Edematous Fibrosclerotic Panniculopathy (EFP)Phase 2NCT01987986
A Phase 2, Randomized, Double-blind, Placebo Controlled Dose Ranging Study of Repeat Doses of AA4500 for the Treatment of Edematous Fibrosclerotic Panniculopathy
A Phase 2 study testing Collagenase Clostridium Histolyticum in people with Edematous Fibrosclerotic Panniculopathy (EFP), run by Endo USA Inc., a Keenova Therapeutics Company at 10 sites in United States.
Trial summary
To assess the safety and effectiveness of repeat doses of AA4500 in the treatment of edematous fibrosclerotic panniculopathy (EFP) commonly known as cellulite in adult women. Read more about this study
- Who is it for?
- Ages 18 to 45 years, women only
- Conditions the study is for
- Edematous Fibrosclerotic Panniculopathy (EFP)
- What is being tested?
- Collagenase Clostridium Histolyticum
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 150 participants
- Where
- 10 sitesUnited States
- Study dates
- Oct 2013to Aug 2014
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 45 years
- Women only
- No healthy volunteers
- Adult
Who may be able to join
- Be a female between 18 to 45 years of age.
- Have at least one well defined dimple that is at least 1 cm but not more than 2 cm along the long axis and that is evident when the subject is standing
- Have a photonumeric cellulite severity scale (CSS) score between 6 and 12
- Have a Body Mass Index (BMI) >19 and <30 kg/m2, and intends to maintain stable body weight throughout the duration of the study
- Be willing to apply appropriate sunscreen to the selected quadrant before each exposure to the sun while participating in the study
- Be judged to be in good health, based upon the results of a medical history, physical examination, and laboratory profile at screening.
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- Have a negative urine pregnancy test at screening and before injection of AA4500 and be using an effective contraception method (ie, abstinence, intrauterine device [IUD], hormonal [estrogen/progestin] contraceptives, or barrier control) for at least one menstrual cycle prior to study enrollment and for the duration of the study.
- Be willing and able to cooperate with the requirements of the study.
- Voluntarily sign and date an informed consent agreement approved by the Institutional Review Board/Independent Ethics Committee/Human Research Ethics Committee (IRB/IEC/HREC).
- Be able to read, complete and understand the Patient Reported Outcomes rating instruments in English.
Who cannot join
- Thyroid disease, unless controlled with medication for ≥ 6 months
- Uncontrolled diabetes mellitus, as determined by the investigator
- Uncontrolled hypertension, as determined by the investigator
- Vascular disorder (eg, phlebitis or varicose veins) in area to be treated
- Lipedema or a lymphatic disorder
- Cushing's disease and/or use of systemic corticosteroids
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- History of lower extremity thrombosis or post-thrombosis syndrome
- Documented autoimmune disorder such as lupus erythematosus, rheumatoid arthritis
- Inflammation or active infection in area to be treated
- Cutaneous alteration in area to be treated
- Rash, eczema, psoriasis, or skin cancer in the area to be treated
- History of keloidal scarring or abnormal wound healing
- Coagulation disorder
- Taking a medication for chronic anticoagulation (except for ≤ 150 mg aspirin daily)
- Known active hepatitis A, B or C
- Known immune deficiency disease or a positive test for human immunodeficiency virus (HIV)
- Other significant conditions including body dysmorphic disorder, which in the investigator's opinion would make the subject unsuitable for enrollment in the study
- Is menopausal defined as 12 months of amenorrhea in the absence of other biological or physiological causes, as determined by the investigator
- Has used any of the following for the treatment of EFP on the legs or buttock within the timelines identified below or intends to use any of the following at any time during the course of the study:
- Liposuction on the side of the body selected for treatment during the 12-month period before injection of AA4500
- Injections (eg, mesotherapy); radiofrequency device treatments; laser treatment; or surgery (including subcision) within the selected treatment quadrant during the 12-month period before injection of AA4500
- Endermologie or similar treatments within the selected treatment quadrant during the 6-months period before injection of AA4500
- Massage therapy within the selected treatment quadrant during the 3-month period before injection of AA4500
- Creams (eg, Celluvera™, TriLastin®) to prevent or mitigate EFP within the selected treatment quadrant during the 2-week period before injection of AA4500
- Has a tattoo located within 2 cm of the site of injection
- Is presently nursing a baby or providing breast milk for a baby.
