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A trial to compare the weekly and daily formulations of setmelanotide in participants with genetic defects in the melanocortin-4 receptor pathway.
This study is designed to compare the safety, pharmacokinetics, and efficacy of weekly and daily formulations of setmelanotide in participants with obesity associated with biallelic or heterozygous POMC (pro-opiomelanocortin), PCSK1 (proprotein convertase subtilisin/kexin Type 1), LEPR (leptin receptor) genetic variants, and participants with Bardet-Biedl Syndrome (BBS).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Run-in Period: Setmelanotide 2 mg QD | Experimental | Participants received subcutaneous (SC) injection of 2 mg setmelanotide once daily (QD) for 1 week in the run-in period. |
|
| Run-in Period: Setmelanotide 2.5 mg QD | Experimental | Participants received SC injection of 2.5 mg setmelanotide QD for 1 week in the run-in period. |
|
| Run-in Period: Setmelanotide 3 mg QD | Experimental | Participants received SC injection of 3 mg setmelanotide QD for 1 week in the run-in period. |
|
| DB Period: Setmelanotide 20 mg QW | Experimental | Participants who received 2 mg setmelanotide QD in the run-in period, received SC injection of 20 mg setmelanotide once weekly (QW) and placebo matched to setmelanotide QD for 13 weeks in the DB period. |
|
| DB Period: Setmelanotide 25 mg QW | Experimental | Participants who received 2.5 mg setmelanotide QD in the run-in period, received SC injection of 25 mg setmelanotide QW and placebo matched to setmelanotide QD for 13 weeks in the DB period. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Setmelanotide 2 mg | Drug | Administered as SC injection |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Maximum Drug Concentration (Cmax) of Setmelanotide After QD Administration in the Run-in Period | Maximum drug concentration determined directly from individual concentration-time data. | Pre-dose (0 hour) and at 0.5, 1, 2, 3, 4, 6, 8, 12, and 24 hours postdose at Week -1 |
| Cmax of Setmelanotide After QW Administration | Maximum drug concentration determined directly from individual concentration-time data. Data are reported by dose level (treatment regimen) in the OL Period and dosing sequence (QD-QD-QW or QD-QW-QW). | Pre-dose (0 hour) and at 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 120, and 168 hours postdose at Week 14 |
| Time to Maximum Plasma Concentration (Tmax) of Setmelanotide After QD Administration in the Run-in Period | Maximum drug concentration determined directly from individual concentration-time data. | Pre-dose (0 hour) and at 0.5, 1, 2, 3, 4, 6, 8, 12, and 24 hours postdose at Week -1 |
| Tmax of Setmelanotide After QW Administration | Maximum drug concentration determined directly from individual concentration-time data. Data are reported by dose level (treatment regimen) in the OL Period and dosing sequence (QD-QD-QW or QD-QW-QW). | Pre-dose (0 hour) and at 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 120, and 168 hours postdose at Week 14 |
| Mean Trough Plasma Concentration (Ctrough) of Setmelanotide After QD or QW Administration at Week 1 | Ctrough is concentration at the end of the dosing interval, prior to subsequent dose administration. Data are reported by dose level (treatment regimen) in the DB Period and dosing sequence (QD-QD-QW or QD-QW-QW). | 30 minutes predose at Week 1 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | An adverse event (AE) is any untoward medical occurrence in a participant or clinical trial participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. The AEs reported after the start of the run-in period were considered TEAEs. |
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Key Inclusion Criteria:
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 |
|---|---|---|---|---|---|---|
| Honor Health Research Institute | Scottsdale | Arizona | 85258 | United States | ||
| Marshfield Clinic Research Institute |
A total of 20 participants were screened and 19 were randomized and treated in this study. The study consisted of a run-in period of up to 1 week in which the participants received once daily (QD) setmelanotide [2 milligrams (mg), 2.5 mg, or 3 mg]; a 13-week double-blind period in which the participants were randomized to either QD or once weekly (QW) setmelanotide; and a 13-week non-randomized open-label period where all participants received open label QW setmelanotide.
