Peyronie's DiseasePhase 3NCT01685437
A Phase 3, Open-label Study of the Safety and Effectiveness of AA4500 Administered Twice Per Treatment Cycle for up to Four Treatment Cycles (2 x 4) in Men With Peyronie's Disease
A Phase 3 study testing AA4500 in people with Peyronie's Disease, run by Endo USA Inc., a Keenova Therapeutics Company.
Trial summary
This study is a Phase 3, open-label study of the safety and efficacy of AA4500 0.58 mg in subjects with Peyronie's disease. Subjects will be screened for study eligibility within 21 days before the initial injection of study drug in the first treatment cycle. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Peyronie's Disease
- What is being tested?
- AA4500
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 189 participants
- Where
- No sites listed yet
- Study dates
- Sep 2012to Dec 2013
- Phase
- Phase 3
- 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 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- No subject should be enrolled until all eligibility criteria have been satisfied. Subjects who completed their participation in Auxilium-sponsored studies AUX-CC-803 or AUX-CC-804 and received placebo in that study may enroll in this study provided they continue to meet the eligibility requirements. To qualify for the study a subject must:
- Have penile curvature of at least 30° in the dorsal, lateral, or dorsal/lateral plane at screening. It must be possible to delineate the single plane of maximal curvature for evaluation during the study
- Be judged to be in good health, based upon the results of a medical history, physical examination,and laboratory profile
- Have participated in Study AUX-CC-803 or Study AUX-CC-804, received placebo in that study,and completed that study
- Voluntarily sign and date an informed consent agreement approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC). The subject must also sign an authorization form to allow disclosure of his protected health information (PHI). The PHI authorization form and informed consent form may be an integrated form or may be separate forms depending on the institution
- Be able to read, complete and understand the various rating instruments in English.
Who cannot join
- A subject will be excluded from study participation if he:
- Has a penile curvature of less than 30° or greater than 90° at the screening visit
- Has any of the following conditions:
- Chordee in the presence or absence of hypospadias
- Thrombosis of the dorsal penile artery and/or vein
- Infiltration by a benign or malignant mass resulting in penile curvature
Show all 32Show fewer
- Infiltration by an infectious agent, such as lymphogranuloma venereum
- Ventral curvature from any cause
- Presence of an active sexually transmitted disease
- Known active hepatitis B or C
- Known immune deficiency disease or be positive for human immunodeficiency virus (HIV)
- Has previously undergone surgery for Peyronie's disease
- Fails to have an erection which in the opinion of the investigator is sufficient to accurately measure the subject's penile deformity after administration of prostaglandin E1 or trimix or another suitable injectable pharmacologic stimulant according to the standard practice at the investigator's institution
- Has a calcified plaque as evident by appropriate radiographic evaluation, penile x-ray or penile ultrasound that would prevent proper injection of study medication. Non-contiguous stippling of calcium is acceptable for inclusion provided the calcium deposit does not interfere with the injection of AA4500 into the plaque
- Has an isolated hourglass deformity of the penis
- Has the plaque causing curvature of the penis located proximal to the base of the penis, so that the injection of the local anesthetic would interfere with the injection of AA4500 into the plaque
- Has previously received alternative medical therapies for Peyronie's disease administered by the intralesional route (including, but not limited to, steroids, verapamil, and the naturally occurring low molecular weight protein, interferon-α2b) within 3 months before the first dose of study drug or plans to use any of these medical therapies at any time during the study
- Has received alternative medical therapies for Peyronie's disease administered by the oral (including, but not limited to, vitamin E [>500 U], potassium aminobenzoate [Potaba], tamoxifen, colchicine, and pentoxifylline, ,) or topical routes (including, but not limited to, verapamil applied as a cream) within 3 months before the first dose of study drug or plans to use any of these medical therapies at any time during the study
