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A Study to Evaluate the Efficacy and Safety of CBL-514 in Participants With EFP; Cellulite (Stage 1)

Primary Purpose

Cellulite

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
40 mg CBL-514
60 mg CBL-514
80 mg CBL-514
Sponsored by
Caliway Biopharmaceuticals Co., Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cellulite

Eligibility Criteria

18 Years - 64 Years (Adult)FemaleDoes not accept healthy volunteers

Inclusion Criteria: Female aged 18 years to 64 years old (at Screening), inclusive. Have a BMI > 18.5 and < 35 kg/m2 and body weight ≥ 50 kg at Screening and Day 1. The participant has both sides of posterolateral thighs assessed according to the modified Hexsel CSS (A) number of evident depressions, (B) depression depth scale, and (C) morphological appearance of skin surface alterations. On assessment by the modified Hexsel CSS, the participant scores at least 4 and no greater than 8 at Screening and Day 1. The total score must contain: Hexsel CSS item (A) 'number of evident depressions' score of ≥ 1 Hexsel CSS item (B) 'depth of depressions' score of ≥ 1 Participant has a stable lifestyle (e.g., exercise, eating patterns, and smoking habit) per participant report for at least 3 months before Screening and during the study. Voluntarily signs the informed consent form and, in the opinion of the Investigator or delegate, is physically and mentally capable of participating in the study, and willing to adhere to study procedures. Exclusion Criteria: Female participant of childbearing potential who is not willing to commit to an acceptable contraceptive regimen with her partner from the time of Screening and throughout study participation until 90 days after the last IP dose, or who is currently pregnant or lactating. Note: females who are not of childbearing potential are not required to use contraception. Females not of childbearing potential are defined as those who have been surgically sterilized (hysterectomy or bilateral oophorectomy) or who are postmenopausal (defined as aged at least 50 years old with ≥ 12 months of amenorrhea and a follicle-stimulating hormone [FSH] > 30 IU/L at Screening). Participant diagnosed with coagulation disorders or is receiving anticoagulant/antiplatelet therapy or medications or dietary supplements, which impede coagulation or platelet aggregation within 14 days prior to the IP administration. Participant has hemoglobin A1c (HbA1c) ≥ 9%, delayed wound healing, or any diabetic risks which, in the opinion of the Investigator (or designee) is inappropriate to participate in the study. Participant has a clinically significant cardiovascular disease and abnormal findings in electrocardiogram (ECG) at Investigator's (or designee's) discretion. Participant with active or prior history of malignancies within 5 years before Screening or being assessed 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 Investigator's (or designee's) discretion. Participant with a history of human immunodeficiency virus (HIV)-1 infection, or participants with active HIV infection at Screening with a positive HIV antigen/antibody (Ag/Ab) combination test. Participant with a history of trypanophobia, the extreme fear of medical procedures involving injections or needles, or who experience vasovagal syncope and pass out at the sight of blood or a needle. Participant with any hepatic medical condition that, in the opinion of the Investigator (or designee), would interfere with assessment of safety or efficacy or compromise the participant's ability to undergo study procedures or provide informed consent. Participant who has a recent history of major depression, anxiety, or other psychiatric disease requiring treatment with prescription medication within 6 months prior to Screening. Participant has abnormal skin or local skin conditions at the treatment area, which in the opinion of the Investigator (or designee), is inappropriate to participate in the study. This includes but is not limited to any of the following: skin manifestations of a systemic disease any abnormality of the skin or soft tissues on the anticipated treatment area skin laxity on treatment area when the participant is in the standing position sensory loss or dysesthesia in the area to be treated evidence of any cause of enlargement in the area to be treated other than localized subcutaneous fat tattoos on the area to be treated participant with a propensity for keloid or hypertrophic scarring. Participant who has had the following surgical or aesthetic procedures: liposuction, cryolipolysis, ultrasonic lipolysis, low level laser therapy (LLLT), or lipolysis injection to the region to be treated before Screening medical device, injection (including but not limited to collagenase clostridium histolyticum injections and collagen stimulating injections), over-the-counter (OTC) cosmetic cream, or cosmetic program to prevent or mitigate EFP to the region to be treated within 12 months before Screening and throughout study participation massage to the region to be treated within 2 weeks before Screening and throughout study participation. Participant is undergoing chronic steroid or immunosuppressive therapy, except for asthma inhaler or topical steroids for skin conditions if the medications are not used on the treatment area. Participating is 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, which in the opinion of the Investigator (or designee), may affect the evaluation of the investigational product or place the participant at undue risk. If a participant needs to use the above-mentioned therapeutic agents during the study for any reason, these therapeutic agents should not be used at least for 2 days before dosing and until 1 day after dosing. Participant receives nonsteroidal anti-inflammatory drug (NSAID) including aspirin within 14 days prior to the IP administration. Participant is unable to receive topical anesthesia (e.g., history of hypersensitivity to lidocaine). Participant with known allergies or sensitivities to the IP or its components. Participant with liver cirrhosis or with inadequate liver function at Screening defined as aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase (ALKP), total bilirubin (TBIL), or gamma-glutamyl transferase (GGT) > 3.0 × upper limit of normal (ULN). Participant with any renal impairment, defined as abnormal serum creatinine, and urea > 1.5 × ULN or estimated glomerular filtration rate (eGFR) < 90 mL/min/1.73 m2. Participants who are currently on dialysis should be excluded. Participants with an eGFR ≥ 60 and < 90 mL/min/1.73 m2 at Screening should be evaluated by the Investigator to exclude pre-existing renal disease or associated dysfunction. If mild decrease in eGFR is assessed by the Investigator as not clinically significant or not related to dysfunction, the participants may be eligible upon the Investigator's assessment. Use of other investigational product or device within 4 weeks prior to Screening.

