Fragile X SyndromePhase 2NCT05832255
An Investigation of Psilocybin on Behavioural and Cognitive Symptoms of Adults With Fragile X Syndrome
A Phase 2 study testing Psilocybin, 1.5 mg in people with Fragile X Syndrome and Behavior, run by Nova Mentis Life Science Corp at 1 site in Canada.
Trial summary
Diverse symptomatology makes Fragile X Syndrome (FXS) difficult to treat, and currently there are no approved prevention or treatment methods for FXS. Current therapies, including pharmaceutical and behavioural interventions, offer a patchwork of solutions that have limited efficacy and high toxicity. Read more about this study
- Who is it for?
- Ages 18 to 50 years
- Conditions the study is for
- Fragile X Syndrome, Behavior, Cognitive Dysfunction
- What is being tested?
- Psilocybin, 1.5 mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteCanada
- Study dates
- 28 Mar 2023to May 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 50 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 18 to 50 years of age
- BMI > 18.3
- Diagnosis of Fragile X syndrome with a molecular genetic confirmation of the full FMR1 mutation (>200 CGG repeats) or the other loss of function mutations of the FMR1 gene (SNVs and deletions of the gene) based on evidence provided by caregiver from prior assessment
- IQ between 40 and 85 points as reported by caregiver based on prior assessment
- Subject has the ability to understand and provides voluntary, written, informed consent to participate in the study
- Or,
Show all 18Show fewer
- 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.
- Caregiver (parent, guardian, or other legally authorized representative) who is willing to participate in the whole study and provides informed consent
- Subject is able to swallow tablets and capsules
- Individual is not of child-bearing potential, defined as those who have undergone a sterilization procedure (e.g. hysterectomy, bilateral oophorectomy, bilateral tubal ligation, complete endometrial ablation) 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 (only in cases when abstinence is the usual and customary form of birth control for the participant)
- Hormonal contraceptives including oral contraceptives, hormone birth control patch (Ortho Evra), vaginal contraceptive ring (NuvaRing), injectable contraceptives (Depo-Provera, Lunelle), or hormone implant (Norplant System)
- Double-barrier method
- Intrauterine devices
- Non-heterosexual lifestyle or agrees to use contraception if planning on changing to heterosexual partner(s)
- Vasectomy of partner at least 6 months prior to screening
Who cannot join
- Individuals who are pregnant, breast feeding, or planning to become pregnant during the study
- Allergy, sensitivity, or intolerance to the investigational product's active or inactive ingredients
- 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
- 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
- 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.
- Have any of the following cardiovascular conditions: coronary artery disease, congenital long QT (time from the start of the Q wave to the end of the T wave) 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
Show all 19Show fewer
- 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
- 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.
- Reported history of moderate to severe hepatic impairment
- Type I or insulin-dependent Type II diabetes
- 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
- Meet DSM-5 criteria for a moderate or severe alcohol or drug use disorder in the past 12 months
- Subjects who plan to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study
- 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. QI assessments will be based on clinical history provided by caregivers and/or physicians. Any participant requiring further assessment will be referred accordingly or excluded by the QI on a case-by-case basis.*
- Current or history of any significant diseases of the gastrointestinal tract, exceptions to be determined by the QI
- 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
- Major surgery in the past 3 months or individuals who have planned surgery during the course of the study. Participants with minor surgery will be considered on a case-by-case basis by the QI
- Participation in other clinical research studies 30 days prior to enrollment as assessed by the QI.
- 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 * Blood draw to obtain laboratory samples for analysis will be avoided in this study due to the undue stress it places on this clinical population and the high safety profile of the medication at the dosage being used.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Open-Label Investigation of the Effects of Sub-Perceptual Repeat Dosing of Psilocybin on the Behavioural and Cognitive Symptoms of Fragile X Syndrome in Adult Patients
Diverse symptomatology makes Fragile X Syndrome (FXS) difficult to treat, and currently there are no approved prevention or treatment methods for FXS. Current therapies, including pharmaceutical and behavioural interventions, offer a patchwork of solutions that have limited efficacy and high toxicity. The current study aims to examine psilocybin as a safe treatment alternative with the ability to improve markers of cognition, communication, mood, behavior as well as markers of neuroinflammation, serotonin levels in exosomes, and neuroplasticity at sub-hallucinogenic doses (microdosing).