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| ID | Title | Description |
|---|---|---|
| FG000 | Run-in Period: Setmelanotide 2 mg QD | Participants received subcutaneous (SC) injection of 2 mg setmelanotide QD for 1 week in the run-in period. |
| FG001 | Run-in Period: Setmelanotide 2.5 mg QD |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Run-in Period (1 Week) |
|
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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 | Jul 7, 2022 | Sep 26, 2024 |
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|
| DB Period: Setmelanotide 3 mg QD | Experimental | Participants who received 3 mg setmelanotide QD in the run-in period, received SC injection of 3 mg setmelanotide QD and placebo matched to setmelanotide QW for 13 weeks in the DB period. |
|
| DB Period: Setmelanotide 30 mg QW | Experimental | Participants who received 3 mg setmelanotide QD in the run-in period, received SC injection of 30 mg setmelanotide QW and placebo matched to setmelanotide QD for 13 weeks in the DB period. |
|
| OL Period: Setmelanotide 20 mg QW | Experimental | Participants who received 2 mg setmelanotide QD in the run-in period and 20 mg setmelanotide QW in the DB period, received SC injection of 20 mg setmelanotide QW for 13 weeks in the OL period. |
|
| OL Period: Setmelanotide 25 mg QW | Experimental | Participants who received 2.5 mg setmelanotide QD in the run-in period and 25 mg setmelanotide QW in the DB period, received SC injection of 25 mg setmelanotide QW for 13 weeks in the OL period. |
|
| OL Period: Setmelanotide 30 mg QW | Experimental | Participants who received 3 setmelanotide QD in the run-in period and 3 mg setmelanotide QD or 30 mg setmelanotide QW in the DB period, received SC injection of 30 mg setmelanotide QW for 13 weeks in the OL period. |
|
| Setmelanotide 2.5 mg |
| Drug |
Administered as SC injection |
|
| Setmelanotide 3 mg | Drug | Administered as SC injection |
|
| Setmelanotide 20 mg | Drug | Administered as SC injection |
|
| Setmelanotide 25 mg | Drug | Administered as SC injection |
|
| Setmelanotide 30 mg | Drug | Administered as SC injection |
|
| Mean Setmelanotide Ctrough After QD or QW Administration at Week 5 |
Ctrough is concentration at the end of the dosing interval, prior to subsequent dose administration. Data are reported by dose level (treatment regimen) in the DB Period and dosing sequence (QD-QD-QW or QD-QW-QW). |
| 30 minutes predose at Week 5 |
| Mean Setmelanotide Ctrough After QD or QW Administration at Week 9 | Ctrough is concentration at the end of the dosing interval, prior to subsequent dose administration. Data are reported by dose level (treatment regimen) in the DB Period and dosing sequence (QD-QD-QW or QD-QW-QW). | 30 minutes predose at Week 9 |
| Mean Setmelanotide Ctrough After QD or QW Administration at Week 18 | Ctrough is concentration at the end of the dosing interval, prior to subsequent dose administration. Data are reported by dose level (treatment regimen) in the DB Period and dosing sequence (QD-QD-QW or QD-QW-QW). | 30 minutes predose at Week 18 |
| Mean Setmelanotide Ctrough After QD or QW Administration at Week 22 | Ctrough is concentration at the end of the dosing interval, prior to subsequent dose administration. Data are reported by dose level (treatment regimen) in the DB Period and dosing sequence (QD-QD-QW or QD-QW-QW). | 30 minutes predose at Week 22 |
| Mean Setmelanotide Ctrough After QD or QW Administration at Week 27 | Ctrough is concentration at the end of the dosing interval, prior to subsequent dose administration. Data are reported by dose level (treatment regimen) in the DB Period and dosing sequence (QD-QD-QW or QD-QW-QW). | 30 minutes predose at Week 27 |
| Area Under the Plasma Concentration-Time Curve Over the Dosing Interval (AUC0-tau) of Setmelanotide After QD Administration in the Run-in Period | AUC0-tau was recorded from collected blood samples. | Pre-dose (0 hour) and at 0.5, 1, 2, 3, 4, 6, and 8 hours postdose at Week -1 |
