The Balance-CAH Study
Enrolling Ages 1 to 17

The Balance-CAH Study is evaluating the safety and effectiveness of an investigational study drug, atumelnant, whether it may reduce the dosage needed for steroid medications like glucocorticoids (GCs), and how it may help manage symptoms of CAH.

Study information for kids

About the Study

The Balance-CAH Study is currently enrolling children and adolescents diagnosed with classic CAH. To learn more and see if you or your child may be eligible to take part, talk to your doctor today.

This study has 3 segments: Parts A, B, and C.

Depending on when you or your child enrolls in the study, you or your child may be eligible to participate in either Part A or B of the study.

If you or your child completes Part A or B of the study, you or your child may be eligible to enroll in a long-term extension study (Part C) where you or your child will continue to receive the study drug for an extended period of time.

Duration

Part A will last about 4 months with up to 9 study visits, 2 of which may be done by telephone.

Study purpose

The purpose of Part A is to determine what the best amount (dose) of the study drug may be for children and adolescents with CAH.

Receive the study drug

All participants in Part A will receive the study drug. There is no placebo in this part of the study. A placebo is a substance that looks just like the study drug but does not contain any medication.

Remain on your current medication

Throughout the study, you or your child will be able to remain on your current GCs as directed by your study doctor.

About the study drug

The study drug is a tablet (or granule for younger children) that will be taken by mouth once daily in the morning as instructed by your study doctor.

No cost to participants

The study drug, study lab tests, imaging (hand x‑ray and ultrasound in male participants), study procedures, and safety assessments are provided at no cost to you. If enrolled, reasonable costs for travel may be reimbursed when you visit the research center for study visits.

If you or your child completes Part A of the Balance-CAH Study, you may be eligible to enroll in a long-term extension study (Part C) where you or your child will continue to receive the study drug for an extended period of time.

Duration

Part B will last about 9 months with up to 16 study visits, 5 of which may be done by telephone.

Study purpose

The purpose of Part B is to evaluate the safety and effectiveness of the study drug in children and adolescents with CAH. We will use the dose from Part A that showed the best results.

Randomized study

Participants will be randomly assigned, like drawing straws, to receive the study drug or a placebo, which is a substance that looks just like the study drug but does not contain any medicine.

Randomization chances

You or your child will have a higher chance of receiving the study drug (about 66%) than the placebo (about 33%).

Remain on your current medication

Throughout the study, you or your child will be allowed to keep taking GCs as directed by your physician, but efforts will be made to reduce the dosage based on how you are feeling.

About the study drug

The assigned study treatment (study drug or placebo) is a tablet (or granule for younger children) that will be taken by mouth once daily in the morning as instructed by your study doctor.

No cost to participants

The assigned study treatment, study lab tests, imaging (hand x‑ray and ultrasound in male participants), study procedures, and safety assessments are provided at no cost to you. Additionally, if enrolled, reasonable costs for travel may be reimbursed when you visit the research center for study visits.

If you or your child completes Part B of the Balance-CAH Study, you may be eligible to enroll in a long-term extension study (Part C) where all participants will be guaranteed to receive the study drug, even if they received the placebo in Part B.

Duration

Part C will last up to 5 years with up to 30 study visits, some of which may be done by telephone.

Long-term extension

Part C is the long-term extension study that you or your child may be eligible for if you complete Part A or B.

Study purpose

The purpose of Part C is to evaluate the long-term safety and effectiveness of the study drug in children and adolescents with CAH.

Receive the study drug

All participants in Part C will receive the study drug, even if they received placebo in Part B.

Remain on your current medication

Throughout the study, you or your child will be allowed to keep taking GCs as directed by your physician, but efforts will be made to reduce the dosage.

About the study drug

The study drug is a tablet (or granule for younger children) that will be taken by mouth once daily in the morning as instructed by your study doctor.

No cost to participants

The study drug, study lab tests, imaging (hand x‑ray and ultrasound in male participants), study procedures, and safety assessments are provided at no cost to you. If enrolled, reasonable costs for travel may be reimbursed when you visit the research center for study visits.

Who May Qualify

To participate in this study, you or your child must:

  • Be 1 to 17 years of age
  • Have received a diagnosis of classic CAH
  • Be on a stable GC regimen (for example: modified-release hydrocortisone, cortisone acetate, prednisolone, prednisone, methylprednisolone, dexamethasone)

Additional requirements apply, which the study team will discuss with you.

Balance-CAH Study Participation

The Balance-CAH Study is made up of the following periods:

Screening period

The study team will perform assessments, such as blood and urine sample collections, to make sure the study is a good match for you or your child. There will be 2 screening visits at the research center.

Treatment period

If you or your child qualifies and decides to participate, you will receive the assigned study treatment to be taken once daily in the morning as instructed by your study doctor. There will be 5 to 12 study visits, several of which may be completed by telephone.

Follow-up period

You or your child will stop taking the assigned study treatment and have 2 follow-up visits so the study team can monitor your or your child’s health. One visit may be completed by telephone.

This period is not required if you or your child enters the long-term extension study (Part C).

See if you may qualify

Balance-CAH Study Part A

Download the Balance-CAH Study Part A flyer to discuss with your child’s doctor.

Download

Balance-CAH Study Part B

Download the Balance-CAH Study Part B flyer to discuss with your child’s doctor.

Download

Balance-CAH Information for Kids

We've provided information to help your child understand the study.

Learn more