Clinical TrialNeurocognitive DisordersPsilocybinTemporarily not available

An Investigation of Psilocybin on Behavioural and Cognitive Symptoms of Adults With Fragile X Syndrome

This open-label trial (n=10) will examine low-dose psilocybin as a safe treatment alternative for Fragile X Syndrome (FXS), aiming to improve markers of cognition, communication, mood, behaviour, neuroinflammation, serotonin levels in exosomes, and neuroplasticity.

Target Enrollment
10 participants
Study Type
Phase II interventional
Design
Non-randomized

Detailed Description

Phase II, open-label, single-group feasibility study of low-dose psilocybin (1.5 mg capsules) administered every other day for 28 days in adults with Fragile X syndrome (n=10).

Assessments include clinician- and caregiver-reported measures (VABS-3, CGI-I, ADAMS, VAS-TS), cognitive tests (NIH-TCB, TMT, MET), safety monitoring (DOA, ECG, vitals) and biomarkers (saliva/buccal swabs, neuroinflammation and serotonin markers in exosomes).

Drug dispensed in blister packs weekly; adherence monitored, unused study medication returned at visits; subjects attend visits at baseline and days 8, 15, 22 and 28.

Study Protocol

Preparation

sessions

Dosing

14 sessions

Integration

sessions

Study Arms & Interventions

Psilocybin 1.5 mg

experimental

Open-label single-group microdosing regimen: 1.5 mg capsule every other day for 28 days.

Interventions

  • Psilocybin1.5 mg
    via Oral• every other day• 14 doses total

    One 1.5 mg capsule on days 1,3,...,27; blister packs dispensed weekly; no more than five capsules (7.5 mg) dispensed at a time; skip missed doses.

Participants

Ages
18 – 50
Sexes
Male & Female

Inclusion Criteria

  • Inclusion Criteria:
  • 1. 18 to 50 years of age
  • 2. BMI > 18.3
  • 3. Diagnosis of Fragile X syndrome with a molecular genetic confirmation of the full FMR1 mutation (>200 CGG repeats) or other loss of function mutations of the FMR1 gene (SNVs and deletions) based on evidence provided by caregiver from prior assessment
  • 4. IQ between 40 and 85 points as reported by caregiver based on prior assessment
  • 5. Subject has the ability to understand and provides voluntary, written, informed consent to participate in the study
  • Or,
  • For subjects who are not their own legal guardian, or do not have the capacity to provide informed consent, subject's parent/legal authorized guardian is able to understand and sign an informed consent form to participate in the study.
  • 6. Caregiver (parent, guardian, or other legally authorized representative) who is willing to participate in the whole study and provides informed consent
  • 7. Subject is able to swallow tablets and capsules
  • 8. Individual is not of child-bearing potential, defined as those who have undergone a sterilization procedure or have been post-menopausal for at least 1 year prior to screening
  • Or,
  • Individuals of child-bearing potential must have a negative baseline urine pregnancy test and agree to use a medically approved method of birth control for the duration of the study. All hormonal birth control must have been in use for a minimum of three months. Acceptable methods of birth control include abstinence, hormonal contraceptives, double-barrier method, intrauterine devices, vasectomy of partner at least 6 months prior to screening, or agreement to use contraception if planning on changing to heterosexual partner(s).

Exclusion Criteria

  • Exclusion Criteria:
  • 1. Individuals who are pregnant, breast feeding, or planning to become pregnant during the study
  • 2. Allergy, sensitivity, or intolerance to the investigational product's active or inactive ingredients
  • 3. Regular use of medications that may have problematic interactions with psilocybin, including but not limited to dopamine agonists, MAO inhibitors, N-methyl-D-aspartate (NMDAR) antagonists, antipsychotics, and stimulants
  • 4. Urine drug test containing non-prescribed drugs of abuse (non-prescribed opioids, benzodiazepines, amphetamines, phencyclidine, cocaine) at screening and day of first treatment. Urine cannabinoid concentrations >50 ng/ml will suggest heavy marijuana use and will be a threshold for excluding potential subjects
  • 5. Having uncontrolled hypertension defined as an average systolic blood pressure ≥140 mmHg or an average diastolic blood pressure ≥90 mmHg, with at least 4 BP assessments completed
  • 6. Have any of the following cardiovascular conditions: coronary artery disease, congenital long QT syndrome, cardiac hypertrophy, cardiac ischemia, congestive heart failure, myocardial infarction, tachycardia, artificial heart valve, a clinically significant screening ECG abnormality, or any other significant cardiovascular condition
  • 7. Significant cardiovascular event in the past 6 months; participants with no significant cardiovascular event on stable medication may be included after assessment by the QI on a case-by-case basis
  • 8. Subjects with a history of seizure disorder except those who are currently receiving treatment with anti-epileptics and have been seizure-free for 3 months preceding screening or have been seizure-free for 3 years if not currently receiving anti-epileptics
  • 9. Reported history of moderate to severe hepatic impairment
  • 10. Type I or insulin-dependent Type II diabetes
  • 11. Meet DSM-5 criteria for schizophrenia spectrum or other psychotic disorders, including major depressive disorder with psychotic features, or Bipolar I or Bipolar II Disorder
  • 12. Meet DSM-5 criteria for a moderate or severe alcohol or drug use disorder in the past 12 months
  • 13. Subjects who plan to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study
  • 14. Medical illness based on physical examination, ECG and routine testing that may complicate cardiovascular safety or drug metabolism or excretion, such as metabolic disease, severe cardiac disease, or kidney or liver failure as assessed by the QI
  • 15. Current or history of any significant diseases of the gastrointestinal tract, exceptions to be determined by the QI
  • 16. Unstable hypertension; treatment on a stable dose of medication for at least 3 months will be considered by the QI on a case-by-case basis
  • 17. Major surgery in the past 3 months or individuals who have planned surgery during the course of the study
  • 18. Participation in other clinical research studies 30 days prior to enrollment
  • 19. Any other condition or lifestyle factor that, in the opinion of the QI, may adversely affect the participant's ability to complete the study or its measures or pose significant risk to the participant

Study Details

  • Status
    Temporarily not available
  • Phase
    Phase II
  • Type
    interventional
  • Design
    Non-randomized
  • Target Enrollment10 participants
  • Timeline
    Start: 2023-03-28
    End: 2023-08-01
  • Compound
    Psilocybin
  • Topic
    Neurocognitive Disorders

Locations

KGK Science Inc. — London, Ontario, Canada

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