The overall objective of this study is to assess the feasibility of low-dose psilocybin as a therapeutic option for individuals living with FXS and to improve diagnostic parameters of FXS, as well as therapeutic responses with the use of biomarkers.
Read the detailed description
A total of 10 subjects who meet all the inclusion criteria and does not meet any of the exclusion criteria will be enrolled into the study. Any subjects prematurely terminated from the study will be replaced to ensure 10 subjects complete the study. A study coordinator will contact referring clinicians, caregivers, and subjects to pre-screen for initial eligibility. Those deemed eligible will be invited for an in-person screening along with the participating caregiver. The screening visit will be approximately two hours long and will consist of informed consent, diagnostic interview, physical examination, drugs of abuse test (DOA), ECG, medical/treatment history review, and demographic forms. A pregnancy test will be performed on females of child-bearing during screening, baseline, and end-of-study visits. All eligible subjects will enter the treatment arm of the study.
Subjects and caregivers will return to the clinic for a baseline visit within three weeks of their screening completion. Baseline visit will include saliva/buccal swab collection, and clinician and self-report assessments for subjects and caregivers. These assessments will include the Vineland Adaptive Behavior Scales-Third Edition (VABS-3), Clinical Global Impressions-Improvement scale (CGI-I), Visual Analog Scale-Treatment Satisfaction (VAS-TS), the Anxiety, Depression and Mood Scale (ADAMS), and the Systematic Assessment For Treatment Emergent Events (SAFTEE). Digital assessments may also be performed at the baseline visit or at home at the discretion of the qualified investigator. Digital assessments will include the NIH Toolbox Cognitive Battery Modified for Intellectual Disabilities (NIH-TCB), the Trail Making Test (TMT), and the Multifaceted Empathy Test (MET).
The study drug will be dispensed in blister packs to monitor adherence and improve subject compliance. Blister packs will be prepared and distributed at each subsequent visit. Subjects will return to the clinic for study visits on day 8, 15, 22, and 28 (study end date). Subjects and caregivers will complete the assessments described above. Subjects will provide additional saliva/buccal swab samples at day 15 and day 28.
- Conditions
- Fragile X Syndrome, Behavior, Cognitive Dysfunction
- Being tested
- Psilocybin, 1.5 mg
- Keywords
- Fragile X Syndrome, Behavior, Cognitive Symptoms, Psilocybin, Microdose
- Medical subject headings
- Fragile X Syndrome, Behavior, Cognitive Dysfunction, Neurobehavioral Manifestations
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Psilocybin, 1.5 mgDrug
Blister packs will contain five capsules of the study drug, Psilocybin 1.5mg. Subjects will be given blister packs with weekly doses at each visit including baseline (day 1), day 8, day 15, day 21, and day 28. Weekly blister packs will contain five doses (1-2 extra capsules depending on the week) to accommodate for flexibility in scheduling. Unused and open packages will be returned to study staff at each visit after baseline.
| Study group | Role | What they receive |
|---|---|---|
| Psilocybin, 1.5 mg | Experimental | Participants will take one capsule containing 1.5 mg psilocybin with a glass of water every other day for a period of 28 days. Dosing schedule will be same for all participants with the drug taken at days 1, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, and no treatment taken on alternating days. No subject shall be provided with more than five capsules (7.5mg psilocybin) at any one time to prevent diversion to the illicit market. If a dose is missed, participants are instructed to skip that dose and continue with their regularly scheduled medications. Participants are not to take >1 capsule per day. Receives: Drug: Psilocybin, 1.5 mg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Retention rate associated with 10 participants progressing to study completion.
Study completion is defined as completing the 28-day treatment period and the final in-clinic study visit.
Measured: baseline to day 28
Compliance with study dosing regimen as measured by the Visual Analogue Scale for Dosing (VAS-D).
Participants will be monitored for compliance using electronic compliance checks via the remote patient reported outcomes system through recording consumption in the treatment diary. Participants and caregivers will receive daily notifications via their preferred format (i.e., email, text) during agreed upon dosage windows. Notification will include a reminder to complete a post-dosage Visual Analogue Scale for Dosing (VAS-D) to monitor for any side effects which can be submitted electronically. Failure to complete the VAS-D within the day of dosing will trigger a call from study personnel to the participant in order to do a compliance check. Study personnel will note any deviations and all points of contact with participants regarding compliance checks.
