Clinical Trials seeking Participants

Novartis mGluR5 Antagonist AFQ056
Date Posted: 1/11/2012, Last Updated: 3/22/2012
Centers around the world are enrolling adults ages 18-45 and children ages 12-17 with Fragile X for a Phase IIb trial of an mGluR5 antagonist.

This randomized, double-blind, placebo-controlled clinical study sponsored by Novartis is designed to assess whether AFQ056 is safe and effective in treating the behavioral symptoms of Fragile X Syndrome. AFQ056 is an mGluR5 antagonist, which decreases mGluR5 activity in the brain. The trial involves 9 visits over 20 weeks.

Patients who complete this trial are offered the option of taking AFQ056 free of charge until the drug is available on the market.

These medical centers are recruiting now and more are coming. You can also see more info and sites at clinicaltrials.gov - here for ages 18-45 and here for ages 12-18. Here is background on mGluR5 written by FRAXA:

Staten Island, NY: NYS Institute for Basic Research
Currently recruiting for participants aged 18-45. Principal Investigator is Dr. Angelo Porto
Contact him at 718-494-8028 or porto_a@medscape.com

Greenwood, SC: Greenwood Genetics Center
Currently recruiting participants aged 18-45. Call study coordinator, Fran Annese, at 1-888-442-4363 or locally at 864-941-8160, or email her at fran@ggc.org

Indiannapolis, IN: Univ. of Indiana
Currently recruiting for participants aged 18-45 and soon adding ages 12-17 with Fragile X. Principal Investigator is Dr. Craig Erickson
Contact: Lauren Mathieu at 317-944-1218 or fragilex@iupui.edu .

Atlanta, Georgia: Emory University
Recruiting for participants aged 12-45 with Fragile X. Principal Investigator is Dr. Jeannie Visootsak
Contacts: Certified Genetic Counselors Heather Clark at 404-778-8484 or hmclark@emory.edu
or Krista Charen at (404) 778-8479 or kharkre@emory.edu
or Lisa Shubeck at (404) 778-8478 or lshubec@emory.edu.

Elwyn, Pennsylvania: Elwyn Fragile X Center, working with Suburban Research Associates (Media, PA)
Recruiting ages 18-45
Contact Mary Delany, Certified Genetic Counselor and Clinical Coordinator, Elwyn Fragile X Center
at 610-891-2349 or mary_delany@elwyn.org
or contact Dana Lewis, Clinical Research Coordinator
dlewis@suburbanresearch.com or 610.891.9024 ext. 103
Suburban Research Associates, Media, PA 19063

Nashville, Tennessee: Vanderbilt University Medical Center
Dr. Jeremy VeenstraVanderWeele, Principal Investigator
Recruiting ages 12-45 with Fragile X for the Phase II study and also recruiting ages 5-11 for a preliminary study.
Study coordinator Jessie Smith
can be reached at 615.936.3288 or Jessica.Smith@vanderbilt.edu

Chicago, IL: Rush Univ. Medical Center Fragile X Clinic and Research
Dr. Elizabeth Berry-Kravis, principal investigator
Recruiting ages 12-45 with Fragile X, and also recruiting ages 5-11 for a preliminary study.
Contact trial/research coordinator Crystal Hervey at 312-942-7250 or Crystal_Hervey@rush.edu

Sacramento, CA: UC Davis Medical Center
Recruiting children and adults, ages 12-45,with Fragile X.
Contact: Emma Hare at 916-703-0223

Omaha, NE: University of Nebraska Dept. of Psychiatry
Psychopharmacology Research Consortium
Recruiting men and women age 19-45 with Fragile X
Contact: Brigette Vaughn at 402-552-6239 or bvaughan@unmc.edu

Phoenix, AZ: Southwest Autism Research and Resource Center, at www.autismcenter.org
contact Sajitha Puthalath, M Pharm, CCRP, at (602) 218-8195 direct line, or 602 340 8717 main line, or SPuthalath@autismcenter.org. Download flyer

Outside the US these sites are also recruiting:

Italy: Roma
For more info (in Italian) visit http://www.xfragile.net/scheda.asp?idprod=381&idpadrerif=39
or please contact Prof. Maria Giulia Torrioli at mgtorrioli@rm.unicatt.it
or Prof. Giovanni Neri at gneri@rm.unicatt.it

Italy: Genoa
Please contact Dr Edvige Veneselli
Child Neuropsychiatry Department
Gaslini Institute
Largo G. Gaslini 5 Genoa, Italy
e-mail: edvigeveneselli@ospedale-gaslini.ge.i or neuropsichiatria@ospedale-gaslini.ge.it
+39-010-5636432

Additional contacts for this study are:
Dr. Michela Stagnaro, stagnaromichela@libero.it
Dr. Lucia Sciarretta, LuciaSciarretta@ospedale-gaslini.ge.it

Canada: Toronto
Fragile X Clinic at Surrey Place Centre
Potential participants must be Canadian residents with Fragile X syndrome between ages 18-45. For further information, please contact Dr. Carlo Paribello at fxrfc@on.aibn.com.

Edinburgh, Scotland
Recruiting children and adults, ages 12-45. Please contact Dr. Andrew Stanfield for more details.
Andrew.Stanfield@ed.ac.uk
Senior Clinical Research Fellow and Honorary Consultant Psychiatrist
Co-director of the Patrick Wild Centre for Research into Autism, Fragile X and Intellectual Disabilities
University of Edinburgh

Australia: Melbourne
Fragile X Alliance Clinic
For information visit www.fragilex.com.au/research/
or by phone at 03 9528 1910

Australia: New South Wales
Carolyn Rogers
Hunter Genetics
Work (Sec) +61 2 4985 3136
(Direct) +61 2 4985 3137 (Mon-Thurs)
The GOLD Service
P O Box 84
WARATAH, NSW 2298
carolyn.rogers@hnehealth.nsw.gov.au

Switzerland: Lausanne
Recruiting ages 12-45 with Fragile X syndrome. Please contact
Sebastien Jacquemont, MD
Service de génétique Médicale
CHUV, Lausanne
email: sebastien.jacquemont@chuv.ch
tel : (+41) 21 314 55 93
tel secr : (+41) 21 314 33 76
fax: (+41) 21 314 33 92

Germany: Berlin
Tubingen, Germany
 Flyer with details in German for the Germany sites is available here

Please email kclapp@fraxa.org if you would like details for any of these sites.

Switzerland: Zurich

Glostrup, Denmark, 2600

Bron Cedex, France, 69677

Paris, France, 75013

Roche mGluR5 Antagonist RO4917523
Date Posted: 2/21/2011, Last Updated: 3/21/2012

The randomized, double-blind, placebo-controlled clinical study sponsored by Roche Pharmaceuticals is testing an mGluR5 antagonist, which decreases mGluR5 activity in the brain and may improve learning and cognition. A phase I trial of this drug was conducted last year in adults without fragile X syndrome, and no serious adverse events were noted. The next trial will assess safety and tolerability of this medicine, as well as its effects on learning, mood, and behavior.

Recruiting for males and females ages 16-50 with Fragile X syndrome has started at the following sites. Please note that clinicaltrials.gov lists all the sites as "Not yet recruiting" ... this will be updated in time.

Atlanta, GA
Emory University School of Medicine
Contact: (404)778-8484 heather.clark@emoryhealthcare.org

Long Beach, CA
Stramski Children’s Development Center
Contact: (562) 933-5607 nodonnell@memorialcare.org

Chicago, IL
Rush University Medical Center
Contact: (312)942-7250 crystal_hervey@rush.edu

Baltimore, MD
Kennedy Krieger Institute
Contact: (443)923-7721 talisa@kennedykrieger.org

Staten Island, NY
New York State Institute for Basic Research
Contact: (718)494-8028 porto_a@medscape.com

New York, NY
Seaver Autism Center at Mount Sinai School of medicine
Contact: (212) 241-3692 lily.schwartz@mssm.edu

Durham, NC
Duke University Medical Center
Contact: (919) 668-0972 wan.liang@duke.edu

Nashville, TN
Vanderbilt Kennedy Center
Contact: (615) 936-3288 sarah.marler@vanderbilt.edu 

Seattle, WA
Seattle Children’s Research Institute
Contact: (206) 884-1168 denise.ward@seattlechildrens.org

Phase 3 Study of STX209 for Social Withdrawal in Fragile X Syndrome
Date Posted: 1/7/2012, Last Updated: 3/20/2012
Seaside Therapeutics is sponsoring a randomized, double-blind, placebo-controlled Phase 3 study to evaluate the effects of STX209 (arbaclofen) on social impairment in people ages 5 to 25 with fragile X syndrome.

