Rettsyndrome.org supports further clinical trials with trofinetide in Rett syndrome

Rettsyndrome.org announces continued financial support for further Rett syndrome clinical trials with trofinetide, a drug developed and manufactured by Neuren Pharmaceuticals.

For Immediate Release – October 12, 2015

(Cincinnati, OH)– Rettsyndrome.org today announced that the Board of Directors has made a financial commitment, through the Research to Reality Campaign, to continue support for further clinical trials with trofinetide, a drug developed and manufactured by Neuren Pharmaceuticals (http://www.neurenpharma.com).

Following a successful Phase 2 trial with trofinetide in adult women with Rett syndrome, and an encouraging meeting with the Food and Drug Administration, Neuren is moving forward with a Phase 2 trial for tolerability in children with Rett syndrome. Concurrently, Neuren is working with the FDA to design a Phase 3 pivotal study with trofinetide as a treatment for Rett.

Rajat Shah, Chairman of Rettsyndrome.org’s, commented "Rettsyndrome.org is proud to continue our partnership with Neuren. We are grateful for their work and commitment to the clinical trials of trofinetide. We are hopeful that these clinical trials will be successful.”

There are no approved drug treatments for Rett syndrome, a devastating neurological disorder. These trial advancements are significant because they bring us closer to the first potential treatments, and the hope of improving quality of life for those suffering from the condition.

This is an optimistic time for the Rett syndrome community. Research efforts are rapidly advancing our understanding of the disorder and the first treatments are closer to a reality. Ultimately, it is hoped they will lead to a cure. Rettsyndrome.org’s Research to Reality Campaign is ensuring that funding is available to advance promising clinical trials like those for trofinetide.

Rettsyndrome.org is committed to a full spectrum approach to research by supporting clinical trials as well as basic, translational and neurohabilitation research to better understand, treat, and ultimately find a cure for Rett syndrome.

About Rettsyndrome.org

Rettsyndrome.org, a 501(c)3 organization, is accelerating research for treatments and a cure for Rett syndrome. As the world’s leading private funder of Rett research, we have funded more than $35 million in peer-reviewed research grants and programs to date. We empower families to make a difference. Visit www.rettsyndrome.org to learn more, or call (800) 818-7388 (RETT)..

About Rett syndrome (RTT)

Rett syndrome is a rare non-inherited genetic postnatal neurological disorder that occurs almost exclusively in girls and leads to lifelong impairments, affecting nearly every aspect of child’s life: their ability to speak, walk, and eat is effected, seizures develop and scoliosis occurs, and many develop irregular breathing patterns. Those diagnosed with Rett syndrome require maximum assistance with basic daily activities. The hallmark sign of Rett syndrome is near constant repetitive hand movements. Cognitive assessment in children with Rett syndrome is complicated, but they understand far more than they can communicate to us, evidenced by their bright and attentive eyes, and their ability to express a wide spectrum of moods and emotions.

UPDATES:
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
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