Sites / Locations

  • Investigational Site 1
  • Investigational Site 2

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

CBL-514 40mg

CBL-514 60mg

CBL-514 80mg

Arm Description

Outcomes

Primary Outcome Measures

Evaluation of the improvement of edematous fibrosclerotic panniculopathy (EFP) measured by cellulite severity scale from baseline following administration of 1 course of CBL-514.
Change in total scores from baseline according to the modified Hexsel cellulite severity scale (CSS) at V4 and V5 based on (A) number of evident depressions, (B) depression depth scale, and (C) morphological appearance of skin surface alterations. (A) Number of evident depressions: '0' = None/no depressions '1' = 1 to 4 depressions are visible '2' = 5 to 9 depressions are visible '3' = 10 or more depressions are visible (B) Depth of depressions: '0' = No depressions '1' = Superficial depressions '2' = Medium depth depressions '3' = Deep depressions (C) Morphological appearance of skin surface alterations: '0' = No raised areas '1' = 'Orange peel' appearance '2' = 'Cottage cheese' appearance '3' = 'Mattress' appearance The total score of (A), (B) and (C) will determine the cellulite severity level as follows : 0 = None 1-3 = Mild 4-6 = Moderate 7-9 = Severe

Secondary Outcome Measures

Percentage of participants' thighs that achieve at least 1-level severity improvement measured by cellulite severity scale from baseline following administration of 1 course of CBL-514.
Percentage of participants' thighs that achieve at least 1-level severity improvement determined by the total scores of modified Hexsel CSS at V4 and V5 compared to baseline.

Full Information

First Posted
November 21, 2022
Last Updated
June 16, 2023
Sponsor
Caliway Biopharmaceuticals Co., Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT05632926
Brief Title
A Study to Evaluate the Efficacy and Safety of CBL-514 in Participants With EFP; Cellulite (Stage 1)
Official Title
A Phase 2a, Open Label Study to Evaluate the Safety, Tolerability, and Efficacy of CBL-514 Injection for the Treatment of Edematous Fibrosclerotic Panniculopathy (EFP) Cellulite (Stage 1)
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Completed
Study Start Date
January 9, 2023 (Actual)
Primary Completion Date
May 3, 2023 (Actual)
Study Completion Date
May 3, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Caliway Biopharmaceuticals Co., Ltd.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
The Stage 1 of this phase 2 study is an open-label single ascending dose (SAD) study to assess the efficacy and safety of CBL-514 in participants with Edematous Fibrosclerotic Panniculopathy (EFP) cellulite.
Detailed Description
This Phase 2a study has an integrated design consisting of a single ascending dose (SAD) part in Stage 1 followed by a single-arm design in Stage 2. The Stage 1 will include a total of 12 participants enrolled in 3 sequential escalating CBL-514 dose groups.The groups will be open label and each group will enroll 4 participants. Eligible participants will be sequentially assigned to receive 1 course of allocated CBL-514 dose administered by subcutaneous injection on both sides of the posterolateral thighs.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cellulite

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
12 (Actual)

