About the Study

The purpose of the STARS clinical research study is to evaluate whether an investigational medication called sarizotan is safe and effective reducing the number of daytime apnea and other breathing episodes in people who have Rett syndrome with irregular breathing.

Who can participate?

A person with Rett syndrome may be able to join the STARS study if he or she:

  • Is at least 4 years old
  • Weighs at least 22 pounds or 10 kilograms
  • Has episodes of irregular breathing while awake during the day
  • Meets other study participation requirements that the study staff will discuss with you

All potential study participants will have their breathing evaluated at home using a device that will count their irregular breathing episodes. This evaluation will determine whether the severity of their symptoms meet the study requirements.

What is the investigational medication?

The investigational medication in the STARS study, called sarizotan, is taken twice-daily and can be given as a capsule, dissolved in a liquid, or through a feeding tube. The study is evaluating a range of doses (2 mg to 10 mg) of sarizotan compared to placebo. Placebo (sometimes called a “sugar pill”) looks like the investigational medication, but contains no active ingredients. Study participants will be randomly assigned to receive sarizotan or placebo. Participants will be twice as likely to receive sarizotan as placebo (ratio of 2:1). No one involved with the study will know which study medication each participant is receiving, but the study doctor can find out this information if needed for medical reasons.

Sarizotan has been previously tested in patients with other medical conditions at doses much higher than are being tested here; however, it has not been tested in patients with Rett syndrome. Researchers believe that sarizotan may help reduce the frequency and severity of irregular breathing episodes in people with Rett syndrome, which may help improve their quality of life.

Speak with the study staff for more information about sarizotan.

What will happen during the study?

Initial participation in the STARS study will last approximately 6 months and include around 7 visits to the study site. After the first 6 months, all participants who are doing well in the study (including those who previously received placebo) will have the option to take the investigational medication (sarizotan) for another 6 months, during which time they will have around 4 additional visits to the study site. All participants will be required to attend a follow-up visit to the study site 2 weeks after their last dose of study medication.

While in the study, participants (with the help of their caregivers) will need to take their study medication twice-daily and attend all required study visits. Caregivers will need to be available for scheduled telephone calls with the study staff. Participants will also be required to wear a device at home to monitor their breathing for at least 15 days over a period of 6 months. The study staff will provide study participants’ caregivers with specific instructions for each visit.

What are the potential risks and benefits of study participation?

The study doctor will explain all of the potential risks and benefits of participating in the study. There is no guarantee that being in the study will help treat irregular breathing in people with Rett syndrome. Participants will receive all study-related treatments and procedures at no cost, and may also qualify for travel expense reimbursement. What we learn from the STARS study may help others who have Rett syndrome with irregular breathing in the future. Talk with the study staff if you have any questions about the risks and benefits of study participation.