Frequently Asked Questions

Please email to see if they may be eligible to participate. You will soon be emailed back with contact information of a study staff member at a site(s) that is currently open to enrollment and closest to you geographically.

Who are we?

Neuren Pharmaceuticals is a specialty biopharmaceutical company with a focus on rare, neurodevelopmental disorders. Our lead medicine, Trofinetide, showed strongly positive results in a Phase III study in Rett syndrome, meeting its co-primary endpoints and demonstrating statistically significant improvements over placebo. On 10 March, 2023, Trofinetide received marketing approval from the U.S. Food and Drug Administration (FDA) for DAYBUE™ to treat Rett syndrome in patients 2 years of age and older. More information can be found at: www.neurenpharma.com.

What is a clinical trial?

Clinical Trials provide information to determine whether a medicine is safe and efficacious. Regulatory Agencies worldwide require the data from such clinical trials to assess a medicine’s benefit to the population being treated.

Clinical trials are carried out in humans after laboratory and animal testing demonstrates that a treatment is likely to be efficacious and safe. The trials move through a series of phases (I, II , III and IV), and follow protocols (approved by an IRB (Institutional Review Board) which is an independent committee established to review and approve research involving human subjects) that balance potential risks with benefits.

All participants in this study will receive thorough study-related physical, psychological, and behavioral evaluations by a study doctor.

While there is no guaranteed benefit to participants in a clinical research study, participation may help to advance scientific knowledge in the area that is being studied.

What is a Phase 2 clinical trial?

The phases of clinical trials are the stages where researchers conduct experiments to find out if a certain medicine or intervention is considered safe and efficacious as a new medical treatment. These range from Phase 1 to Phase 4. The image below summarizes the four phases.

In Phase 2 studies like this, researchers administer the study medication to a group of patients who have the condition for which the medicine is being developed.

The next stage is Phase 3, where researchers will confirm the efficacy of the new treatment.

What is a study protocol?

A protocol details the specific instructions that Clinicians need to carry out the study. It is carefully designed to protect the wellbeing of the participants and answer specific research questions. A protocol explains who can participate in the trial, when different assessments should be taken, which procedures will be performed, details of medicine to be taken and how much to give, as well as information on the duration of the study. While participating in a clinical trial, participants are seen regularly by the study team to monitor their condition and determine the safety and efficacy of the treatment.

How can my child participate in this study?

Once a site has received IRB approval for the study, you will be able to contact them directly to discuss your child’s potential eligibility. They will arrange  a series of screening visits that will assess and confirm that your child meets all the eligibility criteria detailed in the protocol. These criteria are based on age, type of condition, medical history, concurrent medications and a range of clinical and behavioral assessments. If your child meets all these criteria, they will be eligible to participate in the study.

What does this Prader-Willi Syndrome clinical trial involve?

The current study for children and adolescents with Prader-Willi Syndrome is a Phase 2 trial. This trial will include up to 20 male and female participants between the ages of 4-12 years.

This Phase 2 study is intended to confirm that NNZ-2591 is a well-tolerated medicine that is worthy of further development for children and adolescents with Prader-Willi Syndrome. This study is also measuring efficacy (whether individuals see improvement or benefit from taking the study drug). It includes a number of assessments that look at a number of areas of concern in Prader-Willi Syndrome such as overall well-being, communication, behavior, and quality of life.

Your participation will last for approximately 21 weeks. During this time you will attend clinic on 5 occasions. There will also be 12 in-home visits where you will have a telehealth consultation with site staff who will ask questions on your child’s progress. These in-home visits will be supported by a visiting nurse who will carry out a number of assessments and take samples for analysis.

Does gene mutation/deletion affect eligibility?

To be eligible for the study, the participant must have a clinical diagnosis of Prader-Willi Syndrome together with a documented disease-causing genetic abnormality of the 15q11-q13 chromosome region.

What if my child has more than one genetic disorder?

Our main goal in this study is to examine the effectiveness of the new study medication (NNZ-2591) in the treatment of Prader-Willi Syndrome. This means that if your child has been diagnosed with multiple genetic disorders, unfortunately they are unable to participate in this study.

Many of these disorders have overlapping symptoms, and it would then be impossible to accurately measure the effect of the study medication on Prader-Willi Syndrome. 

If you have specific questions or concerns, please speak with your study doctor.

How is the safety of the participant protected?

The ethical and legal codes that govern medical practice also apply to clinical research. Most clinical research is federally regulated with built in safeguards to protect the participants and their information. The trial follows a carefully controlled protocol, a study plan which details what researchers will do throughout the study. Safety data is reviewed by an independent group of clinical experts as the trial progresses. If you or your doctor believe your safety is at risk, you can withdraw from the study at any time. Researchers report the results of the trial at scientific meetings, to medical journals, and to various government agencies. At all times, individual participants' information will be de-identified to remain secret and will not be mentioned in any reports.

Can a participant leave a clinical trial after it has begun?

Yes, a participant can choose to withdraw from a clinical trial at any time. You should speak to the study team to let them know you no longer wish for the participant to continue in the trial.

If my child is responding well to the treatment, can I continue to have access the study medication?

If this study is successful, we will then move forward with opening a phase 3, double-blind, placebo controlled study with a separate but corresponding Open-label Extension study. At that time, all participants that completed the phase 2 trial would have an opportunity to skip the Phase 3 trial and screen for the Open-label extension study.

Can we participate if we don’t live in any of the listed states?

Yes, you can participate in the study. Support for inter-state attendance at site is available.

Trial participation and clinic attendance during the Covid-19 pandemic.

Flexibility has been built into the study protocol and supporting structures to allow for continued subject participation during the Covid-19 pandemic. However, to ensure the integrity of the study, there are a number of study visits where it is critical that on site attendance occur.

Is financial support available to participate?

Yes. Once a participant is confirmed as eligible, financial support for attendance at in-clinic visits is available for that child and their family (inclusive of both parents and siblings, if needed). A company named Clincierge will manage all travel and lodging logistics. Clincierge is a white glove service provider that will work with each participating clinical site in booking flights, ground transportation and hotels for study participants and their families. All up-front costs associated with approved travel and lodging will be paid for by Clincierge. There will be no cost whatsoever to the participant and their family. Receipts of meals for any family member required during the time of travel to a clinical site can be submitted to Clincierge for reimbursement.

How does the COVID-19 vaccine affect my child’s eligibility to participate?

Having the COVID-19 vaccine does not affect your child’s eligibility to participate in this study. Nor is it an issue if received part-way through the study. We’d advise having the vaccination ahead of participation in the trial to avoid any confusion in side effects. The study team will record any vaccines or medications that have been received during the treatment period.