A Trial of Setmelanotide in Acquired Hypothalamic Obesity | NCT05774756

NCT05774756

Status Module

Sponsor/Collaborators Module

Oversight Module

Description Module

Conditions Module

Design Module

Interventional Study Design

Biospecimen

No data available

No data is available for this block.

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Setemelanotide	Experimental	Randomized 2:1 (Setmelanotide: Placebo)	Drug: Setmelanotide
Placebo	Placebo Comparator	Randomized 2:1 (Setmelanotide: Placebo)	Drug: Placebo

Name	Type	Description	Arm Group Labels	Other Names
Setmelanotide	Drug	Solution for daily subcutaneous injection	Setemelanotide

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Pivotal Cohort: Mean Percent Change From Baseline in Body Mass Index (BMI) After 52 Weeks on a Therapeutic Regimen	BMI was calculated as weight (kg)/height (m^2). Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.	Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)

Secondary Outcomes

Measure	Description	Time Frame
Pivotal Cohort: Percentage of Participants With ≥5% Reduction From Baseline in BMI (≥18 Years of Age) or ≥0.2-point Reduction From Baseline in BMI Z-score (<18 Years of Age)	BMI was calculated as weight (kg)/height (m^2). BMI Z-Score calculated for participants <18 years old only is a measure of relative weight adjusted for child's age, sex and height at the time of data collection. The Z-Scores were calculated using the World Health Organization's WHO 2007 BMI SAS Macro Package. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). A decrease in BMI Z-score (< 0) indicates a reduction in BMI from Baseline whereas an increase in BMI-Z score (> 0) indicated an increase in BMI from Baseline. Baseline was defined as the most recent measurement prior to the first administration of study drug. Results below represent the percentage of estimated participants with either ≥5% BMI reduction or ≥0.2 point reduction in BMI Z-score by age in each treatment arm as calculated through the MIANALYZE SAS procedure.

Eligibility Module

Contacts/Locations Module

Name	Affiliation	Role
David Meeker, MD	Rhythm Pharmaceuticals, Inc.	Study Chair

Facility	Status	City	State	ZIP	Country	Contacts
UAN Pediatric Endocrinology		Birmingham	Alabama	35233	United States
Rady Children's Hospital

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
42418774	Derived	Miller JL, van Santen HM, Phillips SA, Hamilton J, Aberle J, Sathyapalan T, Mohamed Z, McCormack SE, Shoemaker AH, Kelsey MM, Ghalib L, Stalla G, Thaker VV, Habiby R, Ode KL, Wabitsch M, Dattani M, Abuzzahab MJ, Abdullatif H, Morgan R, Stefater-Richards M, Aroda VR, Friedrich C, Han JC, Ono H, Nagasaki K, Tanaka T, Isojima T, Arima H, Scimia C, Yuan G, Muller HL, Roth CL; TRANSCEND Trial Group. Setmelanotide for the Treatment of Acquired Hypothalamic Obesity. N Engl J Med. 2026 Jul 9;395(2):138-150. doi: 10.1056/NEJMoa2512275.

IPD Sharing Statement Module

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	Setmelanotide	Participants received setmelanotide once daily (QD) as a subcutaneous (SC) injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
FG001	Placebo

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Annotation Section

No data available

No data is available for this block.

Document Section

Large Document Module

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Dec 19, 2024	Mar 17, 2026

Derived Section

Miscellaneous Info Module

Submission Tracking

Condition Browse Module

Intervention Browse Module

Masking Info

Participants received placebo matched to setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).

Modified intent to treat (mITT) analysis set included all randomized participants who were exposed to at least 1 dose of study treatment.

Arm/Group Information

ID	Title	Description
BG000	Setmelanotide	Participants received setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
BG001	Placebo	Participants received placebo matched to setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
BG002	Total	Total of all reporting groups

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Continuous

Mean

Standard Deviation

years

Title

Denominators

Categories

Title	Measurements
	OG000-16.51± 1.401 OG0013.32± 1.984

Group IDs	Group Description	Statistical Method	Statistical Comment	P-Value	P-Value Comment	Parameter Type	Parameter Value	Dispersion Type	Dispersion Value	Confidence Interval Sides	Confidence Interval %	CI Lower Limit	CI Upper Limit	CI Lower Limit Comment	CI Upper Limit Comment	Estimate Comment	Tested Non-Inferiority	Non-Inferiority Type	Non-Inferiority Comment	Other Analysis Description
OG000 OG001		ANCOVA	The model was adjusted with the stratification factors of age group and subpopulation.	<0.0001		Least square mean difference	-19.83	Standard Error of the Mean	2.411	2-Sided	95	-24.55	-15.10					Other

Secondary

Pivotal Cohort: Percentage of Participants With ≥5% Reduction From Baseline in BMI (≥18 Years of Age) or ≥0.2-point Reduction From Baseline in BMI Z-score (<18 Years of Age)

BMI was calculated as weight (kg)/height (m^2). BMI Z-Score calculated for participants <18 years old only is a measure of relative weight adjusted for child's age, sex and height at the time of data collection. The Z-Scores were calculated using the World Health Organization's WHO 2007 BMI SAS Macro Package. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). A decrease in BMI Z-score (< 0) indicates a reduction in BMI from Baseline whereas an increase in BMI-Z score (> 0) indicated an increase in BMI from Baseline. Baseline was defined as the most recent measurement prior to the first administration of study drug. Results below represent the percentage of estimated participants with either ≥5% BMI reduction or ≥0.2 point reduction in BMI Z-score by age in each treatment arm as calculated through the MIANALYZE SAS procedure.

mITT analysis set included all randomized participants who were exposed to at least 1 dose of study treatment. Participants in pivotal cohort were included in the analysis.

Posted

Number

95% Confidence Interval

Calculated estimate: % of participants

After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)

ID	Title	Description
OG000	Setmelanotide	Participants received setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
OG001

Secondary

Pivotal Cohort: Percentage of Participants With ≥5% Reduction From Baseline in BMI

BMI was calculated as weight (kg)/height (m^2). Results below represent the percentage of estimated participants with ≥5% BMI reduction in each treatment arm as calculated through the MIANALYZE SAS procedure.

mITT analysis set included all randomized participants who were exposed to at least 1 dose of study treatment. Participants in pivotal cohort were included in the analysis.

Posted

Number

95% Confidence Interval

Calculated estimate: % of participants

After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)

ID	Title	Description
OG000	Setmelanotide	Participants received setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
OG001	Placebo	Participants received placebo matched to setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).

Units	Counts
Participants

Secondary

Pivotal Cohort: Change From Baseline in Weekly Average Daily Most Hunger Score in Participants ≥12 Years of Age

Change from baseline in hunger scores for participants ≥12 years of age with acquired hypothalamic obesity was evaluated. Hunger score ranged from 0= "not hungry at all" to 10= "hungriest possible" on an 11-point numeric rating scale. On Daily Hunger Questionnaire, each of the 2 items (average hunger and most hunger) was scored separately and averaged on weekly basis. LSM and SE were calculated using ANCOVA model.

mITT analysis set included all randomized participants who were exposed to at least 1 dose of study treatment. Participants in pivotal cohort who were evaluable for this outcome measure were included in the analysis.

Posted

Least Squares Mean

Standard Error

Score on a scale

Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)

ID	Title	Description
OG000	Setmelanotide	Participants received setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
OG001	Placebo	Participants received placebo matched to setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).

Secondary

Pivotal Cohort: Percentage of Participants (≥12 Years of Age) With a ≥2-point Reduction From Baseline in the Weekly Average Daily Most Hunger Score

Hunger score ranged from 0= "not hungry at all" to 10= "hungriest possible" on an 11-point numeric rating scale. On Daily Hunger Questionnaire, each of the 2 items (average hunger and most hunger) was scored separately and averaged on weekly basis. Results below represent the percentage of estimated participants ≥12 years of age with ≥2-point reduction in each treatment arm as calculated through the MIANALYZE SAS procedure.

Posted

Number

95% Confidence Interval

Calculated estimate: % of participants

After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)

ID	Title	Description
OG000	Setmelanotide	Participants received setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
OG001	Placebo	Participants received placebo matched to setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).

Secondary

Pivotal Cohort: Mean Change From Baseline in the Weekly Average of the Symptoms of Hyperphagia Composite Score in Participants ≥12 Years of Age

Participants ≥12 years of age who were able to self-report were administered the Symptoms of Hyperphagia: Patient (Version 1.0). The questionnaire consisted of 4 items related to the frequency of a participant's hunger symptoms on a scale (Never, 1 or 2 times, 3 or more times) over the past 24 hours. The scores on this scale range from 0 to 2 with higher scores indicating more hyperphagia. Daily composite score was derived as the sum of daily answered questions divided by the number of answered questions per day. The weekly average of the daily composite scores equals to the sum of daily composite scores divided by the number of days with a composite score within the 7 identified days prior to the visit. LSM and SE were calculated using ANCOVA model.

Posted

Least Squares Mean

Standard Error

Score on a scale

Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)

ID	Title	Description
OG000	Setmelanotide	Participants received setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
OG001	Placebo

Secondary

Pivotal Cohort: Percentage of Participants With a ≥10% Reduction From Baseline in BMI

BMI was calculated as weight (kg)/height (m^2). Results below represent the percentage of estimated participants with ≥10% BMI reduction in each treatment arm as calculated through the MIANALYZE SAS procedure.

mITT analysis set included all randomized participants who were exposed to at least 1 dose of study treatment. Participants in pivotal cohort were included in the analysis.

Posted

Number

95% Confidence Interval

Calculated estimate: % of participants

After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)

ID	Title	Description
OG000	Setmelanotide	Participants received setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
OG001	Placebo	Participants received placebo matched to setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).

Units	Counts
Participants

Secondary

Pivotal Cohort: Percentage of Participants With a ≥10% Reduction From Baseline in Body Weight

Body weight was captured for analysis in kilograms. Results below represent the percentage of estimated participants with ≥10% reduction in body weight in each treatment arm as calculated through the MIANALYZE SAS procedure.

mITT analysis set included all randomized participants who were exposed to at least 1 dose of study treatment. Participants in pivotal cohort were included in the analysis.

Posted

Number

95% Confidence Interval

Calculated estimate: % of participants

After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)

ID	Title	Description
OG000	Setmelanotide	Participants received setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
OG001	Placebo	Participants received placebo matched to setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).

Units	Counts
Participants

Secondary

Pivotal Cohort: Mean Percent Change From Baseline in Body Weight in Participants ≥18 Years

Body weight was captured for analysis in kilograms. LSM and SE were calculated using ANCOVA model.

Posted

Least Squares Mean

Standard Error

Percent change

Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)

ID	Title	Description
OG000	Setmelanotide	Participants received setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
OG001	Placebo	Participants received placebo matched to setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).

Units	Counts
Participants

Secondary

Pivotal Cohort: Mean Change From Baseline in BMI Z-Score in Participants <18 Years of Age

BMI was calculated as weight (kg)/height (m^2). BMI Z-Score calculated for participants <18 years old only is a measure of relative weight adjusted for child's age, sex and height at the time of data collection. The Z-Scores were calculated using the World Health Organization's WHO 2007 BMI SAS Macro Package. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). A decrease in BMI Z-score (< 0) indicates a reduction in BMI from Baseline whereas an increase in BMI-Z score (> 0) indicates an increase in BMI from Baseline. Baseline was defined as the most recent measurement prior to the first administration of study drug. LSM and SE were calculated using ANCOVA model.

Posted

Least Squares Mean

Standard Error

Z-Score

Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)

ID	Title	Description
OG000	Setmelanotide	Participants received setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
OG001	Placebo	Participants received placebo matched to setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).

