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Psychedelic

Psilocybin for Fragile X Syndrome

Phase 2

Waitlist Available

Led By David Crowley, MD

Research Sponsored by Nova Mentis Life Science Corp

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

18 to 50 years of age

Must not have

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

Type I or insulin-dependent Type II diabetes

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, day 8, day 15, day 21, day 28

Awards & highlights

No Placebo-Only Group

Summary

This trial seeks to explore if psilocybin can safely treat Fragile X Syndrome, which currently has no approved treatments. It hopes to improve diagnosis & therapeutic responses w/ biomarkers.

Who is the study for?

Adults aged 18-50 with Fragile X syndrome confirmed by genetic testing, able to swallow pills, and not planning pregnancy or using reliable birth control can join. They must have a BMI over 18.3 and an IQ between 40-85. Exclusions include uncontrolled high blood pressure, severe liver or heart conditions, certain mental health disorders, drug abuse, recent major surgery, or unstable medical conditions.

What is being tested?

The trial is exploring the effects of low-dose psilocybin (1.5 mg) on behavior and cognitive symptoms in adults with Fragile X Syndrome. It aims to see if this treatment improves cognition, mood, behavior as well as biological markers related to brain function without causing hallucinations.

What are the potential side effects?

While the study uses sub-hallucinogenic doses of psilocybin aiming for minimal side effects, potential risks may include mild gastrointestinal upset like nausea or diarrhea; short-term changes in mood or perception; headache; fatigue; and possible allergic reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with Fragile X syndrome confirmed by genetic testing.

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I am between 18 and 50 years old.

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I cannot have children due to surgery or being post-menopausal for over a year.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a heart condition such as heart disease, heart attack, or abnormal heart rhythm.

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I have Type I diabetes or insulin-dependent Type II diabetes.

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I have been diagnosed with a schizophrenia spectrum disorder, major depression with psychosis, or bipolar disorder.

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I have a history of serious liver problems.

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My blood pressure is often higher than 140/90 mmHg.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, day 8, day 15, day 21, day 28

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, day 8, day 15, day 21, day 28

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, day 8, day 15, day 21, day 28 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Adherence with completion of diaries monitoring participant and caregiver reported outcomes.

Adherence with completion of questionnaires monitoring participant and caregiver reported outcomes.

Compliance with study dosing regimen as measured by the Visual Analogue Scale for Dosing (VAS-D).

+1 more

Secondary study objectives

Changes in standardized measures of anxiety and depression with corresponding changes in ratings of overall wellbeing.

Changes in standardized measures of behavioral symptoms with particular focus on ratings of aggressive behaviors from baseline to end of study.

Other study objectives

To characterize the role of psilocybin as a modulator of neuroinflammation through analysis of mRNA neuroinflammatory biomarkers using buccal swabs from baseline to study end.

To characterize the role of psilocybin as a modulator of neuroinflammation through analysis of mRNA neuroinflammatory biomarkers using saliva samples from baseline to study end.

To improve diagnostic phenotyping of FXS through analysis of biomarkers using buccal swab and saliva samples.

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Psilocybin, 1.5 mgExperimental Treatment1 Intervention

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.

0 / 8Eligibility criteria met