- Intends to become pregnant during the study.
- Intends to initiate an intensive sport or exercise program during the study.
- Has received an investigational drug or treatment within 30 days before injection of AA4500.
- Has a known systemic allergy to collagenase or any other excipient of AA4500.
- Has received any collagenase treatments within 30 days before treatment.
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Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Charlottesville Dermatology
Charlottesville, Virginia, 22911
Dermatology Research Institute
Coral Gables, Florida, 33146
Tennessee Clinical Research Center
Nashville, Tennessee, 37215
Sadick Research Group
New York, New York, 10075
Dermatology Cosmetic Laser Medical Associates of La Jolla
San Diego, California, 92121
ATS Clinical Research
Santa Monica, California, 90404
Premier Clinical Research
Spokane, Washington, 99204
Stony Brook University Medical Center
Stony Brook, New York, 11794
Mercy Health Research
Washington, Missouri, 63090
Kenneth Beer, MD PA
West Palm Beach, Florida, 33401
About this study
Official title: A Phase 2, Double-blind and Placebo Dose Ranging Study of Repeat Doses of AA4500 for the Treatment of Edematous Fibrosclerotic Panniculopathy
To assess the safety and effectiveness of repeat doses of AA4500 in the treatment of edematous fibrosclerotic panniculopathy (EFP) commonly known as cellulite in adult women.
Read the detailed description
The Phase 2a study is a randomized, double-blind multiple-dose study that is expected to enroll approximately 144 women between the ages of 18 and 45 in the United States. Treatment effectiveness will be evaluated by investigator and patient assessments, as well as 3-D photographic imaging techniques. Once the safety and local tolerability profile from the first stage has been found to be acceptable subjects will be enrolled in stage 2. After an interim safety and local tolerability review was completed of all subjects in stage 1, it was determined that enrollment in stage 2 is acceptable and has been initiated.
- Being tested
- Collagenase Clostridium Histolyticum, Placebo
- Keywords
- cellulite, dimple, orange peel, cottage cheese, mattress texture
- Medical subject headings
- Cellulite
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Collagenase Clostridium HistolyticumBiological
Also called AA4500, Xiaflex, Xiapex
injectible intervention
PlaceboBiological
| Study group | Role | What they receive |
|---|---|---|
| AA4500 0.06 mg (low dose) | Experimental | AA4500 (Collagenase Clostridium Histolyticum)
Each subject could receive up to three treatment sessions. Each treatment session was separated by approximately 21 days. Receives: Biological: Collagenase Clostridium Histolyticum |
| AA4500 0.48 mg (mid-dose) | Experimental | AA4500 (Collagenase Clostridium Histolyticum)
Each subject could receive up to three treatment sessions. Each treatment session was separated by approximately 21 days. Receives: Biological: Collagenase Clostridium Histolyticum |
| AA4500 0.84 mg (high dose) | Experimental | AA4500 (Collagenase Clostridium Histolyticum)
Each subject could receive up to three treatment sessions. Each treatment session was separated by approximately 21 days. Receives: Biological: Collagenase Clostridium Histolyticum |
| Placebo | Placebo comparator | Placebo
Each subject could receive up to three treatment sessions. Each treatment session was separated by approximately 21 days. Receives: Biological: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Investigator Global Assessment of Aesthetic Improvement
Investigators assessment of aesthetic improvement (I-GAIS) scores ranged from 3 to -1 as follows: 3 (very much improved), 2 (much improved), 1 (improved), 0 (no change), -1 (worse).
Measured: Baseline, Day 73
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Nov 2013 (estimated)
- Study startOct 2013
- Primary completionAug 2014
- Study completionAug 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Edematous Fibrosclerotic Panniculopathy (EFP)Studies 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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 150 participants (actual)
Sponsor and investigators
- Lead sponsor
- Endo USA Inc., a Keenova Therapeutics Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Veronica Urdaneta, MD MPH, Study director, Endo USA Inc., a Keenova Therapeutics Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01987986
- Sponsor's protocol ID
- AUX-CC-831
- Status last verified
- Sep 2017
- First submitted
- 30 Oct 2013
- Last updated
- 5 Oct 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Oct 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Double-blind and Placebo Dose Ranging Study of Repeat Doses of AA4500 for the Treatment of Edematous Fibrosclerotic Panniculopathy
- ClinicalTrials.gov ID
- NCT01987986
- Acronym / sponsor ID
- AUX-CC-831
- Keywords
- cellulite, dimple, orange peel, cottage cheese, mattress texture
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 150 participants (actual)
- Lead sponsor
- Endo USA Inc., a Keenova Therapeutics CompanyIndustry (pharma, biotech, devices)
- Study start
- Oct 2013
- Primary completion
- Aug 2014 (actual)
- Study completion
- Aug 2014 (actual)
- First posted
- 20 Nov 2013 (estimated)
- Results
- Posted 2 Jul 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To assess the safety and effectiveness of repeat doses of AA4500 in the treatment of edematous fibrosclerotic panniculopathy (EFP) commonly known as cellulite in adult women.
