Peyronie's DiseasePhase 2NCT00812838
H-22411: BOTOX® for Peyronie's Disease
A Phase 2 study testing 100 units of Botulinum Toxin Type A, Preservative free normal saline and 100 units Botulinum Toxin A in people with Peyronie's Disease, run by Mohit Khera at 1 site in United States.
Trial summary
Peyronie's disease is a condition in which a plaque, or hard lump, forms on the penis. It causes hardened tissue, pain, and an abnormal bending in the penis. These symptoms are more severe during an erection. 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?
- 100 units of Botulinum Toxin Type A, Preservative free normal saline and 100 units Botulinum Toxin A
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Aug 2009to 15 Jan 2019
- 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 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects with stable Peyronie's plaques.
- Males at least 18 years of age
- Must give informed consent.
Who cannot join
- Subjects in the active phase of Peyronie's disease.
- Subjects with less than 1 year history of Peyronie's disease.
- Subjects taking oral medications for Peyronie's disease which include Trental, Viagra, vitamin E, colchicines, L-arginine, and tamoxifen. There will be a 2 week wash-out period if patients are on these medications.
- Subjects with more than 1 penile plaque will be excluded from the study.
- Subjects with calcified plaques demonstrated by ultrasound will be excluded from the study.
- Known allergy or sensitivity to any components of the study medication (botulinum toxin A), anesthetics, or any other product associated with the treatment and general study procedures.
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- Any medical condition or neuromuscular disorder that may put the patient at increased risk with exposure to botulinum toxin A (BTX-A), including myasthenia gravis, Eaton-Lambert syndrome, or amyotrophic lateral sclerosis.
- Patient taking aminoglycosides or any drug known to interfere with neuromuscular transmission.
- Patient has hemophilia or other clotting factor deficiencies or disorders that cause bleeding diathesis.
- Patient must not be taking aspirin, non-steroidal anti-inflammatory drugs, or Coumadin for 7 or more days prior to Botox injection.
- Episode of unstable angina pectoris, myocardial infarction, transient ischemic attack, or cerebrovascular accident within the past 6 months.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: The Efficacy of Botulinum Toxin Type a in Treating Peyronie's Disease
Peyronie's disease is a condition in which a plaque, or hard lump, forms on the penis. It causes hardened tissue, pain, and an abnormal bending in the penis. These symptoms are more severe during an erection. Significant bending of the penis can result in pain, poor erections, and an inability to engage in sexual intercourse.
This disease affects about 3% of the male population. The average age of onset of this disease is 57 years old. The cause of the disease is unknown. However, many believe that it may be due to trauma to the penis (such as injury or extremely vigorous sexual activity).
Read the detailed description
Treatments for this disease have been limited and often unsuccessful. The goal of treatment is to reduce pain and maintain sexual function. Oral medicines that prevent plaque formation and promote plaque breakdown have not been effective. Many patients with the disease will require injections of medicines directly into the plaque. These injections have been used for over 50 years in the treatment of major Peyronie's disease. The disease often resolves on its own without treatment. Surgery may be performed to remove hardened tissue in the penis. However, surgery is not done during the first 12 months of the disease.
There are 2 phases of the disease: the active phase and the inactive phase. The active phase usually occurs during the first 12 months of the disease. The stabilization of the plaque is known as the inactive phase. We are inviting men with stable disease to take part in this study which will test BOTOX® versus a placebo (a placebo contains no medicine).
This will be a randomized, placebo-controlled, cross-over, single-center trial. The placebo group has the option to cross over to the treatment arm (ARM 1) of the study at the end of their 16 weeks of placebo arm (ARM 2). Study drug is Botulinum toxin type A (BOTOX®). Subjects who meet the inclusion criteria for the study will be randomized to either the treatment or placebo arm.
- Treatment: Injection solution will consist of 100 units of BOTOX® in 10 cc of preservative free normal saline, or
- Placebo: Injection solution will consist of 10 cc preservative free normal saline.
- Conditions
- Peyronie's Disease
- Being tested
- 100 units of Botulinum Toxin Type A, Preservative free normal saline, 100 units Botulinum Toxin A
- Medical subject headings
- Penile Induration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
100 units of Botulinum Toxin Type ADrug
Also called BOTOX®
Approximately 20 to 30 injections of 100 units of BOTOX® given with a 20 gage needle directly into the penile plaque
Preservative free normal salineOther
Approximately 20 to 30 injections of 10cc of preservative free normal saline given with a 20 gage needle directly into the penile plaque
100 units Botulinum Toxin ADrug
Also called Cross-over
Approximately 20 to 30 injections of 100 units of BOTOX® given with a 20 gage needle directly into the penile plaque
| Study group | Role | What they receive |
|---|---|---|
| 100 units of Botulinum Toxin Type A | Experimental | Injection solution will consist of 100 units of BOTOX® in 10 cc of preservative free normal saline Receives: Drug: 100 units of Botulinum Toxin Type A |
| Normal saline | Placebo comparator | Injection solution will consist of 10 cc preservative free normal saline
Subjects had the choice of crossing over to ARM 1 at the end of 16 weeks.
