ClinicalTrials.gov record NCT07257978
Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome
A Phase II/III Double-blind, Randomised, Placebo-controlled, Crossover Study Investigating the Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This randomized, double-blind, placebo-controlled crossover study tests NTI164, a full‑spectrum medicinal cannabis extract with very low THC, in people with classical Rett syndrome. It is for females aged 4–25 with Rett syndrome confirmed by an MECP2 mutation. Participants receive both NTI164 and placebo in different treatment periods over a total study time of about 28 to 52 weeks. Blood tests are collected to study how NTI164 affects biology.
What it is studying. Whether NTI164 is safe and affects clinical measures of Rett syndrome compared with placebo, and what biological changes in blood are associated with treatment.
What the researchers hope to learn. To evaluate the efficacy and safety of NTI164 versus placebo for clinical symptoms of Rett syndrome; to investigate biological mechanisms using multi-omic blood analyses; to collect clinician- and caregiver-rated outcome data using validated scales.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
The FENRTT2 study will investigate the efficacy and safety of a medicinal cannabis plant extract with extremely low THC (delta-9-tetrahydrocannabinol), NTI164, on Rett syndrome (RTT) in a crossover design. RTT is a devastating rare genetic condition affecting females and involves debilitating physical and intellectual symptoms. NTI164 is an oil which has demonstrated efficacy in reducing symptoms in several paediatric neurological conditions, including RTT, autism spectrum disorder (ASD), and paediatric acute-onset neuropsychicatric syndrome (PANS). A Phase I/II clinical trial of NTI164 in RTT (FENRTT1/NTIRTT1) showed NTI164 is safe in this population and significantly improved overall clinical severity of illness, as well as core RTT symptoms, including anxiety, mental alertness, communication skills, socialisation/eye contact, and attentiveness. The FENRTT2 study will investigate NTI164 in a larger number of patients, and compare NTI164 to a placebo control. Research tests on patient blood will also be included to further investigate how NTI164 works in the body.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- NTI164
- Placebo
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Females aged 4-25 years of age
- Weight ≥12 kg
- Classical/typical RTT as confirmed with a documented pathogenic variant in the MECP2 gene
- 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)
- Rett Syndrome Clinical Severity Scale rating of 10-36
- Clinical Global Impression - Severity of Illness score ≥4
- Stable pattern of seizures or has had no seizures within 8 weeks of screening, as determined by the participant's primary physician
- 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
- 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
- Known history or symptoms of long QT syndrome
- QTcF interval >450 milliseconds, history of risk factor for torsades de pointes or clinically significant QT prolongation deemed to increase risk
- Currently receiving treatment with DAYBUE™ (Trofinetide)
- Currently using other unregistered drugs for the treatment of Rett syndrome, such as Anavex®
- 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
- A known or suspected hypersensitivity to cannabinoids or any of the excipients
- 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.
- 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
- 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)
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Show the detailed description from the registry
The FENRTT2 study will investigate the efficacy and safety of a full-spectrum medicinal cannabis plant extract with extremely low THC, NTI164, on Rett syndrome (RTT). This study will be a randomised, placebo-controlled, double-blind, crossover study spanning from 28 weeks up t0 52 weeks. RTT is a devastating rare genetic condition affecting females and involves debilitating physical and intellectual symptoms, with inflammation often driving the progression of symptoms. NTI164 is a potently anti-inflammatory oil which has demonstrated efficacy in reducing symptoms in several paediatric neurological conditions, including RTT (Phase I/II), autism spectrum disorder (ASD), and paediatric acute-onset neuropsychicatric syndrome (PANS). A Phase I/II clinical trial of NTI164 in RTT (FENRTT1) showed NTI164 is safe in this population and significantly improved overall clinical severity of illness, as well as core RTT symptoms, including anxiety, mental alertness, communication skills, socialisation/eye contact, and attentiveness. The FENRTT2 study will investigate NTI164 in a larger number of patients and will seek to demonstrate superiority over placebo in clinical outcomes in this cohort of patients. Multi-omic analyses on patient blood will also be included to further investigate the mechanism of action of NTI164, including transcriptomics, proteomics, phosphoproteomics, methylation, and cytokine analyses. Functional and clinical benefit will be measured using several validated, gold-standard assessment tools, rated by both clinicians and parents.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants are randomized into a double‑blind crossover and will receive NTI164 (a low‑THC medicinal cannabis oil) and placebo in different treatment periods over about 28 to 52 weeks. They will attend clinic visits for clinician assessments and caregivers will complete questionnaires at baseline, Week 12, and Week 28; blood samples will be taken for multi‑omic research (transcriptomics, proteomics, phosphoproteomics, methylation, cytokines). Screening and on‑study safety checks (including ECG for QT interval and liver blood tests) are required. Participants must avoid cannabis/cannabinoid products during the trial and maintain stable concomitant medications as specified.
How the study is structured. An interventional (drug) study that is randomized, double-blind and quadruple-masked, with a placebo-controlled crossover design. Each participant receives NTI164 in one treatment period and placebo in another; the study spans roughly 28 to 52 weeks. Outcomes are measured by clinician- and caregiver-rated scales and by blood-based multi-omic analyses.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- The listed sites are in Australia. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Rett syndrome
Important dates
- Study start
- Jul 1, 2027 (estimated)
- Primary completion
- Oct 1, 2028 (estimated)
- Study completion
- Oct 1, 2028 (estimated)
- Target enrollment
- 40 participants (estimated)
- Last updated on registry
- Sep 9, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Fenix Innovation Group
- Collaborators
- Neurotech International Limited
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Kanan Sharma 📞 +61395946666 ✉ MonashChildrensCTC@monashhealth.org
- Michael C Fahey
Participating sites (1)
- Monash Health
Clayton, Victoria, 3168 · Australia
- Kanan Sharma · +61395946666 · MonashChildrensCTC@monashhealth.org
Terms used on this page · explanations by MedicalUpdates.ai
- crossover
- Each participant receives more than one intervention, one after the other, usually with a gap (a washout) in between. Participants act as their own comparison.
- Phase I/II
- A study that combines safety and dose-finding (phase 1) with a first look at whether the treatment has an effect (phase 2).
- placebo
- A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
- at screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- QTcF
- A recording of the heart's electrical activity. QTc is one measurement from it; some drugs are avoided if it is prolonged.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- randomised
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
- placebo-controlled
- A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
- double-blind
- Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
- superiority
- What a comparison is designed to show. A superiority study asks whether the new treatment is better than the comparator; a non-inferiority study asks whether it is not meaningfully worse.
- cohort
- A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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