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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).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Setmelanotide: PPL Group | Experimental | Participants with POMC)/PCSK1/LEPR biallelic mutations collectively referred to as PPL received setmelanotide at a dose of 0.5 milligrams (mg) per day (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. |
|
| Setmelanotide: BBS Group | Experimental | 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 long-term extension (LTE) trial with setmelanotide. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Setmelanotide | Drug | SC injection once daily. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline up to Week 52 |
| 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. | Baseline, Week 52 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Key Inclusion Criteria:
Participants must have obesity due to either:
Age between 2 to <6 years at the time of informed consent
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.
Symptoms or behaviors of hyperphagia
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
Other protocol defined Inclusion/Exclusion criteria may apply.
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| Name | Affiliation | Role |
|---|---|---|
| David Meeker, MD | Rhythm Pharmaceuticals, Inc. | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States | ||
| Columbia University Medical Center, Division of Pediatric Endocrinology, Diabetes and Metabolism |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39549719 | Derived | Argente J, Verge CF, Okorie U, Fennoy I, Kelsey MM, Cokkinias C, Scimia C, Lee HM, Farooqi IS. Setmelanotide in patients aged 2-5 years with rare MC4R pathway-associated obesity (VENTURE): a 1 year, open-label, multicenter, phase 3 trial. Lancet Diabetes Endocrinol. 2025 Jan;13(1):29-37. doi: 10.1016/S2213-8587(24)00273-0. Epub 2024 Nov 13. |
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Of the 13 participants screened, 12 participants were enrolled in the study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Setmelanotide: PPL Group | Participants with pro-opiomelanocortin (POMC)/proprotein convertase subtilisin/kexin type 1 (PCSK1)/leptin receptor (LEPR) biallelic mutations collectively referred to as PPL received setmelanotide at a dose of 0.5 milligrams (mg) per day (QD) via subcutaneous (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. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 22, 2023 | Jun 13, 2024 |
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| Baseline, Week 52 |
| 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. | Baseline, Week 52 |
| 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. | Baseline, Week 52 |
| 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. | From Baseline to Week 52 |
| Change From Baseline in Body Weight | Change from baseline to Week 52 in body weight was reported. | Baseline, Week 52 |
| 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. | From first dose of study drug up to Week 56 |
| 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. | From first dose of study drug up to Week 56 |
| New York |
| New York |
| 10032 |
| United States |
| Marshfield Clinic Research Foundation | Marshfield | Wisconsin | 54449 | United States |
| Sydney Children's Hospital | Randwick | NSW 2031 | Australia |
| Hospital Infantil Niño Jesus | Madrid | 28009 | Spain |
| Addenbrooke's Hospital, Wellcome Trust-MRC Institute of Metabolic Science | Cambridge | CB2 0QQ | United Kingdom |
| FG001 | Setmelanotide: BBS Group | Participants with Bardet-Biedl syndrome (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. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
The safety analysis set included all participants who received at least 1 dose of study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| BG001 | 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. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||
| Body Mass Index (BMI) Z-score | BMI Z-scores were based on the World Health Organization's Child Growth Standards 2007. The BMI Z-score 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. | Mean | Standard Deviation | z-score |
| ||||||||||||||
| BMI | BMI was calculated using participant's weight and height assessments, using the following formula: BMI = kilograms/ square meter (kg/m^2). | Mean | Standard Deviation | kg/m^2 |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | The safety analysis set included all participants who received at least 1 dose of study drug. | Posted | Number | 95% Confidence Interval | percentage of participants | Baseline up to Week 52 |
|
|
| ||||||||||||||||||||||||||||
| 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. | Participants in the Safety Analysis Set with available data were analyzed. | Posted | Mean | Standard Deviation | percent change | Baseline, Week 52 |
| ||||||||||||||||||||||||||||||
| 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. | Participants in the Safety Analysis Set with available data were analyzed. | Posted | Mean | Standard Deviation | Z-score | Baseline, Week 52 |
| ||||||||||||||||||||||||||||||
| 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. | Participants in the Safety Analysis Set with available data were analyzed. | Posted | Mean | Standard Deviation | percentage relative to 95th percentile | Baseline, Week 52 |
| ||||||||||||||||||||||||||||||
