Trial Outcomes & Findings for Study to Evaluate the Safety and Efficacy of Relamorelin in Participants With Diabetic Gastroparesis Study 02 (NCT NCT03426345)

NCT ID: NCT03426345

Last Updated: 2021-08-06

Results Overview

Participants assessed the severity of diabetic gastroparesis symptoms daily using the Diabetic Gastroparesis Symptom Severity Diary (DGSSD), recorded in an electronic diary (e-diary). The DGSSS was derived as the sum of the weekly averages of the 4 DGSSD items: nausea, abdominal pain, postprandial fullness and bloating. Each symptom was scored using an 11-point ordinal scale where: 0= no or not at all uncomfortable to 10= worst possible or most uncomfortable for a total possible DGSSS of 0 (best) to 40 (worst). A negative change from Baseline indicates improvement. Baseline was defined as the average of the 2 weekly DGSSS from the run-in period.

• Recruitment status: TERMINATED
• Study phase: PHASE3
• Target enrollment: 311 participants
• Primary outcome timeframe: Baseline (Day-14 to Day-1) to Week 12
• Results posted on: 2021-08-06

Participant Flow

Participant milestones

Measure	Placebo Following a 2-week placebo run-in, participants received placebo-matching relamorelin injected subcutaneously twice daily for up to 12 weeks.	Relamorelin 10 μg Following a 2-week placebo run-in, participants received relamorelin 10 micrograms (μg) injected subcutaneously twice daily for up to 12 weeks.
Overall Study STARTED	155	156
Overall Study Safety Population	152	155
Overall Study COMPLETED	137	139
Overall Study NOT COMPLETED	18	17

Reasons for withdrawal

Measure	Placebo Following a 2-week placebo run-in, participants received placebo-matching relamorelin injected subcutaneously twice daily for up to 12 weeks.	Relamorelin 10 μg Following a 2-week placebo run-in, participants received relamorelin 10 micrograms (μg) injected subcutaneously twice daily for up to 12 weeks.
Overall Study Adverse Event	4	4
Overall Study Lack of Efficacy	1	0
Overall Study Withdrawal by Subject	5	6
Overall Study Lost to Follow-up	3	1
Overall Study Protocol Deviation	3	4
Overall Study Reason Not Specified	1	2
Overall Study Missing Completion Status	1	0

Baseline Characteristics

Study to Evaluate the Safety and Efficacy of Relamorelin in Participants With Diabetic Gastroparesis Study 02

Baseline characteristics by cohort

Measure	Placebo n=155 Participants Following a 2-week placebo run-in, participants received placebo-matching relamorelin injected subcutaneously twice daily for up to 12 weeks.	Relamorelin 10 μg n=156 Participants Following a 2-week placebo run-in, participants received relamorelin 10 μg injected subcutaneously twice daily for up to 12 weeks.	Total n=311 Participants Total of all reporting groups
Age, Continuous	54.1 years STANDARD_DEVIATION 12.13 • n=5 Participants	55.8 years STANDARD_DEVIATION 12.07 • n=7 Participants	55.0 years STANDARD_DEVIATION 12.11 • n=5 Participants
Sex: Female, Male Female	112 Participants n=5 Participants	114 Participants n=7 Participants	226 Participants n=5 Participants
Sex: Female, Male Male	43 Participants n=5 Participants	42 Participants n=7 Participants	85 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	54 Participants n=5 Participants	51 Participants n=7 Participants	105 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	101 Participants n=5 Participants	105 Participants n=7 Participants	206 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	4 Participants n=5 Participants	7 Participants n=7 Participants	11 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	25 Participants n=5 Participants	17 Participants n=7 Participants	42 Participants n=5 Participants
Race (NIH/OMB) White	123 Participants n=5 Participants	127 Participants n=7 Participants	250 Participants n=5 Participants
Race (NIH/OMB) More than one race	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline (Day-14 to Day-1) to Week 12

Population: Modified Intent-to-treat (mITT) Population included all randomized participants with ≥1 postbaseline assessment of DGSSD.

