Low-THC Cannabis Extract Shows Promise for Rett Syndrome Symptoms in Girls
An open-label trial of 11 girls with Rett syndrome found a full-spectrum cannabis extract (0.08% THC) improved communication, alertness, anxiety, and quality of life over 12 weeks.
Quick Facts
What This Study Found
NTI164 improved global clinical impression (p = 0.028), communication skills (p = 0.003), socialization/eye contact (p = 0.0004), attentiveness (p = 0.001), anxiety (p = 0.004), and quality of life (p = 0.0002) in 11 girls with genetically confirmed Rett syndrome over 12 weeks.
Key Numbers
11 participants; improvements in communication (p = 0.003), eye contact/socialization (p = 0.0004), attentiveness (p = 0.001), anxiety (p = 0.004), quality of life (p = 0.0002), caregiver burden (p = 0.006).
How They Did This
Phase I/II open-label study of 11 female participants aged 5-16 years with pathogenic MECP2 variants. NTI164 administered twice daily for 12 weeks with multiple validated outcome measures.
Why This Research Matters
Rett syndrome is a severe neurodevelopmental disorder with very limited treatment options. While this is a small open-label study, the consistency of improvements across multiple validated outcome measures is noteworthy for a condition with few effective interventions.
The Bigger Picture
Several groups are exploring cannabinoid therapies for rare neurological conditions. Rett syndrome, caused by mutations in a single gene (MECP2), offers a relatively well-defined population for studying potential benefits.
What This Study Doesn't Tell Us
Very small sample (n = 11) with no control group or blinding. Open-label design means placebo and caregiver expectation effects cannot be ruled out. 12-week duration may not capture long-term effects or tolerance.
Questions This Raises
- ?Would a placebo-controlled trial confirm these improvements?
- ?Which components of the full-spectrum extract are most responsible for the observed effects?
Trust & Context
- Key Stat:
- Socialization/eye contact improved with p = 0.0004
- Evidence Grade:
- Small open-label study without a control group, though it used genetically confirmed participants and multiple validated outcome measures.
- Study Age:
- 2025 publication
- Original Title:
- Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) Improves Symptoms of Rett Syndrome: An Open-Label Study.
- Published In:
- Journal of paediatrics and child health (2025)
- Authors:
- Keating, B A, Ogru, Y, Duthy, T G, Douglas, L, Lichkus, K, Isikgel, E, Fahey, M C, Ellaway, C
- Database ID:
- RTHC-06807
Evidence Hierarchy
Frequently Asked Questions
Can cannabis help with Rett syndrome?
In this small open-label trial, a low-THC cannabis extract improved communication, eye contact, attention, anxiety, and quality of life in 11 girls with Rett syndrome. However, without a control group, placebo effects cannot be ruled out.
What type of cannabis product was used for Rett syndrome?
The study used NTI164, a full-spectrum medicinal cannabis extract containing only 0.08% THC, administered twice daily. It contains multiple cannabinoids beyond just CBD and THC.
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Cite This Study
https://rethinkthc.com/research/RTHC-06807APA
Keating, B A; Ogru, Y; Duthy, T G; Douglas, L; Lichkus, K; Isikgel, E; Fahey, M C; Ellaway, C. (2025). Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) Improves Symptoms of Rett Syndrome: An Open-Label Study.. Journal of paediatrics and child health. https://doi.org/10.1111/jpc.70122
MLA
Keating, B A, et al. "Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) Improves Symptoms of Rett Syndrome: An Open-Label Study.." Journal of paediatrics and child health, 2025. https://doi.org/10.1111/jpc.70122
RethinkTHC
RethinkTHC Research Database. "Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NT..." RTHC-06807. Retrieved from https://rethinkthc.com/research/keating-2025-fullspectrum-medicinal-cannabis-plant
Access the Original Study
Study data sourced from PubMed, a service of the U.S. National Library of Medicine, National Institutes of Health.
This study breakdown was produced by the RethinkTHC research team. We analyze and report published research findings without making health recommendations. All interpretations are based solely on the published abstract and study data.