| AUC0-tau of Setmelanotide After QD Administration in the Run-in Period | AUC0-tau was recorded from collected blood samples. Data are reported by dose level (treatment regimen) in the OL Period and dosing sequence (QD-QD-QW or QD-QW-QW). | Pre-dose (0 hour) and 0.5, 1, 2, 6, 8, 12, 24, 48, 72, 96, 120, and 168-hours postdose at Week 14 |
| From first dose of study drug administration up to Week 30 |
| Number of Participants With Injection Site Reactions (ISRs) From Baseline Through Week 13 | The injection site evaluation included the identification of areas of erythema, edema, and induration, as well as the presence of localized pain, tenderness, and itching. Baseline was defined as the last available measurement prior to the first dose of setmelanotide or placebo. Injection site reactions frequency of once daily (QD) and once weekly (QW) were reported. Data are reported by dose level (treatment regimen) in the DB Period. Number of participants with injection site reactions according to severity were reported. | Baseline through Week 13 |
| Number of Participants With ISRs From Week 14 Through Week 27 | The injection site evaluation included the identification of areas of erythema, edema, and induration, as well as the presence of localized pain, tenderness, and itching. Injection site reactions frequency of QD and QW were reported. Data are reported by dose level (treatment regimen) in the OL Period. Number of participants with injection site reactions according to severity were reported. | Week 14 through Week 27 |
| Marshfield |
| Wisconsin |
| 54449 |
| United States |
| Alberta Health Services | Edmonton | Alberta | T6G 2E1 | Canada |
| Charité - Universitätsmedizin Berlin, Campus Virchow-Klinikum | Berlin | 13353 | Germany |
| Erasmus MC | Rotterdam | 3015 CE | Netherlands |
| UPR Medical Sciences Campus | Rio Piedras | 00935 | Puerto Rico |
| Addenbrooke's Hospital | Cambridge | CA2 0QQ | United Kingdom |
Participants received SC injection of 2.5 mg setmelanotide QD for 1 week in the run-in period.
| FG002 | Run-in Period: Setmelanotide 3 mg QD | Participants received SC injection of 3 mg setmelanotide QD for 1 week in the run-in period. |
| FG003 | DB Period: Setmelanotide 20 mg QW | Participants who received 2 mg setmelanotide QD in the run-in period, received SC injection of 20 mg setmelanotide QW and placebo matched to setmelanotide QD for 13 weeks in the DB period. |
| FG004 | DB Period: Setmelanotide 25 mg QW | Participants who received 2.5 mg setmelanotide QD in the run-in period, received SC injection of 25 mg setmelanotide QW and placebo matched to setmelanotide QD for 13 weeks in the DB period. |
| FG005 | DB Period: Setmelanotide 3 mg QD | Participants who received 3 mg setmelanotide QD in the run-in period, received SC injection of 3 mg setmelanotide QD and placebo matched to setmelanotide QW for 13 weeks in the DB period. |
| FG006 | DB Period: Setmelanotide 30 mg QW | Participants who received 3 mg setmelanotide QD in the run-in period, received SC injection of 30 mg setmelanotide QW and placebo matched to setmelanotide QD for 13 weeks in the DB period. |
| FG007 | OL Period: Setmelanotide 20 mg QW | Participants who received 2 mg setmelanotide QD in the run-in period and 20 mg setmelanotide QW in the DB period, received SC injection of 20 mg setmelanotide QW for 13 weeks in the OL period. |
| FG008 | OL Period: Setmelanotide 25 mg QW | Participants who received 2.5 mg setmelanotide QD in the run-in period and 25 mg setmelanotide QW in the DB period, received SC injection of 25 mg setmelanotide QW for 13 weeks in the OL period. |
| FG009 | OL Period: Setmelanotide 30 mg QW | Participants who received 3 setmelanotide QD in the run-in period and 3 mg setmelanotide QD or 30 mg setmelanotide QW in the DB period, received SC injection of 30 mg setmelanotide QW for 13 weeks in the OL period. |
| COMPLETED |
|
| NOT COMPLETED |
|
| Double Blind (DB) Period (13 Weeks) |
|
|
| Open Label (OL) Period (13 Weeks) |
|
|
Full Analysis Set (FAS) included all participants who received at least 1 dose of setmelanotide after the randomization.
