EXPERIENCE-204: Adolescent Study in Fragile X Syndrome

About EXPERIENCE-204
EXPERIENCE-204 (also known as BPN14770-CNS-204 or NCT05163808) is double-blind, which means that neither the participants nor the researchers know which treatment the participants are receiving. Participants will be randomly selected to receive either zatolmilast (the investigational study drug) or placebo (a pill that does not include active drug) for 13 weeks. Approximately two of three participants will receive zatolmilast and one of three participants will receive placebo.
Overview of Criteria
Participants must have a genetic diagnosis of Fragile X syndrome (FXS) and meet the following criteria:
Males aged 9 to 17 with a genetic diagnosis of FXS.
Weigh at least 55 pounds and have a body mass index (BMI) less than or equal to 97 percent of their age group.
Be able to participate in a 13-week study that includes three clinic visits, two home visits and two phone calls.
EXPERIENCE-204 Schedule
Before eligibility can be determined, a participant must attend a screening session (for approximately four–six hours) at a clinic. The screening session will take place approximately two–four weeks before the study begins (Day 1).
Day 1
In-person baseline assessment (approximately four–six hours).
Week 1
Brief phone call.
Week 4
Safety (approximately two hours).*
Week 8
In-person safety and efficacy (approximately four–six hours).
Week 10
Safety (approximately two hours).*
Week 13
In-person safety and efficacy (approximately four–six hours); determine eligibility for continuation to EXPERIENCE-302, the open-label extension.
Week 14
Follow-up phone call for participants not continuing to EXPERIENCE-302, the open-label extension.
Participating Site Locations
Rush University Medical Center
Fragile X Clinic and Research Program
1725 W Harrison Street
Suite 718
Chicago, IL 60612
Recruitment Contact: Loren Escot
loren_escot@rush.edu
312-942-2164
Cincinnati Children's Hospital Medical Center
3333 Burnett Avenue
MLC 7004
Cincinnati, OH 45229
Recruitment Contact: Elizabeth Blank
elizabeth.blank@cchmc.org
513-803-8635
UC Davis Health System
MIND Institute
2825 50th Street
Sacramento, CA 95817
Recruitment Contact: Abigail Higareda Borbe
amoradelhigareda@ucdavis.edu
916-703-0281
Children's Hospital Colorado
Developmental Pediatrics
13123 East 16th Avenue
B140
Aurora, CO 80045
Recruitment Contact: Erika Peterson
erika.peterson@childrenscolorado.org
720-777-8590
Kennedy Krieger Institute
1741 Ashland Avenue
Room 241
Baltimore, MD 21205
Recruitment Contact: Maria Halaguena
halaguena@kennedykrieger.org
443-923-3826
Icahn School of Medicine at Mount Sinai
1 Gustave L. Levy Place
New York, NY 10029
Recruitment Contact: Venus Fan
venus.fan@mssm.edu
929-989-7016
University of Massachusetts Chan Medical School
55 Lake Avenue
North 7th Floor
Worcester, MA 01655
Recruitment Contact: Lindsey Cincotta
lindsey.cincotta@umassmed.edu
508-856-7892
Suburban Research Associates
107 Chesley Drive
Unit 4
Media, PA 19063
Recruitment Contact: Scott Comegys
volunteer@atlas-clinical.com
610-891-7200
Greenwood Genetic Center
14 Edgewood Drive
Greenville, SC 29605
Recruitment Contact: Nicole Johnston
njohnston@ggc.org
864-672-6912
Emory University School of Medicine
101 Woodruff Circle
Suite 7130
Atlanta, GA 30322
Recruitment Contact: Jean Luan McColl
jean.luan@emory.edu
404-778-8619
University of Utah and Primary Children's Hospital
100 N. Mario Capecchi Drive
Salt Lake City, UT 84113
Recruitment Contact: Abbey Loveridge
abbey.loveridge@hsc.utah.edu
801-213-3954
Thompson Autism & Neurodevelopment Center - CHOC
170 S. Main Street
Orange, CA 92868
Recruitment Contact: Ulises Bustos
ulises.bustos@choc.org
714-288-7678
University of Kansas Medical Center
3901 Rainbow Boulevard
Kansas City, KS 66160
Recruitment Contact: Danielle Adam
dadam@kumc.edu
913-588-0494
Clinic for Special Children
20 Community Lane
Gordonville, PA 17529
Recruitment Contact: Joelle Williamson
jwilliamson@clinicforspecialchildren.org
717-687-9407
Amnova Clinical Research
4902 Irvine Center Drive
Suite 104
Irvine, CA 92604
Recruitment Contact: Maggie Wang
maggie@amnovaresearch.com
626-319-5966
Boston Children’s Hospital
300 Longwood Avenue
Boston, MA 02115
Recruitment Contact: Michelle Coughlin
michelle.coughlin@childrens.harvard.edu
617-355-7025