8. Arms, Groups, and Interventions

Arm Title
CBL-514 40mg
Arm Type
Experimental
Arm Title
CBL-514 60mg
Arm Type
Experimental
Arm Title
CBL-514 80mg
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
40 mg CBL-514
Intervention Description
CBL-514 will be administered at the raised area of cellulite.
Intervention Type
Drug
Intervention Name(s)
60 mg CBL-514
Intervention Description
CBL-514 will be administered at the raised area of cellulite.
Intervention Type
Drug
Intervention Name(s)
80 mg CBL-514
Intervention Description
CBL-514 will be administered at the raised area of cellulite.
Primary Outcome Measure Information:
Title
Evaluation of the improvement of edematous fibrosclerotic panniculopathy (EFP) measured by cellulite severity scale from baseline following administration of 1 course of CBL-514.
Description
Change in total scores from baseline according to the modified Hexsel cellulite severity scale (CSS) at V4 and V5 based on (A) number of evident depressions, (B) depression depth scale, and (C) morphological appearance of skin surface alterations. (A) Number of evident depressions: '0' = None/no depressions '1' = 1 to 4 depressions are visible '2' = 5 to 9 depressions are visible '3' = 10 or more depressions are visible (B) Depth of depressions: '0' = No depressions '1' = Superficial depressions '2' = Medium depth depressions '3' = Deep depressions (C) Morphological appearance of skin surface alterations: '0' = No raised areas '1' = 'Orange peel' appearance '2' = 'Cottage cheese' appearance '3' = 'Mattress' appearance The total score of (A), (B) and (C) will determine the cellulite severity level as follows : 0 = None 1-3 = Mild 4-6 = Moderate 7-9 = Severe
Time Frame
Up to 4 weeks from treatment visit
Secondary Outcome Measure Information:
Title
Percentage of participants' thighs that achieve at least 1-level severity improvement measured by cellulite severity scale from baseline following administration of 1 course of CBL-514.
Description
Percentage of participants' thighs that achieve at least 1-level severity improvement determined by the total scores of modified Hexsel CSS at V4 and V5 compared to baseline.
Time Frame
Up to 4 weeks from treatment visit
Other Pre-specified Outcome Measures:
Title
Incidence of adverse events and treatment-emergent adverse events (TEAEs)
Description
Number of participants experiencing TEAEs and number of individual TEAEs
Time Frame
4 weeks from treatment visit
Title
Incidence of adverse events and treatment-emergent adverse events (TEAEs) at the injection site(s)
Description
Number of participants experiencing TEAEs and number of individual TEAEs at the injection site(s)
Time Frame
4 weeks from treatment visit
Title
Incidence of adverse events and treatment-emergent adverse events (TEAEs) related to the investigational product (IP)
Description
Number of participants experiencing TEAEs and number of individual TEAEs related to the investigational product (IP)
Time Frame
4 weeks from treatment visit
Title
Incidence of adverse events of special interest
Description
Number of participants experiencing adverse events of special interest
Time Frame
4 weeks from treatment visit
Title
Number of participants with clinically significant abnormalities in laboratory tests
Description
Laboratory tests include assessment of hematology, biochemistry, coagulation, and urinalysis tests
Time Frame
4 weeks from treatment visit
Title
Number of participants with clinically significant abnormalities in physical examination
Description
Physical examinations include assessment of 12-lead electrocardiogram parameters and vital signs
Time Frame
4 weeks from treatment visit
Title
Incidence of use of concomitant medications to treat treatment-emergent adverse events (TEAEs)
Description
Number of participants experiencing use of concomitant medications to treat treatment-emergent adverse events
Time Frame
4 weeks from treatment visit

