Trial Outcomes & Findings for Assessment of BIM23B065, Given as Repeated Subcutaneous Injection in Subjects With Acromegaly (NCT NCT03045302)

NCT ID: NCT03045302

Last Updated: 2019-03-08

Results Overview

A GH responder was defined as a subject with mean serum GH concentration ≤2.5 micrograms per litre (mcg/L) or \>50% reduction from mean baseline GH concentration after a 6-day titration and an 8-day treatment period with BIM23B065. The mean serum concentration of GH was measured over 6 hours at baseline (Day -1) and on Day 14. The number of subjects who were GH responders at Day 14 of the treatment period is presented.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

4 participants

Primary outcome timeframe

From baseline (Day -1) to Day 14.

Results posted on

2019-03-08

Participant Flow

Subjects with acromegaly were recruited from 26 January 2017 in 4 study centres in 4 countries. Stage 1 of the study consisted of a twice daily (BID) regimen of BIM23B065, and a Stage 2 with a three times daily regimen of BIM23B065 was planned but not conducted due to early termination of the study on 26 May 2017.

7 subjects were screened and 3 failed screening (1 subject did not meet eligibility criteria and 2 subjects did not complete screening procedures due to the early study termination).

Participant milestones

Participant milestones
Measure
BIM23B065 BID Stage 1
Subjects received twice daily administrations of BIM23B065 (at an interval of 12 +/- 1 hour) as subcutaneous injections in the abdominal region during the Stage 1 treatment period which consisted of a titration phase and a treatment phase. Titration phase (Days 1 to 6): Subjects received BIM23B065 0.4 milligram (mg) BID (Days 1 and 2, then BIM23B065 0.6 mg BID (Days 3 and 4) and BIM23B065 0.8 mg BID (Days 5 and 6). Treatment phase (Days 7 to 14): Following titration, a stable target dose of 1.0 mg BID BIM23B065 was planned to be administered from Day 7 to Day 14.
Overall Study
STARTED
4
Overall Study
COMPLETED
3
Overall Study
NOT COMPLETED
1

Reasons for withdrawal

Reasons for withdrawal
Measure
BIM23B065 BID Stage 1
Subjects received twice daily administrations of BIM23B065 (at an interval of 12 +/- 1 hour) as subcutaneous injections in the abdominal region during the Stage 1 treatment period which consisted of a titration phase and a treatment phase. Titration phase (Days 1 to 6): Subjects received BIM23B065 0.4 milligram (mg) BID (Days 1 and 2, then BIM23B065 0.6 mg BID (Days 3 and 4) and BIM23B065 0.8 mg BID (Days 5 and 6). Treatment phase (Days 7 to 14): Following titration, a stable target dose of 1.0 mg BID BIM23B065 was planned to be administered from Day 7 to Day 14.
Overall Study
Pre-treatment adverse event
1

Baseline Characteristics

Assessment of BIM23B065, Given as Repeated Subcutaneous Injection in Subjects With Acromegaly

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
BIM23B065 BID Stage 1
n=4 Participants
Subjects received twice daily administrations of BIM23B065 (at an interval of 12 +/- 1 hour) as subcutaneous injections in the abdominal region during the Stage 1 treatment period which consisted of a titration phase and a treatment phase. Titration phase (Days 1 to 6): Subjects received BIM23B065 0.4 milligram (mg) BID (Days 1 and 2, then BIM23B065 0.6 mg BID (Days 3 and 4) and BIM23B065 0.8 mg BID (Days 5 and 6). Treatment phase (Days 7 to 14): Following titration, a stable target dose of 1.0 mg BID BIM23B065 was planned to be administered from Day 7 to Day 14.
Age, Continuous
53.0 Years
STANDARD_DEVIATION 15.12 • n=99 Participants
Sex: Female, Male
Female
4 Participants
n=99 Participants
Sex: Female, Male
Male
0 Participants
n=99 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=99 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
4 Participants
n=99 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=99 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=99 Participants
Race (NIH/OMB)
Asian
0 Participants
n=99 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=99 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=99 Participants
Race (NIH/OMB)
White
4 Participants
n=99 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=99 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=99 Participants

PRIMARY outcome

Timeframe: From baseline (Day -1) to Day 14.

