mTORopathy Survey Study

Purpose of this Study

This study follows about 40 mothers and their children who have a condition called tuberous sclerosis complex. The children are 10 years old or younger. The study looks at information from pregnancies, including whether the mother took certain medicines during pregnancy or not. To take part, participants will give consent online, have a short phone call, and complete surveys. With permission, the study team will collect medical records about the pregnancy and the child’s health from birth to now. There are no in person visits, treatments, or procedures. The study only collects information to help doctors learn more about how this condition and certain medicines may affect pregnancy and children.

Who Can Participate?

Eligibility

People can join this study if they are mothers and children where the child has a condition called tuberous sclerosis complex or a related genetic condition. The child must have been diagnosed by one year of age and be 10 years old or younger now. Mothers can join if they took certain medicines called sirolimus or everolimus during pregnancy or if they did not take these medicines. Mothers must be willing to give permission for the study team to look at their medical records and their child’s medical records. They also need to be able to complete study surveys. Some people who are currently pregnant can also join if their baby has been diagnosed with this condition and they are willing to have follow up after the baby is born.

Age Range

0-110

Sex/Genders

Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No

What is Involved?

Description

The purpose of this study is to learn if taking certain medicines, called sirolimus or everolimus, during pregnancy affects the health of mothers and their babies. Researchers will compare mothers who took these medicines with those who did not. The goal is to understand any risks and help doctors provide better care for families affected by this condition.

Locations

Duke University Hospital

Visit Timing

Weekdays
Weekends
Evenings

Compensation

No

Spanish Materials Available

No

Study Details

Full Title

Survey study for mTORopathies to explore perinatal and postnatal health

Principal Investigator

Jennifer
Cohen

Protocol Number

PRO00120405

Phase

N/A

Enrollment Status

Pending Open to Enrollment