A Phase 3 interventional study of Setmelanotide and Placebo in Hypothalamic Obesity, sponsored by Rhythm Pharmaceuticals, Inc.. Active, not recruiting at 28 sites in 6 countries. Open to participants aged 4 Years and older. Per ClinicalTrials.gov, last updated 2026-08-10.
Sponsored by Rhythm Pharmaceuticals, Inc. · Phase 3, Interventional, and Treatment
The goal of this trial is to learn how well Setmelanotide works to improve weight reduction, hunger, and quality of life in patients 4 years of age and older with acquired Hypothalamic Obesity (HO). To determine how well setmelanotide works and how safe it is, patients with HO will take a daily injection of either setmelanotide or placebo and complete trial assessments for 52 weeks on a therapeutic regimen.
A separate sub-study in patients with congenital HO is detailed under NCT06760546.
23 studies on the registry are indexed under Sexual Infantilism; 5 are open to participants now.
This study's enrollment of 143 is above the median of 30 across 20 interventional studies indexed under Sexual Infantilism.
Browse Sexual Infantilism studies →Rhythm Pharmaceuticals, Inc. is the lead sponsor of 29 studies on the registry; 2 are open to participants now.
Of its 19 completed or terminated interventional studies of FDA-regulated products, 14 (74%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Randomized 2:1 (Setmelanotide: Placebo)
Drug: Setmelanotide
Randomized 2:1 (Setmelanotide: Placebo)
Drug: Placebo
Solution for daily subcutaneous injection
Also known as: RM-493, Imcivree
Placebo matched to setmelanotide for daily subcutaneous injection
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.
Time frame: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
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.
Time frame: After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
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.
Time frame: After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
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.
Time frame: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
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.
Time frame: After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
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.
Time frame: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
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.
Time frame: After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
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.
Time frame: After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
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.
Time frame: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
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.
Time frame: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
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.
Time frame: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
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.
Time frame: After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
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.
Time frame: After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
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.
Time frame: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
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.
Time frame: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
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.
Time frame: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
Pivotal Cohort: Change From Baseline in Systolic and Diastolic Blood Pressure
Blood pressure was calculated in millimeters of mercury.
Time frame: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
| Milestone | Setmelanotide | Placebo |
|---|---|---|
| Started | 95 | 48 |
| Received at least 1 dose of study drug | 94 | 48 |
| Modified intent to treat pivotal cohort | 81 | 39 |
| Completed | 72 | 38 |
| Not completed | 23 | 10 |
| Withdrew: Adverse event | 3 | 1 |
| Withdrew: Death | 1 | 0 |
| Withdrew: Withdrawal by subject | 4 | 1 |
| Withdrew: Randomized, not treated | 1 | 0 |
| Withdrew: Ongoing | 13 | 8 |
| Withdrew: Withdrawal by parent/guardian | 1 | 0 |
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.
| Percent change | Setmelanotide | Placebo |
|---|---|---|
| Pivotal Cohort: Mean Percent Change From Baseline in Body Mass Index (BMI) After 52 Weeks on a Therapeutic Regimen | -16.51 ± 1.401 | 3.32 ± 1.984 |
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.
| Calculated estimate: % of participants | Setmelanotide | Placebo |
|---|---|---|
| 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) | 82.73 (74.13 to 91.33) | 20.90 (8.01 to 33.78) |
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.
| Calculated estimate: % of participants | Setmelanotide | Placebo |
|---|---|---|
| Pivotal Cohort: Percentage of Participants With ≥5% Reduction From Baseline in BMI | 79.53 (70.60 to 88.47) | 10.41 (0.75 to 20.07) |
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.
| Score on a scale | Setmelanotide | Placebo |
|---|---|---|
| Pivotal Cohort: Change From Baseline in Weekly Average Daily Most Hunger Score in Participants ≥12 Years of Age | -2.73 ± 0.279 | -1.45 ± 0.399 |
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.
