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ROMANA 1 and 2 were two phase 3 trials of an appetite-hormone mimic in lung cancer patients with wasting. Patients gained muscle but not grip strength, which split regulators: approved in Japan, rejected in Europe.
ROMANA 1 (n=484) and ROMANA 2 (n=495) were identical double-blind trials with co-primary endpoints of lean body mass (DXA) and handgrip strength over 12 weeks. Lean body mass increased with anamorelin (ROMANA 1: +0.99 kg vs -0.47 kg; ROMANA 2: +0.65 kg vs -0.98 kg), and body weight, appetite and anorexia-cachexia symptom scores improved, but handgrip strength did not differ from placebo in either trial. Hyperglycaemia and nausea were the main adverse events. The EMA refused marketing authorisation in 2017 citing the absence of a functional benefit; Japan approved anamorelin (Adlumiz) in 2021 for NSCLC, gastric, pancreatic and colorectal cancer cachexia. ROMANA established that lean mass can be pharmacologically increased in cachexia, and that regulators want function or survival, which shaped the endpoint strategy for GDF-15 antagonists.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
979 enrolled.
| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| Change in lean body mass over 12 weeks (ROMANA 1)primary | Anamorelin | 323 | 0.99 kg | - | <0.0001 | link |
| Placebo | 161 | -0.47 kg | ||||
| Change in lean body mass over 12 weeks (ROMANA 2)primary | Anamorelin | 330 | 0.65 kg | - | <0.0001 | link |
| Placebo | 165 | -0.98 kg |
Shares Cachexia-directed therapy (GDF-15 blockade), Cancer cachexia, Body composition (lean mass, fat mass, visceral fat), Cachexia pharmacotherapy: GDF-15 blockade, anamorelin, olanzapine.
Shares Cachexia-directed therapy (GDF-15 blockade), Cancer cachexia, Body composition (lean mass, fat mass, visceral fat), Sarcopenia.
Shares Cachexia-directed therapy (GDF-15 blockade), Sarcopenia, Cancer cachexia, Cachexia, toxicity and the limits of the patient.
Shares Cachexia-directed therapy (GDF-15 blockade), Body composition (lean mass, fat mass, visceral fat), Sarcopenia, Cancer cachexia.
Shares Body composition (lean mass, fat mass, visceral fat), Sarcopenia, Cancer cachexia, Cachexia, toxicity and the limits of the patient.
Shares Cachexia pharmacotherapy: GDF-15 blockade, anamorelin, olanzapine, Cachexia, toxicity and the limits of the patient, Supportive care and survivorship roadmap: making treatment bearable → proving it extends life → caring for tens of millions afterwards, Non-small-cell lung cancer.
Shares Cachexia-directed therapy (GDF-15 blockade), Cancer cachexia, Non-small-cell lung cancer.
Shares Body composition (lean mass, fat mass, visceral fat), Sarcopenia, Cachexia, toxicity and the limits of the patient.