FAQs

Frequently Asked Questions

Who Is Neuren?

Neuren Pharmaceuticals is a specialty biopharmaceutical company that is focused on finding treatments for rare neurodevelopmental disorders. Our first medicine, DAYBUE™ (trofinetide), is approved by the FDA for the treatment of Rett syndrome in adult and pediatric patients aged 2 years and older. Our second medicine, NNZ-2591, is being developed for multiple neurodevelopmental disorders, including Phelan-McDermid syndrome.

Learn more about us at our website: www.neurenpharma.com.

Phelan McDermid syndrome is a genetic disorder that may affect a person’s development, intellectual ability, motor skills, and speech.  

Learn more about us at our website: www.neurenpharma.com.

Clinical research studies (or ‘trials’) look for new ways to develop potential treatment options. This may involve comparing investigational treatments (treatments that have not been approved by a regulatory body) to “comparator” medicines (i.e. an approved medicine) or inactive treatments (i.e. a placebo), by splitting participants into groups and randomly assigning them to one of the treatments. 

Even if participants don’t directly benefit from the results of the clinical study, the information collected adds to scientific knowledge and may help families like yours in the future.  

Caregivers and their children who take part in clinical trials are essential to the process of improving pediatric medical care. 

Participation in the study will last for a total of about 19 weeks. 

If your child completes all the visits in the Koala study and is eligible, you will have the option to enroll them in a long-term extension of the study. All participating children in the extension study will receive NNZ-2591 (there is no placebo group). This study is planned to last 52 weeks but could be extended to allow continued access to NNZ-2591. 

Informed consent is a process in clinical research studies where the participant or their caregiver is provided with all the details about the study, including any potential risks, before making their decision for them or their child to join the study. Participants or their caregivers are required to sign an ‘informed consent form’ before beginning any of the study activities. This is not a contract, and participants can leave the study at any time and for any reason.  

A study protocol details the specific instructions that clinicians need to perform the study. It is carefully designed to protect the well-being of the participants and answer specific research questions. A protocol explains who can participate in the trial, stipulates when different assessments should be taken, directs which procedures will be performed, provides details of medicine to be taken (including the dose and timing), and provides 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.

The study drug, study-related procedures and travel associated with clinic visits are provided at no cost.

All the potential risks and benefits of the study will be explained to you in detail before you decide if you want your child to take part. There are potential risks associated with taking any medical treatment, including the study drug. Your child’s symptoms may get better, stay the same, or get worse. If you choose to have your child join the study, your study doctor will review the potential risks with you.  

There are also potential risks associated with the procedures and tests your child will have during the study. The assessments performed as part of the study may cause your child tiredness, frustration and may be a source of behaviors that are not helpful or are potentially harmful. 

If your child completes all visits in the Koala study and is eligible, you will have the option to enroll them in a long-term extension of the study. All participating children in the extension study will receive NNZ-2591 (there is no placebo group). This is planned to last for 52 weeks, but this could be extended to allow continued access to NNZ-2591. 

You can stop your child from taking part in this study at any time and for any reason. You do not need to give a reason. If they stop, they will not lose any of their regular medical benefits not associated with the study.  

If you decide to take your child out of the study early, you may be asked to attend one more study visit so the study doctor can assess their health. 

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, weight, medical history, concurrent medications prescribed, and a range of clinical and behavioral assessments. If your child meets all the criteria, they will be eligible to participate in the study.

The current study for children and adolescents with Phelan-McDermid syndrome is a phase 3 trial. This trial will include up to XX male and female participants aged 3–12 years.

The phase 3 study is intended to investigate the efficacy (whether individuals see improvement or benefit from taking the study drug) and safety of NNZ-2591 in children and adolescents with Phelan-McDermid syndrome. It includes several assessments that evaluate areas of concern in Phelan-McDermid syndrome such as overall well-being, communication, behavior, and quality of life.

You and your child’s participation will last for approximately 19 weeks. During this time, you will visit the study clinic on 5 occasions. There will also be 6 in-home visits where you will have a telehealth consultation with site staff who will ask questions about your child’s progress.

Multiple genetic tests can be used to confirm a genetic variation associated with Phelan-McDermid syndrome, including chromosome analyses or genetic sequencing . Speak with your doctor about genetic testing options.

The main goal of this study is to examine the effectiveness of the new medication (NNZ-2591) in the treatment of Phelan-McDermid syndrome. Many genetic disorders have overlapping symptoms, and it would be impossible to accurately measure the effect of the study medication on the intended indication, Phelan-McDermid syndrome, in a participant with multiple genetic conditions. This means that if your child has been diagnosed with multiple genetic disorders, they will be unable to participate in this study. If you have specific questions or concerns, please speak with your doctor.

Blood samples will be taken by skilled study nurses who have extensive experience working with children who have neurodevelopmental disorders such as Phelan-McDermid syndrome.

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 medical information. The trial follows a carefully controlled protocol (a study plan that details what researchers will do throughout the study). Safety data are reviewed by an independent group of clinical experts as the trial progresses. If you or your doctor believe your child’s safety is at risk, know that you can withdraw from the study at any time. Researchers report the results of the trial at scientific meetings, to medical journals via scientific articles, and to various government agencies. At all times, participants’ information will be deidentified to remain secret and will not be mentioned in any reports.

Children and adolescents who have participated in a clinical trial of NNZ-2591 for Phelan-McDermid syndrome may be eligible to enroll in an open-label extension study evaluating the long-term safety and efficacy of NNZ-2591. Speak with your study team about the possibility of enrolling in the open-label extension study.

Yes, you can participate in the study. Support for study enrollment at a site in another state is available.

Yes, once your child is confirmed to be eligible to participate in the study, support for attending in-clinic visits will be available. Where required, you can be reimbursed for reasonable travel and accommodation costs. The process will be managed by the PSMF; for more information, please contact the PMFS at the following email: emailtravelprogram@pmsf.org.

Being vaccinated for COVID-19 does not affect eligibility to participate in this study, nor is it an issue to receive a COVID-19 vaccination during the study. We recommend that your child gets the necessary vaccinations before participating in the trial to avoid any confusion regarding side effects. The study team will record any vaccines or medications your child has received during the treatment period.