Detailed description
The Phase 2a study is a randomized, double-blind multiple-dose study that is expected to enroll approximately 144 women between the ages of 18 and 45 in the United States. Treatment effectiveness will be evaluated by investigator and patient assessments, as well as 3-D photographic imaging techniques. Once the safety and local tolerability profile from the first stage has been found to be acceptable subjects will be enrolled in stage 2. After an interim safety and local tolerability review was completed of all subjects in stage 1, it was determined that enrollment in stage 2 is acceptable and has been initiated.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| AA4500 0.06 mg (low dose) | Experimental | Biological: Collagenase Clostridium HistolyticumAA4500 (Collagenase Clostridium Histolyticum) Each subject could receive up to three treatment sessions. Each treatment session was separated by approximately 21 days. |
| AA4500 0.48 mg (mid-dose) | Experimental | Biological: Collagenase Clostridium HistolyticumAA4500 (Collagenase Clostridium Histolyticum) Each subject could receive up to three treatment sessions. Each treatment session was separated by approximately 21 days. |
| AA4500 0.84 mg (high dose) | Experimental | Biological: Collagenase Clostridium HistolyticumAA4500 (Collagenase Clostridium Histolyticum) Each subject could receive up to three treatment sessions. Each treatment session was separated by approximately 21 days. |
| Placebo | Placebo comparator | Biological: PlaceboPlacebo Each subject could receive up to three treatment sessions. Each treatment session was separated by approximately 21 days. |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (10 listed)
- Be a female between 18 to 45 years of age.
- Have at least one well defined dimple that is at least 1 cm but not more than 2 cm along the long axis and that is evident when the subject is standing
- Have a photonumeric cellulite severity scale (CSS) score between 6 and 12
- Have a Body Mass Index (BMI) >19 and <30 kg/m2, and intends to maintain stable body weight throughout the duration of the study
- Be willing to apply appropriate sunscreen to the selected quadrant before each exposure to the sun while participating in the study
Key exclusion criteria (32 listed)
- Thyroid disease, unless controlled with medication for ≥ 6 months
- Uncontrolled diabetes mellitus, as determined by the investigator
- Uncontrolled hypertension, as determined by the investigator
- Vascular disorder (eg, phlebitis or varicose veins) in area to be treated
- Lipedema or a lymphatic disorder
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 |
|---|---|---|
| Collagenase Clostridium HistolyticumAlso: AA4500, Xiaflex, Xiapexinjectible intervention | Biological | AA4500 0.06 mg (low dose), AA4500 0.48 mg (mid-dose), AA4500 0.84 mg (high dose) |
| Placebo | Biological | Placebo |
Primary outcome
Investigator Global Assessment of Aesthetic Improvement
Time frame: Baseline, Day 73
Investigators assessment of aesthetic improvement (I-GAIS) scores ranged from 3 to -1 as follows: 3 (very much improved), 2 (much improved), 1 (improved), 0 (no change), -1 (worse).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Oct 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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.
Veronica Urdaneta, MD MPH study director
Endo USA Inc., a Keenova Therapeutics Company
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Collagenase Clostridium Histolyticum in people with Edematous Fibrosclerotic Panniculopathy (EFP), run by Endo USA Inc., a Keenova Therapeutics Company at 10 sites 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 10 inclusion and 32 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 5 Oct 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites 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. Some participants in this study may receive a placebo. 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?
Yes. Results are posted in the registry (first posted 2 Jul 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Investigator Global Assessment of Aesthetic Improvement (time frame: Baseline, Day 73). 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 5 Oct 2017.