Trial · NCT05194124
Phase 3 Crossover Trial of Two Formulations of Setmelanotide in Participants With Specific Gene Defects in the MC4R Pathway
NCT05194124, Phase 3, Completed, Rhythm Pharmaceuticals, Inc.; results first posted 2024-11-26
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Phase 3 Crossover Trial of Two Formulations of Setmelanotide in Participants With Specific Gene Defects in the MC4R Pathway. Trials, record NCT05194124. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05194124
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase 3 Crossover Trial of Two Formulations of Setmelanotide in Participants With Specific Gene Defects in the MC4R Pathway
- NCT number
- NCT05194124
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 19 participants, actual
- Lead sponsor
- Rhythm Pharmaceuticals, Inc.
- Conditions
- Bardet-Biedl Syndrome, POMC Deficiency
- Interventions
- Setmelanotide 2 mg, Setmelanotide 2.5 mg, Setmelanotide 3 mg, Setmelanotide 20 mg, Setmelanotide 25 mg, Setmelanotide 30 mg
- First posted
- 2022-01-18
- Last updated
- 2024-11-26
- Results first posted
- 2024-11-26
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Maximum Drug Concentration (Cmax) of Setmelanotide After QD Administration in the Run-in Period | Pre-dose (0 hour) and at 0.5, 1, 2, 3, 4, 6, 8, 12, and 24 hours postdose at Week -1 | Maximum drug concentration determined directly from individual concentration-time data. |
| Primary | Cmax of Setmelanotide After QW Administration | 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 | 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). |
| Primary | Time to Maximum Plasma Concentration (Tmax) of Setmelanotide After QD Administration in the Run-in Period | Pre-dose (0 hour) and at 0.5, 1, 2, 3, 4, 6, 8, 12, and 24 hours postdose at Week -1 | Maximum drug concentration determined directly from individual concentration-time data. |
| Primary | Tmax of Setmelanotide After QW Administration | 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 | 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). |
| Primary | Mean Trough Plasma Concentration (Ctrough) of Setmelanotide After QD or QW Administration at Week 1 | 30 minutes predose 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). |
| Primary | Mean Setmelanotide Ctrough After QD or QW Administration at Week 5 | 30 minutes predose 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). |
| Primary | Mean Setmelanotide Ctrough After QD or QW Administration at Week 9 | 30 minutes predose 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). |
| Primary | Mean Setmelanotide Ctrough After QD or QW Administration at Week 18 | 30 minutes predose 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). |
| Primary | Mean Setmelanotide Ctrough After QD or QW Administration at Week 22 | 30 minutes predose 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). |
| Primary | Mean Setmelanotide Ctrough After QD or QW Administration at Week 27 | 30 minutes predose 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). |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 registered outcomesOutcomes15
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Maximum Drug Concentration (Cmax) of Setmelanotide After QD Administration in the Run-in Period | Pre-dose (0 hour) and at 0.5, 1, 2, 3, 4, 6, 8, 12, and 24 hours postdose at Week -1 | 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. | nanograms per milliliter (ng/mL) | MEAN |
| PRIMARY | Cmax of Setmelanotide After QW Administration | 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 | Participants in the PKAS with available data were analyzed. | ng/mL | MEAN |
| PRIMARY | Time to Maximum Plasma Concentration (Tmax) of Setmelanotide After QD Administration in the Run-in Period | Pre-dose (0 hour) and at 0.5, 1, 2, 3, 4, 6, 8, 12, and 24 hours postdose at Week -1 | Participants in the PKAS with available data were analyzed. | hours | MEDIAN |
| PRIMARY | Tmax of Setmelanotide After QW Administration | 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 | Participants in the PKAS with available data were analyzed. | hours | MEDIAN |
| PRIMARY | Mean Trough Plasma Concentration (Ctrough) of Setmelanotide After QD or QW Administration at Week 1 | 30 minutes predose at Week 1 | Participants in the PKAS with available data were analyzed. | ng/mL | MEAN |
| PRIMARY | Mean Setmelanotide Ctrough After QD or QW Administration at Week 5 | 30 minutes predose at Week 5 | Participants in the PKAS with available data were analyzed. | ng/mL | MEAN |
| PRIMARY | Mean Setmelanotide Ctrough After QD or QW Administration at Week 9 | 30 minutes predose at Week 9 | Participants in the PKAS with available data were analyzed. | ng/mL | MEAN |
| PRIMARY | Mean Setmelanotide Ctrough After QD or QW Administration at Week 18 | 30 minutes predose at Week 18 | Participants in the PKAS with available data were analyzed. | ng/mL | MEAN |