Secondary

Pivotal Cohort: Mean Change From Baseline in Percent of BMI 95th Percentile in Participants <18 Years of Age

BMI was calculated using participant's weight and height assessments, using the following formula: BMI = kg/m^2. BMI percentile scores are measures of relative weight adjusted for child's age and gender. The percent of the BMI 95th percentile score expresses the participant's BMI as a percentage of the Centers for Disease Control (CDC) 95th percentile reference population. Baseline was defined as the most recent measurement prior to the first administration of study drug. LSM and SE were calculated using ANCOVA model.

Posted

Least Squares Mean

Standard Error

Percent of BMI 95th Percentile

Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)

ID	Title	Description
OG000	Setmelanotide	Participants received setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
OG001	Placebo	Participants received placebo matched to setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).

Secondary

Pivotal Cohort: Percentage of Participants <18 Years of Age With ≥0.2-Point Reduction From Baseline in BMI Z-Score

BMI was calculated as weight (kg)/height (m^2). BMI Z-Score calculated for participants <18 years old only is a measure of relative weight adjusted for child's age, sex and height at the time of data collection. The Z-Scores were calculated using the World Health Organization's WHO 2007 BMI SAS Macro Package. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). A decrease in BMI Z-score (< 0) indicates a reduction in BMI from Baseline whereas an increase in BMI-Z score (> 0) indicates an increase in BMI from Baseline. Baseline was defined as the most recent measurement prior to the first administration of study drug. Results below represent the percentage of estimated participants with ≥0.2 point reduction in BMI Z-score in each treatment arm and the difference between the treatment arms as calculated through the MIANALYZE SAS procedure.

Posted

Number

95% Confidence Interval

Calculated estimate: % of participants

After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)

ID	Title	Description
OG000	Setmelanotide	Participants received setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
OG001

Secondary

Pivotal Cohort: Percentage of Participants With BMI <30 kg/m^2 (Aged ≥18 Years) or <95th Percentile (Aged <18 Years) From Baseline

BMI was calculated using participant's weight and height assessments, using the following formula: BMI = kg/m^2. BMI Percentile scores are measures of relative weight adjusted for child's age and gender. The percent of the BMI 95th percentile score expresses the participant's BMI as a percentage of the CDC 95th percentile reference population. Baseline was defined as the most recent measurement prior to the first administration of study drug. Results below represent the percentage of estimated participants with either BMI <30 kg/m^2 (aged ≥18 years) or <95th percentile (aged <18 years) in each treatment arm and the difference between the treatment arms as calculated through the MIANALYZE SAS procedure.

mITT analysis set included all randomized participants who were exposed to at least 1 dose of study treatment. Participants in pivotal cohort were included in the analysis.

Posted

Number

95% Confidence Interval

Calculated estimate: % of participants

After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)

ID	Title	Description
OG000	Setmelanotide	Participants received setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
OG001	Placebo	Participants received placebo matched to setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).

Secondary

Pivotal Cohort: Mean Change From Baseline in Physical Functioning Score and Total Score on the Impact of Weight on Quality of Life-Lite (IWQOL)

The IWQOL-Lite-Clinical Trials (administered to participants ≥18 years of age) is a validated 20-item self-report measure of obesity-specific quality of life questionnaire. It assessed 2 primary domains of obesity-related health-related quality of life (HRQoL): physical (7 items) and psychosocial (13 items). Each item was rated on a scale from 0 (worst) to 100 (best), with higher scores indicating better levels of functioning. It provided composite scores for each domain, as well as a total score, all ranging from 0 (worst) to 100 (best). Higher scores reflect better levels of functioning and quality of life. LSM and SE were calculated using ANCOVA model.

Posted

Least Squares Mean

Standard Error

Score on a scale

Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)

ID	Title	Description
OG000	Setmelanotide	Participants received setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
OG001	Placebo	Participants received placebo matched to setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).

Secondary

Pivotal Cohort: Mean Change From Baseline in Total Score on the Impact of Weight on Quality of Life-Kids (IWQOL-Kids)

The IWQOL-Kids (administered to participants between the ages of 11 and <18 years) is a validated 27-item self-report measure of weight-related quality of life for youth. It provided a total score inclusive of 4 domains: physical comfort, body esteem, social life, and family relations. Results below represent the total score, which is rated on a scale from 0 (worst) to 100 (best), with higher scores indicating better levels of functioning. LSM and SE were calculated using ANCOVA model.

