New Details of the Pediatric Phase 2 Clinical Trial Study of Trofinetide

December 8, 2015

Revised March 15, 2016

Neuren Pharmaceuticals is preparing a new Phase 2 clinical trial with trofinetide in a pediatric population with Rett syndrome. This will be their second study in Rett syndrome and supplements the information from the first trial conducted in adolescent and adult women with Rett syndrome.  The age range for the new trial will be from 5 to 15 years old.

This new Phase 2 trial will confirm the tolerability and safety of trofinetide in children and will be informative with respect to the planning of a single Phase 3 trial (a pivotal trial) that will include children, adolescents and adults.  The new Phase 2 trial will examine higher dose levels than the first trial, the results of which showed trends of increasing clinical benefit with increasing dose levels.

The pediatric trial is a placebo-controlled dose-ranging study looking at three fixed-doses of trofinetide (50, 100 and 200 mg/kg) administered orally twice daily. The study is aiming to enroll approximately 60 individuals, all of whom will receive trofinetide. The primary goal of the study will be to assess the tolerability and safety of the study medication in this younger population.  Additionally, Neuren will assess clinical benefit by determining change from baseline on a number of outcome measures including the clinician-completed Motor Behavior Assessment (MBA), the Clinical Global Impression Scale of Improvement and the Caregiver Top 3 Concerns assessment.  Each of these measures exhibited clinical benefit in the first study.  Several of the outcome measures in the study were developed collaboratively with Drs. Alan Percy, Daniel Glaze and Jeffrey Neul who are experienced  PIs from the first Phase 2 trial.

Participating Sites:

Baylor College of Medicine, Houston, TX
University of Alabama, Birmingham, AL
Gillette Children’s Specialty Healthcare, St. Paul, MN
Rush University, Chicago, IL
University of California, San Diego, CA
University of California, San Francisco, CA
Children’s Hospital Colorado, Aurora, CO
Greenwood Genetic Center, Greenwood, SC
Boston Children’s Hosptial, Boston, MA
Vanderbilt University, Nashville, TN
Cincinnati Children’s Hospital Medical Center, Cincinnati, OH
Children's Hospital of Philadelphia, PA

As Neuren is working through the study start-up details, we encourage families who are considering this Clinical Trial to once again read “A Participant’s Guide to Drug Research in Rett Syndrome” .  We look forward to sharing more community announcements as they become known.

For the most current information about this study, please visit: www.rettstudy.com.

UPDATES:

August 12, 2016
We are pleased to announce exciting news for the Neuren Rett-002 study.  The final two sites are now open for recruitment! University of California, San Diego and Cincinnati Children’s Hospital are now recruiting for the Rett-002 study. If you are interested in participating in the study at either of these sites, please find their contact details below.

Recruitment for the Rett-002 study is progressing well. There are still available slots for participation. If you are considering being involved in the study, please contact one of the 12 active sites at your earliest convenience. Site contact details can be found at www.rettstudy.com or clinicaltrials.gov.

University of California, San Diego, CA
Contact: Karen Ditslear
Email: kditslear@ucsd.edu

Cincinnati Children’s Hospital, OH
Contact: Kaela O’Brien
Phone: 513-803-7226
Email: kaela.obrien@cchmc.org

July 14, 2016
We are very excited to announce the tenth site open for recruitment in the Rett-002 study – Children’s Hospital of Philadelphia!

If you are interested in participating in the Rett-002 study at this site, please find their contact details below. For more information on the study, please visit www.rettstudy.com or clinicaltrials.gov.

Children’s Hospital of Philadelphia, PA
Contact: Margaret O’Brien
Phone: 215-590-3057
Email: Obrienm7@email.chop.edu

July 5, 2016
Neuren are pleased to announce another site now active to recruit participants into the Rett-002 study – Children’s Hospital Colorado!

If you are interested in participating in the Rett-002 study at this site, please find their contact details below. For more information on the study, please visit www.rettstudy.com or clinicaltrials.gov.

Children’s Hospital Colorado, Aurora, CO
Contact: Megan Straley
Phone: 720-777-8008
Email: Megan.Straley@childrenscolorado.org
Contact: Nanastasia Welnick
Phone: 720-777-8608
Email: Nanastasia.Welnick@childrenscolorado.org

June 24, 2016
Further exciting news from Neuren with Gillette Children’s Specialty Healthcare, MN now active to enroll participants into the Rett-002 study!

If you are interested in participating in the Rett-002 study at this site, please find their contact details below. For more information on the study, please visit www.rettstudy.com or clinicaltrials.gov.

Gillette Children’s Specialty Healthcare, St. Paul, MN
Contact: Briana Person
Phone: 651-229-1770
Email: BrianaMPerson@gillettechildrens.com

June 20, 2016
Neuren are very pleased to announce the 7th site open for recruitment in the Rett-002 study – UCSF Benioff Children’s Hospital Oakland, CA!

If you are interested in participating in the Rett-002 study at this site, please find their contact details below. For more information on the study, please visit www.rettstudy.com or clinicaltrials.gov.

Contact: Erica Robertson
Phone: 925-979-4055
Email: ERobertson@mail.cho.org

June 9, 2016
The sixth site to become active to recruitment in the Rett-002 study is Vanderbilt University in Nashville, Tennessee!