Measured: baseline to day 28
Compliance with study dosing regimen as measured by the quantity of unused investigational product returned to the study clinic.
Measured: baseline to day 28
Adherence with completion of diaries monitoring participant and caregiver reported outcomes.
Adherence with study requirements for patient reported and caregiver reported outcomes (i.e., diaries) will be monitored using a remote patient reported outcomes system. Adherence will be assessed by determining the number of completed patient/caregiver diaries divided by the number of diaries expected to be completed.
Measured: baseline to day 28
Adherence with completion of questionnaires monitoring participant and caregiver reported outcomes.
Adherence with study requirements for patient reported and caregiver reported outcomes (i.e., questionnaires) will be monitored using a remote patient reported outcomes system. Adherence will be assessed by determining the number of completed patient/caregiver questionnaires divided by the number of questionnaires expected to be completed.
Measured: baseline to day 28
The number of late entries for diaries monitoring patient reported and caregiver reported outcomes.
Measured: baseline to day 28
The number of late entries for questionnaires monitoring patient reported and caregiver reported outcomes.
Measured: baseline to day 28
Satisfaction with study questionnaire.
Measured: baseline to day 28
Secondary outcomes (19)
Change from baseline in standardized measures of behavioral rigidity using the ratings of repetitive and stereotyped behaviors from the revised Repetitive Behavior Scale (RBS-R).
Measured: baseline, day 15, day 28
Change in standardized measures of empathy using the communication domain of the Vineland Adaptive Behavior Scales- Third Edition (VABS-3).
Measured: baseline, day 15, day 28
Change in standardized measures of empathy using the overall score on the Multifaceted Empathy Test (MET).
Measured: baseline, day 15, day 28
Change in standardized measures of expressive and receptive language skills using the communication domain of the Vineland Adaptive Behavior Scales- Third Edition (VABS-3).
Measured: baseline, day 15, day 28
Change in standardized measures of expressive and receptive language skills using the overall score on the Multifaceted Empathy Test (MET).
Measured: baseline, day 15, day 28
Changes in standardized measures of behavioral symptoms as measured by the Aberrant Behavior Checklist (ABC) with particular focus on ratings of repetitive/ritualistic behaviors from baseline to end of study.
Measured: baseline, day 8, day 15, day 21, day 28
Changes in standardized measures of behavioral symptoms as measured by the Aberrant Behavior Checklist (ABC) with particular focus on ratings of aggressive behaviors from baseline to end of study.
Measured: baseline, day 8, day 15, day 21, day 28
Changes in standardized measures of behavioral symptoms as measured by the revised Repetitive Behavior Scale (RBS-R) with particular focus on ratings of repetitive/ritualistic behaviors from baseline to end of study.
Measured: baseline, day 8, day 15, day 21, day 28
Changes in standardized measures of behavioral symptoms as measured by the revised Repetitive Behavior Scale (RBS-R) with particular focus on ratings of aggressive behaviors from baseline to end of study.
Measured: baseline, day 8, day 15, day 21, day 28
Changes in standardized measures of behavioral symptoms as measured by the Clinical Global Impressions (CGI) scale with particular focus on ratings of repetitive/ritualistic behaviors from baseline to end of study.
Measured: baseline, day 8, day 15, day 21, day 28
Changes in standardized measures of behavioral symptoms as measured by the Clinical Global Impressions (CGI) scale with particular focus on ratings of aggressive behaviors from baseline to end of study.
Measured: baseline, day 8, day 15, day 21, day 28
Changes in standardized measures of behavioral symptoms with particular focus on ratings of aggressive behaviors from baseline to end of study.
Changes in behavioral symptoms will be measured via the Aberrant Behavior Checklist (ABC), revised Repetitive Behavior Scale (RBS-R), and Clinical Global Impressions (CGI) scale.
Measured: baseline, day 8, day 15, day 21, day 28
Changes in overall cognition scores as measured by the NIH Toolbox Cognitive Battery Modified for Intellectual Disabilities (NIH-TCB).
Measured: baseline, day 28
Changes in overall cognition scores as measured by the Trail Making Test (TMT)-digital version.
Measured: baseline, day 28
Changes in overall cognition scores as measured by the Behavior Rating Inventory of Executive Function-Adult Version (BRIEF-A).
