Ongoing Clinical Trials for Fragile X

Welcome to the NFXF database for all current clinical trials for Fragile X-associated Disorders. In an effort to help move forward the development of new treatments, the NFXF has asked all pharmaceutical companies conducting Fragile X research to provide us with listings of their current studies.

The NFXF cannot vouch for the currency or accuracy of the information, as trials are regularly started, concluded or closed to new participants. However we will stay in close touch with the companies and update the information on a regular basis.

This announcement has been posted as a courtesy by the National Fragile X Foundation (NFXF) on behalf of the Clinical Trial Sponsors. The announcement and the parties conducting and sponsoring the research meet guidelines established by the NFXF regarding relevancy and appropriateness to Fragile X. The NFXF takes no position and makes no claims as to the potential benefits of the study including those studies involving medications, and cannot be responsible for any outcomes, lack of outcomes or unintended side effects. Your name or other personal information have not been shared with the researchers/company/institution. If interested in participating in this research, it is your responsibility to use the contact information in this announcement. Additional opportunities for being part of research studies can be found at research.fragilex.org.

Clinical Trials 101 Podcast

In early May 2012, we published a podcast featuring Robert Miller of the National Fragile X Foundation and representatives of the the major pharmaceutical companies involved in ongoing Fragile X Clinical Trials. We have Dr. Paul Wang, Vice President, Clinical Development of Seaside Therapeutics; Dr. Eriene Wasef, Associate Clinical Director of Hoffman-La Roche; and Dr. Marla Hochfeld, Executive Medical Director of Novartis. Listeners will hear insightful answers to the following questions:

  1. What is a clinical trial?
  2. Why are clinical trials important?
  3. How do clinical trials contribute to the safety of new drugs?
  4. How do clinical trials contribute to the usefulness of new drugs?
  5. What should I expect to be told about a trial and what should I not expect to be able to know?
  6. Can I drop out of a trial at any time?
  7. What about other medications my child might be taking?
  8. Can my child stay on the trial drug when a trial ends and, if not, why not?
  9. When a trial ends, should I expect to be contacted about the results or will I need to find that out on my own?
  10. What is the best way for listeners to learn about your company’s specific trial or trials?

Listen Now

Play

Check out our state-by-state listing of clinical trials being conducted of potential new drugs for the treatment of fragile X syndrome and see if one might be right for you.

Ongoing Clinical Trials

If you are having trouble funding your trip to this clinic, ask the clinic coordinator about financial assistance through the Justin Silver Fly With Me Fund.


Arizona

Southwest Autism Research & Resource Center

Phoenix, Arizona
Contact: (602) 218-8195 sputhalath@autismcenter.org
Age Range: 18 – 45
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome



California

Miller Children’s Hospital

Long Beach, California
Contact: (562) 728-5034
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome



California

PCSD Feighner Research

San Diego, California
Contact: (619) 692-1003
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome



California

Stanford University

Standford, California
Contact: (650) 704-9736 maik@stanford.edu
Age Range: 12 – 29
Gender: Male or Female
Condition: FXS
Study: Randomized Controlled Study of Donepezil in Fragile X Syndrome



California

Stramski Children’s Development Center

Long Beach, California
Contact: (562) 933-5607 nodonnell@memorialcare.org
Age Range: 16 – 50
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of the Efficacy and Safety of RO4917523 in Patients with Fragile X Syndrome



California

The MIND Institute

Sacramento, California
Contact: (916) 703-0247
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome



California

The MIND Institute

Sacramento, California
Contact: (916) 703-0472
Age Range: 18 – 45
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome



California

The MIND Institute

Sacramento, California
Contact: (602) 218-8170
Age Range: 12 – 17
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome



California

The MIND Institute

Sacramento, California
Contact: (916) 703-0281 alyssa.hamlin@ucdmc.ucdavis.edu
Age Range: 16 – 50
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of the Efficacy and Safety of RO4917523 in Patients with Fragile X Syndrome



Colorado

Children’s Hospital Colorado

Aurora, Colorado
Contact: (720) 777-8486
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome



Georgia

Emory University

Decatur, Georgia
Contact: (404) 778-8484
Age Range: 18 – 45
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome



Georgia

Emory University

Decatur, Georgia
Contact: (404) 778-8590
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome



Georgia

Emory University

Decatur, Georgia
Contact: (404) 778-8484 heather.clark@emoryhealthcare.org
Age Range: 16 – 50
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of the Efficacy and Safety of RO4917523 in Patients with Fragile X Syndrome



Georgia

Emory University

Decatur, Georgia
Contact: (404) 778-8484
Age Range: 12 – 17
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome



Illinois

Rush University Medical Center

Chicago, Illinois
Contact: (312) 942-7250
Age Range: 12 – 17
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome



Illinois

Rush University Medical Center

Chicago, Illinois
Contact: (312) 942-7250
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome



Illinois

Rush University Medical Center

Chicago, Illinois
Contact: (312) 942-7250 crystal_hervey@rush.edu
Age Range: 16 – 50
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of the Efficacy and Safety of RO4917523 in Patients with Fragile X Syndrome



Illinois

Rush University Medical Center

Chicago, Illinois
Contact: (312) 942-7250
Age Range: 18 – 45
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome



Indiana

Indiana University School of Medicine

Indianapolis, Indiana
Contact: (317) 274-8162
Age Range: 18 – 45
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome



Iowa

University of Iowa; Office of Dr. Bruce Luxon

Iowa City, Iowa
Contact: (319) 353-7162
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome



Kentucky

University of Louisville Research Foundation, Inc; Kosair Charities Pediatric Clinical Research Unit

Louisville, Kentucky
Contact: (502) 629-5820
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome



Maryland

Kennedy Krieger Institute

Baltimore, Maryland
Contact: (443) 923-7721 talisa@kennedykrieger.org
Age Range: 16 – 50
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of the Efficacy and Safety of RO4917523 in Patients with Fragile X Syndrome



Maryland

Kennedy Krieger Institute

Baltimore, Maryland
Contact: (443) 923-9315
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome



Massachusetts

Bay State Medical Center

Springfield, Massachusetts
Contact: (413) 794-1136 bruce.waslick@baystatehealth.org
Age Range: 12 – 17
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome



Massachusetts

Children’s Hospital Boston

Boston, Massachusetts
Contact: (857) 218-5328 ramzi.nasir@childrens.harvard.edu
Age Range: 12 – 17
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome



Massachusetts

University of Massachusetts

Worcester, Massachusetts
Contact: (508) 856-5073
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome



Nebraska

University of Nebraska

Omaha, Nebraska
Contact: (402) 552-6239
Age Range: 12 – 17
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome



Nebraska

University of Nebraska

Omaha, Nebraska
Contact: (402) 552-6005
Age Range: 18 – 45
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome



Nebraska

University of Nebraska Medical Center; Dept of Neurological Sciences

Omaha, Nebraska
Contact: (402) 559-4949
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome



New York

New York State Inst. for Basic Research in Dev. Disabilities

Staten Island, New York
Contact: (718) 494-8028 wtbibr@aol.com
Age Range: 12 – 17
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome



New York

New York State Institute for Basic Research in Developmental Disabilities

Staten Island, New York
Contact: (718) 494-8028
Age Range: 18 – 45
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome



New York

New York State Institute for Basic Research in Developmental Disabilities

Staten Island, New York
Contact: (718) 494-8028 porto_a@medscape.com
Age Range: 16 – 50
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of the Efficacy and Safety of RO4917523 in Patients with Fragile X Syndrome



New York

Seaver Autism Center at the Icahn School of Medicine at Mount Sinai

New York, New York
Contact: (212) 241-3692 stacey.lurie@mssm.edu
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome



New York

Seaver Autism Center at the Icahn School of Medicine at Mount Sinai

New York, New York
Contact: (212) 241-3692 stacey.lurie@mssm.edu
Age Range: 14 – 17
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of the Efficacy and Safety of RO4917523 in Patients with Fragile X Syndrome



North Carolina

Duke University Medical Center

Durham, North Carolina
Contact: (919) 668-0972 wan.liang@duke.edu
Age Range: 16 – 50
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of the Efficacy and Safety of RO4917523 in Patients with Fragile X Syndrome



Ohio

Cincinnati Childrens Hospital

Cincinnati, Ohio
Contact: (513) 636-8325
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome



Pennsylvania

Suburban Research / Elwyn Genetics

Media, Pennsylvania
Contact: (610) 891-9024
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome



Pennsylvania

Suburban Research / Elwyn Genetics

Media, Pennsylvania
Contact: (610) 891-9024 x103
Age Range: 18 – 45
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome



South Carolina

Greenwood Genetic Center

Greenwood, South Carolina
Contact: (864) 941-8100 sas@ggc.org
Age Range: 12 – 17
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome



South Carolina

Greenwood Genetic Center

Greenwood, South Carolina
Contact: (864) 941-8100
Age Range: 18 – 45
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome



South Carolina

Greenwood Genetic Center

Greenwood, South Carolina
Contact: (864) 941-4100
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome



Tennessee

Vanderbilt Kennedy Center

Nashville, Tennessee
Contact: (615) 936-3288 sarah.marler@vanderbilt.edu
Age Range: 16 – 50
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of the Efficacy and Safety of RO4917523 in Patients with Fragile X Syndrome



Tennessee

Vanderbilt University

Nashville, Tennessee
Contact: (615) 936-3288
Age Range: 18 – 45
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome



Tennessee

Vanderbilt University

Nashville, Tennessee
Contact: (615) 708-6368
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome



Tennessee

Vanderbilt University

Nashville, Tennessee
Contact: (615) 936-3288
Age Range: 12 – 17
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome



Texas

Red Oaks Psychiatry Associates, P.A.

Houston, Texas
Contact: (281) 893-4111
Age Range: 18 – 45
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome



Texas

Road Runner Research

San Antonio, Texas
Contact: (210) 949-0505
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome



Texas

Texas Children’s Hospital

Houston, Texas
Contact: (832) 822-3752
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome



Washington

Northwest Clinical Research Center

Bellevue, Washington
Contact: (425) 453-0404 akhan@nwcrc.net
Age Range: 12 – 17
Gender: Male or Female
Condition: FXS
Study: Phase 2b Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome



Washington

Seattle Children’s Research Institute

Seattle, Washington
Contact: (206) 884-1168 denise.ward@seattlechildrens.org
Age Range: 16 – 50
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of the Efficacy and Safety of RO4917523 in Patients with Fragile X Syndrome



Washington

Seattle Children’s Research Institute

Seattle, Washington
Contact: (206) 987-1837
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome



Washington, District of Columbia

Children’s National Med Center

, Washington, District of Columbia
Contact: (301) 765-5423
Age Range: 5 – 13
Gender: Male or Female
Condition: FXS
Study: Phase 2 Study of RO4917523 in Pediatric Patients With Fragile X Syndrome