Trial Outcomes & Findings for Open-Label Study of Setmelanotide in Hypothalamic Obesity (NCT NCT04725240)

NCT ID: NCT04725240

Last Updated: 2025-02-07

Results Overview

BMI was calculated using participant's weight and height assessments, using the following formula: BMI = kg/m\^2. Baseline was defined as the most recent measurement prior to the first administration of study drug.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 18 participants
• Primary outcome timeframe: Baseline to 16 weeks
• Results posted on: 2025-02-07

Participant Flow

Of the 19 participants screened, 18 participants were enrolled in the study.

Participant milestones

Measure	Setmelanotide Participants received setmelanotide once daily (QD) via subcutaneous (SC) injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and dose escalated up to a maximum dose of 3.0 milligrams (mg) QD.
Overall Study STARTED	18
Overall Study COMPLETED	16
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure	Setmelanotide Participants received setmelanotide once daily (QD) via subcutaneous (SC) injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and dose escalated up to a maximum dose of 3.0 milligrams (mg) QD.
Overall Study Adverse Event	2

Baseline Characteristics

Participants in the Full Analysis Set with available data were analyzed.

Baseline characteristics by cohort

Measure	Setmelanotide n=18 Participants Participants received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and dose escalated up to a maximum dose of 3.0 mg QD.
Age, Continuous	15.0 years STANDARD_DEVIATION 5.30 • n=18 Participants
Sex: Female, Male Female	7 Participants n=18 Participants
Sex: Female, Male Male	11 Participants n=18 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	4 Participants n=18 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	14 Participants n=18 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=18 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=18 Participants
Race (NIH/OMB) Asian	0 Participants n=18 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants n=18 Participants
Race (NIH/OMB) Black or African American	3 Participants n=18 Participants
Race (NIH/OMB) White	14 Participants n=18 Participants
Race (NIH/OMB) More than one race	0 Participants n=18 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=18 Participants
Body Mass Index (BMI)	37.95 kilograms/square meter (kg/m^2) STANDARD_DEVIATION 6.532 • n=18 Participants
Body Weight	102.76 kilograms (kg) STANDARD_DEVIATION 30.120 • n=18 Participants
BMI Z-Score	3.935 z-score STANDARD_DEVIATION 0.9080 • n=13 Participants • Participants in the Full Analysis Set with available data were analyzed.
Waist Circumference	114.07 centimeters (cm) STANDARD_DEVIATION 16.354 • n=18 Participants

PRIMARY outcome

Timeframe: Baseline to 16 weeks

Population: Participants in the Full Analysis Set were analyzed.

BMI was calculated using participant's weight and height assessments, using the following formula: BMI = kg/m^2. Baseline was defined as the most recent measurement prior to the first administration of study drug.

Outcome measures

Measure	Setmelanotide n=18 Participants Participants received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and dose escalated up to a maximum dose of 3.0 mg QD.
Percentage of Participants With ≥ 5% Reduction in BMI From Baseline After 16 Weeks of Setmelanotide Treatment	88.9 percentage of participants Interval 69.0 to 98.0

SECONDARY outcome

Timeframe: Baseline to Week 16

Population: Participants in the Full Analysis Set were analyzed.

For participants aged ≥6 to <18 years, a participant's BMI Z-score was considered for the analysis; for participants ≥18 years, a participant's body weight (kg) was considered for the analysis. BMI was calculated using participant's weight and height assessments, using the following formula: BMI = kg/m^2. The BMI Z-score indicated the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). A decrease of BMI Z-score indicates a reduction in BMI from Baseline whereas an increase of BMI-Z score indicates an increase in BMI from Baseline. Baseline was defined as the most recent measurement prior to the first administration of study drug.

Outcome measures

Measure	Setmelanotide n=18 Participants Participants received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and dose escalated up to a maximum dose of 3.0 mg QD.
Composite Percentage of Participants Aged ≥6 to <18 Years With ≥0.2 Reduction of BMI Z-Score or Participants Aged ≥18 Years With 5% Reduction of Body Weight From Baseline After 16 Weeks of Setmelanotide Treatment	88.9 percentage of participants Interval 69.0 to 98.0

SECONDARY outcome

Timeframe: Baseline to 16 weeks

Population: Participants in the Full Analysis Set with available data were analyzed.

