Clinical trial NCT05387733: A Study to Evaluate the Efficacy and Safety of CBL-514 in Participants With Dercum's Disease Lipomas
Official title: An Open-Label, Randomized Phase 2a Study to Evaluate the Efficacy and Safety of CBL-514 in Participants With Dercum's Disease Lipomas
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Dercum's Disease
Keywords: Adiposis Dolorosa
Lipedema
Lipomatosis
Skin Diseases
Lipid Metabolism Disorders
Metabolic Diseases
Connective Tissue Diseases
Lead sponsor: Caliway Biopharmaceuticals Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2022-05-24
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Maximum age (years): 64
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: Inclusion Criteria:

1. Male or female, aged 18 years to 64 years old (at Screening), inclusive.
2. Body mass index (BMI) \>18.5 kg/m2 at Screening and Day 1.
3. 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.

   1. Chronic pain (\> 3 months) in the adipose tissue
   2. Pain in and around multiple lipomas.
4. 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.
5. Generally considered healthy according to medical history, physical examination, electrocardiogram (ECG), and laboratory evaluation.
6. 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:

1. 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.
2. Participants that are unable to tolerate subcutaneous injections.
3. Participants that are diagnosed with another disorder with similar characteristics as Dercum's Disease.
4. Participants that are diagnosed with coagulation disorders or is receiving anticoagulant/antiplatelet therapy or medications or dietary supplements, which inhibit coagulation or platelet aggregation.
5. 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.
6. 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.
7. Participants with positive blood screen for Hepatitis B surface antigen (HBsAg), Hepatitis C virus, or Human immunodeficiency virus (HIV).
8. Participants with clinical history of active primary or secondary immunodeficiency, autoimmune disease, or is undergoing chronic steroid or immunosuppressive therapy.
9. 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.
10. 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.
11. Participants requiring continual use of the following therapeutic agents during the study: terfenadine, buspirone, fexofenadine, any medication that is known to strongly inhibit or induce CYP enzymes, sensitive CYP substrates or drugs with narrow therapeutic index, in the opinion of the Investigator, may affect the evaluation of the study product or place the participant at undue risk. If a participant requires the abovementioned therapeutic agents during the study for any reason, they should not be used for at least 2 days before dosing and until 1 day postdose.
12. Participants that undergone aesthetic procedure (eg, liposuction, cryolipolysis, ultrasonic lipolysis, low level laser therapy \[LLLT\], lipolysis injection) to the region to be treated before Screening or during the study.
13. Participants who are unable to receive local anesthesia.
14. Participants with known allergies or sensitivities to the IP or its components.
15. Participants that are in use of other investigational drug or device within 4 weeks prior to Screening.
16. Participant who has undergone vaccination (including with a live-attenuated vaccine) within 14 days prior to administration of the IP.

Primary outcomes:
measure: Efficacy of CBL-514 following injection into lipomas or nodules of participants with Dercum's disease.
timeFrame: 0-84 days
Secondary outcomes:
measure: Efficacy of CBL-514 following injection into lipomas or nodules of participants with Dercum's disease.
timeFrame: 0-84 days
measure: The change in pain of individual lipomas following CBL-514 injection in participants with Dercum's disease.
timeFrame: 0-84 days
measure: The incidence of adverse events of special interests (AESI) as defined in the protocol.
timeFrame: 0-84 days
measure: The incidence of clinically significant abnormal findings as defined in the protocol.
timeFrame: 0-84 days
Other outcomes:
Locations:
city: Tucson
country: United States
facility: Karen L. Herbst, MD, PC
state: Arizona
Study officials:
affiliation: Caliway Biopharmaceuticals Co., Ltd.
name: Anne Sheu
role: STUDY_DIRECTOR
References: