Dercum's DiseasePhase 2NCT05387733
A Study to Evaluate the Efficacy and Safety of CBL-514 in Participants With Dercum's Disease Lipomas
A Phase 2 study testing 10 mg CBL-514 per injection and 15 mg CBL-514 per injection in people with Dercum's Disease, run by Caliway Biopharmaceuticals Co., Ltd. at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 64 years
- Conditions the study is for
- Dercum's Disease
- What is being tested?
- 10 mg CBL-514 per injection and 15 mg CBL-514 per injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Oct 2022to 3 Apr 2023
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 64 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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 1.
- 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.
Who cannot join
- 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.
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- 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.
- 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.
- 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.
- Participants who are unable to receive local anesthesia.
- Participants with known allergies or sensitivities to the IP or its components.
- Participants that are in use of other investigational drug or device within 4 weeks prior to Screening.
- Participant who has undergone vaccination (including with a live-attenuated vaccine) within 14 days prior to administration of the IP.
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
Read the 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.
- Conditions
- Dercum's Disease
- Being tested
- 10 mg CBL-514 per injection, 15 mg CBL-514 per injection
- Keywords
- Adiposis Dolorosa, Lipedema, Lipomatosis, Skin Diseases, Lipid Metabolism Disorders, Metabolic Diseases, Connective Tissue Diseases
- Medical subject headings
- Adiposis Dolorosa, Lipedema, Lipomatosis, Skin Diseases, Lipid Metabolism Disorders, Metabolic Diseases, Connective Tissue Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
10 mg CBL-514 per injectionDrug
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.
15 mg CBL-514 per injectionDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| CBL-514 Group 1 | Experimental | Eligible participants will be enrolled and randomized into one of 2 dose groups. Group 1 with 10 mg CBL-514 per injection. Receives: Drug: 10 mg CBL-514 per injection |
| CBL-514 Group 2 | Experimental | Eligible participants will be enrolled and randomized to one of 2 dose groups. Group 2 with 15 mg CBL-514 per injection. Receives: Drug: 15 mg CBL-514 per injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 0-84 days
Secondary outcomes (4)
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.
Measured: 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".
Measured: 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)).
Measured: 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).
Measured: 0-84 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 May 2022
- Study start3 Oct 2022
- Primary completion3 Apr 2023
- Study completion3 Apr 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dercum's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Caliway Biopharmaceuticals Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Anne Sheu, Study director, Caliway Biopharmaceuticals Co., Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05387733
- Sponsor's protocol ID
- CBL-0201DD
- Status last verified
- Apr 2023
- First submitted
- 19 May 2022
- Last updated
- 20 Apr 2023
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05387733
- Acronym / sponsor ID
- CBL-0201DD
- Conditions
- Dercum's Disease
- Keywords
- Adiposis Dolorosa, Lipedema, Lipomatosis, Skin Diseases, Lipid Metabolism Disorders, Metabolic Diseases, Connective Tissue Diseases
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Caliway Biopharmaceuticals Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 3 Oct 2022 (actual)
- Primary completion
- 3 Apr 2023 (actual)
- Study completion
- 3 Apr 2023 (actual)
- First posted
- 24 May 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| CBL-514 Group 1 | Experimental | Drug: 10 mg CBL-514 per injectionEligible participants will be enrolled and randomized into one of 2 dose groups. Group 1 with 10 mg CBL-514 per injection. |
| CBL-514 Group 2 | Experimental | Drug: 15 mg CBL-514 per injectionEligible participants will be enrolled and randomized to one of 2 dose groups. Group 2 with 15 mg CBL-514 per injection. |
Who may qualify
- Ages
- 18 to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (8 listed)
- 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 1.
- 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.
- 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.
Key exclusion criteria (16 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 10 mg CBL-514 per injectionThe 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. | Drug | CBL-514 Group 1 |
| 15 mg CBL-514 per injectionThe 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. | Drug | CBL-514 Group 2 |
Primary outcome
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.
4 secondary outcomes
Efficacy of CBL-514 following injection into lipomas or nodules of participants with Dercum's disease.
Time frame: 0-84 days
The change in pain of individual lipomas following CBL-514 injection in participants with Dercum's disease.
Time frame: 0-84 days
The incidence of adverse events of special interests (AESI) as defined in the protocol.
Time frame: 0-84 days
The incidence of clinically significant abnormal findings as defined in the protocol.
Time frame: 0-84 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Apr 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Anne Sheu study director
Caliway Biopharmaceuticals Co., Ltd.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 10 mg CBL-514 per injection and 15 mg CBL-514 per injection in people with Dercum's Disease, run by Caliway Biopharmaceuticals Co., Ltd. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy of CBL-514 following injection into lipomas or nodules of participants with Dercum's disease. (time frame: 0-84 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Apr 2023.