Hypothalamic Obesity
Conditions
Brief summary
Open-label, single-arm study designed to evaluate the body weight response to setmelanotide administered subcutaneously (SC) daily in participants with hypothalamic obesity (HO).
Interventions
DRUGSetmelanotide
Setmelanotide for SC injection
Sponsors
Rhythm Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to 40 Years
Healthy volunteers
No
Inclusion criteria
Participants must meet all of the following criteria to be eligible for study participation: * Participant has documented evidence of HO, including: * Recent evidence of hypothalamic injury on magnetic resonance imaging (MRI); AND * Diagnosis of craniopharyngioma or other non-malignant brain tumor affecting the hypothalamic region; AND * Has undergone surgery, or chemotherapy, or radiation ≥6 months and ≤15 years before Screening. * Participant has either unilateral hypothalamic lesions or bilateral hypothalamic lesions, as assessed by MRI. * Obesity, documented by a body mass index (BMI) ≥35 kilograms/square meter (kg/m\^2) for participants ≥18 years of age or BMI ≥95th percentile for age and gender for participants 6 to \<18 years of age. * Documented increase in BMI (change from pre-surgery baseline in BMI z-score ≥0.2 for participants \<18 years of age or BMI \>5% for participants \>18 years of age) either during the first 6 months following surgery or within 1 year before surgery AND still present at Screening. * More than 6 months after the end of post-tumor treatment, including chemotherapy, surgery, or radiation. * Highly effective contraception throughout the study and for 90 days following the study. * Ability to communicate well with the Investigator, understand and comply with the requirements of the study, and understand and sign the written informed consent, or, for participants aged \<18 years, a parent/legal guardian that can sign. * If receiving hormone replacement therapy, the dose of such therapy has remained stable for at least 2 months prior to Screening.
Exclusion criteria
Participants meeting any of the following criteria are not eligible for study participation: * Weight gain \>5% in the previous 3 months. * Weight loss ≥2% in the previous 3 months. * Bariatric surgery or procedure within the last 6 months. * Diagnosis of severe psychiatric disorders * Glycated hemoglobin (HbA1c) \>10.0% at Screening. * Current, clinically significant pulmonary, cardiac, or oncologic disease considered severe enough to interfere with the study and/or confound the results. * Glomerular filtration rate (GFR) \<30mL/min/1.73m\^2 during Screening. * Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions. * History or close family history (parents or siblings) of skin cancer or melanoma * Participation in any clinical study with an investigational drug/device within 3 months or 5 half-lives, whichever is longer, prior to the first setmelanotide dose. * Previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide. * Inability to comply with QD injection regimen. * Pregnant and/or breastfeeding, or desiring to become pregnant during this trial. * Cognitive impairment that, in the Investigator's opinion, precludes participation to the study and completions of study procedures or questionnaires. * Participant is, in Investigator's opinion, otherwise not suitable to participate in the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With ≥ 5% Reduction in BMI From Baseline After 16 Weeks of Setmelanotide Treatment | Baseline to 16 weeks | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants Aged ≥6 to <18 Years With ≥0.2 Reduction of BMI Z-Score From Baseline After 16 Weeks of Setmelanotide Treatment | Baseline to 16 weeks | 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. |
| Percentage of Participants Aged ≥18 Years With ≥5% Reduction of Body Weight From Baseline After 16 Weeks of Setmelanotide Treatment | Baseline to Week 16 | Baseline was defined as the most recent measurement prior to the first administration of study drug. |
| Change From Baseline in Waist Circumference in Participants Aged ≥18 Years After 16 Weeks of Setmelanotide Treatment | Baseline, Week 16 | 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. |
| Change From Baseline in Daily Hunger Questionnaire Score in Participants Aged <12 Years After 16 Weeks of Setmelanotide Treatment | Baseline, Week 16 | 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. |
| 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 | Baseline to Week 16 | 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. |
| Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years | Baseline, Week 16 | 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. |
| Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years | Baseline, Week 16 | 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. |
| Number of Participants With Adverse Events (AEs) | From first dose of study drug up to Day 141 | 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. |
| Change From Baseline in Daily Hunger Questionnaire Scores in Participants Aged ≥12 Years After 16 Weeks of Setmelanotide Treatment | Baseline, Week 16 | 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. |
Countries
United States
Participant flow
Pre-assignment details
Of the 19 participants screened, 18 participants were enrolled in the study.