Participants assessed the severity of diabetic gastroparesis symptoms daily using the Diabetic Gastroparesis Symptom Severity Diary (DGSSD), recorded in an electronic diary (e-diary). The DGSSS was derived as the sum of the weekly averages of the 4 DGSSD items: nausea, abdominal pain, postprandial fullness and bloating. Each symptom was scored using an 11-point ordinal scale where: 0= no or not at all uncomfortable to 10= worst possible or most uncomfortable for a total possible DGSSS of 0 (best) to 40 (worst). A negative change from Baseline indicates improvement. Baseline was defined as the average of the 2 weekly DGSSS from the run-in period.

Outcome measures

Measure	Placebo n=152 Participants Following a 2-week placebo run-in, participants received placebo-matching relamorelin injected subcutaneously twice daily for up to 12 weeks.	Relamorelin 10 μg n=155 Participants Following a 2-week placebo run-in, participants received relamorelin 10 μg injected subcutaneously twice daily for up to 12 weeks.
Change From Baseline to Week 12 in the Weekly Diabetic Gastroparesis Symptom Severity Score (DGSSS) Baseline	23.4 score on a scale Standard Deviation 5.40	24.9 score on a scale Standard Deviation 6.15
Change From Baseline to Week 12 in the Weekly Diabetic Gastroparesis Symptom Severity Score (DGSSS) Change from Baseline to Week 12	-9.3 score on a scale Standard Deviation 10.21	-10.2 score on a scale Standard Deviation 9.24

PRIMARY outcome

Timeframe: Week 6 to Week 12

Population: mITT Population included all randomized participants with ≥1 postbaseline assessment of DGSSD.

The number of vomiting episodes in the previous 24 hours were assessed daily by the participant using the DGSSD and were recorded in the e-diary. A Vomiting Responder was defined as a participant with zero weekly vomiting episodes during each of the last 6 weeks of the 12-week Treatment Period.

Outcome measures

Measure	Placebo n=152 Participants Following a 2-week placebo run-in, participants received placebo-matching relamorelin injected subcutaneously twice daily for up to 12 weeks.	Relamorelin 10 μg n=155 Participants Following a 2-week placebo run-in, participants received relamorelin 10 μg injected subcutaneously twice daily for up to 12 weeks.
Percentage of Participants Meeting the Vomiting Responder Criterion During Each of the Last 6 Weeks of the 12-week Treatment Period	19.1 percentage of participants	18.7 percentage of participants

SECONDARY outcome

Timeframe: Baseline (Day-14 to Day-1) to (Week 6 to Week 12)

Population: mITT Population included all randomized participants with ≥1 postbaseline assessment of DGSSD.

A Nausea Responder was defined as a participant with improvement (decrease) of at least 2-points in the weekly symptom scores for nausea at each of the last 6 weeks of the 12-week Treatment Period. Nausea was one of the items of the DGSSD assessed daily and recorded in the e-diary by the participant using an 11-point ordinal scale where: 0= no nausea to 10= worst possible nausea.

Outcome measures

Measure	Placebo n=152 Participants Following a 2-week placebo run-in, participants received placebo-matching relamorelin injected subcutaneously twice daily for up to 12 weeks.	Relamorelin 10 μg n=155 Participants Following a 2-week placebo run-in, participants received relamorelin 10 μg injected subcutaneously twice daily for up to 12 weeks.
Percentage of Participants Meeting the Nausea Responder Criterion During Each of the Last 6 Weeks of the 12-week Treatment Period	32.9 percentage of participants	38.1 percentage of participants

SECONDARY outcome

Timeframe: Baseline (Day-14 to Day-1) to (Week 6 to Week 12)

Population: mITT Population included all randomized participants with ≥1 postbaseline assessment of DGSSD.

