RECRUITING

A Study to Evaluate the Efficacy and Safety of CBL-514 Compared to Placebo in Participants With Dercum's Disease Lipomas

Conditions

Dercum's Disease Adiposis Dolorosa Lipedema Lipomatosis Skin Diseases Lipid Metabolism Disorders Metabolism Diseases Connective Tissue Diseases

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2024-11-27

Study Completion:2026-04

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06303570

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 64 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT

Inclusion Criteria	Exclusion 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 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. 1. Chronic pain (\>3 months) in the adipose tissue specific to the presence of lipomas and/or 2. Pain in and around multiple lipomas. 4. 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. 5. Generally considered healthy according to medical history, physical examination, ECG, and laboratory evaluation. 6. 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.	1. 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. 2. Unable to tolerate SC injections. 3. Diagnosed with another disorder with similar characteristics as DD as follows. 1. Madelung's disease: multiple symmetric lipomatosis only localized in the upper body ie, shoulders, neck, or head. 2. Panniculitis: inflammation of the SC adipose tissue, characterized by tender nodules and systemic signs. 3. Proteus syndrome: disproportionate and asymmetric overgrowth of skin, and fatty and connective tissue. 4. PTEN hamartoma syndrome: multiple hamartomas which includes segmental overgrowth, lipomatosis, arteriovenous malformation, and epidermal nevus. 5. Gardner syndrome: multiple digestive adenomas with osteomas and multiple skin and soft tissue tumors. 4. Diagnosed with coagulation disorders or is receiving anticoagulant/antiplatelet therapy or medications or dietary supplements, which inhibit coagulation or platelet aggregation. 5. 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. 6. Any clinically significant cardiac, hepatic, renal or neurologic/psychiatric disorders that in the opinion of the Investigator places the participant at significant risk, including but not limited to any of the following: 1. Participants with cirrhosis or with inadequate liver function at screening defined as aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase, or total bilirubin \>3.0 upper limit of normal (ULN). 2. Participants with renal impairment, defined as both serum creatinine and blood urea nitrogen \>1.5× ULN, or estimated glomerular filtration rate (eGFR) \<90 mL/min/1.73 m2, or who are currently on dialysis. 7. Participant with a history of human immunodeficiency virus (HIV)-1 infection or participant with active HIV infection at screening with positive HIV antigen/antibody (Ag/Ab) combo test. 8. Participant is undergoing chronic steroid or immunosuppressive therapy, with the exception of: 1. Use of oral steroid inhalation indicated for asthma management 2. Use of topical steroid application for skin conditions that are not directly applied to or indirectly affect the treatment area 3. Use of steroid as part of treatment for DD, and no side effects from chornic use. 9. Participant with active or prior history of malignancies within 5 years before screening or currently being evaluated for a possible malignancy, with the exception of adequately treated basal cell carcinoma of skin and in situ squamous cell carcinoma of skin at Investigator's discretion. 10. Abnormal skin, local skin conditions, or body modifications at the treatment area, which in the opinion of the Investigator, is inappropriate for participation in the study, including but not limited to any of the following: 1. Prior wound, scar tissue, or infection in the treated area. 2. Tattoo in the treated area. 11. Use of any analgesic except Cannabis within 2 days prior to Day 1 and use of Cannabis within 14 days prior to Day 1. 12. Requiring continual use of any medication that is known to strongly inhibit or induce CYP1A2 enzymes, sensitive CYP1A2 substrates or drugs with narrow therapeutic index during the study that, in the opinion of the Investigator, may affect the evaluation of the study product or place the participant at undue risk. 13. Participant who has undergone liposuction or aesthetic surgery to the region to be treated before screening or during the study, or aesthetic procedure for body contouring (eg, cryolipolysis, ultrasonic lipolysis, low level laser therapy, lipolysis injection) to the region to be treated within 12 months before screening or during the study. 14. Unable to receive local anesthesia. 15. Known allergies or sensitivities to the study drug or its components. 16. Use of other investigational drug or device within 12 weeks prior to screening.