- Has had extracorporeal shock wave therapy (ESWT) for the correction of Peyronie's disease within the 6-month period before screening or plans to have ESWT at any time during the study
- Has used any mechanical type device for correction of Peyronie's disease within the 2-week period before screening or plans to use any these devices at any time during the study
- Has used a mechanical device to induce a passive erection within the 2-week period before screening or plans to use any of these devices at any time during the study
- Has significant erectile dysfunction that has failed to respond to oral treatment with phosphodiesterase type 5 (PDE5) inhibitors
- Has a penile Duplex Doppler ultrasound evaluation at screening that shows compromised penile hemodynamics that in the opinion of the investigator is clinically significant
- Has uncontrolled hypertension, as determined by the investigator
- Has a known recent history of stroke, bleeding, or other significant medical condition, which in the investigator's opinion would make the subject unsuitable for enrollment in the study
- Has received an investigational drug or treatment within 30 days before the first dose of study drug, except for placebo in Studies AUX-CC-803 or AUX-CC-804
- Has a known systemic allergy to collagenase or any other excipient of AA4500
- Has a known allergy to any concomitant medication required as per the protocol
- Has received anticoagulant medication (except for ≤ 150 mg aspirin daily) during the 7 days before each dose of study drug
- Has received any collagenase treatments within 30 days of the first dose of study drug
- Has, at any time, received AA4500 for the treatment of Peyronie's disease
- Is unwilling or unable to cooperate with the requirements of the study including completion of all scheduled study visits
About this study
This study is a Phase 3, open-label study of the safety and efficacy of AA4500 0.58 mg in subjects with Peyronie's disease. Subjects will be screened for study eligibility within 21 days before the initial injection of study drug in the first treatment cycle. Enrollment will include all subjects who meet the eligibility criteria and who received placebo and completed one of the Auxilium-sponsored studies AUX-CC-803 or AUX-CC-804.
Read the detailed description
After the final injection of each treatment cycle, the investigator or qualified designee (ie, qualified by license, education, and training to perform the study procedure according to local, state, and country requirements) will model the plaque in an attempt to stretch or elongate the plaque. If the subject's penile curvature is reduced to <15° after the first, second, or third cycle of injections or if the investigator determines further treatment is not clinically indicated (eg, adverse events; allergic reaction), subsequent treatment cycles will not be administered. Following the maximum of four treatment cycles, each subject will be followed for additional safety and efficacy assessments on Days 168 (± 7 days) and 252 (± 7 days) (nominal weeks 24 and 36).
- Conditions
- Peyronie's Disease
- Being tested
- AA4500
- Keywords
- Peyronie's disease, penile plaque, penile curvature
- Medical subject headings
- Penile Induration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AA4500Biological
Also called Xiaflex, Xiapex
2 injections separated by approximately 24 hours to 72 hours, repeated after 42 days (± 5 days) for up to 4 treatment cycles
| Study group | Role | What they receive |
|---|---|---|
| AA4500 | Experimental | collagenase clostridium histolyticum Receives: Biological: AA4500 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage Change From Baseline in Penile Curvature
A negative value in the percentage change from baseline in penile curvature deformity (angle measured in degrees) indicates less curvature.
Measured: Baseline and Week 36
Change From Baseline in the Peyronie's Disease Bother Domain of the Peyronie's Disease Questionnaire (PDQ)
Peyronie's disease bother score range 0 (no issue or not at all bothered) to 4 (extremely bothered) on 4 questions; total score range 0 to 16. A decrease in the change from baseline total score in the Peyronie's disease bother domain of the PDQ is indicated by a negative number.
Measured: Baseline and Week 36
Secondary outcomes (7)
Change From Baseline in the Severity of Peyronie's Disease Symptoms Domain of the PDQ
Peyronie's disease symptoms (physical and psychological) severity score range 0 (none) to 4 (very severe) on 6 questions; total score range 0 to 24. A decrease in the change from baseline total score in the Peyronie's disease symptoms domain of the PDQ is indicated by a negative number.