Participants by arm
| Arm | Count |
|---|---|
| Setmelanotide 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. | 18 |
| Total | 18 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 2 |
Baseline characteristics
| Characteristic | Setmelanotide |
|---|---|
| Age, Continuous | 15.0 years STANDARD_DEVIATION 5.3 |
| BMI Z-Score | 3.935 z-score STANDARD_DEVIATION 0.908 |
| Body Mass Index (BMI) | 37.95 kilograms/square meter (kg/m^2) STANDARD_DEVIATION 6.532 |
| Body Weight | 102.76 kilograms (kg) STANDARD_DEVIATION 30.12 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 4 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 14 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 3 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 1 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 14 Participants |
| Sex: Female, Male Female | 7 Participants |
| Sex: Female, Male Male | 11 Participants |
| Waist Circumference | 114.07 centimeters (cm) STANDARD_DEVIATION 16.354 |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 18 |
| other Total, other adverse events | 18 / 18 |
| serious Total, serious adverse events | 1 / 18 |
Outcome results
Primary
Percentage of Participants With ≥ 5% Reduction in BMI From Baseline After 16 Weeks of Setmelanotide Treatment
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.
Time frame: Baseline to 16 weeks
Population: Participants in the Full Analysis Set were analyzed.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Setmelanotide | Percentage of Participants With ≥ 5% Reduction in BMI From Baseline After 16 Weeks of Setmelanotide Treatment | 88.9 percentage of participants |
p-value: <0.0001One-sided exact binomial test
Secondary
Change From Baseline in Daily Hunger Questionnaire Score in Participants Aged <12 Years After 16 Weeks of Setmelanotide Treatment
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.
Time frame: Baseline, Week 16
Population: Participants in the Full Analysis Set with available data were analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Setmelanotide | 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
Change From Baseline in Daily Hunger Questionnaire Scores in Participants Aged ≥12 Years After 16 Weeks of Setmelanotide Treatment
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.
Time frame: Baseline, Week 16
Population: Participants in the Full Analysis Set with available data were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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
Change From Baseline in Waist Circumference in Participants Aged ≥18 Years After 16 Weeks of Setmelanotide Treatment
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.
Time frame: Baseline, Week 16
Population: Participants in the Full Analysis Set with available data were analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Setmelanotide | 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
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
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.
Time frame: Baseline to Week 16
Population: Participants in the Full Analysis Set were analyzed.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Setmelanotide | 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 |
p-value: <0.0001One-sided exact binomial test
Secondary
Number of Participants With Adverse Events (AEs)
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.
Time frame: From first dose of study drug up to Day 141
Population: Participants in the Safety Analysis Set were analyzed.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Setmelanotide | Number of Participants With Adverse Events (AEs) | 18 Participants |
Secondary
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years
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.
Time frame: Baseline, Week 16
Population: Participants in the Full Analysis Set with available data were analyzed.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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
Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years
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.
Time frame: Baseline, Week 16
Population: Participants in the Full Analysis Set with available data were analyzed.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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 |
| Setmelanotide | 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
Percentage of Participants Aged ≥18 Years With ≥5% Reduction of Body Weight From Baseline After 16 Weeks of Setmelanotide Treatment
Baseline was defined as the most recent measurement prior to the first administration of study drug.
Time frame: Baseline to Week 16
Population: Participants in the Full Analysis Set with available data were analyzed.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Setmelanotide | 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 |
p-value: <0.0001One-sided exact binomial test
Secondary
Percentage of Participants Aged ≥6 to <18 Years With ≥0.2 Reduction of BMI Z-Score From Baseline After 16 Weeks of Setmelanotide Treatment
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.
Time frame: Baseline to 16 weeks
Population: Participants in the Full Analysis Set with available data were analyzed.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Setmelanotide | 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 |
p-value: <0.0001One-sided exact binomial test