MIND Institute Clinical Trials and Research Studies
Tetra 204: A Randomized, Double-Blind, Placebo-Controlled, Two-Part Study of BPN14770 in Male Adolescents (Aged 12 to <18 Years) with Fragile X Syndrome (BPN14770-CNS-204)(April 2023–May 2025)
BPN14770 (Zatolmilast) is an investigational drug being developed for the treatment of Fragile X syndrome, Alzheimer’s disease, and other cognitive disorders. This study is testing BPN14770 to learn how the study drug acts in the body, how well it works, and if it is safe, compared to a placebo study drug (a treatment with no active ingredients that looks like the study drug).
Eligible participants are males ages 12 to 18 who have been diagnosed with Fragile X syndrome.
Zynerba 17: An Open-Label Extension Study to Assess the Long-Term Safety and Tolerability of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Fragile X Syndrome(Active, closed for recruitment)
for more information about this study.
The purpose of this study is to investigate how safe and effective the study drug, ZYN002, a topical cannabidiol (CBD) gel, is in participants with Fragile X Syndrome (FXS), aged 3 through 17 years old, and how well it is tolerated. We expect the study to be up to 72 months after the screening visit. The visits are every 6 months, and they alternate from onsite to virtual. ZYN002 is an experimental treatment. This means that it is not approved by the Health Authorities including the United States Food and Drug Administration (FDA) and must be tested to see if it is an effective treatment.
Zynerba 33: A Randomized, Double-Blind, Placebo-Controlled Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Fragile X Syndrome – RECONNECT (Open until July 31, 2023)
for more information about this study.
The purpose of this study is to compare how effective and safe the study drug, ZYN002, a topical cannabidiol (CBD) gel, works to treat behavioral symptoms of Fragile X syndrome (FXS), compared to a placebo study drug (a treatment with no active ingredients that looks like the study drug).
This study is for participants who have been diagnosed, via genetic testing, with Fragile X syndrome and are between 3 and 18 years old.
Genotype-Phenotype Relationships in Fragile X Families (March 2017-February 2022)
This study aims to learn more about the fragile X premutation in older adults compared to those without. We will follow over 100 patients with FXTAS ages 55 to 75 in a prospective study to develop innovative clinical measures to identify areas of specific deficits and to identify biological markers for each stage of the. If you participate in this study, you can expect: 2-3 visits to the MIND Institute, which will include a medical history exam, neuropsychological evaluation, behavioral testing, neurological evaluation, balance and tremor assessments, MRI, blood draw, and skin biopsy
We are recruiting males and females with the premutation (ages 55-85 years) who are having symptoms of FXTAS.
Longitudinal Study of Brain and Cognition in Fragile X Premutation Carriers
(Summer 2018–2023)
This study examines changes in the brain and cognition associated with aging, in males with and without the fragile X premutation. The study consists of three 2-day visits over the course of five years to observe changes in the brain and cognition occurring over time. The protocol involves a medical history exam, neuropsychological evaluation, behavioral testing, MRI, and blood draw.
We are recruiting males (ages 45 years and over) with and without the premutation.