fenix Innovation Group Source: ClinicalTrials.gov ↗

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Sep 09, 2026 · Last updated Sep 10, 2026 · Updated 1 time

Summary

Researchers are testing a full-spectrum medicinal cannabis extract called NTI164 in girls and young women with Rett syndrome, a rare genetic disorder that affects movement, speech, and behavior. The study includes 14 participants aged 5 to 20 who take the extract daily for 16 weeks, starting at a low dose and increasing it as tolerated. The goal is to see whether the treatment can reduce symptom severity, measured through questionnaires and blood tests.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
a full-spectrum medicinal cannabis plant extract containing 0.08% THC
What this could lead to
If it works, this could offer a new way to ease the challenging symptoms of Rett syndrome, a rare genetic disorder with few treatment options.
What could go wrong
This is a small, early-stage study with only 14 participants and no control group, so any improvements could be due to chance or placebo. Cannabis-based treatments can also cause drowsiness, digestive issues, or other side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

14 people

The number who actually took part.

Started

Sep 2023

Finished

Dec 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

5 to 20 years

Sex

Female participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

Rett syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Rett syndrome

Contacts and locations

Locations

  • The Childrens Hospital at Westmead

    Sydney, New South Wales, 2145, Australia

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