Measured: baseline, day 28
Changes in standardized measures of cognitive flexibility as measured by the NIH Toolbox Cognitive Battery Modified for Intellectual Disabilities (NIH-TCB) from baseline to end of study.
Measured: baseline, day 28
Changes in standardized measures of cognitive flexibility as measured by the Trail Making Test (TMT)-digital version from baseline to end of study.
Measured: baseline, day 28
Changes in standardized measures of cognitive flexibility as measured by the Behavior Rating Inventory of Executive Function-Adult Version (BRIEF-A) from baseline to end of study.
Measured: baseline, day 28
Changes in standardized measures of anxiety and depression with corresponding changes in ratings of overall wellbeing.
Changes in anxiety, depression, and overall wellbeing will be measured via the Anxiety, Depression and Mood Scale (ADAMS).
Measured: baseline, day 8, day 15, day 21, day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2023
- Study start28 Mar 2023
- Primary completionMay 2024 (estimated)
- Study completionMay 2024 (estimated)
Why it stopped: Indefinite hold due to protocol revisions relating to the exclusion criteria for concurrent medications.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fragile X SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Nova Mentis Life Science Corp
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- KGK Science Inc.
- Responsible party
- Sponsor
- Investigators
- David Crowley, MD, Principal investigator, KGK Science Inc.Marvin Hausman, MD, Study chair, Nova Mentis Life Science Corp
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05832255
- Sponsor's protocol ID
- 22NMCFN01
- Status last verified
- May 2024
- First submitted
- 8 Mar 2023
- Last updated
- 8 May 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Investigation of the Effects of Sub-Perceptual Repeat Dosing of Psilocybin on the Behavioural and Cognitive Symptoms of Fragile X Syndrome in Adult Patients
- ClinicalTrials.gov ID
- NCT05832255
- Acronym / sponsor ID
- 22NMCFN01
- Conditions
- Fragile X Syndrome, Behavior, Cognitive Dysfunction
- Keywords
- Fragile X Syndrome, Behavior, Cognitive Symptoms, Psilocybin, Microdose
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- SuspendedReason stopped: Indefinite hold due to protocol revisions relating to the exclusion criteria for concurrent medications.
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Nova Mentis Life Science CorpIndustry (pharma, biotech, devices)
- Collaborators
- KGK Science Inc.
- Study start
- 28 Mar 2023 (actual)
- Primary completion
- May 2024 (estimated)
- Study completion
- May 2024 (estimated)
- First posted
- 27 Apr 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Diverse symptomatology makes Fragile X Syndrome (FXS) difficult to treat, and currently there are no approved prevention or treatment methods for FXS. Current therapies, including pharmaceutical and behavioural interventions, offer a patchwork of solutions that have limited efficacy and high toxicity. The current study aims to examine psilocybin as a safe treatment alternative with the ability to improve markers of cognition, communication, mood, behavior as well as markers of neuroinflammation, serotonin levels in exosomes, and neuroplasticity at sub-hallucinogenic doses (microdosing).
The overall objective of this study is to assess the feasibility of low-dose psilocybin as a therapeutic option for individuals living with FXS and to improve diagnostic parameters of FXS, as well as therapeutic responses with the use of biomarkers.
Detailed description
A total of 10 subjects who meet all the inclusion criteria and does not meet any of the exclusion criteria will be enrolled into the study. Any subjects prematurely terminated from the study will be replaced to ensure 10 subjects complete the study. A study coordinator will contact referring clinicians, caregivers, and subjects to pre-screen for initial eligibility. Those deemed eligible will be invited for an in-person screening along with the participating caregiver. The screening visit will be approximately two hours long and will consist of informed consent, diagnostic interview, physical examination, drugs of abuse test (DOA), ECG, medical/treatment history review, and demographic forms. A pregnancy test will be performed on females of child-bearing during screening, baseline, and end-of-study visits. All eligible subjects will enter the treatment arm of the study.
Subjects and caregivers will return to the clinic for a baseline visit within three weeks of their screening completion. Baseline visit will include saliva/buccal swab collection, and clinician and self-report assessments for subjects and caregivers. These assessments will include the Vineland Adaptive Behavior Scales-Third Edition (VABS-3), Clinical Global Impressions-Improvement scale (CGI-I), Visual Analog Scale-Treatment Satisfaction (VAS-TS), the Anxiety, Depression and Mood Scale (ADAMS), and the Systematic Assessment For Treatment Emergent Events (SAFTEE). Digital assessments may also be performed at the baseline visit or at home at the discretion of the qualified investigator. Digital assessments will include the NIH Toolbox Cognitive Battery Modified for Intellectual Disabilities (NIH-TCB), the Trail Making Test (TMT), and the Multifaceted Empathy Test (MET).