Measured: Baseline and Week 36
Change From Baseline in the Penile Pain Domain of the PDQ in Subjects With Baseline Penile Pain Score ≥4
Penile pain scale range 0 (no pain) to 10 (extreme pain) on 3 questions; total score range 0 to 30. A decrease in the change from baseline total score in the penile pain domain of the PDQ is indicated by a negative number. Subjects were required to have a penile pain score of 4 or greater at baseline.
Measured: Baseline and Week 36
A Responder Analysis Based on Subject Overall Global Assessment
Subject overall global assessment of Peyronie's disease score range -3 (much worse) to 3 (much improved). A score of 1 (improved in a small but important way), 2 (moderately improved), or 3 indicate a responder.
Measured: Week 36
Change in the Overall Satisfaction Domain of the International Index of Erectile Function (IIEF)
Overall satisfaction domain of the IIEF score range 0 to 5 on 2 questions where higher scores indicate improved function or satisfaction; total score range 0 to 10.
Measured: Baseline and Week 36
Change From Baseline in Penile Plaque Consistency
Penile plaque consistency score range 1 (non-palpable) to 5 (hard). A decrease in the change from baseline in penile plaque consistency is indicated by a negative number.
Measured: Baseline and Week 36
A Composite Responder Analysis Based on Change From Baseline in Penile Curvature and in the Peyronie's Disease Bother Score
A composite responder is indicated by
- a percent reduction from baseline in penile curvature greater than or equal to the threshold, and
- a reduction from baseline in Peyronie's disease bother score greater than or equal to the threshold, or change in the overall sexual activity within the last 3 months to having vaginal intercourse from no vaginal intercourse at screening.
Measured: Week 36
Change From Baseline in Penile Length
A negative value represents a reduction in measurement from baseline.
Measured: Baseline and Week 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Sep 2012 (estimated)
- Study startSep 2012
- Primary completionDec 2013
- Study completionDec 2013
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 Peyronie'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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 189 participants (actual)
Sponsor and investigators
- Lead sponsor
- Endo USA Inc., a Keenova Therapeutics Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Gregory Kaufman, MD, Study director, Auxilium Pharmaceuticals, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01685437
- Sponsor's protocol ID
- AUX-CC-806
- Status last verified
- Sep 2017
- First submitted
- 12 Sep 2012
- 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 3, Open-label Study of the Safety and Effectiveness of AA4500 Administered Twice Per Treatment Cycle for up to Four Treatment Cycles (2 x 4) in Men With Peyronie's Disease
- ClinicalTrials.gov ID
- NCT01685437
- Acronym / sponsor ID
- AUX-CC-806
- Conditions
- Peyronie's Disease
- Keywords
- Peyronie's disease, penile plaque, penile curvature
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 189 participants (actual)
- Lead sponsor
- Endo USA Inc., a Keenova Therapeutics CompanyIndustry (pharma, biotech, devices)
- Study start
- Sep 2012
- Primary completion
- Dec 2013 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 14 Sep 2012 (estimated)
- Results
- Posted 7 Apr 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is a Phase 3, open-label study of the safety and efficacy of AA4500 0.58 mg in subjects with Peyronie's disease. Subjects will be screened for study eligibility within 21 days before the initial injection of study drug in the first treatment cycle. Enrollment will include all subjects who meet the eligibility criteria and who received placebo and completed one of the Auxilium-sponsored studies AUX-CC-803 or AUX-CC-804.
