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.
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 20 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. This means that there will be 10 participants 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).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
20
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.
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.
Investigator Site
Spokane, Washington, United States
RECRUITINGTo estimate the treatment effect, as measured by Partial Response (PR), between CBL-514 and placebo in participants with Dercum's disease (DD).
Lipoma volume will be determined by ultrasound assessment.
Time frame: Week 20
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.
Time frame: Week 20
To estimate the treatment effect, as measured by Complete Response (CR), between CBL-514 and placebo.
Lipoma volume will be determined by ultrasound assessment.
Time frame: Up to 24 weeks
To estimate the treatment effect, as measured by Partial Response (PR), between CBL-514 and placebo.
Lipoma volume will be determined by ultrasound assessment.
Time frame: Week 24
To evaluate the treatment effect, as measured by change in volume, between CBL-514 and placebo.
Lipoma volume will be determined by ultrasound assessment.
Time frame: Up to 24 weeks
To assess the treatment effect, as measured by pain, between CBL-514 and placebo.
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.
Time frame: Up to 24 weeks
To evaluate the incidence of adverse events of special interests (AESI) as defined in the protocol.
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Adverse events (AEs) will be assessed by recording of clinical responses (e.g. treatment-emergent adverse events (TEAEs), and injection site reactions (ISRs)).
Time frame: 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.
Assessed by significant clinical changes in safety parameter (e.g. laboratory assessments, vital signs, ECGs, physical examinations).
Time frame: From baseline to 8 weeks post final treatment