Numerous clinical trials evaluate setmelanotide from Phase 1 safety evaluations through Phase 4 post-marketing research, assessing therapeutic efficacy in rare genetic disorders of obesity and hyperphagia linked to leptin-melanocortin pathway defects. Key targeted conditions include hypothalamic obesity, pro-opiomelanocortin deficiency obesity, Bardet-Biedl syndrome, Alström syndrome, and Prader-Willi syndrome. Sponsored primarily by Rhythm Pharmaceuticals Inc. alongside academic institutions such as Massachusetts General Hospital and Charite University, these studies encompass recruiting, completed, and active not recruiting recruitment statuses.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT07674290 | RECRUITING | Bardet Biedl Syndrome (BBS); Bardet Biedl Syndrome; Bardet-Biedl Syndrome (BBS); Alstrom Syndrome |
Tom Hühne |
2023-01-01 | PHASE4 |
| NCT07496463 | ENROLLING_BY_INVITATION | Pseudohypoparathyroidism Type 1a; Obesity |
Massachusetts General Hospital |
2026-07-21 | PHASE2 |
| NCT06760546 | RECRUITING | Hypothalamic Obesity; Multiple Pituitary Hormone Deficiency Genetic Form; Septo-Optic Dysplasia; Optic Nerve Hypoplasia; Childhood-onset Combined Pituitary Hormone Deficiency; Pituitary Stalk Interruption Syndrome |
Rhythm Pharmaceuticals, Inc. |
2025-09-23 | PHASE3 |
| NCT06772597 | ACTIVE_NOT_RECRUITING | Prader-Willi Syndrome; Obesity; Hyperphagia |
Rhythm Pharmaceuticals, Inc. |
2025-03-04 | PHASE2 |
| NCT06596135 | COMPLETED | Obesity Associated With Defects in Leptin-melanocortin Pathway |
Rhythm Pharmaceuticals, Inc. |
2024-10-21 | PHASE3 |
| NCT05093634 | COMPLETED | Obesity; Genetic Obesity |
Rhythm Pharmaceuticals, Inc. |
2021-12-10 | PHASE3 |
| NCT05774756 | ACTIVE_NOT_RECRUITING | Hypothalamic Obesity |
Rhythm Pharmaceuticals, Inc. |
2023-04-26 | PHASE3 |
| NCT03651765 | COMPLETED | Obesity Associated With Defects in Leptin-melanocortin Pathway |
Rhythm Pharmaceuticals, Inc. |
2018-07-15 | PHASE2; PHASE3 |
| NCT05194124 | COMPLETED | Bardet-Biedl Syndrome; POMC Deficiency |
Rhythm Pharmaceuticals, Inc. |
2021-12-21 | PHASE3 |
| NCT04966741 | COMPLETED | Bardet-Biedl Syndrome; POMC Deficiency Obesity; PCSK1 Deficiency Obesity; LEPR Deficiency Obesity |
Rhythm Pharmaceuticals, Inc. |
2022-03-08 | PHASE3 |
| NCT04963231 | COMPLETED | Genetic Obesity |
Rhythm Pharmaceuticals, Inc. |
2021-11-30 | PHASE2 |
| NCT05774756 | Enrolling by invitation | Hypothalamic Obesity |
Rhythm Pharmaceuticals Inc. |
2023-04-26 | Phase 3 |
| NCT04725240 | COMPLETED | Hypothalamic Obesity |
Rhythm Pharmaceuticals, Inc. |
2021-06-07 | PHASE2 |
| NCT05046132 | COMPLETED | Healthy |
Rhythm Pharmaceuticals, Inc. |
2021-08-05 | PHASE4 |
| NCT03013543 | COMPLETED | Genetic Obesity; Obesity; Obesity Due to Melanocortin 4 Receptor Deficiency |
Rhythm Pharmaceuticals, Inc. |
2017-02-10 | PHASE2 |
| NCT05093634 | Recruiting | Obesity|Genetic Obesity |
Rhythm Pharmaceuticals Inc. |
2021-12-10 | Phase 3 |
| NCT04725240 | Completed | Hypothalamic Obesity |
Rhythm Pharmaceuticals Inc. |
2021-06-07 | Phase 2 |
| NCT04348175 | COMPLETED | Renal Insufficiency |
Rhythm Pharmaceuticals, Inc. |
2020-07-09 | PHASE1 |
| NCT03746522 | COMPLETED | Bardet Biedl Syndrome (BBS); Alström Syndrome (AS) |
Rhythm Pharmaceuticals, Inc. |
2018-11-23 | PHASE3 |
| NCT03287960 | COMPLETED | Leptin Receptor Deficiency Obesity |
Rhythm Pharmaceuticals, Inc. |
2018-01-30 | PHASE3 |
| NCT02896192 | COMPLETED | Pro-opiomelanocortin (POMC) Deficiency Obesity |