An Abdominal Pain Responder was defined as a participant with an improvement (decrease) of at least 2-points in the weekly symptom scores for abdominal pain at each of the last 6 weeks of the 12-week Treatment Period. Abdominal pain was one of the items of the DGSSD assessed daily and recorded in the e-diary by the participant using an 11-point ordinal scale where: 0= no abdominal pain to 10= the worst possible abdominal pain and was recorded in an e-diary.

Outcome measures

Measure	Placebo n=152 Participants Following a 2-week placebo run-in, participants received placebo-matching relamorelin injected subcutaneously twice daily for up to 12 weeks.	Relamorelin 10 μg n=155 Participants Following a 2-week placebo run-in, participants received relamorelin 10 μg injected subcutaneously twice daily for up to 12 weeks.
Percentage of Participants Meeting the Abdominal Pain Responder Criterion During Each of the Last 6 Weeks of the 12-week Treatment Period	27.0 percentage of participants	36.1 percentage of participants

SECONDARY outcome

Timeframe: Baseline (Day-14 to Day-1) to (Week 6 to Week 12)

Population: mITT Population included all randomized participants with ≥1 postbaseline assessment of DGSSD.

A Bloating Responder was defined as a participant with an improvement (decrease) of at least 2-points in the weekly symptom scores for bloating at each of the last 6 weeks of the 12-week Treatment Period. Bloating was one of the items of the DGSSD assessed daily and recorded by the participant in the e-diary using an 11-point ordinal scale where: 0= no bloating and 10= the worst possible bloating and was recorded in the e-diary.

Outcome measures

Measure	Placebo n=152 Participants Following a 2-week placebo run-in, participants received placebo-matching relamorelin injected subcutaneously twice daily for up to 12 weeks.	Relamorelin 10 μg n=155 Participants Following a 2-week placebo run-in, participants received relamorelin 10 μg injected subcutaneously twice daily for up to 12 weeks.
Percentage of Participants Meeting the Bloating Responder Criterion During Each of the Last 6 Weeks of the 12-week Treatment Period	27.0 percentage of participants	31.6 percentage of participants

SECONDARY outcome

Timeframe: Baseline (Day-14 to Day-1) to (Week 6 to Week 12)

Population: mITT Population included all randomized participants with ≥1 postbaseline assessment of DGSSD.

A Postprandial Fullness Responder was defined as a participant with an improvement (decrease) of at least 2-points in the weekly symptom scores for Postprandial Fullness at each of the last 6 weeks of the 12-week Treatment Period. Postprandial Fullness was one of the items of the DGSSD assessed daily and recorded by the participant in the e-diary using an 11-point ordinal scale where: 0= no feeling of fullness until finishing a meal (best) to 10= feeling full after only a few bites (worst).

Outcome measures

Measure	Placebo n=152 Participants Following a 2-week placebo run-in, participants received placebo-matching relamorelin injected subcutaneously twice daily for up to 12 weeks.	Relamorelin 10 μg n=155 Participants Following a 2-week placebo run-in, participants received relamorelin 10 μg injected subcutaneously twice daily for up to 12 weeks.
Percentage of Participants Meeting the Postprandial Fullness Responder Criterion During Each of the Last 6 Weeks of the 12-week Treatment Period	23.7 percentage of participants	27.7 percentage of participants

SECONDARY outcome

Timeframe: Up to approximately 16 weeks

Population: Safety Population included all participants who received ≥1 administration of double-blind study treatment.

An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE is an AE that begins or worsens after receiving study drug.

Outcome measures

Measure	Placebo n=152 Participants Following a 2-week placebo run-in, participants received placebo-matching relamorelin injected subcutaneously twice daily for up to 12 weeks.	Relamorelin 10 μg n=155 Participants Following a 2-week placebo run-in, participants received relamorelin 10 μg injected subcutaneously twice daily for up to 12 weeks.
Number of Participants Who Experienced One or More Treatment-Emergent Adverse Events (TEAE)	75 Participants	86 Participants

SECONDARY outcome

Timeframe: Up to 12 weeks

Population: Safety Population included all participants who received ≥1 administration of double-blind study treatment. Number analyzed is the number of participants with non-PCS baseline values and at least one post-baseline assessment.

Clinical Laboratory tests included Hematology, Chemistry and Urinalysis tests. The investigator determined if the results were clinically significant. Only those categories where at least 1 person had a non-PCS value at Baseline and met the PCS criterion at least once during postbaseline are reported.

Outcome measures

Measure	Placebo n=152 Participants Following a 2-week placebo run-in, participants received placebo-matching relamorelin injected subcutaneously twice daily for up to 12 weeks.	Relamorelin 10 μg n=155 Participants Following a 2-week placebo run-in, participants received relamorelin 10 μg injected subcutaneously twice daily for up to 12 weeks.
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Eosinophils Absolute Cell Count [10^9/liter (L)]: >3×Upper Limit of Normal Value (ULN)	0 Participants	1 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Hematocrit (RATIO): >1.1×ULN	1 Participants	0 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Hematocrit (RATIO): <0.9×Lower Limit of Normal Value (LLN)	4 Participants	4 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Hemoglobin [grams (g)/L]: <0.9×LLN	2 Participants	5 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Lymphocytes Absolute Cell Count (10^9/L): <0.8×LLN	1 Participants	3 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Mean Corpuscular Volume [femtoliter(fL)]: >1.1×ULN	1 Participants	1 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Neutrophils Absolute Cell Count (10^9/L): >1.5×ULN	0 Participants	1 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Neutrophils Absolute Cell Count (10^9/L): <0.8×LLN	2 Participants	2 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Red Blood Cell Count (10^12/L): <0.9×LLN	1 Participants	1 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Alanine Aminotransferase [Serum Glutamic Pyruvic Transaminase (SGPT)] [unit(U)/L]: >=3×ULN	1 Participants	0 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Albumin (g/L): <0.9×LLN	0 Participants	1 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Alkaline Phosphatase (U/L): >=3×ULN	1 Participants	0 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Aspartate Aminotransferase [Serum Glutamic Oxaloacetic Transaminase (SGOT)] (U/L): >=3×ULN	1 Participants	2 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Bicarbonate (HCO3) [millimoles (mmol)/L]: >1.1×ULN	0 Participants	1 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Bicarbonate (HCO3) (mmol/L): <0.9×LLN	1 Participants	0 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Blood Urea Nitrogen (mmol/L): >1.2×ULN	15 Participants	3 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Chloride (mmol/L): <0.9×LLN	2 Participants	0 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Cholesterol, Total, Fasting (mmol/L): >1.6×ULN	2 Participants	1 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Creatinine [micromoles(μmol)/L]: >1.3×ULN	8 Participants	7 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Glucose-Chemistry, Fasting (mmol/L): >2.5×ULN	11 Participants	21 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Glucose-Chemistry, Fasting (mmol/L): <0.9×LLN	6 Participants	5 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Glycohemoglobin A1C: Increase of >=0.5%	80 Participants	111 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Glycohemoglobin A1C: Increase of >=1%	80 Participants	111 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Phosphorus (mmol/L): >1.1×ULN	5 Participants	5 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Phosphorus (mmol/L): <0.9×LLN	1 Participants	1 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Potassium (mmol/L): <0.9×LLN	1 Participants	0 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Sodium (mmol/L): <0.9×LLN	1 Participants	0 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Triglycerides, Fasting (mmol/L): >=3×ULN	3 Participants	5 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Uric Acid (Urate) (μmol/L): >1.1×ULN	18 Participants	13 Participants
Number of Participants With Potential Clinically Significant (PCS) Clinical Laboratory Results Uric Acid (Urate) (μmol/L): <0.9×LLN	3 Participants	0 Participants

SECONDARY outcome

Timeframe: Up to 12 weeks

Population: Safety Population included all participants who received ≥1 administration of double-blind study treatment.

