Bardet-Biedl Syndrome, LEPR Deficiency Obesity, PCSK1 Deficiency Obesity, POMC Deficiency Obesity
Conditions
Keywords
Bardet Biedl Syndrome, POMC Deficiency, PCSK1, LEPR, Obesity, Pediatric Obesity
Brief summary
This is a phase 3 open-label, clinical study to evaluate the efficacy, safety and tolerability of setmelanotide over 1 year of treatment, in pediatric participants aged 2 to \<6 years with obesity due to either biallelic variants of the pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) genes or Bardet-Biedl Syndrome (BBS).
Detailed description
Pediatric participants aged 2 to \<6 years with obesity due to either biallelic variants of the POMC, PCSK1 or LEPR genes or BBS will be enrolled into this phase 3 open-label clinical trial at one of approximately 8 clinical centers in North America, Europe, or Australia. All participants will be assigned to receive setmelanotide via daily subcutaneous (SC) injection for 1 year.
Interventions
DRUGSetmelanotide
SC injection once daily.
Sponsors
Rhythm Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 5 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: 1. Participants must have obesity due to either: 1. POMC, PCSK1, or LEPR deficiency, confirmed by genetic testing demonstrating biallelic variants that are interpreted as pathogenic, likely pathogenic, or of undetermined significance (VUS) by the American College of Medical Genetics and Genomics criteria (ACMG), or 2. BBS confirmed clinical and genetic diagnosis 2. Age between 2 to \<6 years at the time of informed consent 3. Obesity, defined as body mass index (BMI) ≥97th percentile for age and gender and body weight of at least 15 kilograms (kg) at the time of enrollment. 4. Symptoms or behaviors of hyperphagia 5. Parent or guardian of study participant is able to understand and comply with the requirements of the study (including QD injection regimen and all other study procedures) and is able to understand and sign the written consent/assent. Key
Exclusion criteria
1. Glycated hemoglobin (HbA1c) \>9.0% at screening 2. History of significant liver disease 3. Glomerular filtration rate (GFR) \<60 milliliter per minute per 1.73 meter square (mL/min/1.73 m\^2) 4. History or close family history of melanoma, or participant history of oculocutaneous albinism. 5. Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion) 6. Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing. 7. Previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide. 8. Significant hypersensitivity to any excipient in the study drug. 9. Inadequate hepatic function 10. Any other uncontrolled endocrine, metabolic or medical condition(s) known to impact body weight Other protocol defined Inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Mean Percent Change From Baseline in BMI | Baseline, Week 52 | Mean percent change from baseline to Week 52 in BMI was reported. 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. |
| Percentage of Participants With Greater Than or Equal to (≥) 0.2 Reduction of BMI Z-Score From Baseline to Week 52 | Baseline up to Week 52 | A responder was defined as a decrease from baseline to 52 weeks in the participant's BMI z-score of ≥0.2. BMI was calculated using participant's weight and height assessments, using the following formula: BMI = kg/m\^2. The BMI Z-scores were based on the World Health Organization's Child Growth Standards 2007 and indicated the number of standard deviations away from the mean. A Z-score of 0 was equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). A decrease of BMI Z-score (\< 0) indicated a reduction in BMI from Baseline whereas an increase of 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Mean Change From Baseline in Bone Age | Baseline, Week 52 | Mean change from baseline to Week 52 in bone age was reported. A standard bone age measurement (of the hand/wrist area) was obtained at the beginning and the end of the trial to monitor for growth related safety concerns. Baseline was defined as the most recent measurement prior to the first administration of study drug. |
| Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | From Baseline to Week 52 | ASQ-3: developmental screening questionnaire that consists of 5 areas: communication, gross motor, fine motor, problem solving, and personal-social. Each area has 6 questions scored as Yes=10 points, Sometimes=5 points, and Not yet=0 points. A child can score between 0-60 points for each area with total score range: 0 to 300; higher scores are indicative of improvement. Total area score is then compared to age-adjusted standardized score cutoff (determined by developers of tool) which indicate whether child's development appears to be on schedule according to these categories: Below=Total analysis score (TAS) is below cutoff. Further assessment with professional may be needed; Monitor=TAS is close to cutoff. Provide learning activities and monitor; Above= TAS is above cutoff, and child's development appears to be on schedule. Shift from baseline for each developmental area of assessment according to these 3 outcome categories was reported. Total score was not applicable. |
