Phase 3 study
Not yet recruiting Phase 3
This trial at a glance
Where this trial is running
| Site | City | State or country | Status at this site |
|---|---|---|---|
| Monash Health | Clayton | VIC | Status not stated |
voxsanity.com.au · trial sites from public registries · registry record synced 11 October 2026 · not medical advice
Who can join
The official eligibility criteria
Registry text, reproduced exactly. These are the words the trial's sponsor published on ClinicalTrials.gov. They have not been edited, shortened or rewritten, and they are written for researchers rather than for patients.
Inclusion Criteria:
1. Females aged 4-25 years of age
2. Weight ≥12 kg
3. Classical/typical RTT as confirmed with a documented pathogenic variant in the MECP2 gene
4. At least 6 months post-regression at screening (i.e. no loss or degradation in ambulation, hand function, speech, non-verbal communication, or social skills within 6 months of screening)
5. Rett Syndrome Clinical Severity Scale rating of 10-36
6. Clinical Global Impression - Severity of Illness score ≥4
7. Stable pattern of seizures or has had no seizures within 8 weeks of screening, as determined by the participant's primary physician
8. Other patient medications must be stable (i.e. no dose adjustments) for at least 8 weeks prior to screening, including steroids, anti-inflammatories, anxiolytics etc
Exclusion Criteria:
1. 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 coeliac disease or inflammatory bowel disease), or major surgery planned
2. Known history or symptoms of long QT syndrome
3. QTcF interval \>450 milliseconds, history of risk factor for torsades de pointes or clinically significant QT prolongation deemed to increase risk
4. Currently receiving treatment with DAYBUE™ (Trofinetide)
5. Currently using other unregistered drugs for the treatment of Rett syndrome, such as Anavex®
6. Currently using or has used recreational or medicinal cannabis or 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
7. A known or suspected hypersensitivity to cannabinoids or any of the excipients
8. Moderate-severe impairment in hepatic function at screening, defined as serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> 2 x upper limit of normal (ULN), or total bilirubin (TBL) \> 2 x ULN. This criterion can only be confirmed once laboratory results are available, participants enrolled into the trial who are later found to meet this criterion will be screen-failed.
9. Participant is enrolled in another clinical trial within 14 days of screening or becomes enrolled in another clinical trial throughout the duration of this study
10. Infection and/or antibiotic use in the 2 weeks prior to screening (participants can be recruited following 2 weeks without infection and/or antibiotic use)
Dates on the record
Results, publications and the official record
- No results are posted on the official record yet.
- No publications are listed on the official record yet.
- Official record and contacts on ClinicalTrials.gov – contact details for each site are kept there, and are not copied onto this page.
Sources and dates
- ClinicalTrials.gov – the record this page reformats
- ClinicalTrials.gov processing date: 9 September 2026.
- This page last checked by Voxsanity: 11 October 2026.
Voxsanity reformats data from public registries into this layout. It does not edit, interpret or add to what the registry published.
Not medical advice.
Individual trial records are refreshed on a rolling basis rather than all at once, so a record may be up to several weeks old. Each trial page and trial card shows the date its own record was last synced. Where a record has lagged, its status, sites and enrolment can differ from the current registry state in either direction, so check the registry link on the trial page before acting on them.
Letter for your doctor
A one-page summary of this trial to print or email to your GP. It carries the registry's own record of the trial and its eligibility criteria, and nothing Voxsanity has written about whether it suits you.