If you are interested in participating in the Rett-002 study at this site, please find their contact details below:

Contact: Dawn Lischke-Redmond
Phone: 615-875-9311
Email: dawn.m.lischke-redmond@vanderbilt.edu

Neuren recently announced that 14 subjects have already enrolled in the Rett-002 study of trofinetide. A further 14 are scheduled for their first visit. This is great news as the trial moves forward. Neuren anticipates completion of the trial in the fourth quarter of 2016.

To read Neuren's full announcement visit http://www.neurenpharma.com/irm/PDF/1603/ChairmansAddresstoShareholders.

For more information on the study, please visit www.rettstudy.com or clinicaltrials.gov.

May 16, 2016
More good news from the Rett-002 study. Neuren have just activated our fifth site in the study. Rush University Medical Center, IL are now open for enrolment!

If you are interested in participating in the Rett-002 study at this site, please find their contact details below. For more information on the study, please visit www.rettstudy.com or clinicaltrials.gov.

Rush University, IL
Contact: Anna DeSonia
Phone: 312-563-6636
Email: Anna_M_DeSonia@rush.edu

April 22, 2016
We are delighted to announce that Boston Children’s Hospital, MA and Baylor College of Medicine, TX are now recruiting for the Rett-002 study!

If you are interested in participating in the Rett-002 study at either of these sites, please find their contact details below. For more information on the study, please visit www.rettstudy.com or clinicaltrials.gov.

Boston Children’s Hospital, MA
Contact: Lindsay Swanson
Phone: 617-355-5230
Email: RettResearch@childrens.harvard.edu

Baylor College of Medicine, TX
Contact: Aryn Knight
Phone: 832-822-3751 
Email: axknight@texaschildrens.org

April 8, 2016
More exciting news from Neuren about the Rett-002 study: Greenwood Genetic Center in South Carolina is now open for recruitment!

If you are interested in participating in the Rett-002 study at this site, please find their contact details below. For more information on the study, please visit www.rettstudy.com.

Greenwood Genetic Center, Greenwood, SC
Contact: Fran Annese
Phone: 864-941-8100
Email: fran@ggc.org

April 7, 2016
Exciting news from Neuren about the trofinetide trial Rett-002: the first subject has been randomized! The subject was enrolled at the University of Alabama at Birmingham, AL.

For more information about the trial, study locations and how to register your interest in participating, please visit www.rettstudy.com.

April 4, 2016
Updates from Neuren about the trofinetide trial Rett-002: We’re up and running! The Rett-002 study is now open for enrollment. In the past week, the University of Alabama at Birmingham, AL has been initiated and are active to recruitment! If you are interested in participating in the Rett-002 study at this site, please find their contact details below. For more information on the study, please visit

www.rettstudy.com.

University of Alabama at Birmingham, Birmingham, AL
Contact: Jane Lane
Phone: 205-934-1130
Email: jlane@uab.edu

March 7, 2016
We are pleased to announce that Cincinnati Children’s Hospital Medical Center in Cincinnati, OH is now an official participating site in the Phase 2 Trofinetide Trial. They are working closely with Neuren to receive IRB approval. When available, we look forward to sharing contact information and announcing when this site will start recruiting.

February 18, 2016
We are pleased to announce that Vanderbilt University in Nashville, TN is now a participating site in the Phase 2 Trofinetide Trial. They are working closely with Neuren to receive IRB approval. When available, we look forward to sharing contact information and announcing when this site will start recruiting.

February 2, 2016
Each Rett-002 study site requires approval through their respective Institutional Review Boards (IRBs) in order to conduct the study. In addition, contracts and all financial arrangements with the site need to be settled before recruitment can commence. Therefore, sites will become open to recruitment at different times, depending on the review times of each individual IRB and institutional legal and financial groups.

A Contact Form will be made available on the Rett-002 study website (www.rettstudy.com) when each site becomes active to recruitment. Families will then be able to register their interest in participating in the study at a particular site.

We will keep you updated as each site becomes active to recruitment and will provide their contact details as this occurs.

January 15, 2016

Neuren Pharmaceuticals announces Boston Children's Hospital as a participating site in the ‪#‎rettsyndrome‬ Phase 2 clinical trial of trofinetide.‬

Remember that all sites are currently preparing submissions to their individual Institutional Review Boards and can begin recruiting participants once this process is complete. Contact details will be provided once they receive approval to conduct the study.

January 13, 2016

Neuren Pharmaceuticals announced today the sites that have been selected so far to participate in the Phase 2 clinical trial of trofinetide for girls age 5-15 years.

All sites are currently preparing submissions to their individual Institutional Review Boards and can begin recruiting participants once this process is complete, which can take from weeks to months. As they receive approval to conduct the study, and can begin discussing enrollment, contact details will be provided through the study website - www.rettstudy.com.

Participating sites:
Baylor College of Medicine, Houston, TX
University of Alabama, Birmingham, AL
Gillette Children’s Specialty Healthcare, St. Paul, MN
Rush University, Chicago, IL
University of California, San Diego, CA
University of California, San Francisco, CA
Children’s Hospital Colorado, Aurora, CO
Greenwood Genetic Center, Greenwood, SC