| Mean Absolute Change From Baseline in BMI Z-score | Baseline, Week 52 | Mean absolute change from baseline to Week 52 in BMI Z-score was reported. BMI was calculated using participant's weight and height assessments, using the following formula: BMI = kg/m\^2. The BMI Z-scores were based on the World Health Organization's Child Growth Standards 2007 and indicated the number of standard deviations away from the mean. A Z-score of 0 was equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). A decrease of BMI Z-score (\< 0) indicated a reduction in BMI from Baseline whereas an increase of 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. |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | From first dose of study drug up to Week 56 | An adverse event (AE) was any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention. A TEAE was defined as any AE that started or worsened in intensity on or after the date of the first administration of study drug. |
| Number of Participants With TEAEs Graded by Severity | From first dose of study drug up to Week 56 | A TEAE was any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention. A TEAE was defined as any AE that started or worsened in intensity on or after the date of the first administration of study drug. TEAEs were graded according to Common Terminology Criteria for Adverse Events (CTCAE) criteria. Grade 1- Mild; Grade 2- Moderate; Grade 3- Severe; Grade 4- Life Threatening; Grade 5- Death related to AE. |
| Change From Baseline in Body Weight | Baseline, Week 52 | Change from baseline to Week 52 in body weight was reported. |
| Mean Change From Baseline in Percent of the 95th Percentile of BMI | Baseline, Week 52 | Mean change from baseline to Week 52 in percent of the 95th percentile of BMI was reported. 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 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. |
Countries
Australia, Spain, United Kingdom, United States
Participant flow
Pre-assignment details
Of the 13 participants screened, 12 participants were enrolled in the study.
Participants by arm
| Arm | Count |
|---|---|
| Setmelanotide: PPL Group Participants with POMC/PCSK1/LEPR biallelic mutations collectively referred to as PPL received setmelanotide at a dose of 0.5 mg QD via SC injection for 52 weeks. The dose was escalated by increments of 0.5 mg every 2 weeks, if tolerated, at the dose escalation visits (Weeks 2, 4, and 6) to a maximum dose of 0.5 to 2.0 mg QD with the maximum dose based on body weight. Following the last dose in this study, participants who were considered likely to benefit from continued setmelanotide treatment and who had completed this trial could be eligible to enter an open-label long-term extension (LTE) trial with setmelanotide. | 7 |
| Setmelanotide: BBS Group Participants with BBS received setmelanotide at a dose of 0.5 mg QD via SC injection for 52 weeks. The dose was escalated by increments of 0.5 mg every 2 weeks, if tolerated, at the dose escalation visits (Weeks 2, 4, and 6) to a maximum dose of 0.5 to 2.0 mg QD with the maximum dose based on body weight. Following the last dose in this study, participants who were considered likely to benefit from continued setmelanotide treatment and who had completed this trial could be eligible to enter an open-label LTE trial with setmelanotide. | 5 |
| Total | 12 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Lost to Follow-up | 1 | 0 |
Baseline characteristics
| Characteristic | Setmelanotide: PPL Group | Setmelanotide: BBS Group | Total |
|---|---|---|---|
| Age, Continuous | 3.4 years STANDARD_DEVIATION 0.53 | 3.8 years STANDARD_DEVIATION 1.3 | 3.6 years STANDARD_DEVIATION 0.9 |
| BMI | 34.347 kg/m^2 STANDARD_DEVIATION 7.0673 | 23.716 kg/m^2 STANDARD_DEVIATION 3.5184 | 29.918 kg/m^2 STANDARD_DEVIATION 7.8559 |
| Body Mass Index (BMI) Z-score | 10.749 z-score STANDARD_DEVIATION 3.84 | 4.233 z-score STANDARD_DEVIATION 1.0742 | 8.034 z-score STANDARD_DEVIATION 4.4408 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants | 0 Participants | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 4 Participants | 5 Participants | 9 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 2 Participants | 0 Participants | 2 Participants |
| Race/Ethnicity, Customized Race American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized Race Asian | 0 Participants | 1 Participants | 1 Participants |
| Race/Ethnicity, Customized Race Other | 2 Participants | 0 Participants | 2 Participants |
| Race/Ethnicity, Customized Race Unknown or Not Reported | 2 Participants | 0 Participants | 2 Participants |
