Congenital Adrenal Hyperplasia
Conditions
Keywords
ATR-101, classic congenital adrenal hyperplasia, 21-hydroxylase deficiency, CAH
Brief summary
This is a Phase 2 multicenter, single-blind, multiple dose study to evaluate the safety and efficacy of orally administered ATR-101 in subjects with classic congenital adrenal hyperplasia (CAH). Treatment duration will range from a minimum of approximately 2 months to 6 months per subject. A subject may receive a minimum of one dose level or up to a maximum of 5 dose levels, in sequentially increasing dose strengths. Each dose level will last 28 days.
Interventions
DRUGATR-101
125-1000 mg twice per week
Sponsors
Millendo Therapeutics, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Single treatment arm with up to 5 planned dosing periods with escalating doses. Subjects were dosed with each dose level for 2 weeks, followed by 2 weeks of matching placebo (subjects blinded).
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* Documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency based on: Documented genetic mutation in the CYP21A2 enzyme consistent with a diagnosis of classic CAH, or historical documentation of elevated 17-hydroxyprogesterone * Biochemical marker of disease status of 17-hydroxyprogesterone ≥ 4 times the upper limit of normal * Chronic glucocorticoid replacement therapy for at least 6 consecutive months * Stable glucocorticoid and mineralocorticoid regimen for at least 1 month
Exclusion criteria
* Non-classic CAH * Other causes of adrenal insufficiency * Surgery within the previous 3 months prior to screening or planned surgery during study participation * History of active cancer requiring medical or surgical therapy within the past 6 months
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Reduction of 17-hydroxyprogesterone to </= 2 Times the Upper Limit of Normal at Any Time Following 2 Weeks of Dosing With ATR-101 | Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels. | 17-hydroxyprogesterone was measured predose in the morning at the beginning and end of each dose level. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| ATR-101 Ascending dose levels of ATR-101 beginning with 125 mg by mouth twice per day up to 1000 mg twice per day. | 10 |
| Total | 10 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
Baseline characteristics
| Characteristic | ATR-101 |
|---|---|
| 17-hydroxyprogesterone | 6591.0 ng/dL STANDARD_DEVIATION 5371.8 |
| Age, Continuous | 30.3 years STANDARD_DEVIATION 13.76 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 9 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 9 Participants |
| Region of Enrollment United States | 10 participants |
| Sex: Female, Male Female | 5 Participants |
| Sex: Female, Male Male | 5 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 10 |
| other Total, other adverse events | 9 / 10 |
| serious Total, serious adverse events | 1 / 10 |
Outcome results
Primary
Number of Participants With Reduction of 17-hydroxyprogesterone to </= 2 Times the Upper Limit of Normal at Any Time Following 2 Weeks of Dosing With ATR-101
17-hydroxyprogesterone was measured predose in the morning at the beginning and end of each dose level.
Time frame: Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| ATR-101 | Number of Participants With Reduction of 17-hydroxyprogesterone to </= 2 Times the Upper Limit of Normal at Any Time Following 2 Weeks of Dosing With ATR-101 | 2 Participants |