Vital Signs included assessments of heart rate, respiratory rate, systolic and diastolic blood pressure, and body temperature. The investigator determined if the abnormal results were clinically significant.

Outcome measures

Measure	Placebo n=152 Participants Following a 2-week placebo run-in, participants received placebo-matching relamorelin injected subcutaneously twice daily for up to 12 weeks.	Relamorelin 10 μg n=155 Participants Following a 2-week placebo run-in, participants received relamorelin 10 μg injected subcutaneously twice daily for up to 12 weeks.
Number of Participants With Clinically Meaningful Trends for Vital Signs	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Up to 12 weeks

Population: Safety Population included all participants who received ≥1 administration of double-blind study treatment.

A standard 12-lead ECG was performed. The investigator determined if the abnormal results were clinically significant.

Outcome measures

Measure	Placebo n=152 Participants Following a 2-week placebo run-in, participants received placebo-matching relamorelin injected subcutaneously twice daily for up to 12 weeks.	Relamorelin 10 μg n=155 Participants Following a 2-week placebo run-in, participants received relamorelin 10 μg injected subcutaneously twice daily for up to 12 weeks.
Number of Participants With Clinically Significant Abnormal Electrocardiogram (ECG) Results	1 Participants	2 Participants

SECONDARY outcome

Timeframe: Baseline (Day 1) up to 12 weeks

Population: Safety Population included all participants who received ≥1 administration of double-blind study treatment. Overall number analyzed is the number of participants with non-PCS baseline values and at least one post-baseline assessment.

Outcome measures

Measure	Placebo n=146 Participants Following a 2-week placebo run-in, participants received placebo-matching relamorelin injected subcutaneously twice daily for up to 12 weeks.	Relamorelin 10 μg n=151 Participants Following a 2-week placebo run-in, participants received relamorelin 10 μg injected subcutaneously twice daily for up to 12 weeks.
Number of Participants With a ≥1% Increase in Glycosylated Hemoglobin A1c (HBA1c)	80 Participants	111 Participants

SECONDARY outcome

Timeframe: Baseline (Day 1), Day 14, Day 28, Day 84, and End of Treatment (Up to Day 84)

Population: Safety Population included all participants who received ≥1 administration of double-blind study treatment (N=155 in the Relamorelin 10 μg arm). Anti-relamorelin antibody testing was only done for those participants who received treatment with relamorelin. Number analyzed is the number of participants with data available at the given timepoint. Due to a laboratory issue not all positive screening tests were confirmed.

A blood sample was collected that was sent to a laboratory for an anti-relamorelin antibody screening test. A positive screening test was confirmed by an immunodepletion assay. The number of participants in each of the following categories are reported: Negative Screening Test, Positive Screening Test, Negative Confirmatory Test, and Positive Confirmatory Test at each time point.

Outcome measures

Measure	Placebo n=155 Participants Following a 2-week placebo run-in, participants received placebo-matching relamorelin injected subcutaneously twice daily for up to 12 weeks.	Relamorelin 10 μg Following a 2-week placebo run-in, participants received relamorelin 10 μg injected subcutaneously twice daily for up to 12 weeks.
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Screening Test (Baseline) · Negative	127 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Screening Test (Baseline) · Positive	18 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Confirmatory Test (Baseline) · Negative	12 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Confirmatory Test (Baseline) · Positive	0 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Screening Test (Day 14) · Negative	115 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Screening Test (Day 14) · Positive	18 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Confirmatory Test (Day 14) · Negative	13 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Confirmatory Test (Day 14) · Positive	0 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Screening Test (Day 28) · Negative	104 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Screening Test (Day 28) · Positive	17 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Confirmatory Test (Day 28) · Negative	12 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Confirmatory Test (Day 28) · Positive	0 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Screening Test (Day 84) · Negative	87 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Screening Test (Day 84) · Positive	17 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Confirmatory Test (Day 84) · Negative	11 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Confirmatory Test (Day 84) · Positive	1 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Screening Test (End of Treatment) · Negative	6 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Screening Test (End of Treatment) · Positive	1 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Confirmatory Test (End of Treatment) · Negative	1 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Confirmatory Test (End of Treatment) · Positive	0 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Screening Test (Unscheduled) · Negative	6 Participants	—
Number of Participants With Anti-relamorelin Antibody Testing Results by Visit Screening Test (Unscheduled) · Positive	0 Participants	—