| Race/Ethnicity, Customized Race White | 3 Participants | 4 Participants | 7 Participants |
| Sex: Female, Male Female | 2 Participants | 3 Participants | 5 Participants |
| Sex: Female, Male Male | 5 Participants | 2 Participants | 7 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 7 | 0 / 5 |
| other Total, other adverse events | 7 / 7 | 5 / 5 |
| serious Total, serious adverse events | 0 / 7 | 0 / 5 |
Outcome results
Primary
Mean Percent Change From Baseline in BMI
Mean percent change from baseline to Week 52 in BMI was reported. 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, Week 52
Population: Participants in the Safety Analysis Set with available data were analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Setmelanotide: PPL Group | Mean Percent Change From Baseline in BMI | -25.597 percent change | Standard Deviation 11.4911 |
| Setmelanotide: BBS Group | Mean Percent Change From Baseline in BMI | -9.719 percent change | Standard Deviation 8.8383 |
Primary
Percentage of Participants With Greater Than or Equal to (≥) 0.2 Reduction of BMI Z-Score From Baseline to Week 52
A responder was defined as a decrease from baseline to 52 weeks in the participant's BMI z-score of ≥0.2. BMI was calculated using participant's weight and height assessments, using the following formula: BMI = kg/m\^2. The BMI Z-scores were based on the World Health Organization's Child Growth Standards 2007 and indicated the number of standard deviations away from the mean. A Z-score of 0 was equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). A decrease of BMI Z-score (\< 0) indicated a reduction in BMI from Baseline whereas an increase of 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.
Time frame: Baseline up to Week 52
Population: The safety analysis set included all participants who received at least 1 dose of study drug.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Setmelanotide: PPL Group | Percentage of Participants With Greater Than or Equal to (≥) 0.2 Reduction of BMI Z-Score From Baseline to Week 52 | 85.7 percentage of participants |
| Setmelanotide: BBS Group | Percentage of Participants With Greater Than or Equal to (≥) 0.2 Reduction of BMI Z-Score From Baseline to Week 52 | 80.0 percentage of participants |
Secondary
Change From Baseline in Body Weight
Change from baseline to Week 52 in body weight was reported.
Time frame: Baseline, Week 52
Population: Participants in the Safety Analysis Set with available data were analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Setmelanotide: PPL Group | Change From Baseline in Body Weight | -7.139 kilograms | Standard Deviation 5.0834 |
| Setmelanotide: BBS Group | Change From Baseline in Body Weight | -0.327 kilograms | Standard Deviation 2.9572 |
Secondary
Mean Absolute Change From Baseline in BMI Z-score
Mean absolute change from baseline to Week 52 in BMI Z-score was reported. BMI was calculated using participant's weight and height assessments, using the following formula: BMI = kg/m\^2. The BMI Z-scores were based on the World Health Organization's Child Growth Standards 2007 and indicated the number of standard deviations away from the mean. A Z-score of 0 was equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). A decrease of BMI Z-score (\< 0) indicated a reduction in BMI from Baseline whereas an increase of 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.
Time frame: Baseline, Week 52
Population: Participants in the Safety Analysis Set with available data were analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Setmelanotide: PPL Group | Mean Absolute Change From Baseline in BMI Z-score | -5.185 Z-score | Standard Deviation 1.8585 |
| Setmelanotide: BBS Group | Mean Absolute Change From Baseline in BMI Z-score | -1.331 Z-score | Standard Deviation 1.2295 |
Secondary
Mean Change From Baseline in Bone Age
Mean change from baseline to Week 52 in bone age was reported. A standard bone age measurement (of the hand/wrist area) was obtained at the beginning and the end of the trial to monitor for growth related safety concerns. Baseline was defined as the most recent measurement prior to the first administration of study drug.
Time frame: Baseline, Week 52
Population: Participants in the Safety Analysis Set with available data were analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Setmelanotide: PPL Group | Mean Change From Baseline in Bone Age | 1.020 years | Standard Deviation 1.0733 |
| Setmelanotide: BBS Group | Mean Change From Baseline in Bone Age | 0.700 years | Standard Deviation 0.8367 |
Secondary
Mean Change From Baseline in Percent of the 95th Percentile of BMI
Mean change from baseline to Week 52 in percent of the 95th percentile of BMI was reported. 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 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.