Posted

Least Squares Mean

Standard Error

Score on a scale

Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)

ID	Title	Description
OG000	Setmelanotide	Participants received setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
OG001	Placebo	Participants received placebo matched to setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).

Secondary

Pivotal Cohort: Mean Change From Baseline in Waist Circumference

Waist circumference was captured for analysis in centimeters. LSM and SE were calculated using ANCOVA model.

Posted

Least Squares Mean

Standard Error

Centimeters

Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)

ID	Title	Description
OG000	Setmelanotide	Participants received setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
OG001	Placebo	Participants received placebo matched to setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).

Units	Counts
Participants

Secondary

Pivotal Cohort: Change From Baseline in Systolic and Diastolic Blood Pressure

Blood pressure was calculated in millimeters of mercury.

Posted

Mean

Standard Deviation

Millimeters of mercury

Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)

ID	Title	Description
OG000	Setmelanotide	Participants received setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
OG001	Placebo	Participants received placebo matched to setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).

Units	Counts
Participants	OG000

Arm/Groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Setmelanotide	Participants received setmelanotide solution QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).	2	95	26	94	94	94
EG001	Placebo	Participants received placebo matched to setmelanotide solution QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).	0	48	3	48	46	48

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
COVID-19	Infections and infestations	MedDRA 27.0	Systematic Assessment	EG0002 affected94 at risk EG0010 affected48 at risk
Cellulitis	Infections and infestations	MedDRA 27.0	Systematic Assessment	EG0002 affected94 at risk EG0010 affected48 at risk
Appendicitis	Infections and infestations	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Gastroenteritis	Infections and infestations	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Localised infection	Infections and infestations	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Lower respiratory tract infection	Infections and infestations	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Norovirus infection	Infections and infestations	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Pyelonephritis	Infections and infestations	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Streptococcal sepsis	Infections and infestations	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Viral infection	Infections and infestations	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Altered state of consciousness	Nervous system disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Carotid artery aneurysm	Nervous system disorders	MedDRA 27.0	Systematic Assessment	EG0000 affected94 at risk EG0011 affected48 at risk
Cerebral artery stenosis	Nervous system disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Headache	Nervous system disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Loss of consciousness	Nervous system disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Seizure	Nervous system disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Status migrainosus	Nervous system disorders	MedDRA 27.0	Systematic Assessment	EG0000 affected94 at risk EG0011 affected48 at risk
Adrenal insufficiency	Endocrine disorders	MedDRA 27.0	Systematic Assessment	EG0003 affected94 at risk EG0010 affected48 at risk
Adrenocortical insufficiency acute	Endocrine disorders	MedDRA 27.0	Systematic Assessment	EG0002 affected94 at risk EG0010 affected48 at risk
Hypernatraemia	Metabolism and nutrition disorders	MedDRA 27.0	Systematic Assessment	EG0002 affected94 at risk EG0011 affected48 at risk
Hyponatraemia	Metabolism and nutrition disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Vomiting	Gastrointestinal disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0011 affected48 at risk
Haematemesis	Gastrointestinal disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Nausea	Gastrointestinal disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Vision blurred	Eye disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Catheter site erosion	General disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Cholecystitis	Hepatobiliary disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Anaphylactic reaction	Immune system disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Concussion	Injury, poisoning and procedural complications	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Blood sodium abnormal	Investigations	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Acute respiratory failure	Respiratory, thoracic and mediastinal disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk
Hypertension	Vascular disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected94 at risk EG0010 affected48 at risk

