Deformity; Skin, Fat Redistribution, Lipodystrophy
Conditions
Brief summary
The purpose of this study is to evaluate the efficacy and safety of deoxycholate for the treatment of lipocytic ("fatty") deformities of the trunk and lower extremities; this includes areas such as cellulite or body contour deformities following liposuction. Ten participants will be enrolled in the pilot (part 1) portion of this study, and 60 participants will be enrolled in part 2 of this study (the blinded study). Those who are eligible to be in the pilot (part 1) study will receive an injection with deoxycholate \[concentration of 10 mg/mL (1.0%)\] directly into the zone of fatty deformity. Those who are eligible to be in the second part of this study will be randomly assigned to receive an injection with deoxycholate \[concentration of 10 mg/mL (1.0%)\] to one fatty deformity site, and an injection with a placebo (0.9% benzyl alcohol) without deoxycholate to a separate fatty deformity site. Thus, at each treatment session, participants will receive an injection with deoxycholate to one lipocytic deformity site, and an injection with a placebo to a separate (similar appearing) fatty deformity site. The placebo is provided for the comparator group. Subsequent treatment visits will be conducted at approximately 28-day intervals for up to 5 treatment sessions (Visits 2, 3, 4, and 5 \[±5 days, relative to the date of the previous treatment session\]). Approximately 7 (±3) days after each treatment visit, participants will be required to come to the research site for Visits 1A, 2A, 3A, 4A, and 5A (relative to each treatment session) for evaluations of the treatment area, recording of vital signs and weight, and to update records on adverse events and concomitant medications. A post-treatment follow-up visit at 6 months (Visit 6 on Day 168) relative to the date of the initial treatment session will be required.
Detailed description
In the pilot portion of the study: * 10 participants (either male or female, between 18-55 years old ) with lipoocytic deformities of the lower extremities or trunk will receive unilateral treatment with deoxycholate (less than or equal to 2mg/cm2) to assess for safety and proof of concept; this includes participants who have less than or equal to 10-cm2 area of grade I, II, or III cellulite (based on the Nurnberger & Muller grading scale), and / or post-surgical deformities associated with liposuction. * Participants will receive less than or equal to 2mg/cm2 deoxycholate in this study. This dose has been shown in previous large scale clinical trials evaluating deoxycholate injections to the submental fat to be a safe and effective dose for treatment with minimal side effects. * Injections will be 1.0cm apart, 0.2mL/injection into the fatty deformity zone. * Participants may be injected every 4 weeks for up to 2 treatments. * The participants will be followed closely for 6 months after the last treatment session to assess the safety and efficacy of deoxycholate. Follow up visits are conducted at Day 7, 14, 28, 35, 42, 56, 84, 112, 140, 168, and 196. * If the pilot participants show any improvement (1-point improvement on the Nurnberger Muller Lipocytic Deformities grading scale or a 20% improvement on the linear analog scale) in their fatty deformity within the first 2 months after receiving the initial deoxycholate treatment, the investigator will continue with the split-body, placebo-controlled portion of the study (study part 2). Please note that participants enrolled in the pilot study may also be eligible for enrollment in the placebo-controlled study if the participant has more than 2 other fatty deformities. Part 2, single-center, prospective, double blind, split body study: * 60 participants (either male or female participants 18 years of age or older) with lipocytic deformities of the lower extremities or trunk will receive injections with deoxycholate and placebo. Participants must have at least two lipocytic deformities that are of a similar nature, and less than or equal to 10cm2 in surface area. This includes participants with grade I, II, or III cellulite (on the Nurnberger Muller Scale) and/or post surgical deformities associated with liposuction. * These two or more sites will be labeled numerically and blinded, and participants will be randomized to receive injections of deoxycholate (site # 1), and placebo (site # 2) or vice versa to lipocytic deformity zones. * Participants will receive no more than 10 mL in any treatment session and will not undergo more than 5 treatments. Participants may be injected every 4 weeks for up to 5 treatments. * All participants will undergo treatment as follows: * Placebo: 0.2mL / injection to a zone of lipocytic deformity, approximately 1.0cm apart. Patients may be injected every 4 weeks for up to 5 treatments; no participant will receive more than 10 mL in any treatment session * Deoxycholate (2mg/cm2): 0.2mL / injection to a zone of lipocytic deformity, approximately 1.0cm apart. Participants may be injected every 4 weeks for up to 5 treatments; no participant will receive more than 10 mL in any treatment session * Study duration: 6 months with 11 Visits
Interventions
DRUGKybella
Less than or equal to 2 mg/cm\^2 sodium deoxycholate subcutaneous injection into the zone of lipocytic deformity every four weeks for up to five treatments.