Time frame: Baseline, Week 52
Population: Participants in the Safety Analysis Set with available data were analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Setmelanotide: PPL Group | Mean Change From Baseline in Percent of the 95th Percentile of BMI | -47.595 percentage relative to 95th percentile | Standard Deviation 17.328 |
| Setmelanotide: BBS Group | Mean Change From Baseline in Percent of the 95th Percentile of BMI | -14.462 percentage relative to 95th percentile | Standard Deviation 13.8571 |
Secondary
Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3)
ASQ-3: developmental screening questionnaire that consists of 5 areas: communication, gross motor, fine motor, problem solving, and personal-social. Each area has 6 questions scored as Yes=10 points, Sometimes=5 points, and Not yet=0 points. A child can score between 0-60 points for each area with total score range: 0 to 300; higher scores are indicative of improvement. Total area score is then compared to age-adjusted standardized score cutoff (determined by developers of tool) which indicate whether child's development appears to be on schedule according to these categories: Below=Total analysis score (TAS) is below cutoff. Further assessment with professional may be needed; Monitor=TAS is close to cutoff. Provide learning activities and monitor; Above= TAS is above cutoff, and child's development appears to be on schedule. Shift from baseline for each developmental area of assessment according to these 3 outcome categories was reported. Total score was not applicable.
Time frame: From Baseline to Week 52
Population: Participants in the Safety Analysis Set with available data were analyzed.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Communication | Baseline (Above): Week 52 (Above) | 5 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Communication | Baseline (Above): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Communication | Baseline (Above): Week 52 (Below) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Communication | Baseline (Monitor): Week 52 (Above) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Communication | Baseline (Monitor): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Communication | Baseline (Monitor): Week 52 (Below) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Communication | Baseline (Below): Week 52 (Above) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Communication | Baseline (Below): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Communication | Baseline (Below): Week 52 (Below) | 1 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Fine Motor | Baseline (Above): Week 52 (Above) | 4 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Fine Motor | Baseline (Above): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Fine Motor | Baseline (Above): Week 52 (Below) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Fine Motor | Baseline (Monitor): Week 52 (Above) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Fine Motor | Baseline (Monitor): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Fine Motor | Baseline (Monitor): Week 52 (Below) | 1 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Fine Motor | Baseline (Below): Week 52 (Above) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Fine Motor | Baseline (Below): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Fine Motor | Baseline (Below): Week 52 (Below) | 1 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Gross Motor | Baseline (Above): Week 52 (Above) | 1 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Gross Motor | Baseline (Above): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Gross Motor | Baseline (Above): Week 52 (Below) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Gross Motor | Baseline (Monitor): Week 52 (Above) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Gross Motor | Baseline (Monitor): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Gross Motor | Baseline (Monitor): Week 52 (Below) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Gross Motor | Baseline (Below): Week 52 (Above) | 3 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Gross Motor | Baseline (Below): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Gross Motor | Baseline (Below): Week 52 (Below) | 2 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Personal - Social | Baseline (Above): Week 52 (Above) | 3 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Personal - Social | Baseline (Above): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Personal - Social | Baseline (Above): Week 52 (Below) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Personal - Social | Baseline (Monitor): Week 52 (Above) | 2 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Personal - Social | Baseline (Monitor): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Personal - Social | Baseline (Monitor): Week 52 (Below) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Personal - Social | Baseline (Below): Week 52 (Above) | 1 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Personal - Social | Baseline (Below): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Personal - Social | Baseline (Below): Week 52 (Below) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Problem Solving | Baseline (Above): Week 52 (Above) | 4 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Problem Solving | Baseline (Above): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Problem Solving | Baseline (Above): Week 52 (Below) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Problem Solving | Baseline (Monitor): Week 52 (Above) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Problem Solving | Baseline (Monitor): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Problem Solving | Baseline (Monitor): Week 52 (Below) | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Problem Solving | Baseline (Below): Week 52 (Above) | 1 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Problem Solving | Baseline (Below): Week 52 (Monitor) | 1 Participants |
