Clinical research sites across the United States are seeking qualified volunteers to participate in a clinical study testing an investigational medication for Rett syndrome.

If your daughter or loved one has classical/typical Rett syndrome, she may be eligible to participate.

Those who qualify may receive study-related care at no cost. Travel assistance and reimbursement may also be available for volunteers that do not live near a research site.

Participation Requirements:

Your daughter or loved one may qualify if she:

Is 2 to under 5 years old at the screening visit

Has classical/typical Rett syndrome

Weighs at least 9 kg (19.8 lbs)

Can swallow study medication (provided as a liquid solution) or can take it by gastrostomy tube

There are additional requirements to participate. A study representative will discuss them with you.

See if your loved one qualifies

Find out more about the DAFFODIL study and site locations:

Or call us at 442-232-2001

Research staff will be in contact with you directly to determine whether your loved one qualifies for the study.

About the Study

The DAFFODIL study is a 26-month open-label study evaluating the efficacy (how well it works) and safety of trofinetide, an investigational medication administered as an oral solution to treat Rett syndrome. The DAFFODIL Study is also designed to investigate the potential benefit of the study medication on overall quality of life for the participants.

Qualified individuals will participate in a screening period of up to four (4) weeks, an initial treatment period of twelve (12) weeks, an extended treatment period up to twenty-one (21) months and a safety follow-up period of thirty (30) days.

What is Rett Syndrome?

Rett syndrome is a seriously debilitating neurodevelopmental disorder that occurs primarily in females following apparently normal development for the first six months of life. Currently, there are no approved medicines for the treatment of Rett syndrome. Rett syndrome occurs in 1 in 10,000 to 15,000 girls born in the United States. (1,2) Rett syndrome is mostly seen in girls and causes problems with brain function affecting speech, movement, moods and emotions, breathing, swallowing, heart function, and digestion. (3)

To learn more about Rett Syndrome visit:

1. Bienvenu T, Philippe C, De Roux N, et al. The incidence of Rett syndrome in France.

2. Neul JL, Kaufmann WE, Glaze DG, et al. Rett syndrome: revised diagnostic criteria and nomenclature. Ann Neurol. 2010;68(6):944-950.

3. Motil KJ, Caeg E, Barrish JO, et al. Gastrointestinal and nutritional problems occur frequently throughout life in girls and women with Rett syndrome. J Pediatr Gastroenterol Nutr. 2012;55(3):292-298.

What is a Clinical Study?

A clinical study tests the efficacy (how well it works), safety, and/or tolerability of an investigational medication in a population of volunteers. Every investigational medicine goes through the clinical study process. Therefore, participants like you and your loved one play an important role in advancing medicine for present and future generations.

What is an Investigational Medication?

An investigational medication can be a new drug or a drug being tested for a different use than what it was previously approved by the U.S. Food and Drug Administration (the “FDA”) to treat. All investigational medications used in this phase of the clinical research study process have been tested in a laboratory and the FDA is allowing it to be tested in people. It is not approved for sale by the FDA or other government agencies. A clinical study tests how well an investigational medication works and whether it is safe to use for a specific disease or disorder.

Why Participate in a Clinical Study?

By having your child participate in a clinical study, you play a more active role in their healthcare, gain access to new research treatments before they are widely available, and help others by contributing to medical research. As a participant in a clinical study, your child can play an important role in getting new drugs approved for use in treating other patients.

It is important to remember that the main purpose of clinical studies is to study new drugs or new uses of approved drugs. Often, drugs must be tested under very specific conditions, so not all patients with a given disease will be able to enroll in a clinical study. In general, each clinical study has inclusion and exclusion requirements that are intended to make the study more likely to demonstrate the effect of the treatment, or to protect patients by minimizing risks to certain populations.

If you are considering having your child participate in a clinical study, you should carefully read information about the drug study being considered and ask questions about exactly what will happen during the study during a process known as “informed consent.” You are entitled to receive detailed information regarding what is known about the drug before you decide to enter your loved one in a clinical study. On behalf of your child, you must provide a signed informed consent document indicating that you understand what will happen during the clinical study, before participating.

You should also get advice from your healthcare professional.

To read more about clinical research visit:

Social Media and Clinical Studies

If your loved one participates in a clinical study it is important that you do not share information with anyone other than people who need to know, like your doctors and the study team at the clinic. Although social media communication programs such as Facebook, Twitter, Instagram, YouTube, Snapchat, etc., are fun and easy to use, it is important that information is not shared this way or any other way, like with other people in the waiting room during study visits. This can impact the outcome of a study and its chances of success.

Study Locations

This study is being conducted at the following locations:

Aurora, CO*

Baltimore, MD*

Birmingham, AL*

Boston, MA*

Chicago, IL*

Cincinnati, OH*

Houston, TX*

La Jolla, CA*

Nashville, TN

Philadelphia, PA*

St. Louis, MO

St. Paul, MN*

*not yet recruiting