A Single-Blind, Placebo-Control, Randomized Phase 2 Study to Evaluate the Efficacy and Safety of CBL-514 in Participants With Dercum's Disease Lipomas

Caliway Biopharmaceuticals Co., Ltd.2 sites in 1 country19 target enrollmentNovember 27, 2024

ConditionsDercum's Disease

InterventionsCBL-514 injection 0.9% Sodium chloride

DrugsCBL-514 injection 0.9% Sodium chloride

Overview

Phase: Phase 2
Intervention: CBL-514 injection
Conditions: Dercum's Disease
Sponsor: Caliway Biopharmaceuticals Co., Ltd.
Enrollment: 19
Locations: 2
Primary Endpoint: To estimate the treatment effect, as measured by pain, between CBL-514 and placebo in participants with DD.
Status: Active, not recruiting
Last Updated: 18 days ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials Related News

Overview

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.

Detailed Description

This is a phase 2 study to evaluate the efficacy and safety of CBL-514 injections in participants with Dercum's disease lipomas. A total of approximately 25 participants will be randomized. Eligible participants will be randomized (1:1) to receive either CBL-514 or placebo once every 4 weeks for up to 5 treatments for each selected lipoma. Participant numbers are expected to be approximately balanced in each dose group (CBL-514 group and placebo group). Eligible participants must have at least 4 and up to 10 painful individual lipomas. The injection volume per lipoma will depend on the lipoma size (as determined by ultrasound).

Registry

clinicaltrials.gov

Start Date

November 27, 2024

End Date

April 1, 2026

Last Updated

18 days ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Caliway Biopharmaceuticals Co., Ltd.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•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
•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.

Exclusion Criteria

•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.

Arms & Interventions

CBL-514 injection

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.

Intervention: CBL-514 injection

0.9% Sodium Chloride

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.

Intervention: 0.9% Sodium chloride

Outcomes

Primary Outcomes

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

Time Frame: Week 20

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.

To estimate the treatment effect, as measured by Partial Response (PR), between CBL-514 and placebo in participants with Dercum's disease (DD).

Time Frame: Week 20

Lipoma volume will be determined by ultrasound assessment.

Secondary Outcomes

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)
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 estimate the treatment effect, as measured by Partial Response (PR), between CBL-514 and placebo.(Week 24)
To assess the treatment effect, as measured by pain, 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)
To estimate the treatment effect, as measured by pain, between CBL-514 and placebo in participants with DD.(Week 20)
To estimate the treatment effect, as measured by Complete Response (CR), between CBL-514 and placebo.(Up to 24 weeks)
To evaluate the treatment effect, as measured by change in volume, between CBL-514 and placebo.(Up to 24 weeks)