BMI was calculated using participant's weight and height assessments, using the following formula: BMI = kg/m^2. The BMI Z-score indicated the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). A decrease of BMI Z-score indicates a reduction in BMI from Baseline whereas an increase of BMI-Z score indicates an increase in BMI from Baseline. Baseline was defined as the most recent measurement prior to the first administration of study drug.

Outcome measures

Measure	Setmelanotide n=13 Participants Participants received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and dose escalated up to a maximum dose of 3.0 mg QD.
Percentage of Participants Aged ≥6 to <18 Years With ≥0.2 Reduction of BMI Z-Score From Baseline After 16 Weeks of Setmelanotide Treatment	92.3 percentage of participants Interval 68.4 to 99.6

SECONDARY outcome

Timeframe: Baseline to Week 16

Population: Participants in the Full Analysis Set with available data were analyzed.

Baseline was defined as the most recent measurement prior to the first administration of study drug.

Outcome measures

Measure	Setmelanotide n=5 Participants Participants received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and dose escalated up to a maximum dose of 3.0 mg QD.
Percentage of Participants Aged ≥18 Years With ≥5% Reduction of Body Weight From Baseline After 16 Weeks of Setmelanotide Treatment	80.0 percentage of participants Interval 34.3 to 99.0

SECONDARY outcome

Timeframe: Baseline, Week 16

Population: Participants in the Full Analysis Set with available data were analyzed.

Waist circumference was measured after participants had fasted for at least 8 hours and at approximately the same time at each visit. Baseline was defined as the most recent measurement prior to the first administration of study drug.

Outcome measures

Measure	Setmelanotide n=4 Participants Participants received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and dose escalated up to a maximum dose of 3.0 mg QD.
Change From Baseline in Waist Circumference in Participants Aged ≥18 Years After 16 Weeks of Setmelanotide Treatment	-10.275 cm Standard Deviation 4.2272

SECONDARY outcome

Timeframe: Baseline, Week 16

Population: Participants in the Full Analysis Set with available data were analyzed.

Daily Hunger Questionnaire for participants <12 years of age: Three aspects of hunger (average hunger in the last 24 hours, most/worst hunger in the last 24 hours, and morning hunger) were assessed daily using a pictorial (smiley face) version of the Likert rating scale with scores from 0 to 4, with 0 = not hungry at all and 4 = hungriest possible. Responses were recorded in the electronic diary. Each of the 3 items (average hunger, most/worst hunger, and morning hunger) was scored separately and averaged to calculate a total score ranging from 0 (not hungry at all) to 4 (hungriest possible) with higher score indicating more hunger. Baseline daily hunger score was calculated as the average of 7 days prior to Baseline visit.

Outcome measures

Measure	Setmelanotide n=5 Participants Participants received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and dose escalated up to a maximum dose of 3.0 mg QD.
Change From Baseline in Daily Hunger Questionnaire Score in Participants Aged <12 Years After 16 Weeks of Setmelanotide Treatment	-1.490 units on a scale Standard Deviation 1.2126

SECONDARY outcome

Timeframe: Baseline, Week 16

Population: Participants in the Full Analysis Set with available data were analyzed.

Daily Hunger Questionnaire for participants ≥12 years of age: Three aspects of hunger (average hunger in the last 24 hours, most/worst hunger in the last 24 hours, and morning hunger) were assessed daily using a numeric rating score for each from 0 to 10, with 0 = not hungry at all and 10 = hungriest possible. The responses to the Daily Hunger Questionnaire were recorded in the electronic diary. Baseline daily hunger score was calculated as the average of 7 days prior to Baseline visit.