| Setmelanotide: PPL Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Problem Solving | Baseline (Below): Week 52 (Below) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Gross Motor | Baseline (Monitor): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Communication | Baseline (Above): Week 52 (Above) | 2 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Problem Solving | Baseline (Monitor): Week 52 (Above) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Communication | Baseline (Above): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Gross Motor | Baseline (Monitor): Week 52 (Below) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Communication | Baseline (Above): Week 52 (Below) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Personal - Social | Baseline (Below): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Communication | Baseline (Monitor): Week 52 (Above) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Gross Motor | Baseline (Below): Week 52 (Above) | 1 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Communication | Baseline (Monitor): Week 52 (Monitor) | 1 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Problem Solving | Baseline (Below): Week 52 (Above) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Communication | Baseline (Monitor): Week 52 (Below) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Gross Motor | Baseline (Below): Week 52 (Monitor) | 2 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Communication | Baseline (Below): Week 52 (Above) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Personal - Social | Baseline (Below): Week 52 (Below) | 2 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Communication | Baseline (Below): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Gross Motor | Baseline (Below): Week 52 (Below) | 2 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Communication | Baseline (Below): Week 52 (Below) | 2 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Problem Solving | Baseline (Monitor): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Fine Motor | Baseline (Above): Week 52 (Above) | 2 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Personal - Social | Baseline (Above): Week 52 (Above) | 2 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Fine Motor | Baseline (Above): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Problem Solving | Baseline (Above): Week 52 (Above) | 3 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Fine Motor | Baseline (Above): Week 52 (Below) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Personal - Social | Baseline (Above): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Fine Motor | Baseline (Monitor): Week 52 (Above) | 1 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Problem Solving | Baseline (Below): Week 52 (Below) | 2 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Fine Motor | Baseline (Monitor): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Personal - Social | Baseline (Above): Week 52 (Below) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Fine Motor | Baseline (Monitor): Week 52 (Below) | 1 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Problem Solving | Baseline (Above): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Fine Motor | Baseline (Below): Week 52 (Above) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Personal - Social | Baseline (Monitor): Week 52 (Above) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Fine Motor | Baseline (Below): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Problem Solving | Baseline (Monitor): Week 52 (Below) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Fine Motor | Baseline (Below): Week 52 (Below) | 1 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Personal - Social | Baseline (Monitor): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Gross Motor | Baseline (Above): Week 52 (Above) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Problem Solving | Baseline (Above): Week 52 (Below) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Gross Motor | Baseline (Above): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Personal - Social | Baseline (Monitor): Week 52 (Below) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Gross Motor | Baseline (Above): Week 52 (Below) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Problem Solving | Baseline (Below): Week 52 (Monitor) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Gross Motor | Baseline (Monitor): Week 52 (Above) | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) | Personal - Social | Baseline (Below): Week 52 (Above) | 1 Participants |
Secondary
Number of Participants With TEAEs Graded by Severity
A TEAE was any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention. A TEAE was defined as any AE that started or worsened in intensity on or after the date of the first administration of study drug. TEAEs were graded according to Common Terminology Criteria for Adverse Events (CTCAE) criteria. Grade 1- Mild; Grade 2- Moderate; Grade 3- Severe; Grade 4- Life Threatening; Grade 5- Death related to AE.
Time frame: From first dose of study drug up to Week 56
Population: Participants in the Safety Analysis Set were analyzed.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Setmelanotide: PPL Group | Number of Participants With TEAEs Graded by Severity | Moderate | 5 Participants |
| Setmelanotide: PPL Group | Number of Participants With TEAEs Graded by Severity | Life Threatening | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With TEAEs Graded by Severity | Severe | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With TEAEs Graded by Severity | Death | 0 Participants |
| Setmelanotide: PPL Group | Number of Participants With TEAEs Graded by Severity | Mild | 2 Participants |
| Setmelanotide: BBS Group | Number of Participants With TEAEs Graded by Severity | Death | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With TEAEs Graded by Severity | Mild | 5 Participants |
| Setmelanotide: BBS Group | Number of Participants With TEAEs Graded by Severity | Moderate | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With TEAEs Graded by Severity | Severe | 0 Participants |
| Setmelanotide: BBS Group | Number of Participants With TEAEs Graded by Severity | Life Threatening | 0 Participants |
Secondary
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
An adverse event (AE) was any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention. A TEAE was defined as any AE that started or worsened in intensity on or after the date of the first administration of study drug.
Time frame: From first dose of study drug up to Week 56
Population: Participants in the Safety Analysis Set were analyzed.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Setmelanotide: PPL Group | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | 7 Participants |
| Setmelanotide: BBS Group | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | 5 Participants |