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.

Travel to and from sites for a study participant and his caregiver may be covered by Shionogi and may include transportation and lodging arrangements, as well as reimbursement for expenses, such as meals (limitations may apply).
Zatolmilast is an investigational drug. Safety and efficacy have not been established. There is no guarantee that zatolmilast will be approved by any health authority.

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.

* Safety visits may be completed remotely using a designated visiting nurse service, followed by a call from a clinic.

Participating Site Locations

Don’t see a site near you? Travel support to and from sites for study participant and his caregiver may be covered by Shionogi and may include travel and lodging arrangements, as well as reimbursement for expenses, such as meals (limitations may apply). Contact medinfo@shionogi.com or 1-800-849-9707 if you have any questions.

Rush University Medical Center

Fragile X Clinic and Research Program
1725 W Harrison Street
Suite 718
Chicago, IL 60612

Recruitment Contact: Loren Escot

312-942-2164

Cincinnati Children's Hospital Medical Center

3333 Burnett Avenue
MLC 7004
Cincinnati, OH 45229

Recruitment Contact: Elizabeth Blank

513-803-8635

UC Davis Health System

MIND Institute
2825 50th Street
Sacramento, CA 95817

Recruitment Contact: Abigail Higareda Borbe

916-703-0281

Children's Hospital Colorado

Developmental Pediatrics
13123 East 16th Avenue
B140
Aurora, CO 80045

Recruitment Contact: Erika Peterson

720-777-8590

Kennedy Krieger Institute

1741 Ashland Avenue
Room 241
Baltimore, MD 21205

Recruitment Contact: Maria Halaguena

443-923-3826

Icahn School of Medicine at Mount Sinai

1 Gustave L. Levy Place
New York, NY 10029

Recruitment Contact: Venus Fan

929-989-7016

University of Massachusetts Chan Medical School

55 Lake Avenue
North 7th Floor
Worcester, MA 01655

Recruitment Contact: Lindsey Cincotta

508-856-7892

Suburban Research Associates

107 Chesley Drive
Unit 4
Media, PA 19063

Recruitment Contact: Scott Comegys

610-891-7200

Greenwood Genetic Center

14 Edgewood Drive
Greenville, SC 29605

Recruitment Contact: Nicole Johnston

864-672-6912

Emory University School of Medicine

101 Woodruff Circle
Suite 7130
Atlanta, GA 30322

Recruitment Contact: Jean Luan McColl

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

801-213-3954

Thompson Autism & Neurodevelopment Center - CHOC

170 S. Main Street
Orange, CA 92868

Recruitment Contact: Ulises Bustos

714-288-7678

University of Kansas Medical Center

3901 Rainbow Boulevard
Kansas City, KS 66160

Recruitment Contact: Danielle Adam

913-588-0494

Clinic for Special Children

20 Community Lane
Gordonville, PA 17529

Recruitment Contact: Joelle Williamson

717-687-9407

Amnova Clinical Research

4902 Irvine Center Drive
Suite 104
Irvine, CA 92604

Recruitment Contact: Maggie Wang

626-319-5966

Boston Children’s Hospital

300 Longwood Avenue
Boston, MA 02115

Recruitment Contact: Michelle Coughlin

617-355-7025

  • Participants in EXPERIENCE-204 will be eligible to enroll in EXPERIENCE-302, an up to two-year, open-label extension, after completing Week 13.

    EXPERIENCE-302
  • Additional study details can be found on ClinicalTrials.gov.

    ClinicalTrials.gov