Research
Clinical Trials
After decades with no approved treatments, cachexia research has entered a historic phase. Multiple trials are now enrolling. Below is information on clinical trials, including a list of trials that are enrolling.
About Trials
What is a clinical trial?
Clinical trials are research studies that test new treatments to find out whether they are safe and whether they work. For cancer cachexia, trials are testing whether new drugs — alone or alongside nutrition and exercise — can help patients preserve muscle and weight, feel stronger, and tolerate their cancer treatment better.
Most cachexia studies are add-on trials, meaning the study drug is given on top of your current cancer treatment, not in place of it. Participants are typically followed by both the trial team and their regular oncology team.
What are the phases?
Phase 1 trials test safety in small groups. Phase 2 trials look for an early signal that the treatment works. Phase 3 (sometimes called pivotal or registrational) trials are large studies designed to support FDA or EMA approval.
Will I get a placebo?
Some cachexia trials are placebo-controlled, meaning some participants receive an inactive treatment for comparison. You will always be told whether the trial uses a placebo, and you will continue to receive your standard cancer care either way.
What does it cost?
Study drugs and trial-related visits, tests, and procedures are typically provided at no cost to participants. Your routine cancer care is billed the way it normally would be.
What does participating look like?
Each trial differs, but participation generally involves regular visits to the trial site, scans, lab work, and questionnaires. Some trials also include a wearable device or home-monitoring component to track activity and strength.
For a deeper overview, you can also watch our Introduction to Clinical Trials for Cachexia video on the Patient Resources page.
The Science
How these drugs work
Cachexia is driven by signals the tumor sends into your brain and muscles — signals that switch off appetite, ramp up inflammation, and tell muscle to break down. Most of the drugs in trials today aim to block one of those signals so the body can hold on to weight and strength.
Ponsegromab, Visugromab
Blocking GDF-15
GDF-15 is a hormone that many tumors flood the bloodstream with. It reaches the brainstem and tells the body to stop eating and start wasting muscle. These drugs are antibodies that grab onto GDF-15 and block it, restoring appetite and helping preserve weight and muscle. Ponsegromab’s Phase 2 results in 2024 were the first to show patients actually gaining weight instead of losing it — a milestone for the field.
TCMCB07 (Mifomelatide)
Blocking brain shut-off signals
Deep in the brain, receptors called MC3R and MC4R act as an appetite and energy shut-off switch when the body detects inflammation. In cachexia, that switch gets stuck on. This drug crosses into the brain and turns the signal down, telling the body it’s safe to eat and hold on to muscle again.
GFS202A (bispecific antibody)
Hitting two signals at once
Cachexia is driven by more than one signal. A bispecific antibody is a hybrid designed to block two things at the same time — in this case GDF-15 and IL-6, a major inflammation driver. The idea is to shut down two of the biggest drivers of cachexia with a single medicine.
MMIEAD and similar studies
Non-drug trials
Not every cachexia trial tests a drug. Some test coordinated programs of exercise, nutrition counseling, and anti-inflammatory support delivered together. The reasoning: cachexia has many drivers, and hitting several at once may work better than any one alone.
Where
Trial site locations
Every site listed on clinicaltrials.gov for the trials below. Click a marker for site details. Use the chips to filter by trial.
Currently enrolling — April 2026
List of clinical trials
Trial information is updated periodically. Always verify current status at clinicaltrials.gov and speak with your oncologist before pursuing enrollment.
RIVER-mPDAC
Ponsegromab — Anti-GDF-15 monoclonal antibody How it works →
% change in body weight at week 12; appetite symptoms
The largest cachexia trial currently enrolling. Ponsegromab showed +11.4 lb average weight gain at 64 weeks in Phase 2. Combined with first-line chemotherapy.
VINCIT
Visugromab (CTL-002) — Anti-GDF-15 monoclonal antibody How it works →
Change in body weight and appetite at week 12
Just opened April 2026 — first patient dosed. Adaptive design with up to 52 weeks of treatment in Part II.
Mifomelatide Phase 2
Mifomelatide (TCMCB07) — MC3R/MC4R antagonist peptide (crosses blood-brain barrier) How it works →
Change in body weight at 12 weeks; safety and tolerability
Randomized, double-blind, placebo-controlled basket trial enrolling two cohorts (CRC and PDAC). Patients begin daily subcutaneous injections of either mifomelatide (12.5, 25, or 50 mg) or placebo at cycle 2 of first-line chemotherapy for 12 weeks, followed by an optional 26-week open-label extension at 25 mg. Primary completion estimated October 2027.
GFS202A Phase 1
GFS202A — Bispecific antibody: anti-GDF-15 × anti-IL-6 How it works →
Safety, tolerability, PK
World's first bispecific antibody targeting both GDF-15 and IL-6 simultaneously. China only at this stage.
GDFATHER-HCC-01
Visugromab + Nivolumab + Lenvatinib — Anti-GDF-15 mAb + PD-1 inhibitor + TKI How it works →
Progression-free survival (cachexia as secondary endpoint)
Second-line HCC trial. Cachexia explicitly assessed via FAACT-ACS questionnaire as a secondary endpoint.
MMIEAD (Kidney Cachexia)
Exercise + anti-inflammatory + dietary counseling — Non-pharmacological multimodal intervention How it works →
Recruitment feasibility; intervention adherence
Non-cancer cachexia trial. Non-pharmacological intervention — the only such trial on this list.
How to explore participating
Talk to your oncologist first
Your oncologist needs to know you're interested in a cachexia trial. They can help assess your eligibility and connect you with the trial team.
Check eligibility criteria
Each trial has specific requirements — cancer type, stage, ECOG performance status, and weight loss criteria. Read the full listing on clinicaltrials.gov.
Contact the trial site
Each trial has site-specific contacts listed on clinicaltrials.gov. Reach out directly to ask about availability and screening.
Ask your community
Many CCN community members have enrolled in trials or researched them. Our Facebook group is a great place to ask questions from people who have been through it.