Trial · NCT03287960
Setmelanotide for the Treatment of Leptin Receptor (LEPR) Deficiency Obesity
NCT03287960, Phase 3, Completed, Rhythm Pharmaceuticals, Inc.; results first posted 2023-05-23
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Setmelanotide for the Treatment of Leptin Receptor (LEPR) Deficiency Obesity. Trials, record NCT03287960. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03287960
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Setmelanotide for the Treatment of Leptin Receptor (LEPR) Deficiency Obesity
- NCT number
- NCT03287960
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 15 participants, actual
- Lead sponsor
- Rhythm Pharmaceuticals, Inc.
- Conditions
- Leptin Receptor Deficiency Obesity
- Interventions
- Setmelanotide, Placebo
- First posted
- 2017-09-19
- Last updated
- 2023-05-23
- Other registries' IDs
- eudract:2017-002005-36
- Results first posted
- 2023-05-23
- Limitations and caveats
Based on meetings with the Federal Drug Administration (FDA), the structure of the endpoints was slightly different in the statistical analysis plan (SAP) and the protocol.~In addition, there was no planned comparison to a randomized placebo group given the rarity of monogenic LEPR deficiency obesity.
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants Who Reached ≥10% Weight Loss Threshold After 1 Year (Pivotal Cohort) | Week 52 | The percentage of participants who met the ≥10% weight loss threshold (responders) after approximately Week 52 (\~1 year) of treatment were analyzed. |
| Secondary | Percentage of Participants Who Reached ≥10% Weight Loss Threshold After 1 Year (Pivotal + Supplemental Cohort) | Week 52 | The percentage of participants who met the ≥10% weight loss threshold (responders) after approximately Week 52 (\~1 year) of treatment were analyzed. |
| Secondary | Mean Percent Change From Baseline in Body Weight | Baseline and Week 52 | The mean percent change from baseline in body weight at 52 weeks was analyzed. |
| Secondary | Mean Percent Change From Baseline in Hunger Score ('Most Hunger') | Baseline and Week 52 | The mean percent change in hunger scores for participants ≥12 years of age with leptin receptor (LEPR) deficiency obesity in treatment with setmelanotide was evaluated. Hunger score ranged from 0 - 10 on a Likert-type scale; 0 = not hungry at all and 10 = hungriest possible. On the Daily Hunger Questionnaire, each of the 3 items (average hunger, most/worst hunger, and morning hunger) was scored separately and averaged on a weekly basis. |
| Secondary | Percentage of Participants Achieving at Least 25% Improvement in Daily Hunger From Baseline | Baseline and Week 52 | The percentage of participants (≥12 years of age) achieving a ≥25% improvement from baseline in hunger score at Week 52 (i.e., after treatment with setmelanotide for 52 weeks at the therapeutic dose) was assessed. Hunger ranged from 0 - 10 on a Likert-type scale; 0 = not hungry at all and 10 = hungriest possible. On the Daily Hunger Questionnaire, each of the 3 items (average hunger, most/worst hunger, and morning hunger) was scored separately and averaged on a weekly basis. |
| Secondary | Absolute Change From Baseline in Waist Circumference | Baseline and Week 52 | Waist circumference (cm) was measured according to the National Heart, Lung, and Blood Institute (NHLBI) criteria. Waist circumference was measured when participants were fasting at approximately the same time at each visit. The absolute change from baseline in waist circumference was assessed. |
| Secondary | Absolute Change in Body Weight (Reversal of Weight Loss) During Double-Blind Placebo-Controlled Withdrawal Period | Baseline and Week 8 of withdrawal period | A comparison of weight change was evaluated during the 8 week placebo-controlled withdrawal period for each participant, during which each participant received 4 weeks of placebo and 4 weeks active therapy in a blinded fashion. |