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| ID | Title | Description |
|---|---|---|
| BG000 | Setmelanotide 2 mg QD | Run-in Period: Participants received SC injection of 2 mg setmelanotide QD for 1 week. DB Period: Participants received SC injection of 20 mg setmelanotide QW and placebo matched to setmelanotide QD for 13 weeks. OL Period: Participants received SC injection of 20 mg setmelanotide QW for 13 weeks. |
| BG001 | Setmelanotide 2.5 mg QD | Run-in Period: Participants received SC injection of 2.5 mg setmelanotide QD for 1 week. DB Period: Participants received SC injection of 25 mg setmelanotide QW and placebo matched to setmelanotide QD for 13 weeks. OL Period: Participants received SC injection of 25 mg setmelanotide QW for 13 weeks. |
| BG002 | Setmelanotide 3 mg QD | Run-in Period: Participants received SC injection of 3 mg setmelanotide QD for 1 week. DB Period: Participants received SC injection of 3 mg setmelanotide QD and placebo matched to setmelanotide QW or 30 mg setmelanotide QW and placebo matched to setmelanotide QD for 13 weeks. OL Period: Participants received SC injection of 30 mg setmelanotide QW for 13 weeks. |
| BG003 | 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 |
|
| 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 | Maximum Drug Concentration (Cmax) of Setmelanotide After QD Administration in the Run-in Period | Maximum drug concentration determined directly from individual concentration-time data. | Participants in the Pharmacokinetic Analysis Set (PKAS) (all participants who received at least 1 dose of setmelanotide and who had a sufficient number of measurable plasma concentrations to permit assessment of noncompartmental parameters) with available data were analyzed. | Posted | Mean | Standard Deviation | nanograms per milliliter (ng/mL) | Pre-dose (0 hour) and at 0.5, 1, 2, 3, 4, 6, 8, 12, and 24 hours postdose at Week -1 |
|
|
| |||||||||||||||||||||||||||||||
| Primary | Cmax of Setmelanotide After QW Administration | Maximum drug concentration determined directly from individual concentration-time data. Data are reported by dose level (treatment regimen) in the OL Period and dosing sequence (QD-QD-QW or QD-QW-QW). | Participants in the PKAS with available data were analyzed. | Posted | Mean | Standard Deviation | ng/mL | Pre-dose (0 hour) and at 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 120, and 168 hours postdose at Week 14 |
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| Primary | Time to Maximum Plasma Concentration (Tmax) of Setmelanotide After QD Administration in the Run-in Period | Maximum drug concentration determined directly from individual concentration-time data. | Participants in the PKAS with available data were analyzed. | Posted | Median | Full Range | hours | Pre-dose (0 hour) and at 0.5, 1, 2, 3, 4, 6, 8, 12, and 24 hours postdose at Week -1 |
|
| ||||||||||||||||||||||||||||||||
| Primary | Tmax of Setmelanotide After QW Administration | Maximum drug concentration determined directly from individual concentration-time data. Data are reported by dose level (treatment regimen) in the OL Period and dosing sequence (QD-QD-QW or QD-QW-QW). | Participants in the PKAS with available data were analyzed. | Posted | Median | Full Range | hours | Pre-dose (0 hour) and at 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 120, and 168 hours postdose at Week 14 |
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| Primary | Mean Trough Plasma Concentration (Ctrough) of Setmelanotide After QD or QW Administration at Week 1 | Ctrough is concentration at the end of the dosing interval, prior to subsequent dose administration. Data are reported by dose level (treatment regimen) in the DB Period and dosing sequence (QD-QD-QW or QD-QW-QW). | Participants in the PKAS with available data were analyzed. | Posted | Mean | Standard Deviation | ng/mL | 30 minutes predose at Week 1 |
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| Primary | Mean Setmelanotide Ctrough After QD or QW Administration at Week 5 | Ctrough is concentration at the end of the dosing interval, prior to subsequent dose administration. Data are reported by dose level (treatment regimen) in the DB Period and dosing sequence (QD-QD-QW or QD-QW-QW). | Participants in the PKAS with available data were analyzed. | Posted | Mean | Standard Deviation | ng/mL | 30 minutes predose at Week 5 |