| Calculated estimate: % of participants | Setmelanotide | Placebo |
|---|---|---|
| Pivotal Cohort: Percentage of Participants (≥12 Years of Age) With a ≥2-point Reduction From Baseline in the Weekly Average Daily Most Hunger Score | 61.04 (47.01 to 75.06) | 45.04 (24.56 to 65.52) |
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.
| Score on a scale | Setmelanotide | Placebo |
|---|---|---|
| Pivotal Cohort: Mean Change From Baseline in the Weekly Average of the Symptoms of Hyperphagia Composite Score in Participants ≥12 Years of Age | -0.30 ± 0.029 | -0.14 ± 0.042 |
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.
| Calculated estimate: % of participants | Setmelanotide | Placebo |
|---|---|---|
| Pivotal Cohort: Percentage of Participants With a ≥10% Reduction From Baseline in BMI | 63.02 (52.50 to 73.55) | 5.18 (0.00 to 12.17) |
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.
| Calculated estimate: % of participants | Setmelanotide | Placebo |
|---|---|---|
| Pivotal Cohort: Percentage of Participants With a ≥10% Reduction From Baseline in Body Weight | 55.60 (44.77 to 66.44) | 0.05 (0.00 to 1.05) |
Body weight was captured for analysis in kilograms. LSM and SE were calculated using ANCOVA model.
| Percent change | Setmelanotide | Placebo |
|---|---|---|
| Pivotal Cohort: Mean Percent Change From Baseline in Body Weight in Participants ≥18 Years | -16.51 ± 2.297 | 2.44 ± 3.228 |
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.
| Z-Score | Setmelanotide | Placebo |
|---|---|---|
| Pivotal Cohort: Mean Change From Baseline in BMI Z-Score in Participants <18 Years of Age | -1.47 ± 0.129 | -0.12 ± 0.180 |
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.
| Percent of BMI 95th Percentile | Setmelanotide | Placebo |
|---|---|---|
| Pivotal Cohort: Mean Change From Baseline in Percent of BMI 95th Percentile in Participants <18 Years of Age | -26.30 ± 2.279 | -0.14 ± 3.284 |
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.
| Calculated estimate: % of participants | Setmelanotide | Placebo |
|---|---|---|
| Pivotal Cohort: Percentage of Participants <18 Years of Age With ≥0.2-Point Reduction From Baseline in BMI Z-Score | 89.31 (80.08 to 98.54) | 31.09 (11.93 to 50.24) |
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.
| Calculated estimate: % of participants | Setmelanotide | Placebo |
|---|---|---|
| Pivotal Cohort: Percentage of Participants With BMI <30 kg/m^2 (Aged ≥18 Years) or <95th Percentile (Aged <18 Years) From Baseline | 39.95 (29.18 to 50.72) | 12.82 (4.30 to 27.43) |
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.
| Score on a scale | Setmelanotide | Placebo |
|---|---|---|
| IWQOL-Lite-CT-Physical Function Score | 31.32 ± 3.702 | -4.46 ± 5.145 |
| IWQOL-Lite-CT-Total Score | 32.04 ± 3.059 | 3.00 ± 4.249 |
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.
| Score on a scale | Setmelanotide | Placebo |
|---|---|---|
| Pivotal Cohort: Mean Change From Baseline in Total Score on the Impact of Weight on Quality of Life-Kids (IWQOL-Kids) | 22.05 ± 4.708 | 7.92 ± 4.001 |
Waist circumference was captured for analysis in centimeters. LSM and SE were calculated using ANCOVA model.
| Centimeters | Setmelanotide | Placebo |
|---|---|---|
| Pivotal Cohort: Mean Change From Baseline in Waist Circumference | -11.58 ± 1.161 | 3.85 ± 1.584 |
Blood pressure was calculated in millimeters of mercury.