| Secondary | Absolute Daily Hunger Reduction Score During the Double-Blind Placebo-Controlled Withdrawal Period | Week 8 of withdrawal period | The absolute score in daily hunger reduction during the double-blind placebo-controlled withdrawal period (≥12 Years of Age) was assessed. Hunger ranged from 0 - 10 on a Likert-type scale; 0 = not hungry at all and 10 = hungriest possible. On the Daily Hunger Questionnaire, each of the 3 items (average hunger, most/worst hunger, and morning hunger) was scored separately and averaged on a weekly basis. The weekly average hunger score of the daily worst (most) hunger score in 24 hours is the hunger score used to assess this study endpoint. Lower scores represent lower hunger, higher scores represent greater hunger. |
| Secondary | Mean Percent Change From Baseline in Body Mass Index | Baseline and Week 52 | The mean percent change from baseline in body mass index (BMI) was assessed. |
| Secondary | Change From Baseline in Glucose Parameters | Baseline and Week 52 | Glucose parameters included glucose, glycated hemoglobin (HbA1c) and Oral glucose tolerance test (OGTT). Data is planned to be reported only for change from baseline in glucose levels. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants Who Reached ≥10% Weight Loss Threshold After 1 Year (Pivotal Cohort) | Week 52 | The full analysis set (FAS) consisted of participants who received any of the study drug injections and at least one baseline assessment (included those who did and did not demonstrate ≥5 kg weight loss or 5% of body weight if weight is \<100 kg at baseline over the initial12-week open label treatment period and proceeded into the double blind, placebo-controlled withdrawal period). Participants in pivotal cohort were included in the analysis. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Who Reached ≥10% Weight Loss Threshold After 1 Year (Pivotal + Supplemental Cohort) | Week 52 | FAS Population. Participants in pivotal and supplemental cohort were included in the analysis. | percentage of participants | NUMBER |
| SECONDARY | Mean Percent Change From Baseline in Body Weight | Baseline and Week 52 | The Designated Use Set (DUS) consisted of participants who received any of the study drug injections, demonstrated ≥5 kg weight loss \[or 5% of body weight if weight was \<100 kg at baseline\] during the initial 12-week open label treatment period, and proceeded into the double-blind, placebo-controlled withdrawal period. Participants in pivotal and supplemental cohort were included in the analysis. | Percent change | MEAN |
| SECONDARY | Mean Percent Change From Baseline in Hunger Score ('Most Hunger') | Baseline and Week 52 | DUS Population. Participants in pivotal and supplemental cohort were included in the analysis. | Percent Change | MEAN |
| SECONDARY | Percentage of Participants Achieving at Least 25% Improvement in Daily Hunger From Baseline | Baseline and Week 52 | FAS population with available data at specified time point. Participants in pivotal and supplemental cohort were included in the analysis. | percentage of participants | NUMBER |
| SECONDARY | Absolute Change From Baseline in Waist Circumference | Baseline and Week 52 | DUS population with available data at specified time point. Participants in pivotal and supplemental cohort were included in the analysis. | Centimeter | MEAN |
| SECONDARY | Absolute Change in Body Weight (Reversal of Weight Loss) During Double-Blind Placebo-Controlled Withdrawal Period | Baseline and Week 8 of withdrawal period | DUS population with available data at specified time point. Participants in pivotal and supplemental cohort were included in the analysis. | Kilogram | MEAN |
| SECONDARY | Absolute Daily Hunger Reduction Score During the Double-Blind Placebo-Controlled Withdrawal Period | Week 8 of withdrawal period | DUS population with available data at specified time point. Participants in pivotal and supplemental cohort were included in the analysis. | units on a scale | MEAN |
| SECONDARY | Mean Percent Change From Baseline in Body Mass Index | Baseline and Week 52 | DUS population with available data at specified time point. Participants in pivotal and supplemental cohort were included in the analysis. | Percent change | MEAN |
| SECONDARY | Change From Baseline in Glucose Parameters | Baseline and Week 52 | The Safety Analysis Set (SAS) was defined as all participants who received any study drug injections at least one post-dose safety assessment. Participants with available data were analyzed. Participants in pivotal and supplemental cohort were included in the analysis. | millimole per liter | MEAN |