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| Primary | Mean Setmelanotide Ctrough After QD or QW Administration at Week 9 | Ctrough is concentration at the end of the dosing interval, prior to subsequent dose administration. Data are reported by dose level (treatment regimen) in the DB Period and dosing sequence (QD-QD-QW or QD-QW-QW). | Participants in the PKAS with available data were analyzed. | Posted | Mean | Standard Deviation | ng/mL | 30 minutes predose at Week 9 |
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| Primary | Mean Setmelanotide Ctrough After QD or QW Administration at Week 18 | Ctrough is concentration at the end of the dosing interval, prior to subsequent dose administration. Data are reported by dose level (treatment regimen) in the DB Period and dosing sequence (QD-QD-QW or QD-QW-QW). | Participants in the PKAS with available data were analyzed. | Posted | Mean | Standard Deviation | ng/mL | 30 minutes predose at Week 18 |
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| Primary | Mean Setmelanotide Ctrough After QD or QW Administration at Week 22 | Ctrough is concentration at the end of the dosing interval, prior to subsequent dose administration. Data are reported by dose level (treatment regimen) in the DB Period and dosing sequence (QD-QD-QW or QD-QW-QW). | Participants in the PKAS with available data were analyzed. | Posted | Mean | Standard Deviation | ng/mL | 30 minutes predose at Week 22 |
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| Primary | Mean Setmelanotide Ctrough After QD or QW Administration at Week 27 | Ctrough is concentration at the end of the dosing interval, prior to subsequent dose administration. Data are reported by dose level (treatment regimen) in the DB Period and dosing sequence (QD-QD-QW or QD-QW-QW). | Participants in the PKAS with available data were analyzed. | Posted | Mean | Standard Deviation | ng/mL | 30 minutes predose at Week 27 |
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| Primary | Area Under the Plasma Concentration-Time Curve Over the Dosing Interval (AUC0-tau) of Setmelanotide After QD Administration in the Run-in Period | AUC0-tau was recorded from collected blood samples. | Participants in the PKAS with available data were analyzed. | Posted | Mean | Standard Deviation | hours*ng/mL | Pre-dose (0 hour) and at 0.5, 1, 2, 3, 4, 6, and 8 hours postdose at Week -1 |
|
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| Primary | AUC0-tau of Setmelanotide After QD Administration in the Run-in Period | AUC0-tau was recorded from collected blood samples. Data are reported by dose level (treatment regimen) in the OL Period and dosing sequence (QD-QD-QW or QD-QW-QW). | Participants in the PKAS with available data were analyzed. | Posted | Mean | Standard Deviation | hours*mg/mL | Pre-dose (0 hour) and 0.5, 1, 2, 6, 8, 12, 24, 48, 72, 96, 120, and 168-hours postdose at Week 14 |
| |||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | An adverse event (AE) is any untoward medical occurrence in a participant or clinical trial participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. The AEs reported after the start of the run-in period were considered TEAEs. | Participants in the Safety Analysis Set (all participants who received at least 1 dose of protocol-specified study drug) were analyzed. | Posted | Count of Participants | Participants | From first dose of study drug administration up to Week 30 |
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| Secondary | Number of Participants With Injection Site Reactions (ISRs) From Baseline Through Week 13 | The injection site evaluation included the identification of areas of erythema, edema, and induration, as well as the presence of localized pain, tenderness, and itching. Baseline was defined as the last available measurement prior to the first dose of setmelanotide or placebo. Injection site reactions frequency of once daily (QD) and once weekly (QW) were reported. Data are reported by dose level (treatment regimen) in the DB Period. Number of participants with injection site reactions according to severity were reported. | Participants in the Safety Analysis Set with available data were analyzed. | Posted | Count of Participants | Participants | Baseline through Week 13 |
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| Secondary | Number of Participants With ISRs From Week 14 Through Week 27 | The injection site evaluation included the identification of areas of erythema, edema, and induration, as well as the presence of localized pain, tenderness, and itching. Injection site reactions frequency of QD and QW were reported. Data are reported by dose level (treatment regimen) in the OL Period. Number of participants with injection site reactions according to severity were reported. | Participants in the Safety Analysis Set with available data were analyzed. | Posted | Count of Participants | Participants | Week 14 through Week 27 |
