Trial Outcomes & Findings for A Study of ATR-101 for the Treatment of Endogenous Cushing's Syndrome (NCT NCT03053271)

NCT ID: NCT03053271

Last Updated: 2021-03-02

Results Overview

The number of subjects meeting the criterion was divided by the total number of subjects.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 4 participants
• Primary outcome timeframe: Through Day 85
• Results posted on: 2021-03-02

Participant Flow

Participant milestones

Measure	ATR-101 (Nevanimibe HCl) Subjects received nevanimibe orally 250 mg BID for 2 weeks, then 500 mg BID for 2 weeks, then 1000 mg BID for 2-4 weeks.
Overall Study STARTED	4
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Measure	ATR-101 (Nevanimibe HCl) Subjects received nevanimibe orally 250 mg BID for 2 weeks, then 500 mg BID for 2 weeks, then 1000 mg BID for 2-4 weeks.
Overall Study Did not meet randomization criteria	3
Overall Study Withdrawal by Subject	1

Baseline Characteristics

A Study of ATR-101 for the Treatment of Endogenous Cushing's Syndrome

Baseline characteristics by cohort

Measure	ATR-101 (Nevanimibe HCl) n=4 Participants Subjects received nevanimibe orally 250 mg BID for 2 weeks, then 500 mg BID for 2 weeks, then 1000 mg BID for 2-4 weeks.
Age, Continuous	44.3 years STANDARD_DEVIATION 9.18 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	4 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	3 participants n=5 Participants
Region of Enrollment United Kingdom	1 participants n=5 Participants
Weight	86.42 kg STANDARD_DEVIATION 7.755 • n=5 Participants
Body Mass Index	30.79 kg/m^2 STANDARD_DEVIATION 3.172 • n=5 Participants
24-hr Urinary Free Cortisol	198.84 ug/day STANDARD_DEVIATION 147.071 • n=5 Participants

PRIMARY outcome

Timeframe: Through Day 85

Population: 4 patients were analyzed. None ("0") of them showed a normal 24-hr urinary free cortisol (UFC) or a reduction in 24-hr UFC of ≥ 50% relative to their baseline value

The number of subjects meeting the criterion was divided by the total number of subjects.

Outcome measures

Measure	ATR-101 (Nevanimibe HCl) n=4 Participants Subjects received nevanimibe orally 250 mg BID for 2 weeks, then 500 mg BID for 2 weeks, then 1000 mg BID for 2-4 weeks.
The Proportion of Subjects With Either a Normal 24-hr Urinary Free Cortisol (UFC) or a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value	0 Participants

SECONDARY outcome

Timeframe: Through Day 85

Population: 4 patients were analyzed. None ("0") of them showed a normal 24-hr urinary free cortisol (UFC)

The number of subjects meeting the criterion was divided by the total number of subjects.

Outcome measures

Measure	ATR-101 (Nevanimibe HCl) n=4 Participants Subjects received nevanimibe orally 250 mg BID for 2 weeks, then 500 mg BID for 2 weeks, then 1000 mg BID for 2-4 weeks.
The Proportion of Subjects With a Normal 24-hr UFC	0 Participants

SECONDARY outcome

Timeframe: Through Day 85

Population: 4 patients were analyzed. None ("0") of them showed a reduction in 24-hr UFC of ≥ 50% relative to their baseline value

The number of subjects meeting the criterion was divided by the total number of subjects.

Outcome measures

Measure	ATR-101 (Nevanimibe HCl) n=4 Participants Subjects received nevanimibe orally 250 mg BID for 2 weeks, then 500 mg BID for 2 weeks, then 1000 mg BID for 2-4 weeks.
The Proportion of Subjects With a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value	0 Participants

SECONDARY outcome

Timeframe: Through Day 57 and Day 85

Population: 4 patients were analyzed at Day 57 and Day 85. None ("0") of them showed a normal 24-hr urinary free cortisol (UFC).

The number of subjects meeting the criterion was divided by the total number of subjects.

Outcome measures

Measure	ATR-101 (Nevanimibe HCl) n=4 Participants Subjects received nevanimibe orally 250 mg BID for 2 weeks, then 500 mg BID for 2 weeks, then 1000 mg BID for 2-4 weeks.
The Proportion of Subjects With a Normal 24-hr UFC Day 57	0 Participants
The Proportion of Subjects With a Normal 24-hr UFC Day 85	0 Participants

SECONDARY outcome

Timeframe: Through Day 57 and Day 85

Population: 4 patients were analyzed at Day 57 and Day 85. None ("0") of them showed a reduction in 24-hr UFC of ≥ 50% relative to their baseline value

The number of subjects meeting the criterion was divided by the total number of subjects.

Outcome measures

Measure	ATR-101 (Nevanimibe HCl) n=4 Participants Subjects received nevanimibe orally 250 mg BID for 2 weeks, then 500 mg BID for 2 weeks, then 1000 mg BID for 2-4 weeks.
The Proportion of Subjects With a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value Day 57	0 Participants
The Proportion of Subjects With a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value Day 85	0 Participants

Adverse Events

ATR-101 (Nevanimibe HCl)

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	ATR-101 (Nevanimibe HCl) n=4 participants at risk Subjects received nevanimibe orally 250 mg BID for 2 weeks, then 500 mg BID for 2 weeks, then 1000 mg BID for 2-4 weeks.
Skin and subcutaneous tissue disorders Hyperhidrosis	25.0% 1/4 • Number of events 1 • Adverse events were collected from the time of informed consent until 30 days after the last dose of study drug
Skin and subcutaneous tissue disorders Rash pruritic	25.0% 1/4 • Number of events 1 • Adverse events were collected from the time of informed consent until 30 days after the last dose of study drug
Ear and labyrinth disorders Ear discomfort	25.0% 1/4 • Number of events 1 • Adverse events were collected from the time of informed consent until 30 days after the last dose of study drug
Gastrointestinal disorders Abdominal distention	25.0% 1/4 • Number of events 1 • Adverse events were collected from the time of informed consent until 30 days after the last dose of study drug
Gastrointestinal disorders Diarrhea	25.0% 1/4 • Number of events 1 • Adverse events were collected from the time of informed consent until 30 days after the last dose of study drug
General disorders Fatigue	25.0% 1/4 • Number of events 1 • Adverse events were collected from the time of informed consent until 30 days after the last dose of study drug
General disorders Peripheral swelling	25.0% 1/4 • Number of events 1 • Adverse events were collected from the time of informed consent until 30 days after the last dose of study drug
Injury, poisoning and procedural complications Laceration	25.0% 1/4 • Number of events 1 • Adverse events were collected from the time of informed consent until 30 days after the last dose of study drug
Investigations Hepatic enzyme increased	25.0% 1/4 • Number of events 1 • Adverse events were collected from the time of informed consent until 30 days after the last dose of study drug
Metabolism and nutrition disorders Decreased appetite	25.0% 1/4 • Number of events 1 • Adverse events were collected from the time of informed consent until 30 days after the last dose of study drug
Nervous system disorders Headache	25.0% 1/4 • Number of events 1 • Adverse events were collected from the time of informed consent until 30 days after the last dose of study drug

Additional Information

Clinical Trial Information

Millendo Therapeutics

Phone: +1 734 845-9000

Email: millendo@millendo.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place