Detailed description
After the final injection of each treatment cycle, the investigator or qualified designee (ie, qualified by license, education, and training to perform the study procedure according to local, state, and country requirements) will model the plaque in an attempt to stretch or elongate the plaque. If the subject's penile curvature is reduced to <15° after the first, second, or third cycle of injections or if the investigator determines further treatment is not clinically indicated (eg, adverse events; allergic reaction), subsequent treatment cycles will not be administered. Following the maximum of four treatment cycles, each subject will be followed for additional safety and efficacy assessments on Days 168 (± 7 days) and 252 (± 7 days) (nominal weeks 24 and 36).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AA4500 | Experimental | Biological: AA4500collagenase clostridium histolyticum |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- No subject should be enrolled until all eligibility criteria have been satisfied. Subjects who completed their participation in Auxilium-sponsored studies AUX-CC-803 or AUX-CC-804 and received placebo in that study may enroll in this study provided they continue to meet the eligibility requirements. To qualify for the study a subject must:
- Have penile curvature of at least 30° in the dorsal, lateral, or dorsal/lateral plane at screening. It must be possible to delineate the single plane of maximal curvature for evaluation during the study
- Be judged to be in good health, based upon the results of a medical history, physical examination,and laboratory profile
- Have participated in Study AUX-CC-803 or Study AUX-CC-804, received placebo in that study,and completed that study
- Voluntarily sign and date an informed consent agreement approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC). The subject must also sign an authorization form to allow disclosure of his protected health information (PHI). The PHI authorization form and informed consent form may be an integrated form or may be separate forms depending on the institution
Key exclusion criteria (32 listed)
- Has a penile curvature of less than 30° or greater than 90° at the screening visit
- Has any of the following conditions:
- Has previously undergone surgery for Peyronie's disease
- Fails to have an erection which in the opinion of the investigator is sufficient to accurately measure the subject's penile deformity after administration of prostaglandin E1 or trimix or another suitable injectable pharmacologic stimulant according to the standard practice at the investigator's institution
- Has a calcified plaque as evident by appropriate radiographic evaluation, penile x-ray or penile ultrasound that would prevent proper injection of study medication. Non-contiguous stippling of calcium is acceptable for inclusion provided the calcium deposit does not interfere with the injection of AA4500 into the plaque
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 |
|---|---|---|
| AA4500Also: Xiaflex, Xiapex2 injections separated by approximately 24 hours to 72 hours, repeated after 42 days (± 5 days) for up to 4 treatment cycles | Biological | AA4500 |
Primary outcome
Percentage Change From Baseline in Penile Curvature
Time frame: Baseline and Week 36
A negative value in the percentage change from baseline in penile curvature deformity (angle measured in degrees) indicates less curvature.
Change From Baseline in the Peyronie's Disease Bother Domain of the Peyronie's Disease Questionnaire (PDQ)
Time frame: Baseline and Week 36
Peyronie's disease bother score range 0 (no issue or not at all bothered) to 4 (extremely bothered) on 4 questions; total score range 0 to 16. A decrease in the change from baseline total score in the Peyronie's disease bother domain of the PDQ is indicated by a negative number.
7 secondary outcomes
Change From Baseline in the Severity of Peyronie's Disease Symptoms Domain of the PDQ
Time frame: Baseline and Week 36
Change From Baseline in the Penile Pain Domain of the PDQ in Subjects With Baseline Penile Pain Score ≥4
Time frame: Baseline and Week 36
A Responder Analysis Based on Subject Overall Global Assessment
Time frame: Week 36
Change in the Overall Satisfaction Domain of the International Index of Erectile Function (IIEF)
Time frame: Baseline and Week 36
Change From Baseline in Penile Plaque Consistency
Time frame: Baseline and Week 36
A Composite Responder Analysis Based on Change From Baseline in Penile Curvature and in the Peyronie's Disease Bother Score
Time frame: Week 36
Change From Baseline in Penile Length
Time frame: Baseline and Week 36
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.
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Assessment of the Value of FAPI and FDG-PET-CT in Diagnosis of Acute Phase Peyronie's Disease
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Concurrent Low Intensity Shockwave Therapy on Collagenase Clostridium Histolyticum for Peyronie's Disease
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Platelet-Rich Plasma for Peyronie's Disease
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Delayed Dose Collagenase Clostridium Histolyticum (CCH) Protocol for Men With Peyronie's Disease
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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.
Gregory Kaufman, MD study director
Auxilium Pharmaceuticals, Inc.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing AA4500 in people with Peyronie's Disease, run by Endo USA Inc., a Keenova Therapeutics Company.
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 6 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.
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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 7 Apr 2015). 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?
Percentage Change From Baseline in Penile Curvature (time frame: Baseline and Week 36). The study lists 2 primary outcomes in all. 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.