|
From first dose of study drug administration up to week 30
Safety Analysis Set included all participants who received at least 1 dose of protocol-specified study drug.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Run-in Period: Setmelanotide 2 mg QD | Participants received SC injection of 2 mg setmelanotide QD for 1 week in the run-in period. | 0 | 2 | 0 | 2 | 0 | 2 |
| EG001 | Run-in Period: Setmelanotide 2.5 mg QD | Participants received SC injection of 2.5 mg setmelanotide QD for 1 week in the run-in period. | 0 | 1 | 0 | 1 | 0 | 1 |
| EG002 | Run-in Period: Setmelanotide 3 mg QD | Participants received SC injection of 3 mg setmelanotide QD for 1 week in the run-in period. | 0 | 16 | 0 | 16 | 1 | 16 |
| EG003 | DB Period: Setmelanotide 20 mg QW | Participants who received 2 mg setmelanotide QD in the run-in period, received SC injection of 20 mg setmelanotide QW and placebo matched to setmelanotide QD for 13 weeks in the DB period. | 0 | 2 | 0 | 2 | 2 | 2 |
| EG004 | DB Period: Setmelanotide 25 mg QW | Participants who received 2.5 mg setmelanotide QD in the run-in period, received SC injection of 25 mg setmelanotide QW and placebo matched to setmelanotide QD for 13 weeks in the DB period. | 0 | 1 | 0 | 1 | 1 | 1 |
| EG005 | DB Period: Setmelanotide 3 mg QD | Participants who received 3 mg setmelanotide QD in the run-in period, received SC injection of 3 mg setmelanotide QD and placebo matched to setmelanotide QW for 13 weeks in the DB period. | 0 | 9 | 0 | 9 | 9 | 9 |
| EG006 | DB Period: Setmelanotide 30 mg QW | Participants who received 3 mg setmelanotide QD in the run-in period, received SC injection of 30 mg setmelanotide QW and placebo matched to setmelanotide QD for 13 weeks in the DB period. | 0 | 7 | 0 | 7 | 6 | 7 |
| EG007 | OL Period: Setmelanotide 20 mg QW | Participants who received 2 mg setmelanotide QD in the run-in period and 20 mg setmelanotide QW in the DB period, received SC injection of 20 mg setmelanotide QW for 13 weeks in the OL period. | 0 | 1 | 0 | 1 | 0 | 1 |
| EG008 | OL Period: Setmelanotide 25 mg QW | Participants who received 2.5 mg setmelanotide QD in the run-in period and 25 mg setmelanotide QW in the DB period, received SC injection of 25 mg setmelanotide QW for 13 weeks in the OL period. | 0 | 1 | 0 | 1 | 1 | 1 |
| EG009 | OL Period: Setmelanotide 30 mg QW | Participants who received 3 setmelanotide QD in the run-in period and 30 mg setmelanotide QD or QW in the DB period, received SC injection of 30 mg setmelanotide QW for 13 weeks in the OL period. | 0 | 14 | 0 | 14 | 10 | 14 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Injection site erythema | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Injection site induration | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Injection site haemorrhage | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Injection site mass | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Injection site pruritus | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Injection site bruising | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Injection site inflammation | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Tinea versicolour | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Subcutaneous abscess | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Skin hyperpigmentation | Skin and subcutaneous tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Meniscus injury | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Dry throat | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Appetite disorder | Metabolism and nutrition disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Melanocytic naevus | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Sleep paralysis | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Mood altered | Psychiatric disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Depressed mood | Psychiatric disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Libido decreased | Psychiatric disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Menstruation irregular | Reproductive system and breast disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Food allergy | Immune system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA 26.1 | Systematic Assessment |
|
Not provided
