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Setmelanotide for the Treatment of Leptin Receptor (LEPR) Deficiency Obesity
Phase 3 trial testing Setmelanotide in Leptin Receptor Deficiency Obesity in 15 participants. Completed in 25 September 2020.
| Lead sponsor | Rhythm Pharmaceuticals, Inc. |
|---|---|
| Phase | Phase 3 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 15 |
| Start date | 30 January 2018 |
| Primary completion | 25 September 2020 |
| Estimated completion | 25 September 2020 |
| Sites | 6 locations across France, Netherlands, United Kingdom, Germany, Canada, Reunion |
Rhythm Pharmaceuticals, Inc. — full company profile →
6 and older, any sex, with Leptin Receptor Deficiency Obesity. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
The percentage of participants who met the ≥10% weight loss threshold (responders) after approximately Week 52 (\~1 year) of treatment were analyzed.
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | 45.5 | 19.96 – 72.88 |
The percentage of participants who met the ≥10% weight loss threshold (responders) after approximately Week 52 (\~1 year) of treatment were analyzed.
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | 53.3 | 30.00 – 75.63 |
The mean percent change from baseline in body weight at 52 weeks was analyzed.
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | -12.34 | ± 7.534 |
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.
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | -42.7 | ± 27.49 |
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.
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | 71.4 | 46.00 – 89.60 |
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.
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | 131.58 | ± 24.047 |
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | -9.80 | ± 6.095 |
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.
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Double-Blind) | -1.2 | ± 3.04 |
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Double-Blind) | 4.9 | ± 2.82 |
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 repre
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Double-Blind) | 3.4 | ± 1.63 |
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Double-Blind) | 6.3 | ± 2.13 |
The mean percent change from baseline in body mass index (BMI) was assessed.
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | -14.24 | ± 9.096 |
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.
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | 5.586 | ± 2.3740 |
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | -0.465 | ± 1.4350 |
Time frame: From first dose up to ~30 days after last dose of study drug (Up to ~ 1 year). Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Setmelanotide |
|---|---|---|
| Cholecystitis | Hepatobiliary disorders | — |
| Road Traffic Accident | Injury, poisoning and procedural complications | — |
| Suicidal Ideation | Psychiatric disorders | — |
| Gastric Banding Reversal | Surgical and medical procedures | — |
| Reaction | System | Setmelanotide |
|---|---|---|
| Injection site erythema | General disorders | — |
| Skin hyperpigmentation | Skin and subcutaneous tissue disorders | — |
| Injection site pruritus | General disorders | — |
| Nausea | Gastrointestinal disorders | — |
| Injection site induration | General disorders | — |
| Injection site pain | General disorders | — |
| Diarrhea | Gastrointestinal disorders | — |
| Injection site oedema | General disorders | — |
| Injection site bruising | General disorders | — |
| Headache | Nervous system disorders | — |
| Asthenia | General disorders | — |
| Insomnia | Psychiatric disorders | — |
| Spontaneous penile erection | Reproductive system and breast disorders | — |
| Dizziness | Nervous system disorders | — |
| Abdominal pain upper | Gastrointestinal disorders | — |
| Back pain | Musculoskeletal and connective tissue disorders | — |
| Arthralgia | Musculoskeletal and connective tissue disorders | — |
| Nasopharyngitis | Infections and infestations | — |
| Influenza like illness | General disorders | — |
| Injection site hypersensitivity | General disorders | — |
| Anxiety | Psychiatric disorders | — |
| Anemia | Blood and lymphatic system disorders | — |
| Muscle spasms | Musculoskeletal and connective tissue disorders | — |
| Melanocytic naevus | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | — |
| Fatigue | General disorders | — |
| Injection site haematoma | General disorders | — |
| Malaise | General disorders | — |
| Depression | General disorders | — |
| Depressed mood | Psychiatric disorders | — |
| Constipation | Gastrointestinal disorders | — |
| Vomiting | Gastrointestinal disorders | — |
| Erythema | Skin and subcutaneous tissue disorders | — |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | — |
| Hypothyroidism | Endocrine disorders | — |
| Gastrointestinal infection | Infections and infestations | — |
| Pharyngitis | Infections and infestations | — |
| Rhinitis | Infections and infestations | — |
| Flushing | Vascular disorders | — |
| Eye naevus | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | — |
| Chest pain | General disorders | — |
Most-reported serious reactions: Cholecystitis, Road Traffic Accident, Suicidal Ideation, Gastric Banding Reversal.
Data from ClinicalTrials.gov NCT03287960 adverse events section.
To demonstrate statistically significant and clinically meaningful effects of setmelanotide on percent body weight change in participants with LEPR deficiency obesity due to rare bi-allelic or loss-of function mutations at the end of 1 year of treatment.
8 peer-reviewed publications reference this trial (live from Europe PMC):
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