Rhythm Pharmaceuticals, Inc. |
2017-02-14 | PHASE3 |
| NCT03651765 | Active not recruiting | Obesity Associated With Defects in Leptin-melanocortin Pathway |
Rhythm Pharmaceuticals Inc. |
2018-07-15 | Phase 2|Phase 3 |
| NCT03262610 | COMPLETED | Hypertriglyceridemia; Obesity |
Rhythm Pharmaceuticals, Inc. |
2017-08-15 | PHASE2 |
| NCT02507492 | UNKNOWN | Homozygous or Compound Heterozygous POMC, LEPR or PCSK1 Gene Mutation |
Charite University, Berlin, Germany |
2015-01 | PHASE2 |
| NCT03013543 | Completed | Genetic Obesity|Obesity|Obesity Due to Melanocortin 4 Receptor Deficiency |
Rhythm Pharmaceuticals Inc. |
2017-02-10 | Phase 2 |
| NCT02311673 | COMPLETED | Prader-Willi Syndrome |
Rhythm Pharmaceuticals, Inc. |
2015-03-19 | PHASE2 |
| NCT02041195 | COMPLETED | Overweight and Obesity |
Rhythm Pharmaceuticals, Inc. |
2014-01 | PHASE1; PHASE2 |
| NCT01867437 | COMPLETED | Obesity; Morbid Obesity |
Rhythm Pharmaceuticals, Inc. |
2013-05 | PHASE1 |
| NCT02431442 | COMPLETED | Overweight; Obese |
Rhythm Pharmaceuticals, Inc. |
2012-01 | PHASE1 |
| NCT01749137 | COMPLETED | Overweight |
Rhythm Pharmaceuticals, Inc. |
2013-01-14 | PHASE2 |
(data from https://clinicaltrials.gov, updated on 2026-06-29)
Check the
Setmelanotide (RM-493)
product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.
Mechanism and Biochemical Profile
Appendix RUO and cGMP Quality Standards
| Quality Dimension | RUO (Research Use Only) | cGMP (Current Good Manufacturing Practice) |
|---|---|---|
| Clinical Applicability | Prohibited in human clinical trials or medical diagnostics. | Mandatory for human clinical trials (Phase I–III) and therapies. |
| Regulatory Status | Non-regulated grade; exempt from drug manufacturing laws. | Legally enforced by health authorities (e.g., FDA, EMA, NMPA). |
| Facility Environment | Unclassified analytical or research laboratories. | Validated Cleanrooms (ISO Class 5–8) with continuous monitoring. |
| Quality Control | Basic purity and activity testing. | Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma). |
| Process Validation | Basic equipment calibration; no process validation required. | Full qualification (IQ/OQ/PQ) and complete batch records. |
| Quality Assurance | Vendor self-declared without required formal QMS. | Mandatory QA/QC unit, Change Control, CAPA, and vendor audits. |
| Regulatory Impact | High risk of IND rejection if used as a critical raw material. | Required for IND/NDA filings, supported by Drug Master Files (DMF). |
Footnotes
Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).
- Setmelanotide (RM-493) Mechanism of Action: MC4R Activation in Hypothalamic Neurons Research
- Setmelanotide (RM-493) Working Concentration | Cell Culture | Treatment
- Setmelanotide (RM-493) Solubility in DMSO
- Setmelanotide (RM-493) Stock Solution
- Setmelanotide (RM-493) Storage
- Setmelanotide (RM-493) Stability
- Setmelanotide (RM-493) Combination Database
- Setmelanotide (RM-493) Molecular Weight
- Setmelanotide (RM-493) SMILES
- Setmelanotide (RM-493) CAS Number
- Setmelanotide (RM-493) Chemical Structure (2D and 3D)
- Setmelanotide (RM-493) IC50 for Cell-based and Cell-free Assays
- Setmelanotide (RM-493) Sigma (aabh9a959d1e)? Try a better option.
- Setmelanotide (RM-493) MedChemExpress (HY-19870)? Try a better option.
- Setmelanotide (RM-493) Cayman (35564)? Try a better option.
- Setmelanotide (RM-493) APExBIO (C8966)? Try a better option.
- Setmelanotide (RM-493) SDS｜MSDS
Note: Technical data last updated: Sep 1, 2026.