10. Eligibility

Sex
Female
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
64 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Female aged 18 years to 64 years old (at Screening), inclusive. Have a BMI > 18.5 and < 35 kg/m2 and body weight ≥ 50 kg at Screening and Day 1. The participant has both sides of posterolateral thighs assessed according to the modified Hexsel CSS (A) number of evident depressions, (B) depression depth scale, and (C) morphological appearance of skin surface alterations. On assessment by the modified Hexsel CSS, the participant scores at least 4 and no greater than 8 at Screening and Day 1. The total score must contain: Hexsel CSS item (A) 'number of evident depressions' score of ≥ 1 Hexsel CSS item (B) 'depth of depressions' score of ≥ 1 Participant has a stable lifestyle (e.g., exercise, eating patterns, and smoking habit) per participant report for at least 3 months before Screening and during the study. Voluntarily signs the informed consent form and, in the opinion of the Investigator or delegate, is physically and mentally capable of participating in the study, and willing to adhere to study procedures. Exclusion Criteria: Female participant of childbearing potential who is not willing to commit to an acceptable contraceptive regimen with her partner from the time of Screening and throughout study participation until 90 days after the last IP dose, or who is currently pregnant or lactating. Note: females who are not of childbearing potential are not required to use contraception. Females not of childbearing potential are defined as those who have been surgically sterilized (hysterectomy or bilateral oophorectomy) or who are postmenopausal (defined as aged at least 50 years old with ≥ 12 months of amenorrhea and a follicle-stimulating hormone [FSH] > 30 IU/L at Screening). Participant diagnosed with coagulation disorders or is receiving anticoagulant/antiplatelet therapy or medications or dietary supplements, which impede coagulation or platelet aggregation within 14 days prior to the IP administration. Participant has hemoglobin A1c (HbA1c) ≥ 9%, delayed wound healing, or any diabetic risks which, in the opinion of the Investigator (or designee) is inappropriate to participate in the study. Participant has a clinically significant cardiovascular disease and abnormal findings in electrocardiogram (ECG) at Investigator's (or designee's) discretion. Participant with active or prior history of malignancies within 5 years before Screening or being assessed 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 Investigator's (or designee's) discretion. Participant with a history of human immunodeficiency virus (HIV)-1 infection, or participants with active HIV infection at Screening with a positive HIV antigen/antibody (Ag/Ab) combination test. Participant with a history of trypanophobia, the extreme fear of medical procedures involving injections or needles, or who experience vasovagal syncope and pass out at the sight of blood or a needle. Participant with any hepatic medical condition that, in the opinion of the Investigator (or designee), would interfere with assessment of safety or efficacy or compromise the participant's ability to undergo study procedures or provide informed consent. Participant who has a recent history of major depression, anxiety, or other psychiatric disease requiring treatment with prescription medication within 6 months prior to Screening. Participant has abnormal skin or local skin conditions at the treatment area, which in the opinion of the Investigator (or designee), is inappropriate to participate in the study. This includes but is not limited to any of the following: skin manifestations of a systemic disease any abnormality of the skin or soft tissues on the anticipated treatment area skin laxity on treatment area when the participant is in the standing position sensory loss or dysesthesia in the area to be treated evidence of any cause of enlargement in the area to be treated other than localized subcutaneous fat tattoos on the area to be treated participant with a propensity for keloid or hypertrophic scarring. Participant who has had the following surgical or aesthetic procedures: liposuction, cryolipolysis, ultrasonic lipolysis, low level laser therapy (LLLT), or lipolysis injection to the region to be treated before Screening medical device, injection (including but not limited to collagenase clostridium histolyticum injections and collagen stimulating injections), over-the-counter (OTC) cosmetic cream, or cosmetic program to prevent or mitigate EFP to the region to be treated within 12 months before Screening and throughout study participation massage to the region to be treated within 2 weeks before Screening and throughout study participation. Participant is undergoing chronic steroid or immunosuppressive therapy, except for asthma inhaler or topical steroids for skin conditions if the medications are not used on the treatment area. Participating is 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, which in the opinion of the Investigator (or designee), may affect the evaluation of the investigational product or place the participant at undue risk. If a participant needs to use the above-mentioned therapeutic agents during the study for any reason, these therapeutic agents should not be used at least for 2 days before dosing and until 1 day after dosing. Participant receives nonsteroidal anti-inflammatory drug (NSAID) including aspirin within 14 days prior to the IP administration. Participant is unable to receive topical anesthesia (e.g., history of hypersensitivity to lidocaine). Participant with known allergies or sensitivities to the IP or its components. Participant with liver cirrhosis or with inadequate liver function at Screening defined as aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase (ALKP), total bilirubin (TBIL), or gamma-glutamyl transferase (GGT) > 3.0 × upper limit of normal (ULN). Participant with any renal impairment, defined as abnormal serum creatinine, and urea > 1.5 × ULN or estimated glomerular filtration rate (eGFR) < 90 mL/min/1.73 m2. Participants who are currently on dialysis should be excluded. Participants with an eGFR ≥ 60 and < 90 mL/min/1.73 m2 at Screening should be evaluated by the Investigator to exclude pre-existing renal disease or associated dysfunction. If mild decrease in eGFR is assessed by the Investigator as not clinically significant or not related to dysfunction, the participants may be eligible upon the Investigator's assessment. Use of other investigational product or device within 4 weeks prior to Screening.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Anne Sheu
Organizational Affiliation
Caliway Biopharmaceuticals Co., Ltd.
Official's Role
Study Director
Facility Information:
Facility Name
Investigational Site 1
City
Encino
State/Province
California
ZIP/Postal Code
91436
Country
United States
Facility Name
Investigational Site 2
City
New York
State/Province
New York
ZIP/Postal Code
10022
Country
United States

12. IPD Sharing Statement

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A Study to Evaluate the Efficacy and Safety of CBL-514 in Participants With EFP; Cellulite (Stage 1)

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