CBL-514 for Adiposis Dolorosa

Overseen By Shannon Wu

Age: 18 - 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Caliway Biopharmaceuticals Co., Ltd.

Must not be taking: Anticoagulants, Antiplatelets, CYP inhibitors, others

Disqualifiers: Pregnancy, Coagulation disorders, Diabetes, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called CBL-514, an experimental therapy, to determine its effectiveness and safety in reducing painful lumps under the skin known as lipomas, a symptom of Dercum's Disease. Participants will receive one of two different doses to identify which is more effective. Individuals with Dercum's Disease and at least four painful lipomas not near sensitive body areas may be suitable for this trial. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications, such as terfenadine, buspirone, fexofenadine, and any drugs that strongly affect liver enzymes, at least 2 days before and until 1 day after the study dose. If you are on these medications, you may need to pause them during the study.

Is there any evidence suggesting that CBL-514 is likely to be safe for humans?

Research has shown that CBL-514 was safe in earlier studies. These studies aimed to reduce subcutaneous belly fat and found that participants generally tolerated the treatment well. Side effects were mostly mild, especially compared to other injection treatments, causing only minor discomfort or issues for participants.

In the new trial, two groups will receive different doses of CBL-514, either 10 mg or 15 mg per injection. Past evidence suggests that both doses are similarly safe. However, since this study is ongoing, the researchers will continue monitoring to ensure participant safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Dercum's Disease?

Researchers are excited about CBL-514 for treating Dercum's Disease lipomas because it offers a potentially new approach compared to standard options like surgery, corticosteroids, or liposuction. Most current treatments focus on removing or shrinking the lipomas mechanically or with medication that has systemic effects. CBL-514, however, is an injectable treatment that targets the lipomas directly, with the potential to dissolve them from within using a novel mechanism. This targeted action could mean fewer side effects and a more straightforward treatment process for patients. Additionally, the study is exploring different dosing regimens, which could optimize effectiveness and minimize discomfort.

What evidence suggests that CBL-514 might be an effective treatment for Dercum's Disease?

Research has shown that CBL-514 can help reduce unwanted fat. In earlier studies, many participants experienced a noticeable decrease in fat after receiving CBL-514 injections. For instance, one study found that 69.6% of participants lost at least 150mL of subcutaneous fat within eight weeks of treatment. Another study demonstrated that 87.5% of treated thighs showed less severe cellulite. These findings suggest that CBL-514 might also help shrink fatty lumps, known as lipomas, in conditions like Dercum's Disease. Participants in this trial will be randomized into two groups to receive different doses of CBL-514, either 10 mg or 15 mg per injection, to further evaluate its effectiveness. ^{1 2 5 6 7}

Who Is on the Research Team?

Anne Sheu

Principal Investigator

Caliway Biopharmaceuticals Co., Ltd.

Are You a Good Fit for This Trial?

Inclusion Criteria

Has at least 4 painful and well defined lipomas with a diameter of ≥1.0 cm and ≤6.0 cm as determined by ultrasound. Excluding lipomas in proximity to vulnerable anatomic structures, including the salivary glands, lymph nodes, muscles, and along the anatomic landmarks of the marginal mandibular nerve.

Generally considered healthy according to medical history, physical examination, electrocardiogram (ECG), and laboratory evaluation.

You have pain in and around many lumps of fat in your body.

Exclusion Criteria

Participants requiring continual use of the following therapeutic agents during the study: terfenadine, buspirone, fexofenadine, any medication that is known to strongly inhibit or induce CYP enzymes, sensitive CYP substrates or drugs with narrow therapeutic index, in the opinion of the Investigator, may affect the evaluation of the study product or place the participant at undue risk. If a participant requires the abovementioned therapeutic agents during the study for any reason, they should not be used for at least 2 days before dosing and until 1 day postdose.

You have had cosmetic procedures such as liposuction or laser therapy in the area being studied before or during the trial.

Participant who has undergone vaccination (including with a live-attenuated vaccine) within 14 days prior to administration of the IP.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive up to 2 courses of CBL-514 injections into selected lipomas, with dosage based on lipoma size

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• CBL-514

How Is the Trial Designed?

Group I: CBL-514 Group 2 Experimental Treatment 1 Intervention

Eligible participants will be enrolled and randomized to one of 2 dose groups. Group 2 with 15 mg CBL-514 per injection.

Group II: CBL-514 Group 1 Experimental Treatment 1 Intervention

Eligible participants will be enrolled and randomized into one of 2 dose groups. Group 1 with 10 mg CBL-514 per injection.

CBL-514 is already approved in European Union, United States for the following indications:

Approved in European Union as CBL-514 for:

• Dercum's disease

Approved in United States as CBL-514 for:

• Dercum's disease

Who Is Running the Clinical Trial?

Caliway Biopharmaceuticals Co., Ltd.

Lead Sponsor

Trials

11

Recruited

550+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12080885/

Efficacy and Safety of CBL-514 Injection in Reducing ... CBL-514 treatment significantly reduced abdominal subcutaneous fat volume with a favorable safety profile. As a noninvasive treatment, CBL-514 could be a new, ...

2. caliwaybiopharma.com caliwaybiopharma.com/en/news-detail/94/

Caliway Announces Positive Data from CBL-0201EFP ... The highest dose of CBL-514 treatment demonstrated the best efficacy, 87.5% of thighs achieved at least 1-level improvement in cellulite severity two weeks ...

3. biospace.com biospace.com/press-releases/caliway-announces-cbl-514-second-phase-2b-study-results-cbl-0205-met-endpoints

Caliway Announces CBL-514 Second Phase 2b Study ... Nearly 80% of the CBL-514 treatment group achieved at least 1 grade improvement in CR-AFRS in the PP population. These study results align with ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT04897412

NCT04897412 | A Study to Evaluate the Efficacy, Safety ... A Study to Evaluate the Efficacy, Safety, and Tolerability of CBL-514 Injection for Reducing Subcutaneous Fat (Stage 2). ClinicalTrials.gov ID NCT04897412.

5. academic.oup.com academic.oup.com/asj/advance-article/doi/10.1093/asj/sjaf032/8051634

Efficacy and Safety of CBL-514 Injection in Reducing ... At 8 weeks post-final treatment, 69.6% of CBL-514-treated participants lost ≥150mL subcutaneous fat, compared with none in the placebo group (p< ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT04698642

NCT04698642 | A Study to Evaluate the Safety, Tolerability ... A Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of CBL-514 Injection for Reducing Convexity or Fullness of Abdominal Subcutaneous Fat.

7. clinicaltrials.gov clinicaltrials.gov/study/NCT05736107

NCT05736107 | A Study to Evaluate the Efficacy, Safety ... This is a randomized, placebo-controlled, Phase 2b study to evaluate the efficacy, safety, and tolerability of CBL-514 injection for reducing subcutaneous fat.