A Study to Evaluate the Efficacy and Safety of CBL-514 Compared to Placebo in Participants With Dercum's Disease Lipomas

NCT06303570 | Active Not Recruiting Phase 2 FDA Drug

• 1 other identifier: CBL-0202DD
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 1

Brief Summary

This is a single-blind, placebo-controlled, randomized phase 2 study to evaluate the efficacy and safety of CBL-514 injections in participants with Dercum's Disease lipomas.

Conditions

Dercum's Disease

Keywords

Adiposis Dolorosa; Lipedema; Lipomatosis; Skin Diseases; Lipid Metabolism Disorders; Metabolism Diseases; Connective Tissue Diseases

Outcome Measures

Primary Outcomes (1)

• To estimate the treatment effect, as measured by pain, between CBL-514 and placebo in participants with DD.

  The Comparative Pain Scale is a 11-point scale (0-10) to assess pain from 0 being "pain free" to 10 being unimaginable/unspeakable pain.

  Week 20

Secondary Outcomes (4)

• To estimate the treatment effect, as measured by pain, between CBL-514 and placebo in participants with DD.

  Up to 24 weeks

• To estimate the treatment effect, as measured by Partial Response (PR), between CBL-514 and placebo.

  Up to 24 weeks

• To evaluate the incidence of adverse events of special interests (AESI) as defined in the protocol.

  From baseline to 8 weeks post final treatment

• To evaluate the incidence of clinically significant abnormal findings as defined in the protocol as defined in the protocol.

  From baseline to 8 weeks post final treatment

Study Arms (2)

CBL-514 injection

EXPERIMENTAL

Eligible participants will receive CBL-514 administered in doses ranging from 1 mL to a maximum of 12 mL per lipoma, with treatments scheduled at intervals of approximately 4 weeks, up to 5 treatments.

Drug: CBL-514 injection

0.9% Sodium Chloride

PLACEBO COMPARATOR

Eligible participants will receive 0.9% Sodium Chloride administered in doses ranging from 1 mL to a maximum of 12 mL per lipoma, with treatments scheduled at intervals of approximately 4 weeks, up to 5 treatments.

Drug: 0.9% Sodium chloride

Interventions

CBL-514 injection DRUG

The total injection volume per lipoma will based on the lipoma size,as determined by ultrasound. Dosing scheme is presented as below: Lipoma diameter of \>0mm and \<10mm - total injection volume per lipoma: 1 mL ; total of 5 mg CBL-514. Lipoma diameter of ≥10mm and ≤20mm - total injection volume per lipoma: 3 mL ; total of 15 mg CBL-514. Lipoma diameter of \>20mm and ≤30mm - total injection volume per lipoma: 5 mL ; total of 25 mg CBL-514. Lipoma diameter of \>30mm and ≤40mm - total injection volume per lipoma: 8 mL ; total of 40 mg CBL-514. Lipoma diameter of \>40mm and ≤50mm - total injection volume per lipoma: 12 mL ; total of 60 mg CBL-514.

CBL-514 injection

0.9% Sodium chloride DRUG

The total injection volume per lipoma will based on the lipoma size,as determined by ultrasound. Dosing scheme is presented as below: Lipoma diameter of \>0mm and \<10mm - total injection volume per lipoma: 1 mL. Lipoma diameter of ≥10mm and ≤20mm - total injection volume per lipoma: 3 mL. Lipoma diameter of \>20mm and ≤30mm - total injection volume per lipoma: 5 mL. Lipoma diameter of \>30mm and ≤40mm - total injection volume per lipoma: 8 mL. Lipoma diameter of \>40mm and ≤50mm - total injection volume per lipoma: 12 mL.

0.9% Sodium Chloride

Eligibility Criteria

• Age: 18 Years - 64 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Male or female, aged 18 years to 64 years old (at screening), inclusive.
• Body mass index (BMI) \>18.5 kg/m2 at screening and Day 1.
• Has confirmed DD and/or fulfills the following clinical criteria of DD in localized nodular form. The final diagnosis of disease is in the opinion of the Investigator.
• Chronic pain (\>3 months) in the adipose tissue specific to the presence of lipomas and/or
• Pain in and around multiple lipomas.
• Has at least 4 and up to 10 painful and well-defined lipomas with dimension of ≥10 mm and ≤50 mm as measured by ultrasound (read by the Investigator) at screening.
• Generally considered healthy according to medical history, physical examination, ECG, and laboratory evaluation.
• Voluntarily signs the informed consent form (ICF) and, in the opinion of the Investigator or designee, is physically and mentally capable of participating in the study, and willing to adhere to study procedures.

You may not qualify if:

• Female participant of childbearing potential who is not willing to commit to an acceptable contraceptive regimen from the time of screening and throughout study participation until 90 days after the last IP dose, or who is currently pregnant or lactating. Female participant of childbearing potential who is breastfeeding or anticipates breastfeeding from the time of screening and throughout study participation until 90 days after the last IP dose. Male participant who is not willing to commit to using of a condom and refraining from sperm donation from the time of the first dose of IP, throughout study participation until 90 days after the last IP dose.
• Unable to tolerate SC injections.
• Diagnosed with another disorder with similar characteristics as DD as follows.
• Madelung's disease: multiple symmetric lipomatosis only localized in the upper body ie, shoulders, neck, or head.
• Panniculitis: inflammation of the SC adipose tissue, characterized by tender nodules and systemic signs.
• Proteus syndrome: disproportionate and asymmetric overgrowth of skin, and fatty and connective tissue.
• PTEN hamartoma syndrome: multiple hamartomas which includes segmental overgrowth, lipomatosis, arteriovenous malformation, and epidermal nevus.
• Gardner syndrome: multiple digestive adenomas with osteomas and multiple skin and soft tissue tumors.
• Diagnosed with coagulation disorders or is receiving anticoagulant/antiplatelet therapy or medications or dietary supplements, which inhibit coagulation or platelet aggregation.
• Has fasting glucose concentration \>200 mg/dL, delayed wound healing, bleeding risk, or any diabetic risks which, in the opinion of the Investigator or designee, is inappropriate to participate in the study.
• Any clinically significant cardiac, hepatic, renal or neurologic/psychiatric disorders that in the opinion of the Investigator places the participant at significant risk, including but not limited to any of the following:
• Participants with cirrhosis or with inadequate liver function at screening defined as aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase, or total bilirubin \>3.0 upper limit of normal (ULN).
• Participants with renal impairment, defined as both serum creatinine and blood urea nitrogen \>1.5× ULN, or estimated glomerular filtration rate (eGFR) \<90 mL/min/1.73 m2, or who are currently on dialysis.
• Participant with a history of human immunodeficiency virus (HIV)-1 infection or participant with active HIV infection at screening with positive HIV antigen/antibody (Ag/Ab) combo test.
• Participant is undergoing chronic steroid or immunosuppressive therapy, with the exception of:

Sponsors & Collaborators

• Caliway Biopharmaceuticals Co., Ltd.: lead

Study Sites (1)

Investigator Site

Spokane, Washington, 99204, United States

Location

MeSH Terms

Conditions

Adiposis Dolorosa; Lipedema; Lipomatosis; Skin Diseases; Lipid Metabolism Disorders; Connective Tissue Diseases

Interventions

Sodium Chloride

Condition Hierarchy (Ancestors)

Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 26, 2024
• First Posted: March 12, 2024
• Study Start: November 27, 2024
• Primary Completion: March 23, 2026
• Study Completion: April 1, 2026
• Last Updated: April 14, 2026

Record last verified: 2026-03

Locations

US (1)