These studies will enroll more than 120 subjects. Patients will be randomized to receive STX209 or placebo and then treated over an eight-week period. Subjects who complete the entire study may be eligible to enroll in a subsequent open-label study. The study is designed to measure the efficacy, safety and tolerability of STX209. The primary efficacy endpoint will evaluate social withdrawal. Details of the study can be obtained at clinicaltrials.gov and at www.seasidetherapeutics.com or by calling 1-877-713-9009, ext. 8.

Trial Centers recruiting males and females ages 5-50 with Fragile X syndrome:

Phoenix, AZ: Southwest Autism Research & Resource Center
Dr. Raun Melmed, Principal Investigator
Contact: Sajitha Puthalath at 602-218-8195 or SPuthalath@autismcenter.org

Long Beach, CA: Miller Children’s Hospital
Dr. Gary Feldman, Principal Investigator
Contact: Nan O’Donnell at 562-933-5607 or NODonnell@memorialcare.org

Sacramento, CA: MIND Institute, UC Davis
Dr. Randi Hagerman, Principal Investigator
Contact: Lindsey Partington at 916-703-0471 or Lindsey.Partington@ucdmc.ucdavis.edu

Denver, CO: Children’s Hospital Colorado
Dr. Nicole Tartaglia, Principal Investigator
Contact: Susan Howell at 720-777-8361 or Susan.howell@childrenscolorado.org

Miami, FL: University of Miami
Dr. Deborah Barbouth, Principal Investigator
Contact: Elsa Salazar at 305-243-6562 or ESalazar2@med.miami.edu

Orange City, FL: Lake Mary Pediatrics
Dr. Miles Landis, Principal Investigator
Contact: Heidi Andersen at 386-960-8282 or lmpresearch2@gmail.com

Decatur, GA: Emory University
Dr. Jeannie Visootsak, Principal Investigator
Contact: Heather Clark at 404-778-8484 or hmclark@emory.edu

Chicago, IL: Rush University
Dr. Elizabeth Berry-Kravis, Principal Investigator
Contact: Crystal Hervey at 312-942-7250 or Crystal_Hervey@rush.edu

Indianapolis, IN: University of Indiana
Dr. Craig Erickson, Principal Investigator
Contact: Vanessa Patrick at 317-948-4888 or vpatrick@iupui.edu

Kansas City, KS: Kansas University
Dr. Jessica Hellings, Principal Investigator
Contact: Anne Nugent at 913-588-5751 or anugent@kumc.edu

Worcester, MA: University of Massachusetts
Dr. Jean Frazier, Principal Investigator
Recruiting children and adults, ages 5-25 with Fragile X Syndrome.
Contact: Michael Hill at 508-856-2041 or michael.hill@umassmed.edu

Baltimore, MD: Kennedy Krieger Institute
Dr. Michael Johnston, Principal Investigator
Contact: Tanjala Gipson at 443-923-7613 or ResearchTrials@kennedykrieger.org

Columbia, MO: University of Missouri
Dr. David Beversdorf, Principal Investigator
Contact: Holly Rice at 573-884-3006 or riceh@missouri.edu

Durham, NC: Duke University
Dr. Ave Lachiewicz, Principal Investigator
Contact: Wan Lan Liang at 919-668-0972 or Wan.liang@duke.edu

Akron, OH: Akron Children’s Hospital
Dr. Carol Delahunty, Principal Investigator
Contact: Jen Michel at 330-543-4738 or jmichel@chmca.org

Media, PA: Suburban Research Associates
Dr. Shivkumar Hatti, Principal Investigator
Contact: Maureen O’Donnell at 610-891-9024 x 103 or modonnell@suburbanresearch.com

New York, NY: Mount Sinai School of Medicine, Seaver Autism Center
Dr. Alexander Kolevzon, Principal Investigator
Contact: Lily Schwartz at 212-241-3692 or lily.schwartz@exchange.mssm.edu