Outcome measures

Measure	Setmelanotide n=11 Participants Participants received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and dose escalated up to a maximum dose of 3.0 mg QD.
Change From Baseline in Daily Hunger Questionnaire Scores in Participants Aged ≥12 Years After 16 Weeks of Setmelanotide Treatment Most hunger	-2.922 units on a scale Standard Deviation 2.2851
Change From Baseline in Daily Hunger Questionnaire Scores in Participants Aged ≥12 Years After 16 Weeks of Setmelanotide Treatment Average hunger	-3.122 units on a scale Standard Deviation 1.9999
Change From Baseline in Daily Hunger Questionnaire Scores in Participants Aged ≥12 Years After 16 Weeks of Setmelanotide Treatment Morning hunger	-2.717 units on a scale Standard Deviation 1.9393

SECONDARY outcome

Timeframe: Baseline, Week 16

Population: Participants in the Full Analysis Set with available data were analyzed.

Parents or caregivers of participants <12 years old answered the Caregiver Reported Global Hunger Question for participants who were <12 years old. The following question was asked: How hungry is your child acting now? Possible responses were: Not hungry at all; A little hungry; Moderately hungry; and Extremely hungry. Baseline was defined as the most recent measurement prior to the first administration of study drug.

Outcome measures

Measure	Setmelanotide n=5 Participants Participants received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and dose escalated up to a maximum dose of 3.0 mg QD.
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years Not hungry at all (Baseline) - Not hungry at all (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years A little hungry (Baseline) - Not hungry at all (week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years Moderately hungry (Baseline) - Not hungry at all (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years Extremely hungry (Baseline) - Not hungry at all (Week 16)	2 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years Not hungry at all (Baseline) - A little hungry (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years A little hungry (Baseline) - A little hungry (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years Moderately hungry (Baseline) - A little hungry (Week 16)	1 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years Extremely hungry (Baseline) - A little hungry (Week 16)	2 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years Not hungry at all (Baseline) - Moderately hungry (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years A little hungry (Baseline) - Moderately hungry (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years Moderately hungry (Baseline) - Moderately hungry (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years Extremely hungry (Baseline) - Moderately hungry (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years Not hungry at all (Baseline) - Extremely hungry (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years A little hungry (Baseline) - Extremely hungry (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years Moderately hungry (Baseline) - Extremely hungry (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years Extremely hungry (Baseline) - Extremely hungry (Week 16)	0 Participants

SECONDARY outcome

Timeframe: Baseline, Week 16

Population: Participants in the Full Analysis Set with available data were analyzed.

Global Hunger Questionnaire for participants ≥12 years of age. The following question was asked: Overall, how would you rate the hunger you experience now? Possible responses were: No hunger; Mild hunger; Moderate hunger; and Severe hunger. Baseline was defined as the most recent measurement prior to the first administration of study drug.

Outcome measures

Measure	Setmelanotide n=11 Participants Participants received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and dose escalated up to a maximum dose of 3.0 mg QD.
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years No hunger (Baseline) - No hunger (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years Mild hunger (Baseline) - No hunger (Week 16)	1 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years Moderate hunger (Baseline) - No hunger (Week 16)	3 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years Severe hunger (Baseline) - No hunger (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years No hunger (Baseline) - Mild hunger (Week 16)	1 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years Mild hunger (Baseline) - Mild hunger (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years Moderate hunger (Baseline) - Mild hunger (Week 16)	3 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years Severe hunger (Baseline) - Mild hunger (Week 16)	2 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years No hunger (Baseline) - Moderate hunger (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years Mild hunger (Baseline) - Moderate hunger (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years Moderate hunger (Baseline) - Moderate hunger (Week 16)	1 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years Severe hunger (Baseline) - Moderate hunger (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years No hunger (Baseline) - Severe hunger (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years Mild hunger (Baseline) - Severe hunger (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years Moderate hunger (Baseline) - Severe hunger (Week 16)	0 Participants
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years Severe hunger (Baseline) - Severe hunger (Week 16)	0 Participants

SECONDARY outcome

Timeframe: From first dose of study drug up to Day 141

Population: Participants in the Safety Analysis Set were analyzed.

An AE was defined as any untoward medical occurrence or clinically significant worsening of an existing condition associated with the use of a drug in humans, whether or not considered drug related. An AE (also referred to as an adverse experience) could be any unfavorable and unintended sign (e.g., an abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, without any judgment about causality.