Source ClinicalTrials.gov
Results by group14
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants Who Reached ≥10% Weight Loss Threshold After 1 Year (Pivotal Cohort) | Setmelanotide (Entire Study) | 45.5 | 90% Confidence Interval 19.96 to 72.88 | percentage of participants | |
| Percentage of Participants Who Reached ≥10% Weight Loss Threshold After 1 Year (Pivotal + Supplemental Cohort) | Setmelanotide (Entire Study) | 53.3 | 90% Confidence Interval 30.00 to 75.63 | percentage of participants | |
| Mean Percent Change From Baseline in Body Weight | Setmelanotide (Entire Study) | -12.34 | Standard Deviation 7.534 | Percent change | |
| Mean Percent Change From Baseline in Hunger Score ('Most Hunger') | Setmelanotide (Entire Study) | -42.7 | Standard Deviation 27.49 | Percent Change | |
| Percentage of Participants Achieving at Least 25% Improvement in Daily Hunger From Baseline | Setmelanotide (Entire Study) | 71.4 | 90% Confidence Interval 46.00 to 89.60 | percentage of participants | |
| Absolute Change From Baseline in Waist Circumference | Setmelanotide (Entire Study) | Baseline | 131.58 | Standard Deviation 24.047 | Centimeter |
| Absolute Change From Baseline in Waist Circumference | Setmelanotide (Entire Study) | Change at Week 52 | -9.80 | Standard Deviation 6.095 | Centimeter |
| Absolute Change in Body Weight (Reversal of Weight Loss) During Double-Blind Placebo-Controlled Withdrawal Period | Setmelanotide (Double-Blind) | Change at Week 4: Setmelanotide | -1.2 | Standard Deviation 3.04 | Kilogram |
| Absolute Change in Body Weight (Reversal of Weight Loss) During Double-Blind Placebo-Controlled Withdrawal Period | Setmelanotide (Double-Blind) | Change at Week 4: Placebo | 4.9 | Standard Deviation 2.82 | Kilogram |
| Absolute Daily Hunger Reduction Score During the Double-Blind Placebo-Controlled Withdrawal Period | Setmelanotide (Double-Blind) | Setmelanotide | 3.4 | Standard Deviation 1.63 | units on a scale |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Titration Period (2 to 12 Weeks) | STARTED | Setmelanotide | 15 | |
| Titration Period (2 to 12 Weeks) | COMPLETED | Setmelanotide | 15 | |
| Titration Period (2 to 12 Weeks) | NOT COMPLETED | Setmelanotide | 0 | |
| Open-Label Period (10 Weeks) | STARTED | Setmelanotide | 15 | |
| Open-Label Period (10 Weeks) | COMPLETED | Setmelanotide | 14 | |
| Open-Label Period (10 Weeks) | NOT COMPLETED | Setmelanotide | 1 | |
| Double-Blind Withdrawal Period (8 Weeks) | STARTED | Setmelanotide | 14 | |
| Double-Blind Withdrawal Period (8 Weeks) | COMPLETED | Setmelanotide | 14 | |
| Double-Blind Withdrawal Period (8 Weeks) | NOT COMPLETED | Setmelanotide | 0 | |
| Open-Label Period (32 Weeks) | STARTED | Setmelanotide | 14 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events136
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 | Flushing | Vascular disorders | Setmelanotide | 1 | 15 | 1 |
| other | Melanocytic naevus | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Setmelanotide | 2 | 15 | 3 |
| other | Eye naevus | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Setmelanotide | 1 | 15 | 1 |
| other | Injection site erythema | General disorders | Setmelanotide | 11 | 15 | 32 |
| other | Injection site induration | General disorders | Setmelanotide | 7 | 15 | 16 |
| other | Injection site pain | General disorders | Setmelanotide | 7 | 15 | 14 |
| other | Injection site oedema | General disorders | Setmelanotide | 6 | 15 | 13 |
| other | Injection site pruritus | General disorders | Setmelanotide | 8 | 15 | 13 |
| other | Fatigue | General disorders | Setmelanotide | 2 | 15 | 9 |
| other | Injection site bruising | General disorders | Setmelanotide | 5 | 15 | 8 |
1 to 10 of 136Source ClinicalTrials.gov
See all 136 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Setmelanotide | 1 | 15 |
| Total, other adverse events | Setmelanotide | 15 | 15 |
| Total, serious adverse events | Setmelanotide | 3 | 15 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
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/NCT03287960
GET https://conductscience.org/api/trials/v1/NCT03287960/events?page=2