| PRIMARY | Mean Setmelanotide Ctrough After QD or QW Administration at Week 22 | 30 minutes predose at Week 22 | Participants in the PKAS with available data were analyzed. | ng/mL | MEAN |
| PRIMARY | Mean Setmelanotide Ctrough After QD or QW Administration at Week 27 | 30 minutes predose at Week 27 | Participants in the PKAS with available data were analyzed. | ng/mL | MEAN |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group364
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Maximum Drug Concentration (Cmax) of Setmelanotide After QD Administration in the Run-in Period | Setmelanotide 2 mg QD | 29.4 | Standard Deviation 3.49 | nanograms per milliliter (ng/mL) | |
| Maximum Drug Concentration (Cmax) of Setmelanotide After QD Administration in the Run-in Period | Setmelanotide 2.5 mg QD | 44.8 | nanograms per milliliter (ng/mL) | ||
| Maximum Drug Concentration (Cmax) of Setmelanotide After QD Administration in the Run-in Period | Setmelanotide 3 mg QD | 59.9 | Standard Deviation 29.6 | nanograms per milliliter (ng/mL) | |
| Cmax of Setmelanotide After QW Administration | Setmelanotide 20 mg QW (QD-QW-QW) | 22.4 | ng/mL | ||
| Cmax of Setmelanotide After QW Administration | Setmelanotide 25 mg QW (QD-QW-QW) | 32.7 | ng/mL | ||
| Cmax of Setmelanotide After QW Administration | Setmelanotide 30 mg QW (QD-QD-QW) | 31.8 | Standard Deviation 28.6 | ng/mL | |
| Cmax of Setmelanotide After QW Administration | Setmelanotide 30 mg QW (QD-QW-QW) | 57.3 | Standard Deviation 33.0 | ng/mL | |
| Time to Maximum Plasma Concentration (Tmax) of Setmelanotide After QD Administration in the Run-in Period | Setmelanotide 2 mg QD | 6.91 | Full Range 5.92 to 7.90 | hours | |
| Time to Maximum Plasma Concentration (Tmax) of Setmelanotide After QD Administration in the Run-in Period | Setmelanotide 2.5 mg QD | 5.88 | hours | ||
| Time to Maximum Plasma Concentration (Tmax) of Setmelanotide After QD Administration in the Run-in Period | Setmelanotide 3 mg QD | 6.02 | Full Range 2.00 to 8.00 | hours |
1 to 10 of 364Source ClinicalTrials.gov
See all 364 results by groupParticipant flow90
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Run-in Period (1 Week) | STARTED | Run-in Period: Setmelanotide 2 mg QD | 2 | |
| Run-in Period (1 Week) | STARTED | Run-in Period: Setmelanotide 2.5 mg QD | 1 | |
| Run-in Period (1 Week) | STARTED | Run-in Period: Setmelanotide 3 mg QD | 16 | |
| Run-in Period (1 Week) | STARTED | DB Period: Setmelanotide 20 mg QW | 0 | |
| Run-in Period (1 Week) | STARTED | DB Period: Setmelanotide 25 mg QW | 0 | |
| Run-in Period (1 Week) | STARTED | DB Period: Setmelanotide 3 mg QD | 0 | |
| Run-in Period (1 Week) | STARTED | DB Period: Setmelanotide 30 mg QW | 0 | |
| Run-in Period (1 Week) | STARTED | OL Period: Setmelanotide 20 mg QW | 0 | |
| Run-in Period (1 Week) | STARTED | OL Period: Setmelanotide 25 mg QW | 0 | |
| Run-in Period (1 Week) | STARTED | OL Period: Setmelanotide 30 mg QW | 0 |
1 to 10 of 90Source ClinicalTrials.gov
See all 90 participant flowAdverse events470
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Injection site erythema | General disorders | Run-in Period: Setmelanotide 2 mg QD | 0 | 2 | |
| other | Injection site erythema | General disorders | Run-in Period: Setmelanotide 2.5 mg QD | 0 | 1 | |
| other | Injection site erythema | General disorders | Run-in Period: Setmelanotide 3 mg QD | 1 | 16 | |
| other | Injection site erythema | General disorders | DB Period: Setmelanotide 20 mg QW | 1 | 2 | |
| other | Injection site erythema | General disorders | DB Period: Setmelanotide 25 mg QW | 1 | 1 | |
| other | Injection site erythema | General disorders | DB Period: Setmelanotide 3 mg QD | 4 | 9 | |
| other | Injection site erythema | General disorders | DB Period: Setmelanotide 30 mg QW | 2 | 7 | |
| other | Injection site erythema | General disorders | OL Period: Setmelanotide 20 mg QW | 0 | 1 | |
| other | Injection site erythema | General disorders | OL Period: Setmelanotide 25 mg QW | 1 | 1 | |
| other | Injection site erythema | General disorders | OL Period: Setmelanotide 30 mg QW | 0 | 14 |
1 to 10 of 470Source ClinicalTrials.gov
See all 470 adverse eventsAdverse event totals30
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Run-in Period: Setmelanotide 2 mg QD | 0 | 2 |
| Total, all-cause mortality | Run-in Period: Setmelanotide 2.5 mg QD | 0 | 1 |
| Total, all-cause mortality | Run-in Period: Setmelanotide 3 mg QD | 0 | 16 |
| Total, all-cause mortality | DB Period: Setmelanotide 20 mg QW | 0 | 2 |
| Total, all-cause mortality | DB Period: Setmelanotide 25 mg QW | 0 | 1 |
| Total, all-cause mortality | DB Period: Setmelanotide 3 mg QD | 0 | 9 |
| Total, all-cause mortality | DB Period: Setmelanotide 30 mg QW | 0 | 7 |
| Total, all-cause mortality | OL Period: Setmelanotide 20 mg QW | 0 | 1 |
| Total, all-cause mortality | OL Period: Setmelanotide 25 mg QW | 0 | 1 |
| Total, all-cause mortality | OL Period: Setmelanotide 30 mg QW | 0 | 14 |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT05194124
GET https://conductscience.org/api/trials/v1/NCT05194124/results?page=2