Adverse Events

Placebo

Serious events: 6 serious events

Other events: 14 other events

Deaths: 0 deaths

Relamorelin 10 μg

Serious events: 8 serious events

Other events: 13 other events

Deaths: 0 deaths

Serious adverse events

Measure	Placebo n=152 participants at risk Following a 2-week placebo run-in, participants received placebo-matching relamorelin injected subcutaneously twice daily for up to 12 weeks.	Relamorelin 10 μg n=155 participants at risk Following a 2-week placebo run-in, participants received relamorelin 10 μg injected subcutaneously twice daily for up to 12 weeks.
Cardiac disorders Cardiac failure congestive	0.66% 1/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.65% 1/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Gastrointestinal disorders Abdominal pain	0.00% 0/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.65% 1/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Gastrointestinal disorders Diarrhoea	0.00% 0/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.65% 1/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Gastrointestinal disorders Nausea	0.00% 0/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.65% 1/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Gastrointestinal disorders Small intestinal obstruction	0.00% 0/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.65% 1/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Gastrointestinal disorders Vomiting	0.00% 0/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.65% 1/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Infections and infestations COVID-19	0.00% 0/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.65% 1/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Infections and infestations Pneumonia	0.00% 0/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.65% 1/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Infections and infestations Urinary tract infection	0.00% 0/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.65% 1/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Infections and infestations Influenza	0.66% 1/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.00% 0/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Infections and infestations Sepsis	0.66% 1/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.00% 0/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Infections and infestations Upper respiratory tract infection	0.66% 1/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.00% 0/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Metabolism and nutrition disorders Dehydration	0.66% 1/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.65% 1/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Metabolism and nutrition disorders Diabetic ketoacidosis	0.66% 1/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.65% 1/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Metabolism and nutrition disorders Malnutrition	0.66% 1/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.00% 0/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Musculoskeletal and connective tissue disorders Muscular weakness	0.66% 1/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.00% 0/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Nervous system disorders Headache	0.66% 1/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.00% 0/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Renal and urinary disorders Acute kidney injury	0.00% 0/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.65% 1/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Renal and urinary disorders Renal failure	0.66% 1/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.00% 0/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Reproductive system and breast disorders Vaginal haemorrhage	0.00% 0/109 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.88% 1/113 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Respiratory, thoracic and mediastinal disorders Asthma	0.66% 1/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	0.00% 0/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.

Other adverse events

Measure	Placebo n=152 participants at risk Following a 2-week placebo run-in, participants received placebo-matching relamorelin injected subcutaneously twice daily for up to 12 weeks.	Relamorelin 10 μg n=155 participants at risk Following a 2-week placebo run-in, participants received relamorelin 10 μg injected subcutaneously twice daily for up to 12 weeks.
Gastrointestinal disorders Diarrhoea	5.9% 9/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	3.2% 5/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.
Infections and infestations Urinary tract infection	3.3% 5/152 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.	5.8% 9/155 • Up to approximately 16 weeks All-Cause Mortality is reported for all randomized participants. The Safety Population, all participants who received ≥1 administration of double-blind study treatment, was used to determine the number of participants at risk for Serious Adverse Events and Other Adverse Events.

Additional Information

Therapeutic Area, Head

Allergan

Phone: 714-246-4500

Email: clinicaltrials@allergan.com

Results disclosure agreements

• Principal investigator is a sponsor employee A disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 90 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
• Publication restrictions are in place

Restriction type: OTHER