Staten Island, NY: NYS Institute of Basic Research
Dr. Ted Brown, Principal Investigator
Contact: Angelo Porto at 718-494-8028 or porto_a@medscape.com

Oklahoma City, OK: OU Physician's Child Study Center
Dr. Thomas Lock, Principal Investigator
Contact: Lora Tusing at 405 271-4401 or Lora-Tusing@ouhsc.edu

Nashville, TN: Vanderbilt University
Dr. Jeremy Veenstra-VanderWeele, Principal Investigator
Contact: Jessica Smith at 615-936-3288 or Jessica.smith@Vanderbilt.Edu

Houston, TX: Red Oak
Dr. Lawrence Ginsberg, Principal Investigator
Contact: Elissa Molloy at 281-893-7559 x 267 or Elissa.molloy@redoakpsychiatry.com

San Antonio, TX: Road Runner Research
Dr. Jerry Tomasovic, Principal Investigator
Contact: Kristin McGinley at 210-949-0505 or kmcginley@rrresearchsa.com

Seattle, WA: Seattle Children’s Research Institute
Dr. Bryan King, Principal Investigator
Contact: Denise Ward at 206-884-1168 or denise.ward@SeattleChildrens.org

General Information About Participating in Clinical Trials
Date Posted: 1/11/2012, Last Updated: 1/11/2012
Thank you for being interested in participation in a clinical trial for Fragile X! FRAXA has been working since 1994 to discover targeted medications and get them to market. We are getting so close! But this is a challenging new phase for all of us. This part now involves our children and getting them enrolled in clinical trials. As exciting as it is, we know many of you have questions about what it really means to be in a clinical trial and how to even get started.

Look here for Freqently Asked Questions about Trials

Please read below for Fragile X trials that are accepting participants.

We are looking forward to this voyage with all of you on board – this is the great result of all of your support throughout the years and we all remain hopeful that these trials will be successful for the sake of all affected by Fragile X.
Brain Imaging Study at NIH, Bethesda, MD
Date Posted: 1/24/2009, Last Updated: 4/29/2011

Protein Synthesis in the Brain of Patients With Fragile X Syndrome

The National Institutes of Health is seeking men (ages 18-24 years who are not taking psychotropic medications) with fragile X syndrome for a research study at the NIH Clinical Center in Bethesda, Maryland. This study involves brain imaging in subjects with fragile X syndrome. For more information visit clinicaltrials.gov

Fragile X Research Registry - The Waisman Center, Madison, WI, and the Carolina Institute for Developmental Disabilities, Chapel Hill, NC
Date Posted: 1/1/2011, Last Updated: 3/30/2011
The registry is a database of people who want to be notified about fragile X research studies. Children and adults with fragile X-associated disorders may join. The Registry is for families and individuals with Fragile X syndrome, the Fragile X premutation, Fragile X-associated primary ovarian insufficiency (FXPOI), and/or Fragile X-associated tremor/ ataxia syndrome (FXTAS). Learn more at www.fragileXregistry.org
Multi-center Early Development Study - Chapel Hill, NC
Date Posted: 12/9/2010, Last Updated: 12/9/2010
This multi-center study involves the collaboration of two sites which are part of an NIH-funded network (University of North Carolina at Chapel Hill, and Washington University in St. Louis, MO). At UNC, the lead investigator is Heather Cody Hazlett, PhD. Currently, we are searching for 30 participants in the US who meet the following criteria: * Are between 0-6 months of age (parents who are expecting may also be eligible) * Have a diagnosis of fragile X Once a family is enrolled in this study, they will travel to Chapel Hill, NC for a comprehensive series of developmental, non-invasive assessments completed on the infant at the 6, 12 and 24 month time periods. Also, during this trip the infant will receive an MRI scan while they sleep. Between trips to Chapel Hill there will be phone conversations, genetic and environmental data collection. There is no cost for family participation. All travel and lodging costs are reimbursed by the study, and any services the study provides are at no charge to the family. Families will receive feedback on the assessments and each MRI scan. The family will also receive $150 for each completed visit. Please contact Leslie Braddy at 1.800.793.5715 or lbraddy@med.unc.edu for more information. View/print flyer here

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