| Millimeters of mercury | Setmelanotide | Placebo |
|---|---|---|
| Systolic blood pressure | -2.16 ± 11.587 | 2.84 ± 11.438 |
| Diastolic blood pressure | -0.88 ± 7.480 | 2.71 ± 9.789 |
Collected over Baseline up to Week 62. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Setmelanotide | 2/95 (2.1%) | 26/94 (27.7%) | 94/94 (100%) |
| Placebo | 0/48 (0%) | 3/48 (6.3%) | 46/48 (95.8%) |
| Event | Setmelanotide | Placebo |
|---|---|---|
| Adrenal insufficiencyEndocrine disorders | 3/94 | 0/48 |
| COVID-19Infections and infestations | 2/94 | 0/48 |
| CellulitisInfections and infestations | 2/94 | 0/48 |
| Adrenocortical insufficiency acuteEndocrine disorders | 2/94 | 0/48 |
| HypernatraemiaMetabolism and nutrition disorders | 2/94 | 1/48 |
| Carotid artery aneurysmNervous system disorders | 0/94 | 1/48 |
| Status migrainosusNervous system disorders | 0/94 | 1/48 |
| VomitingGastrointestinal disorders | 1/94 | 1/48 |
| AppendicitisInfections and infestations | 1/94 | 0/48 |
| GastroenteritisInfections and infestations | 1/94 | 0/48 |
| Event | Setmelanotide | Placebo |
|---|---|---|
| Skin hyperpigmentationSkin and subcutaneous tissue disorders | 52/94 | 11/48 |
| NauseaGastrointestinal disorders | 49/94 | 17/48 |
| VomitingGastrointestinal disorders | 36/94 | 14/48 |
| HeadacheNervous system disorders | 34/94 | 18/48 |
| DiarrhoeaGastrointestinal disorders | 19/94 | 14/48 |
| Injection site reactionGeneral disorders | 21/94 | 11/48 |
| FatigueGeneral disorders | 10/94 | 9/48 |
| Erection increasedReproductive system and breast disorders | 4/40 | 3/17 |
| Injection site erythemaGeneral disorders | 16/94 | 8/48 |
| Injection site pruritusGeneral disorders | 16/94 | 8/48 |
Modified intent to treat (mITT) analysis set included all randomized participants who were exposed to at least 1 dose of study treatment.
| Age, Continuous(years) | Setmelanotide | Placebo | Total |
|---|---|---|---|
| Mean | 20.6 ± 13.35 | 22.9 ± 16.31 | 21.4 ± 14.40 |
| Sex: Female, Male(Participants) | Setmelanotide | Placebo | Total |
|---|---|---|---|
| Female | 54 | 31 | 85 |
| Male | 40 | 17 | 57 |
| Ethnicity (NIH/OMB)(Participants) | Setmelanotide | Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 8 | 8 | 16 |
| Not Hispanic or Latino | 85 | 40 | 125 |
| Unknown or Not Reported | 1 | 0 | 1 |
| Race (NIH/OMB)(Participants) | Setmelanotide | Placebo | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 11 | 5 | 16 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 6 | 1 | 7 |
| White | 71 | 36 | 107 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 6 | 6 | 12 |
| Region of Enrollment(participants) | Setmelanotide | Placebo | Total |
|---|---|---|---|
| Canada | 5 | 3 | 8 |
| Netherlands | 7 | 5 | 12 |
| United States | 57 | 31 | 88 |
| Japan | 8 | 4 | 12 |
| United Kingdom | 9 | 1 | 10 |
| Germany | 8 | 4 | 12 |
| Body Mass Index(kilograms (kg)/square meter (m^2)) | Setmelanotide | Placebo | Total |
|---|---|---|---|
| Mean | 35.73 ± 9.173 | 36.78 ± 9.332 | 36.07 ± 9.199 |
PDFs hosted by the registry, ClinicalTrials.gov.
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Studies of the drug tested here: Setmelanotide
Rhythm Pharmaceuticals, Inc. →