The study drug will be dispensed in blister packs to monitor adherence and improve subject compliance. Blister packs will be prepared and distributed at each subsequent visit. Subjects will return to the clinic for study visits on day 8, 15, 22, and 28 (study end date). Subjects and caregivers will complete the assessments described above. Subjects will provide additional saliva/buccal swab samples at day 15 and day 28.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Psilocybin, 1.5 mg | Experimental | Drug: Psilocybin, 1.5 mgParticipants will take one capsule containing 1.5 mg psilocybin with a glass of water every other day for a period of 28 days. Dosing schedule will be same for all participants with the drug taken at days 1, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, and no treatment taken on alternating days. No subject shall be provided with more than five capsules (7.5mg psilocybin) at any one time to prevent diversion to the illicit market. If a dose is missed, participants are instructed to skip that dose and continue with their regularly scheduled medications. Participants are not to take >1 capsule per day. |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (18 listed)
- 18 to 50 years of age
- BMI > 18.3
- Diagnosis of Fragile X syndrome with a molecular genetic confirmation of the full FMR1 mutation (>200 CGG repeats) or the other loss of function mutations of the FMR1 gene (SNVs and deletions of the gene) based on evidence provided by caregiver from prior assessment
- IQ between 40 and 85 points as reported by caregiver based on prior assessment
- Subject has the ability to understand and provides voluntary, written, informed consent to participate in the study
Key exclusion criteria (19 listed)
- Individuals who are pregnant, breast feeding, or planning to become pregnant during the study
- Allergy, sensitivity, or intolerance to the investigational product's active or inactive ingredients
- 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
- 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
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Psilocybin, 1.5 mgBlister packs will contain five capsules of the study drug, Psilocybin 1.5mg. Subjects will be given blister packs with weekly doses at each visit including baseline (day 1), day 8, day 15, day 21, and day 28. Weekly blister packs will contain five doses (1-2 extra capsules depending on the week) to accommodate for flexibility in scheduling. Unused and open packages will be returned to study staff at each visit after baseline. | Drug | Psilocybin, 1.5 mg |
Primary outcome
Retention rate associated with 10 participants progressing to study completion.
Time frame: baseline to day 28
Study completion is defined as completing the 28-day treatment period and the final in-clinic study visit.
Compliance with study dosing regimen as measured by the Visual Analogue Scale for Dosing (VAS-D).
Time frame: baseline to day 28
Participants will be monitored for compliance using electronic compliance checks via the remote patient reported outcomes system through recording consumption in the treatment diary. Participants and caregivers will receive daily notifications via their preferred format (i.e., email, text) during agreed upon dosage windows. Notification will include a reminder to complete a post-dosage Visual Analogue Scale for Dosing (VAS-D) to monitor for any side effects which can be submitted electronically. Failure to complete the VAS-D within the day of dosing will trigger a call from study personnel to the participant in order to do a compliance check. Study personnel will note any deviations and all points of contact with participants regarding compliance checks.
Compliance with study dosing regimen as measured by the quantity of unused investigational product returned to the study clinic.
Time frame: baseline to day 28
Adherence with completion of diaries monitoring participant and caregiver reported outcomes.
Time frame: baseline to day 28
Adherence with study requirements for patient reported and caregiver reported outcomes (i.e., diaries) will be monitored using a remote patient reported outcomes system. Adherence will be assessed by determining the number of completed patient/caregiver diaries divided by the number of diaries expected to be completed.
Adherence with completion of questionnaires monitoring participant and caregiver reported outcomes.
Time frame: baseline to day 28
Adherence with study requirements for patient reported and caregiver reported outcomes (i.e., questionnaires) will be monitored using a remote patient reported outcomes system. Adherence will be assessed by determining the number of completed patient/caregiver questionnaires divided by the number of questionnaires expected to be completed.
The number of late entries for diaries monitoring patient reported and caregiver reported outcomes.