| 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. | Participants in the Safety Analysis Set with available data were analyzed. | Posted | Mean | Standard Deviation | years | Baseline, Week 52 |
| ||||||||||||||||||||||||||||||
| 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. | Participants in the Safety Analysis Set with available data were analyzed. | Posted | Count of Participants | Participants | No | From Baseline to Week 52 |
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Body Weight | Change from baseline to Week 52 in body weight was reported. | Participants in the Safety Analysis Set with available data were analyzed. | Posted | Mean | Standard Deviation | kilograms | Baseline, Week 52 |
|
| |||||||||||||||||||||||||||||
| 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. | Participants in the Safety Analysis Set were analyzed. | Posted | Count of Participants | Participants | From first dose of study drug up to Week 56 |
| |||||||||||||||||||||||||||||||
| 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. | Participants in the Safety Analysis Set were analyzed. | Posted | Count of Participants | Participants | From first dose of study drug up to Week 56 |
|
From first dose of study drug up to Week 56
The safety analysis set included all participants who received at least 1 dose of study drug.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Setmelanotide: PPL Group | Participants with POMC/PCSK1/LEPR biallelic mutations collectively referred 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. | 0 | 7 | 0 | 7 | 7 | 7 |
| EG001 | 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. | 0 | 5 | 0 | 5 | 5 | 5 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nasopharyngitis | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Skin candida | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Skin hyperpigmentation | Skin and subcutaneous tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Injection site bruising | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Injection site pruritus | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Injection site discoloration | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Injection site erythema | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Appetite disorder | Metabolism and nutrition disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Polydipsia | Metabolism and nutrition disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Melanocytic naevus | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.1 | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA 26.1 | Systematic Assessment |
|
All information regarding setmelanotide supplied by Rhythm to the investigator is privileged and confidential information. The investigator agrees to use this information to accomplish the study and will not use it for other purposes without consent from Rhythm. The information obtained from the clinical study will be used towards the development of setmelanotide and may be disclosed to regulatory authority(ies), other investigators, corporate partners, or consultants as required.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Rhythm Clinical Trials | Rhythm Pharmaceuticals, Inc. | 857-264-4280 | clinicaltrials@rhythmtx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Aug 21, 2023 | Jun 13, 2024 | SAP_001.pdf |
| ID | Term |
|---|---|
| D020788 | Bardet-Biedl Syndrome |
| C565726 | Proopiomelanocortin Deficiency |
| D009765 | Obesity |
| D063766 | Pediatric Obesity |
| ID | Term |
|---|---|
| D007027 | Hypothalamic Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D012174 | Retinitis Pigmentosa |
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D000072661 | Ciliopathies |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
| D050177 | Overweight |
| D044343 | Overnutrition |
| D009748 | Nutrition Disorders |
| D009750 | Nutritional and Metabolic Diseases |
| D001835 | Body Weight |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| C579663 | setmelanotide |
Not provided
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| White |
|
| Other |
|
| Unknown or Not Reported |
|
| Units |
|---|
| Counts |
|---|
| Participants |
|
|
|
|
|
|
| Units | Counts |
|---|
| Participants |
|
|
| OG001 | 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. |
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
| Baseline (Above): Week 52 (Below) |
|
| Baseline (Monitor): Week 52 (Above) |
|
| Baseline (Monitor): Week 52 (Monitor) |
|
| Baseline (Monitor): Week 52 (Below) |
|
| Baseline (Below): Week 52 (Above) |
|
| Baseline (Below): Week 52 (Monitor) |
|
| Baseline (Below): Week 52 (Below) |
|
| Baseline (Above): Week 52 (Below) |
|
| Baseline (Monitor): Week 52 (Above) |
|
| Baseline (Monitor): Week 52 (Monitor) |
|
| Baseline (Monitor): Week 52 (Below) |
|
| Baseline (Below): Week 52 (Above) |
|
| Baseline (Below): Week 52 (Monitor) |
|
| Baseline (Below): Week 52 (Below) |
|
| Baseline (Above): Week 52 (Below) |
|
| Baseline (Monitor): Week 52 (Above) |
|
| Baseline (Monitor): Week 52 (Monitor) |
|
| Baseline (Monitor): Week 52 (Below) |
|
| Baseline (Below): Week 52 (Above) |
|
| Baseline (Below): Week 52 (Monitor) |
|
| Baseline (Below): Week 52 (Below) |
|
| Baseline (Above): Week 52 (Below) |
|
| Baseline (Monitor): Week 52 (Above) |
|
| Baseline (Monitor): Week 52 (Monitor) |
|
| Baseline (Monitor): Week 52 (Below) |
|
| Baseline (Below): Week 52 (Above) |
|
| Baseline (Below): Week 52 (Monitor) |
|
| Baseline (Below): Week 52 (Below) |
|