This Phase 2 study will be an open-label and randomized study to assess the efficacy and safety of CBL-514 in participants with Dercum's Disease lipomas.
This is a Phase 2a study to evaluate the efficacy and safety of CBL-514 injections in participants with Dercum's disease. It will employ an open-label, randomized study design. Eligible participants will have at least 4 painful lipomas of appropriate size and up to 8 lipomas will be treated per subject. Eligible participants will be enrolled and randomized to 2 dose groups, with 6 participants per group. Each enrolled participant will receive up to 2 courses of their allocated dose of CBL-514 administered into selected lipomas, which the injection volume per lipoma will be based on the lipoma size, determined by ultrasound.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
12
The total injection volume per lipoma will be based on the lipoma size, as determined by ultrasound. Dosing sceme is presented as below: Lipoma diameter of ≥1.0cm and ≤2.0cm - 1 injection (2 mL each); total of 10 mg CBL-514. Lipoma diameter of \>2.0cm and ≤3.5cm - 2 injections (2 mL each); total of 20 mg CBL-514. Lipoma diameter of \>3.5cm and ≤5.0cm - 3 injections (2 mL each); total of 30 mg CBL-514. Lipoma diameter of \>5.0cm and ≤6.0cm - 5 injections (2 mL each); total of 50 mg CBL-514.
The total injection volume per lipoma will be based on the lipoma size, as determined by ultrasound. Dosing sceme is presented as below: Lipoma diameter of ≥1.0cm and ≤2.0cm - 1 injection (3 mL each); total of 15 mg CBL-514. Lipoma diameter of \>2.0cm and ≤3.5cm - 2 injections (3 mL each); total of 30 mg CBL-514. Lipoma diameter of \>3.5cm and ≤5.0cm - 3 injections (3 mL each); total of 45 mg CBL-514. Lipoma diameter of \>5.0cm and ≤6.0cm - 5 injections (3 mL each); total of 75 mg CBL-514.
Karen L. Herbst, MD, PC
Tucson, Arizona, United States
Efficacy of CBL-514 following injection into lipomas or nodules of participants with Dercum's disease.
Evaluated the change of lipomas or nodules dimensions (as measured by ultrasound) after treatment with CBL-514 compared to Baseline.
Time frame: 0-84 days
Efficacy of CBL-514 following injection into lipomas or nodules of participants with Dercum's disease.
Evaluated by percentage of lipomas or nodules with dimensions change and/or complete clearance (as measured by ultrasound) after treatment with CBL-514 compared to Baseline.
Time frame: 0-84 days
The change in pain of individual lipomas following CBL-514 injection in participants with Dercum's disease.
Assessed by change in pain assessment of individual lipomas evaluated by the Comparative Pain Scale compared to Baseline, where 0 is "no pain" and 10 is "worst pain".
Time frame: 0-84 days
The incidence of adverse events of special interests (AESI) as defined in the protocol.
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: 0-84 days
The incidence of clinically significant abnormal findings as defined in the protocol.
Assessed by significant clinical changes in safety parameter (e.g. laboratory assessments, vital signs, ECGs, physical examinations).
Time frame: 0-84 days
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