NCT05387733
Completed
Phase 2
An Open-Label, Randomized Phase 2a Study to Evaluate the Efficacy and Safety of CBL-514 in Participants With Dercum's Disease Lipomas
ConditionsDercum's Disease
Overview
- Phase
- Phase 2
- Intervention
- 10 mg CBL-514 per injection
- Conditions
- Dercum's Disease
- Sponsor
- Caliway Biopharmaceuticals Co., Ltd.
- Enrollment
- 12
- Locations
- 1
- Primary Endpoint
- Efficacy of CBL-514 following injection into lipomas or nodules of participants with Dercum's disease.
- Status
- Completed
- Last Updated
- 3 years ago
Overview
Brief Summary
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.
Detailed Description
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.
Investigators
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 Dercum's disease and/or fulfills the following clinical criteria of Dercum's disease in localized nodular form. The final diagnosis of disease is in the opinion of the PI.
- •Chronic pain (\> 3 months) in the adipose tissue
- •Pain in and around multiple lipomas.
- •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.
- •Voluntarily signs the Informed Consent Form (ICF) and, in the opinion of the PI or designee, is physically and mentally capable of participating in the study, and willing to adhere to study procedures.
Exclusion Criteria
- •Women of childbearing potential (WOCBP) who are 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 are currently pregnant or lactating. Male participants who are not willing to commit to an acceptable contraceptive method. Female participants who are not WOCBP are not required to use contraception.
- •Participants that are unable to tolerate subcutaneous injections.
- •Participants that are diagnosed with another disorder with similar characteristics as Dercum's Disease.
- •Participants that are diagnosed with coagulation disorders or is receiving anticoagulant/antiplatelet therapy or medications or dietary supplements, which inhibit coagulation or platelet aggregation.
- •Participants who has fasting glucose concentration \>200 mg/dL, delayed wound healing, bleeding risk, or any diabetic risks which, in the opinion of PI or designee, is inappropriate to participate in the study.
- •Participants with any clinically significant cardiac, hepatic, renal or neurologic/psychiatric disorders that in the opinion of the PI places the participant at significant risk.
- •Participants with positive blood screen for Hepatitis B surface antigen (HBsAg), Hepatitis C virus, or Human immunodeficiency virus (HIV).
- •Participants with clinical history of active primary or secondary immunodeficiency, autoimmune disease, or is undergoing chronic steroid or immunosuppressive therapy.
- •Participants with active, suspected, or prior history of malignancies within 5 years before Screening or being worked-up for a possible malignancy. Except adequately treated basal cell carcinoma of the skin and in situ squamous cell carcinoma of the skin would be eligible as per PI's discretion.
- •Participants with abnormal skin, local skin conditions, or body modifications at the treatment area, which in the opinion of the PI, is inappropriate for participation in the study, including but not limited to prior wound, scar tissue, infection in the treated area, or tatoo in the treated area.
Arms & Interventions
CBL-514 Group 1
Eligible participants will be enrolled and randomized into one of 2 dose groups. Group 1 with 10 mg CBL-514 per injection.
Intervention: 10 mg CBL-514 per injection
CBL-514 Group 2
Eligible participants will be enrolled and randomized to one of 2 dose groups. Group 2 with 15 mg CBL-514 per injection.
Intervention: 15 mg CBL-514 per injection
Outcomes
Primary Outcomes
Efficacy of CBL-514 following injection into lipomas or nodules of participants with Dercum's disease.
Time Frame: 0-84 days
Evaluated the change of lipomas or nodules dimensions (as measured by ultrasound) after treatment with CBL-514 compared to Baseline.
Secondary Outcomes
- Efficacy of CBL-514 following injection into lipomas or nodules of participants with Dercum's disease.(0-84 days)
- The change in pain of individual lipomas following CBL-514 injection in participants with Dercum's disease.(0-84 days)
- The incidence of adverse events of special interests (AESI) as defined in the protocol.(0-84 days)
- The incidence of clinically significant abnormal findings as defined in the protocol.(0-84 days)
Study Sites (1)
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Related News
Caliway's CBL-514 Receives EMA Orphan Drug Designation for Dercum's Disease- Caliway Biopharmaceuticals' CBL-514 has been granted Orphan Drug Designation by the EMA for the treatment of Dercum's disease, a rare and painful condition.
- CBL-514 is a novel small molecule drug that induces adipocyte apoptosis and lipolysis, showing promising efficacy in reducing painful lipomas associated with Dercum's disease.
- A Phase 2 study demonstrated that CBL-514 reduced the size of painful lipomas by over 50% in 64.5% of patients, with 38.7% experiencing complete clearance and significant pain reduction.
- With both EMA and FDA Orphan Drug Designations, Caliway anticipates accelerated clinical development for CBL-514, positioning it as a potential first-in-class therapy.Caliway's CBL-514 Receives EMA Orphan Drug Designation for Dercum's Disease- Caliway Biopharmaceuticals' CBL-514 receives Orphan Drug Designation from the EMA for Dercum's disease, marking it as the first drug with this designation.
- CBL-514 previously received both FDA Orphan Drug and Fast Track Designations for Dercum's disease, potentially accelerating its clinical development.
- Phase 2 study results showed CBL-514 significantly reduced lipoma dimension by over 50% and improved pain scores by 4.7 points in Dercum's disease patients.
- A Phase 2b study is currently underway, with results anticipated in Q4 2025, further evaluating CBL-514's efficacy in treating Dercum's disease.Caliway's CBL-514 Receives EMA Orphan Drug Designation for Dercum's Disease- Caliway Biopharmaceuticals' CBL-514 has been granted Orphan Drug Designation by the EMA for the treatment of Dercum's disease, a rare and painful adipose tissue disorder.
- CBL-514 is the first drug to receive EMA Orphan Drug Designation for Dercum's disease, complementing its prior FDA Fast Track and Orphan Drug Designations.
- Phase 2 study results showed CBL-514 significantly reduced lipoma dimension by 50% and improved pain scores by 4.7 points in patients with Dercum's disease.
- A Phase 2b study of CBL-514 is currently underway, with results expected in Q4 2025, potentially positioning it as a first-in-class therapy.Caliway's CBL-514 Receives EMA Orphan Drug Designation for Dercum's Disease- Caliway Biopharmaceuticals' CBL-514 has been granted Orphan Drug Designation by the EMA for the treatment of Dercum's disease, a rare and painful adipose tissue disorder.
- CBL-514 is the first drug to receive this designation from the EMA and also holds both FDA Orphan Drug and Fast Track Designations for Dercum's disease.
- Phase 2 study results showed CBL-514 significantly reduced lipoma dimension by over 50% and improved pain scores by 4.7 points in Dercum's disease patients.
- A Phase 2b study is currently underway, with results expected in Q4 2025, further evaluating CBL-514's efficacy in treating Dercum's disease.Caliway's CBL-514 Receives EMA Orphan Drug Designation for Dercum's Disease- Caliway Biopharmaceuticals' CBL-514 has been granted Orphan Drug Designation by the EMA for the treatment of Dercum's disease, a rare and painful condition.
- CBL-514 is the first drug to receive this designation from the EMA and also holds both FDA Orphan Drug and Fast Track Designations for Dercum's disease.
- Phase 2 study results showed CBL-514 significantly reduced lipoma dimension by 50% and improved pain scores by 4.7 points in patients with Dercum's disease.
- A Phase 2b study is currently underway, with results expected in Q4 2025, further evaluating CBL-514's efficacy in treating Dercum's disease.