Sponsors
The Maas Clinic
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Masking description
In Part 1 (pilot) the investigator and the participant will know which zone is receiving the active treatment. In part 2 of the study, the investigator and participants are blinded to which of the two matched zones of lipocytic deformity is receiving placebo treatment and which is receiving active treatment.
Intervention model description
In the pilot portion of this study, 10 participants will receive treatment with deoxycholate into the zone of lipocytic deformity. In part two of this study, 60 participants with at least two lipocytic deformities will receive injections with deoxycholate into one zone of lipocytic deformity and placebo into the other zone of lipocytic deformity.
Eligibility
Sex/Gender
ALL
Age
18 Years to 85 Years
Healthy volunteers
Yes
Inclusion criteria
* Patients with Grade I, II, or III cellulite (based on the Nurnberger \& Muller Scale) by physician assessment, affecting less than or equal to 10 cm2 surface area, and / or the presence of post surgical deformities associated with liposuction. * Males and nonpregnant, nonlactating females 18 to 55 years of age, inclusive, on the day of randomization (Visit 1). Females of childbearing potential must have a negative human chorionic gonadotropin (hCG) test result within 28 days before Visit 1 and agree to practice adequate contraception, in the judgment of the investigator, during the course of the study. Females of childbearing potential who are not sexually active need not practice contraception. * Time and ability to complete the study and comply with instructions. * Understanding of the study and contents of the informed consent. * History of stable body weight, in the judgment of the investigator, for at least 6 months before randomization. * Expected to understand and agree to comply with the visit schedule and all protocol specified tests and procedures and agreement by the subject to refrain from making significant changes, in the judgment of the investigator, to his or her dietary or exercise habits during the course of the subject's participation. * Agreement to forgo any treatment or behavior (e.g., unshaven body hair) during the subject's participation in the study that may affect the assessments of the zones of area. * Medically able to undergo the administration of study material determined by clinical and laboratory tests obtained within 28 days before randomization for which the investigator identified no clinically significant abnormality. * Signed informed consent obtained before any study-specific procedure is performed.
Exclusion criteria
* Patients who present with adipose tumors (lipoma) * Human immunodeficiency virus (HIV)- or medication-related lipodystrophies * Concurrent therapy that, in the investigator's opinion, would interfere with evaluation of the efficacy or safety of the medication. * Active infection involving the fatty deformity zones. * Pregnant women, nursing mothers, or women who are planning pregnancy during the study, or think they may be pregnant at the start of the study. Throughout the course of the study, women of childbearing potential must use reliable forms of contraception (e.g. abstinence, oral contraceptives for more than 12 consecutive weeks prior to enrollment, or spermicide and condoms). * Current history of chronic drug or alcohol abuse. * Clinically diagnosed anxiety disorder, or any other significant psychiatric disorder (e.g. depression) that, in the opinion of the investigator, might interfere with the patient's participation in the study. * History of any intervention to treat cellulite (e.g., liposuction, surgery, laser, or lipolytic agents). * History of trauma associated with the cellulite areas that in the judgment of the investigator may affect evaluation of safety or efficacy of treatment. * Body mass index of \>40.0 as determined on Visit 1. * A result on coagulation tests (PT, PTT) obtained within 28 days before randomization that indicates the presence of any clinically significant bleeding disorder. * Any medical condition (e.g., respiratory, cardiovascular, hepatic, neurological disease, or thyroid dysfunction) that would interfere with assessment of safety or efficacy or compromise the subject's ability to undergo study procedures or give informed consent. * Treatment with radio frequency or laser procedures in the cellulite zones within 12 months before randomization. * History of sensitivity to any components of the study material * History of sensitivity to topical or local anesthetics (e.g., lidocaine, benzocaine, procaine). * Previous participation in a deoxycholate trial. * Treatment with an investigational device or agent within 30 days before randomization.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Clinician-Reported Cellulite Grading Scale (CR-CGS) - Assessing Change in Appearance of Lipocytic Deformity | Will be measured every 30 days up to 7 months. | Grading performed by investigator using the 4-point Nürnberger and Müller Lipocytic Deformities Grading Scale, assessing the appearance of the zone of lipocytic deformity. |
| Patient-Reported Visual Analog Scale - Assessing Change in Appearance of Lipocytic Deformity | Will be measured every 30 days up to 7 months. | Grading performed by subject using the 10-point Visual Analog Grading Scale, assessing the appearance of the zone of lipocytic deformity. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Subject Global Questions | Up to 7 months | Global assessment of patient satisfaction based upon an analog linear scale. |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORCorey S Maas, M.D.
The Maas Clinic
Outcome results
None listed