Do You Have a Patient Facing Challenges Due to CAH?

These clinical research studies may interest you.

Refer a Patient

If you have a patient with classic congenital adrenal hyperplasia (CAH), they may be eligible to participate in the Calm-CAH or Balance-CAH Study.

About the Studies

The Calm-CAH and Balance-CAH studies are 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.

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Study Purposes

This is a phase 3, double-blind, placebo-controlled study evaluating the safety, efficacy, pharmacokinetics, and pharmacodynamics of an investigational study drug, atumelnant, in adults living with CAH. The primary objective of this study is to reduce daily supraphysiological glucocorticoid (GCs) dosage while maintaining adrenal androgen control.

This is a phase 2/3 study with 3 parts. Part A (phase 2) will evaluate the safety and tolerability of atumelnant in pediatric patients living with CAH. Part B (phase 3) will evaluate the efficacy of atumelnant in reducing the daily supraphysiological GC dose while maintaining adrenal androgen normalization. Part C (open-label extension [OLE]) will evaluate the long-term safety and efficacy of atumelnant in pediatric patients that completed participation in either Part A or Part B of this study. Participants will have the opportunity to enter directly into this OLE period, where they will receive atumelnant for up to 5 years.

Study Designs

Participation in the study will last up to 42 weeks and enroll approximately 150 participants. The study includes up to 14 visits that will be conducted at the research center, at home if allowed and available, or over the phone.

Participants will be randomized 2:1 to receive either atumelnant 80 mg or placebo, which will be taken orally once daily in the evening. All participants will remain on their pre-study GC therapies. However, the dose level of these GC therapies will be adjusted during specific time points in the study based on biomarkers and clinical presentation of the participants. An option for dose escalation to either atumelnant 120 mg or placebo 120 mg will occur later in the study. Participants who complete the 32-week treatment period of this study may be eligible to take part in a separate long-term OLE study, which allows them to continue treatment.

Part A (phase 2) will last up to 18 weeks and will enroll a maximum of 48 participants. Part A includes up to 9 visits that will be conducted at the research center, at home if allowed and available, or over the phone. Participants will be placed into 1 of 4 cohorts based on their age and weight and will receive a dosage of atumelnant between 6 mg and 80 mg, which will be taken orally once daily in the morning. All participants will remain on their pre-study GC therapies.

Part B (phase 3) will last up to 38 weeks and will enroll approximately 105 new participants. Part B includes up to 16 visits that will be conducted at the research center, at home if allowed and available, or over the phone. Participants will be randomized 2:1 to receive either atumelnant or placebo, which will be taken orally once daily in the morning. The dose level of atumelnant will be based on the results of Part A. All participants will remain on their pre-study GC therapies. However, the dose level of these GC therapies will be adjusted during specific time points in the study based on biomarkers and clinical presentation of the participants.

Part C (OLE) will last up to 5 years and will enroll up to 153 participants who completed either Part A or Part B. Part C includes up to 30 visits. The exact number of visits will depend on if participants were enrolled in Part A or Part B previously, and will be conducted at the research center, at home if allowed and available, or over the phone. All participants will receive atumelnant, which will be taken orally once daily in the morning. All participants will remain on their GC therapies. However, the dose level of these GC therapies may be adjusted based on biomarkers and clinical presentation of the participants.

Key Eligibility Criteria

Eligible participants must:

  • Be 18 to 74 years of age
  • Have classic CAH due to 21-OHD
  • Be on a stable regimen of GC replacement (e.g., modified-release hydrocortisone, cortisone acetate, prednisolone, prednisone, methylprednisolone, dexamethasone)
  • No history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic GC therapy

Other eligibility criteria will apply.

Eligible participants must:

  • Be 1 to 17 years of age
  • Have classic CAH due to 21-OHD
  • Have elevated morning serum A4 level > upper limit of normal (ULN) during screening obtained prior to GC administration
  • Be on stable supraphysiologic GC replacement therapy (e.g., modified-release hydrocortisone, cortisone acetate, prednisolone, prednisone, methylprednisolone, dexamethasone)
  • Have a body weight of at least 10 kg

Other eligibility criteria will apply.

Healthcare Provider Resources

If you would like a hard copy of study information, download the study fact sheets for reference or to share with other physicians.

Calm-CAH Study Fact Sheet

Download

Balance-CAH Study Fact Sheet

Download