Outcome measures

Measure	Setmelanotide n=18 Participants Participants received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and dose escalated up to a maximum dose of 3.0 mg QD.
Number of Participants With Adverse Events (AEs)	18 Participants

Adverse Events

Setmelanotide

Serious events: 1 serious events

Other events: 18 other events

Deaths: 0 deaths

Serious adverse events

Measure	Setmelanotide n=18 participants at risk Participants received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and dose escalated up to a maximum dose of 3.0 mg QD.
Infections and infestations Clostridium difficile colitis	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.

Other adverse events

Measure	Setmelanotide n=18 participants at risk Participants received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and dose escalated up to a maximum dose of 3.0 mg QD.
General disorders Fatigue	16.7% 3/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
General disorders Injection site pain	16.7% 3/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
General disorders Injection site pruritus	11.1% 2/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
General disorders Pain	11.1% 2/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
General disorders Pyrexia	11.1% 2/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
General disorders Injection site erythema	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
General disorders Injection site induration	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
General disorders Injection site swelling	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Reproductive system and breast disorders Erection increased	27.3% 3/11 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Respiratory, thoracic and mediastinal disorders Cough	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Respiratory, thoracic and mediastinal disorders Epistaxis	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Respiratory, thoracic and mediastinal disorders Nasal congestion	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Psychiatric disorders Conversion disorder	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Investigations Alanine aminotransferase increased	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Investigations Blood creatine phosphokinase increased	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Investigations Blood uric acid increased	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Investigations Hepatic enzyme increased	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Investigations Weight decreased	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Injury, poisoning and procedural complications Contusion	11.1% 2/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Injury, poisoning and procedural complications Radius fracture	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Congenital, familial and genetic disorders Type V hyperlipidaemia	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Nervous system disorders Headache	11.1% 2/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Nervous system disorders Balance disorder	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Nervous system disorders Seizure	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Nervous system disorders Speech disorder	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Blood and lymphatic system disorders Anaemia	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Gastrointestinal disorders Nausea	61.1% 11/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Gastrointestinal disorders Vomiting	33.3% 6/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Gastrointestinal disorders Diarrhoea	22.2% 4/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Gastrointestinal disorders Abdominal pain	16.7% 3/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Gastrointestinal disorders Abdominal pain upper	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Gastrointestinal disorders Gastritis	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Gastrointestinal disorders Portal hypertensive gastropathy	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Hepatobiliary disorders Hypertransaminasaemia	11.1% 2/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Skin and subcutaneous tissue disorders Skin hyperpigmentation	33.3% 6/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Skin and subcutaneous tissue disorders Acne	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Skin and subcutaneous tissue disorders Rash	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Skin and subcutaneous tissue disorders Rash erythematous	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Musculoskeletal and connective tissue disorders Back pain	11.1% 2/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Musculoskeletal and connective tissue disorders Myalgia	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Musculoskeletal and connective tissue disorders Pain in extremity	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Musculoskeletal and connective tissue disorders Scoliosis	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Infections and infestations COVID-19	22.2% 4/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Infections and infestations Upper respiratory tract infection	11.1% 2/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Infections and infestations Administration site cellulitis	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Infections and infestations Gastroenteritis	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Infections and infestations Sinusitis	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Infections and infestations Staphylococcal abscess	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Metabolism and nutrition disorders Dehydration	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.
Hepatobiliary disorders Hepatic steatosis	5.6% 1/18 • From first dose of study drug up to Day 141 Participants in the Safety Analysis Set were analyzed.

Additional Information

Rhythm Clinical Trials

Rhythm Pharmaceuticals, Inc.

Phone: 857-264-4280

Email: clinicaltrials@rhythmtx.com

Results disclosure agreements

• Principal investigator is a sponsor employee All information regarding setmelanotide supplied by Rhythm to the investigator is privileged and confidential information. The investigator agrees to use this information to accomplish the study and will not use it for other purposes without consent from Rhythm. The information obtained from the clinical study will be used towards the development of setmelanotide and may be disclosed to regulatory authority(ies), other investigators, corporate partners, or consultants as required.
• Publication restrictions are in place

Restriction type: OTHER