Inclusion Criteria

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

1. 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.
2. Unable to tolerate SC injections.
3. Diagnosed with another disorder with similar characteristics as DD as follows.
1. Madelung's disease: multiple symmetric lipomatosis only localized in the upper body ie, shoulders, neck, or head.
2. Panniculitis: inflammation of the SC adipose tissue, characterized by tender nodules and systemic signs.
3. Proteus syndrome: disproportionate and asymmetric overgrowth of skin, and fatty and connective tissue.
4. PTEN hamartoma syndrome: multiple hamartomas which includes segmental overgrowth, lipomatosis, arteriovenous malformation, and epidermal nevus.
5. Gardner syndrome: multiple digestive adenomas with osteomas and multiple skin and soft tissue tumors.
4. Diagnosed with coagulation disorders or is receiving anticoagulant/antiplatelet therapy or medications or dietary supplements, which inhibit coagulation or platelet aggregation.
5. 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.
6. Any clinically significant cardiac, hepatic, renal or neurologic/psychiatric disorders that in the opinion of the Investigator places the participant at significant risk, including but not limited to any of the following:
1. Participants with cirrhosis or with inadequate liver function at screening defined as aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase, or total bilirubin \>3.0 upper limit of normal (ULN).
2. Participants with renal impairment, defined as both serum creatinine and blood urea nitrogen \>1.5× ULN, or estimated glomerular filtration rate (eGFR) \<90 mL/min/1.73 m2, or who are currently on dialysis.
7. Participant with a history of human immunodeficiency virus (HIV)-1 infection or participant with active HIV infection at screening with positive HIV antigen/antibody (Ag/Ab) combo test.
8. Participant is undergoing chronic steroid or immunosuppressive therapy, with the exception of:
1. Use of oral steroid inhalation indicated for asthma management
2. Use of topical steroid application for skin conditions that are not directly applied to or indirectly affect the treatment area
3. Use of steroid as part of treatment for DD, and no side effects from chornic use.
9. Participant with active or prior history of malignancies within 5 years before screening or currently being evaluated for a possible malignancy, with the exception of adequately treated basal cell carcinoma of skin and in situ squamous cell carcinoma of skin at Investigator's discretion.
10. Abnormal skin, local skin conditions, or body modifications at the treatment area, which in the opinion of the Investigator, is inappropriate for participation in the study, including but not limited to any of the following:
1. Prior wound, scar tissue, or infection in the treated area.
2. Tattoo in the treated area.
11. Use of any analgesic except Cannabis within 2 days prior to Day 1 and use of Cannabis within 14 days prior to Day 1.
12. Requiring continual use of any medication that is known to strongly inhibit or induce CYP1A2 enzymes, sensitive CYP1A2 substrates or drugs with narrow therapeutic index during the study that, in the opinion of the Investigator, may affect the evaluation of the study product or place the participant at undue risk.
13. Participant who has undergone liposuction or aesthetic surgery to the region to be treated before screening or during the study, or aesthetic procedure for body contouring (eg, cryolipolysis, ultrasonic lipolysis, low level laser therapy, lipolysis injection) to the region to be treated within 12 months before screening or during the study.
14. Unable to receive local anesthesia.
15. Known allergies or sensitivities to the study drug or its components.
16. Use of other investigational drug or device within 12 weeks prior to screening.

Contacts and Locations

Study Contact

Sandy Liu

CONTACT

+886 2 26971355

cr@caliway.com.tw

Study Locations (Sites)

Investigator Site

Spokane, Washington, 99204

United States

Collaborators and Investigators

Sponsor: Caliway Biopharmaceuticals Co., Ltd.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-11-27

Study Completion Date2026-04

Study Record Updates

Study Start Date2024-11-27

Study Completion Date2026-04

Terms related to this study

Keywords Provided by Researchers

Adiposis Dolorosa
Lipedema
Lipomatosis
Skin Diseases
Lipid Metabolism Disorders
Metabolism Diseases
Connective Tissue Diseases

Additional Relevant MeSH Terms

Dercum's Disease