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Notes	Statistical Information
Nausea	Gastrointestinal disorders	MedDRA 27.0	Systematic Assessment		EG00049 affected94 at risk EG00117 affected48 at risk
Vomiting	Gastrointestinal disorders	MedDRA 27.0	Systematic Assessment		EG00036 affected94 at risk EG00114 affected48 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA 27.0	Systematic Assessment		EG00019 affected94 at risk EG00114 affected48 at risk
Abdominal pain	Gastrointestinal disorders	MedDRA 27.0	Systematic Assessment		EG00012 affected94 at risk EG0017 affected48 at risk
Constipation	Gastrointestinal disorders	MedDRA 27.0	Systematic Assessment		EG00011 affected94 at risk EG0014 affected48 at risk
Abdominal pain upper	Gastrointestinal disorders	MedDRA 27.0	Systematic Assessment		EG0007 affected94 at risk EG0016 affected48 at risk
Injection site reaction	General disorders	MedDRA 27.0	Systematic Assessment		EG00021 affected94 at risk EG00111 affected48 at risk
Injection site erythema	General disorders	MedDRA 27.0	Systematic Assessment		EG00016 affected94 at risk EG0018 affected48 at risk
Injection site pruritus	General disorders	MedDRA 27.0	Systematic Assessment		EG00016 affected94 at risk EG0018 affected48 at risk
Fatigue	General disorders	MedDRA 27.0	Systematic Assessment		EG00010 affected94 at risk EG0019 affected48 at risk
Injection site pain	General disorders	MedDRA 27.0	Systematic Assessment		EG00010 affected94 at risk EG0017 affected48 at risk
Pyrexia	General disorders	MedDRA 27.0	Systematic Assessment		EG0007 affected94 at risk EG0016 affected48 at risk
Injection site induration	General disorders	MedDRA 27.0	Systematic Assessment		EG0008 affected94 at risk EG0013 affected48 at risk
Injection site oedema	General disorders	MedDRA 27.0	Systematic Assessment		EG0007 affected94 at risk EG0014 affected48 at risk
Influenza like illness	General disorders	MedDRA 27.0	Systematic Assessment		EG0006 affected94 at risk EG0014 affected48 at risk
Injection site swelling	General disorders	MedDRA 27.0	Systematic Assessment		EG0005 affected94 at risk EG0011 affected48 at risk
Oedema peripheral	General disorders	MedDRA 27.0	Systematic Assessment		EG0000 affected94 at risk EG0014 affected48 at risk
Skin hyperpigmentation	Skin and subcutaneous tissue disorders	MedDRA 27.0	Systematic Assessment		EG00052 affected94 at risk EG00111 affected48 at risk
Rash	Skin and subcutaneous tissue disorders	MedDRA 27.0	Systematic Assessment		EG0004 affected94 at risk EG0014 affected48 at risk
Dry skin	Skin and subcutaneous tissue disorders	MedDRA 27.0	Systematic Assessment		EG0005 affected94 at risk EG0011 affected48 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA 27.0	Systematic Assessment		EG00015 affected94 at risk EG0017 affected48 at risk
COVID-19	Infections and infestations	MedDRA 27.0	Systematic Assessment		EG00011 affected94 at risk EG0015 affected48 at risk
Nasopharyngitis	Infections and infestations	MedDRA 27.0	Systematic Assessment		EG0008 affected94 at risk EG0017 affected48 at risk
Influenza	Infections and infestations	MedDRA 27.0	Systematic Assessment		EG0006 affected94 at risk EG0015 affected48 at risk
Sinusitis	Infections and infestations	MedDRA 27.0	Systematic Assessment		EG0003 affected94 at risk EG0017 affected48 at risk
Gastroenteritis	Infections and infestations	MedDRA 27.0	Systematic Assessment		EG0007 affected94 at risk EG0012 affected48 at risk
Pneumonia	Infections and infestations	MedDRA 27.0	Systematic Assessment		EG0004 affected94 at risk EG0013 affected48 at risk
Urinary tract infection	Infections and infestations	MedDRA 27.0	Systematic Assessment		EG0004 affected94 at risk EG0013 affected48 at risk
Viral rhinitis	Infections and infestations	MedDRA 27.0	Systematic Assessment		EG0005 affected94 at risk EG0012 affected48 at risk
Otitis media	Infections and infestations	MedDRA 27.0	Systematic Assessment		EG0005 affected94 at risk EG0011 affected48 at risk
Conjunctivitis	Infections and infestations	MedDRA 27.0	Systematic Assessment		EG0002 affected94 at risk EG0013 affected48 at risk
Ear infection	Infections and infestations	MedDRA 27.0	Systematic Assessment		EG0005 affected94 at risk EG0010 affected48 at risk
Gastroenteritis viral	Infections and infestations	MedDRA 27.0	Systematic Assessment		EG0001 affected94 at risk EG0013 affected48 at risk
Headache	Nervous system disorders	MedDRA 27.0	Systematic Assessment		EG00034 affected94 at risk EG00118 affected48 at risk
Dizziness	Nervous system disorders	MedDRA 27.0	Systematic Assessment		EG00011 affected94 at risk EG0014 affected48 at risk
Blood creatine phosphokinase increased	Investigations	MedDRA 27.0	Systematic Assessment		EG0002 affected94 at risk EG0014 affected48 at risk
Alanine aminotransferase increased	Investigations	MedDRA 27.0	Systematic Assessment		EG0005 affected94 at risk EG0010 affected48 at risk
Depressive symptom	Psychiatric disorders	MedDRA 27.0	Systematic Assessment		EG0006 affected94 at risk EG0015 affected48 at risk
Depression	Psychiatric disorders	MedDRA 27.0	Systematic Assessment		EG0005 affected94 at risk EG0013 affected48 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA 27.0	Systematic Assessment		EG00011 affected94 at risk EG0017 affected48 at risk
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	MedDRA 27.0	Systematic Assessment		EG0009 affected94 at risk EG0013 affected48 at risk
Nasal congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 27.0	Systematic Assessment		EG0004 affected94 at risk EG0016 affected48 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA 27.0	Systematic Assessment		EG0007 affected94 at risk EG0012 affected48 at risk
Myalgia	Musculoskeletal and connective tissue disorders	MedDRA 27.0	Systematic Assessment		EG0004 affected94 at risk EG0014 affected48 at risk
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA 27.0	Systematic Assessment		EG0004 affected94 at risk EG0014 affected48 at risk
Back pain	Musculoskeletal and connective tissue disorders	MedDRA 27.0	Systematic Assessment		EG0006 affected94 at risk EG0011 affected48 at risk
Decreased appetite	Metabolism and nutrition disorders	MedDRA 27.0	Systematic Assessment		EG00010 affected94 at risk EG0011 affected48 at risk
Hyponatraemia	Metabolism and nutrition disorders	MedDRA 27.0	Systematic Assessment		EG0006 affected94 at risk EG0011 affected48 at risk
Hypernatraemia	Metabolism and nutrition disorders	MedDRA 27.0	Systematic Assessment		EG0005 affected94 at risk EG0010 affected48 at risk
Melanocytic naevus	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA 27.0	Systematic Assessment		EG00015 affected94 at risk EG0013 affected48 at risk
Erection increased	Reproductive system and breast disorders	MedDRA 27.0	Systematic Assessment	This is a sex-specific AE. Only male participants were at risk.	EG0004 affected40 at risk EG0013 affected17 at risk

First MCP Posted Date

ID	Term
D050035	Sexual Infantilism

Ancestor Terms

ID	Term
D006059	Gonadal Dysgenesis
D012734	Disorders of Sex Development
D014564	Urogenital Abnormalities
D052776	Female Urogenital Diseases
D005261	Female Urogenital Diseases and Pregnancy Complications
D000091642	Urogenital Diseases
D052801	Male Urogenital Diseases
D000013	Congenital Abnormalities
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D006058	Gonadal Disorders
D004700	Endocrine System Diseases
D007006	Hypogonadism

ID	Term
C579663	setmelanotide

Units	Counts
Participants	OG00061 OG00128

Participants received placebo matched to setmelanotide QD as a SC injection for approximately 52 weeks on a therapeutic regimen (up to approximately 60 weeks).

Units	Counts
Participants	OG00048 OG00123

Units	Counts
Participants	OG00017 OG00111