Population: The Efficacy Evaluable population consisted of all subjects with at least 1 BIM23B065 administration with available pharmacodynamic (PD) data and no protocol deviations with relevant impact on PD data, who had an evaluable primary efficacy endpoint (mean concentration of GH over 6 hours) at baseline and at Day 14.

A GH responder was defined as a subject with mean serum GH concentration ≤2.5 micrograms per litre (mcg/L) or \>50% reduction from mean baseline GH concentration after a 6-day titration and an 8-day treatment period with BIM23B065. The mean serum concentration of GH was measured over 6 hours at baseline (Day -1) and on Day 14. The number of subjects who were GH responders at Day 14 of the treatment period is presented.

Outcome measures

Outcome measures
Measure
BIM23B065 BID Stage 1
n=3 Participants
Subjects received twice daily administrations of BIM23B065 (at an interval of 12 +/- 1 hour) as subcutaneous injections in the abdominal region during the Stage 1 treatment period which consisted of a titration phase and a treatment phase. Titration phase (Days 1 to 6): Subjects received BIM23B065 0.4 milligram (mg) BID (Days 1 and 2, then BIM23B065 0.6 mg BID (Days 3 and 4) and BIM23B065 0.8 mg BID (Days 5 and 6). Treatment phase (Days 7 to 14): Following titration, a stable target dose of 1.0 mg BID BIM23B065 was planned to be administered from Day 7 to Day 14.
The Number of Subjects Who Were Growth Hormone (GH) Responders at Day 14 of the Treatment Period
1 Participants

Adverse Events

BIM23B065 BID Stage 1

Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
BIM23B065 BID Stage 1
n=4 participants at risk
Subjects received twice daily administrations of BIM23B065 (at an interval of 12 +/- 1 hour) as subcutaneous injections in the abdominal region during the Stage 1 treatment period which consisted of a titration phase and a treatment phase. Titration phase (Days 1 to 6): Subjects received BIM23B065 0.4 milligram (mg) BID (Days 1 and 2, then BIM23B065 0.6 mg BID (Days 3 and 4) and BIM23B065 0.8 mg BID (Days 5 and 6). Treatment phase (Days 7 to 14): Following titration, a stable target dose of 1.0 mg BID BIM23B065 was planned to be administered from Day 7 to Day 14.
Vascular disorders
Hypotension
25.0%
1/4 • Number of events 1 • Treatment emergent adverse events were collected from Day 1 of the treatment period up to the end of Week 4 (approximately 28 days).
The Safety population consisted of all subjects with at least 1 BIM23B065 administration.
Blood and lymphatic system disorders
Anaemia
25.0%
1/4 • Number of events 1 • Treatment emergent adverse events were collected from Day 1 of the treatment period up to the end of Week 4 (approximately 28 days).
The Safety population consisted of all subjects with at least 1 BIM23B065 administration.
Cardiac disorders
Bradycardia
25.0%
1/4 • Number of events 2 • Treatment emergent adverse events were collected from Day 1 of the treatment period up to the end of Week 4 (approximately 28 days).
The Safety population consisted of all subjects with at least 1 BIM23B065 administration.
Gastrointestinal disorders
Abdominal pain upper
25.0%
1/4 • Number of events 1 • Treatment emergent adverse events were collected from Day 1 of the treatment period up to the end of Week 4 (approximately 28 days).
The Safety population consisted of all subjects with at least 1 BIM23B065 administration.
Gastrointestinal disorders
Nausea
25.0%
1/4 • Number of events 1 • Treatment emergent adverse events were collected from Day 1 of the treatment period up to the end of Week 4 (approximately 28 days).
The Safety population consisted of all subjects with at least 1 BIM23B065 administration.
Hepatobiliary disorders
Cholelithiasis
25.0%
1/4 • Number of events 1 • Treatment emergent adverse events were collected from Day 1 of the treatment period up to the end of Week 4 (approximately 28 days).
The Safety population consisted of all subjects with at least 1 BIM23B065 administration.

Additional Information

Medical Director

Ipsen

Phone: Use email address below

Results disclosure agreements

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

Restriction type: GT60