Time frame: baseline to day 28
The number of late entries for questionnaires monitoring patient reported and caregiver reported outcomes.
Time frame: baseline to day 28
Satisfaction with study questionnaire.
Time frame: baseline to day 28
19 secondary outcomes
Change from baseline in standardized measures of behavioral rigidity using the ratings of repetitive and stereotyped behaviors from the revised Repetitive Behavior Scale (RBS-R).
Time frame: baseline, day 15, day 28
Change in standardized measures of empathy using the communication domain of the Vineland Adaptive Behavior Scales- Third Edition (VABS-3).
Time frame: baseline, day 15, day 28
Change in standardized measures of empathy using the overall score on the Multifaceted Empathy Test (MET).
Time frame: baseline, day 15, day 28
Change in standardized measures of expressive and receptive language skills using the communication domain of the Vineland Adaptive Behavior Scales- Third Edition (VABS-3).
Time frame: baseline, day 15, day 28
Change in standardized measures of expressive and receptive language skills using the overall score on the Multifaceted Empathy Test (MET).
Time frame: baseline, day 15, day 28
Changes in standardized measures of behavioral symptoms as measured by the Aberrant Behavior Checklist (ABC) with particular focus on ratings of repetitive/ritualistic behaviors from baseline to end of study.
Time frame: baseline, day 8, day 15, day 21, day 28
Changes in standardized measures of behavioral symptoms as measured by the Aberrant Behavior Checklist (ABC) with particular focus on ratings of aggressive behaviors from baseline to end of study.
Time frame: baseline, day 8, day 15, day 21, day 28
Changes in standardized measures of behavioral symptoms as measured by the revised Repetitive Behavior Scale (RBS-R) with particular focus on ratings of repetitive/ritualistic behaviors from baseline to end of study.
Time frame: baseline, day 8, day 15, day 21, day 28
Changes in standardized measures of behavioral symptoms as measured by the revised Repetitive Behavior Scale (RBS-R) with particular focus on ratings of aggressive behaviors from baseline to end of study.
Time frame: baseline, day 8, day 15, day 21, day 28
Changes in standardized measures of behavioral symptoms as measured by the Clinical Global Impressions (CGI) scale with particular focus on ratings of repetitive/ritualistic behaviors from baseline to end of study.
Time frame: baseline, day 8, day 15, day 21, day 28
Changes in standardized measures of behavioral symptoms as measured by the Clinical Global Impressions (CGI) scale with particular focus on ratings of aggressive behaviors from baseline to end of study.
Time frame: baseline, day 8, day 15, day 21, day 28
Changes in standardized measures of behavioral symptoms with particular focus on ratings of aggressive behaviors from baseline to end of study.
Time frame: baseline, day 8, day 15, day 21, day 28
Changes in overall cognition scores as measured by the NIH Toolbox Cognitive Battery Modified for Intellectual Disabilities (NIH-TCB).
Time frame: baseline, day 28
Changes in overall cognition scores as measured by the Trail Making Test (TMT)-digital version.
Time frame: baseline, day 28
Changes in overall cognition scores as measured by the Behavior Rating Inventory of Executive Function-Adult Version (BRIEF-A).
Time frame: baseline, day 28
Changes in standardized measures of cognitive flexibility as measured by the NIH Toolbox Cognitive Battery Modified for Intellectual Disabilities (NIH-TCB) from baseline to end of study.
Time frame: baseline, day 28
Changes in standardized measures of cognitive flexibility as measured by the Trail Making Test (TMT)-digital version from baseline to end of study.
Time frame: baseline, day 28
Changes in standardized measures of cognitive flexibility as measured by the Behavior Rating Inventory of Executive Function-Adult Version (BRIEF-A) from baseline to end of study.
Time frame: baseline, day 28
Changes in standardized measures of anxiety and depression with corresponding changes in ratings of overall wellbeing.
Time frame: baseline, day 8, day 15, day 21, day 28
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 May 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
David Crowley, MD principal investigator
KGK Science Inc.
Marvin Hausman, MD study chair
Nova Mentis Life Science Corp
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Psilocybin, 1.5 mg in people with Fragile X Syndrome and Behavior, run by Nova Mentis Life Science Corp at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: paused, as of the sponsor's last update on 8 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Retention rate associated with 10 participants progressing to study completion. (time frame: baseline to day 28). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 May 2024.