Research

Clinical Trials

After decades with no approved treatments, cachexia research has entered a historic phase. Multiple trials are now enrolling — including the first-ever pivotal-scale studies. Here is what is currently open.

Why now? Ponsegromab’s Phase 2 results — published in the New England Journal of Medicine in 2024 — validated GDF-15 as a driver of cachexia and triggered a wave of pivotal-scale trials. 2025–2026 is the most active period of cachexia drug development in history.
Search ClinicalTrials.gov →

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, plain-language overview, you can also watch our Introduction to Clinical Trials for Cachexia video on the Patient Resources page.

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.

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List of clinical trials

Trial information is updated periodically. Always verify current status at clinicaltrials.gov and speak with your oncologist before pursuing enrollment.

RecruitingPhase 2b / 3Pfizer

RIVER-mPDAC

PonsegromabAnti-GDF-15 monoclonal antibody

Cancer typesPancreatic cancer
StartedOct 2025
Target enrollment~1,000 participants
RegionsUnited States, Canada, Europe, Asia-Pacific
Primary endpoint

% 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.

RecruitingPhase 2 / 3CatalYm

VINCIT

Visugromab (CTL-002)Anti-GDF-15 monoclonal antibody

Cancer typesNSCLC, Colorectal cancer, Other solid tumors
StartedApr 2026
Target enrollment~518 participants
RegionsUnited States, Europe, Asia-Pacific
Primary endpoint

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.

EnrollingPhase 2Endevica Bio

B07 (TCMCB07 Phase 2)

TCMCB07MC3R/MC4R antagonist peptide (crosses blood-brain barrier)

Cancer typesStage IV metastatic colorectal cancer
StartedApr 2025
Target enrollment100 participants
RegionsUnited States
Primary endpoint

Weight maintenance / body composition during chemotherapy

Unique prophylactic design — enrolling patients before cachexia develops, at chemotherapy initiation. Phase 3 planned Q1 2027.

EnrollingPhase 1GenFleet Therapeutics

GFS202A Phase 1

GFS202ABispecific antibody: anti-GDF-15 × anti-IL-6

Cancer typesAdvanced solid tumors
StartedEarly 2025
Target enrollmentTBD participants
RegionsChina
Primary endpoint

Safety, tolerability, PK

World's first bispecific antibody targeting both GDF-15 and IL-6 simultaneously. China only at this stage.

RecruitingPhase 2bCatalYm

GDFATHER-HCC-01

Visugromab + Nivolumab + LenvatinibAnti-GDF-15 mAb + PD-1 inhibitor + TKI

Cancer typesHepatocellular carcinoma (HCC)
StartedApr 2026
Target enrollment104 participants
RegionsUnited States, Europe, Asia-Pacific
Primary endpoint

Progression-free survival (cachexia as secondary endpoint)

Second-line HCC trial. Cachexia explicitly assessed via FAACT-ACS questionnaire as a secondary endpoint.

EnrollingPhase Feasibility RCTAcademic (UK)

MMIEAD (Kidney Cachexia)

Exercise + anti-inflammatory + dietary counselingNon-pharmacological multimodal intervention

Cancer typesAdvanced chronic kidney disease
StartedJul 2025
Target enrollment48 participants
RegionsUnited Kingdom
Primary endpoint

Recruitment feasibility; intervention adherence

Non-cancer cachexia trial. Non-pharmacological intervention — the only such trial on this list.

How to explore participating

1

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.

2

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.

3

Contact the trial site

Each trial has site-specific contacts listed on clinicaltrials.gov. Reach out directly to ask about availability and screening.

4

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.

Ask the Community