Cross-over: For subjects in Arm 2, crossover to BOTOX treatment will begin after the Week 16 Visit by repeating the study schedule as for Week 0 to Week 16. Receives: Other: Preservative free normal saline; Drug: 100 units Botulinum Toxin A |
What is being measured
The primary outcome is the main question the study is designed to answer.
Average Percent Change of Penile Curvature in Degrees
Measured by a protractor from pictures taken at baseline (pre-treatment screening visit) and end of treatment at week 16
*Crossover subjects were added to Experimental Group for analysis*
Negative value equates to a reduction in curvature Positive value equates to an increase in curvature
Measured: Baseline (Pre-Treatment Screening Visit) to end of treatment at week 16
Secondary outcomes (4)
Change in Penile Blood Flow for Peak Systolic Velocity (PSV) and End-diastolic Velocity (EDV)
Results of penile doppler ultrasound from baseline/screening visit to end of treatment at week 16 will be compared.
peak systolic velocity (PSV) and end-diastolic velocity (EDV) are assessed here.
Change is calculated as week 16 values - screening visit values
Negative values are a decrease in velocity Positive values are an increase in velocity
Measured: Baseline (Pre-Treatment Screening Visit) to end of treatment at week 16
Change in Penile Blood Flow for Diameter
Results of penile doppler ultrasound from the baseline/screening visit to end of treatment at week 16 will be compared.
Diameter is assessed here.
Change is calculated as week 16 values - screening visit values
Negative values are a decrease in diameter Positive values are an increase in diameter
Measured: Baseline (Pre-Treatment Screening Visit) to end of treatment at week 16
Change in Penile Plaque Size
Results of ultrasound at baseline/screening visit to end of treatment at week 16 will be compared.
*Crossover subjects were added to Experimental Group for analysis*
Change is calculated as week 16 values values minus screening visit values
Increase in value equates to increased plaque size Decrease in value equates to decreased plaque size
Measured: Baseline (Pre-Treatment Screening Visit) to end of treatment at week 16
Changes in International Index of Erectile Function Scores (IIEF)
Subject's average scores on IIEF at baseline/screening visit to end of treatment at week 16 are compared
The subscale measures self-reported erectile function. Maximum score is 30, Minimum is 0. A score of 0 is the absolute best outcome. A score of 30 is the absolute worst outcome. Erectile Function score is a summation of questions 1,2,3,4, and 15. This study is assessing their erectile function and not the other subscales.
The other subscale scores are :
- Orgasmic Function (Questions 9, 10); Maximum score = 10, Minimum score = 0
- Sexual Desire (Questions 11, 12); Maximum score = 10, Minimum score = 0
- Intercourse Satisfaction (Questions 6, 7, 8); Maximum score = 15, Minimum score = 0
- Overall Satisfaction (Question 13, 14): Maximum score = 10, Minimum score = 0
Subscales are not combined to make a total composite score.
*Crossover subjects were added to Experimental Group for analysis*
Measured: Baseline (Pre-Treatment Screening Visit) to end of treatment at week 16
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Dec 2008 (estimated)
- Study start7 Aug 2009
- Primary completion15 Feb 2017
- Study completion15 Jan 2019
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.
- Publications (1)Papers the sponsor has linked to this study.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mohit Khera
- Sponsor type
- University, hospital or other
- Collaborators
- Allergan
- Responsible party
- Sponsor-investigator, Mohit Khera, Baylor College of Medicine
- Investigators
- Mohit Khera, MD, MBA, Principal investigator, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00812838
- Sponsor's protocol ID
- 11-07-40-04
- Status last verified
- Apr 2024
- First submitted
- 18 Dec 2008
- Last updated
- 8 Apr 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No: Data will be published in aggregate.
Publications
Khera M, Boone TB, Smith CP. Botulinum toxin type A: a novel approach to the treatment of recurrent urethral strictures. J Urol. 2004 Aug;172(2):574-5. doi: 10.1097/01.ju.0000130652.27541.22. No abstract available.
PubMed 15247734
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Efficacy of Botulinum Toxin Type a in Treating Peyronie's Disease
- ClinicalTrials.gov ID
- NCT00812838
- Acronym / sponsor ID
- 11-07-40-04
- Conditions
- Peyronie's Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Mohit KheraUniversity, hospital or other
- Collaborators
- Allergan
- Study start
- 7 Aug 2009 (actual)
- Primary completion
- 15 Feb 2017 (actual)
- Study completion
- 15 Jan 2019 (actual)
- First posted
- 22 Dec 2008 (estimated)
- Results
- Posted 17 Feb 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Peyronie's disease is a condition in which a plaque, or hard lump, forms on the penis. It causes hardened tissue, pain, and an abnormal bending in the penis. These symptoms are more severe during an erection. Significant bending of the penis can result in pain, poor erections, and an inability to engage in sexual intercourse.
This disease affects about 3% of the male population. The average age of onset of this disease is 57 years old. The cause of the disease is unknown. However, many believe that it may be due to trauma to the penis (such as injury or extremely vigorous sexual activity).
