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Setmelanotide for the Treatment of Early-Onset Pro-Opiomelanocortin (POMC) Deficiency Obesity
Phase 3 trial testing Setmelanotide in Pro-opiomelanocortin (POMC) Deficiency Obesity in 15 participants. Completed in 25 May 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 | 14 February 2017 |
| Primary completion | 25 May 2020 |
| Estimated completion | 25 May 2020 |
| Sites | 7 locations across France, Belgium, United Kingdom, Germany, Canada, United States, Spain |
Rhythm Pharmaceuticals, Inc. — full company profile →
6 and older, any sex, with Pro-opiomelanocortin (POMC) 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 after approximately Week 52 (\~1 year) of treatment were analyzed.
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | 80.0 | 49.31 – 96.32 |
The percentage of participants who met the ≥ 10% weight loss threshold after approximately Week 52 (\~1 year) of treatment were analyzed.
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | 85.7 | 61.46 – 97.40 |
The mean percent change from baseline in body weight at 52 weeks was analyzed.
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | -25.83 | ± 9.721 |
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. 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.
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | -27.1 | ± 28.11 |
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. The weekly average hunger score of the daily worst (most) hunger score in 24 hours is the hunger score used to assess th
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | 50.0 | 19.29 – 80.71 |
Waist circumference (centimeters) 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) | 115.48 | ± 17.920 |
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | -17.51 | ± 9.112 |
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) | -2.8 | ± 2.41 |
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Double-Blind) | 6.2 | ± 3.87 |
The absolute score in daily hunger 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 great
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Double-Blind) | 4.5 | ± 2.57 |
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Double-Blind) | 6.7 | ± 2.22 |
The mean percent change from baseline in BMI was assessed.
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | -27.32 | ± 8.971 |
The AUC change from baseline for the OGTT was assessed. The AUC was calculated by the linear trapezoidal method and includes pre-dose and post-dose assessments. At each visit, oral glucose was captured pre-meal, and post-meal at the following times: 30 minutes, 60 minutes, 90 minutes, and 120 minutes. OGTT was not performed for participants with a diagnosis of Type 1 or Type 2 diabetes.
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | 951.900 | ± 464.9342 |
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | -35.284 | ± 442.6334 |
Change from baseline in serum glucose at Week 52 was assessed.
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | 6.606 | ± 5.0024 |
| Group | Value | 95% CI |
|---|---|---|
| Setmelanotide (Entire Study) | -1.527 | ± 1.9374 |
Time frame: From first dose up to ~30 days after last dose of study drug (Up to ~ 1 year). Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Setmelanotide |
|---|---|---|
| Pleurisy | Respiratory, thoracic and mediastinal disorders | — |
| Depression | Psychiatric disorders | — |
| Major Depression | Psychiatric disorders | — |
| Panic Attack | Psychiatric disorders | — |
| Adrenocortical Insufficiency Acute | Endocrine disorders | — |
| Pneumonia | Infections and infestations | — |
| Hypoglycaemia | Metabolism and nutrition disorders | — |
| Reaction | System | Setmelanotide |
|---|---|---|
| Skin Hyperpigmentation | Skin and subcutaneous tissue disorders | — |
| Injection Site Erythema | General disorders | — |
| Injection Site Oedema | General disorders | — |
| Injection Site Pruritus | General disorders | — |
| Headache | Nervous system disorders | — |
| Nausea | Gastrointestinal disorders | — |
| Vomiting | Gastrointestinal disorders | — |
| Upper Respiratory Tract Infection | Infections and infestations | — |
| Melanocytic Naevus | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | — |
| Fatigue | General disorders | — |
| Diarrhoea | Gastrointestinal disorders | — |
| Back Pain | Musculoskeletal and connective tissue disorders | — |
| Injection Site Pain | General disorders | — |
| Abdominal Pain | Gastrointestinal disorders | — |
| Dry Mouth | Gastrointestinal disorders | — |
| Dry Skin | Skin and subcutaneous tissue disorders | — |
| Chills | General disorders | — |
| Asthenia | General disorders | — |
| Injection Site Bruising | General disorders | — |
| Vertigo | Ear and labyrinth disorders | — |
| Alopecia | Skin and subcutaneous tissue disorders | — |
| Pyrexia | General disorders | — |
| Injection Site Induration | General disorders | — |
| Suicidal Ideation | Psychiatric disorders | — |
| Depressed Mood | Psychiatric disorders | — |
| Restlessness | Psychiatric disorders | — |
| Sleep Disorder | Psychiatric disorders | — |
| Spontaneous Penile Erection | Reproductive system and breast disorders | — |
| Lip Dry | Gastrointestinal disorders | — |
| Rash Papular | Skin and subcutaneous tissue disorders | — |
| Arthralgia | Musculoskeletal and connective tissue disorders | — |
| Muscle Contracture | Musculoskeletal and connective tissue disorders | — |
| Pain in Extremity | Musculoskeletal and connective tissue disorders | — |
| Haematoma | Vascular disorders | — |
| Multiple Allergies | Immune system disorders | — |
| Hyperthermia | General disorders | — |
| Injection Site Discolouration | General disorders | — |
| Injection Site Nodule | General disorders | — |
| Temperature Intolerance | General disorders | — |
| Thirst | General disorders | — |
Most-reported serious reactions: Pleurisy, Depression, Major Depression, Panic Attack, Adrenocortical Insufficiency Acute, Pneumonia, Hypoglycaemia.
Data from ClinicalTrials.gov NCT02896192 adverse events section.
To demonstrate statistically significant and clinically meaningful effects of setmelanotide on percent body weight change in participants with pro-opiomelanocortin (POMC) deficiency or proprotein convertase subtilisin/kexin type 1 (PCSK1) deficiency obesity due to rare biallelic 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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