Not provided
| 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 | Jul 24, 2023 | Sep 26, 2024 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D020788 | Bardet-Biedl Syndrome |
| C565726 | Proopiomelanocortin Deficiency |
| D006963 | Hyperphagia |
| 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 |
| D012817 | Signs and Symptoms, Digestive |
| 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 |
|
| White |
|
| Other |
|
| OG003 | Setmelanotide 30 mg QW (QD-QW-QW) | Participants who received 3 mg setmelanotide QD in the run-in period and 30 mg setmelanotide QW in the DB period, received SC injection of 30 mg setmelanotide QW for 13 weeks in the OL period. |
|
|
|
| OG003 | Setmelanotide 30 mg QW (QD-QW-QW) | Participants who received 3 mg setmelanotide QD in the run-in period and 30 mg setmelanotide QW in the DB period, received SC injection of 30 mg setmelanotide QW for 13 weeks in the OL period. |
|
|
| OG003 | Setmelanotide 30 mg QW (QD-QW-QW) | Participants who received 3 mg setmelanotide QD in the run-in period and 30 mg setmelanotide QW in the DB period, received SC injection of 30 mg setmelanotide QW for 13 weeks in the OL period. |
|
|
| OG003 |
| Setmelanotide 30 mg QW (QD-QW-QW) |
Participants who received 3 mg setmelanotide QD in the run-in period and 30 mg setmelanotide QW in the DB period, received SC injection of 30 mg setmelanotide QW for 13 weeks in the OL period. |
|
|
| OG003 |
| Setmelanotide 30 mg QW (QD-QW-QW) |
Participants who received 3 mg setmelanotide QD in the run-in period and 30 mg setmelanotide QW in the DB period, received SC injection of 30 mg setmelanotide QW for 13 weeks in the OL period. |
|
|
| OG003 | Setmelanotide 30 mg QW (QD-QW-QW) | Participants who received 3 mg setmelanotide QD in the run-in period and 30 mg setmelanotide QW in the DB period, received SC injection of 30 mg setmelanotide QW for 13 weeks in the OL period. |
|
|
| OG003 | Setmelanotide 30 mg QW (QD-QW-QW) | Participants who received 3 mg setmelanotide QD in the run-in period and 30 mg setmelanotide QW in the DB period, received SC injection of 30 mg setmelanotide QW for 13 weeks in the OL period. |
|
|
| OG003 | Setmelanotide 30 mg QW (QD-QW-QW) | Participants who received 3 mg setmelanotide QD in the run-in period and 30 mg setmelanotide QW in the DB period, received SC injection of 30 mg setmelanotide QW for 13 weeks in the OL period. |
|
|
|
| OG003 | Setmelanotide 30 mg QW (QD-QW-QW) | Participants who received 3 mg setmelanotide QD in the run-in period and 30 mg setmelanotide QW in the DB period, received SC injection of 30 mg setmelanotide QW for 13 weeks in the OL period. |
|
|
| OG003 | DB Period: Setmelanotide 20 mg QW | Participants who received 2 mg setmelanotide QD in the run-in period, received SC injection of 20 mg setmelanotide QW and placebo matched to setmelanotide QD for 13 weeks in the DB period. |
| OG004 | DB Period: Setmelanotide 25 mg QW | Participants who received 2.5 mg setmelanotide QD in the run-in period, received SC injection of 25 mg setmelanotide QW and placebo matched to setmelanotide QD for 13 weeks in the DB period. |
| OG005 | DB Period: Setmelanotide 3 mg QD | Participants who received 3 mg setmelanotide QD in the run-in period, received SC injection of 3 mg setmelanotide QD and placebo matched to setmelanotide QW for 13 weeks in the DB period. |
| OG006 | DB Period: Setmelanotide 30 mg QW | Participants who received 3 mg setmelanotide QD in the run-in period, received SC injection of 30 mg setmelanotide QW and placebo matched to setmelanotide QD for 13 weeks in the DB period. |
| OG007 | OL Period: Setmelanotide 20 mg QW | Participants who received 2 mg setmelanotide QD in the run-in period and 20 mg setmelanotide QW in the DB period, received SC injection of 20 mg setmelanotide QW for 13 weeks in the OL period. |
| OG008 | OL Period: Setmelanotide 25 mg QW | Participants who received 2.5 mg setmelanotide QD in the run-in period and 25 mg setmelanotide QW in the DB period, received SC injection of 25 mg setmelanotide QW for 13 weeks in the OL period. |
| OG009 | OL Period: Setmelanotide 30 mg QW | Participants who received 3 setmelanotide QD in the run-in period and 3 mg setmelanotide QD or 30 mg setmelanotide QW in the DB period, received SC injection of 30 mg setmelanotide QW for 13 weeks in the OL period. |
|
|
| OG003 | Setmelanotide 30 mg QW | Participants who received 3 mg setmelanotide QD in the run-in period, received 30 mg setmelanotide QW in the DB period. |
|
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