Detailed description
Treatments for this disease have been limited and often unsuccessful. The goal of treatment is to reduce pain and maintain sexual function. Oral medicines that prevent plaque formation and promote plaque breakdown have not been effective. Many patients with the disease will require injections of medicines directly into the plaque. These injections have been used for over 50 years in the treatment of major Peyronie's disease. The disease often resolves on its own without treatment. Surgery may be performed to remove hardened tissue in the penis. However, surgery is not done during the first 12 months of the disease.
There are 2 phases of the disease: the active phase and the inactive phase. The active phase usually occurs during the first 12 months of the disease. The stabilization of the plaque is known as the inactive phase. We are inviting men with stable disease to take part in this study which will test BOTOX® versus a placebo (a placebo contains no medicine).
This will be a randomized, placebo-controlled, cross-over, single-center trial. The placebo group has the option to cross over to the treatment arm (ARM 1) of the study at the end of their 16 weeks of placebo arm (ARM 2). Study drug is Botulinum toxin type A (BOTOX®). Subjects who meet the inclusion criteria for the study will be randomized to either the treatment or placebo arm.
- Treatment: Injection solution will consist of 100 units of BOTOX® in 10 cc of preservative free normal saline, or
- Placebo: Injection solution will consist of 10 cc preservative free normal saline.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 100 units of Botulinum Toxin Type A | Experimental | Drug: 100 units of Botulinum Toxin Type AInjection solution will consist of 100 units of BOTOX® in 10 cc of preservative free normal saline |
| Normal saline | Placebo comparator | Other: Preservative free normal saline, Drug: 100 units Botulinum Toxin AInjection solution will consist of 10 cc preservative free normal saline Subjects had the choice of crossing over to ARM 1 at the end of 16 weeks. Cross-over: For subjects in Arm 2, crossover to BOTOX treatment will begin after the Week 16 Visit by repeating the study schedule as for Week 0 to Week 16. |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Subjects with stable Peyronie's plaques.
- Males at least 18 years of age
- Must give informed consent.
Key exclusion criteria (11 listed)
- Subjects in the active phase of Peyronie's disease.
- Subjects with less than 1 year history of Peyronie's disease.
- Subjects taking oral medications for Peyronie's disease which include Trental, Viagra, vitamin E, colchicines, L-arginine, and tamoxifen. There will be a 2 week wash-out period if patients are on these medications.
- Subjects with more than 1 penile plaque will be excluded from the study.
- Subjects with calcified plaques demonstrated by ultrasound will be excluded from 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 |
|---|---|---|
| 100 units of Botulinum Toxin Type AAlso: BOTOX®Approximately 20 to 30 injections of 100 units of BOTOX® given with a 20 gage needle directly into the penile plaque | Drug | 100 units of Botulinum Toxin Type A |
| Preservative free normal salineApproximately 20 to 30 injections of 10cc of preservative free normal saline given with a 20 gage needle directly into the penile plaque | Other | Normal saline |
| 100 units Botulinum Toxin AAlso: Cross-overApproximately 20 to 30 injections of 100 units of BOTOX® given with a 20 gage needle directly into the penile plaque | Drug | Normal saline |
Primary outcome
Average Percent Change of Penile Curvature in Degrees
Time frame: Baseline (Pre-Treatment Screening Visit) to end of treatment at week 16
Measured by a protractor from pictures taken at baseline (pre-treatment screening visit) and end of treatment at week 16 *Crossover subjects were added to Experimental Group for analysis* Negative value equates to a reduction in curvature Positive value equates to an increase in curvature
4 secondary outcomes
Change in Penile Blood Flow for Peak Systolic Velocity (PSV) and End-diastolic Velocity (EDV)
Time frame: Baseline (Pre-Treatment Screening Visit) to end of treatment at week 16
Change in Penile Blood Flow for Diameter
Time frame: Baseline (Pre-Treatment Screening Visit) to end of treatment at week 16
Change in Penile Plaque Size
Time frame: Baseline (Pre-Treatment Screening Visit) to end of treatment at week 16
Changes in International Index of Erectile Function Scores (IIEF)
Time frame: Baseline (Pre-Treatment Screening Visit) to end of treatment at week 16
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 15247734Khera M, Boone TB, Smith CP. Botulinum toxin type A: a novel approach to the treatment of recurrent urethral strictures. J Urol. 2004 Aug;172(2):574-5. doi: 10.1097/01.ju.0000130652.27541.22. No abstract available.
- Individual participant dataSharing planned: No. Data will be published in aggregate.
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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.
Mohit Khera, MD, MBA principal investigator
Baylor College of Medicine
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 100 units of Botulinum Toxin Type A, Preservative free normal saline and 100 units Botulinum Toxin A in people with Peyronie's Disease, run by Mohit Khera 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 3 inclusion and 11 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 8 Apr 2024. 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. 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 17 Feb 2020). 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?
Average Percent Change of Penile Curvature in Degrees (time frame: Baseline (Pre-Treatment Screening Visit) to end of treatment at week 16). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00812838, which also lists 1 linked publication.
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 8 Apr 2024.