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H-22411: BOTOX® for Peyronie's Disease

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ClinicalTrials.gov Identifier: NCT00812838
Recruitment Status : Completed
First Posted : December 22, 2008
Results First Posted : February 17, 2020
Last Update Posted : February 17, 2020
Sponsor:
Collaborator:
Allergan
Information provided by (Responsible Party):
Mohit Khera, Baylor College of Medicine

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Peyronie's Disease
Interventions Drug: 100 units of Botulinum Toxin Type A
Other: Preservative free normal saline
Drug: 100 units Botulinum Toxin A
Enrollment 12
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Experimental Placebo Comparator
Hide Arm/Group Description 100 units of Botulinum Toxin Type A Injection solution will consist of 100 units of BOTOX® in 10 cc of preservative free normal saline

Normal saline Injection solution will consist of 10 cc preservative free normal saline.

Optional 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.

Period Title: Treatment Period of 16 Weeks
Started 6 6
Completed 6 6
Not Completed 0 0
Period Title: Optional Crossover of 16 Weeks
Started [1] 0 4
Completed 0 4
Not Completed 0 0
[1]
Optional crossover did not apply to the experimental arm (ARM 1)
Arm/Group Title Experimental Placebo Comparator Total
Hide Arm/Group Description

100 units of Botulinum Toxin Type A Injection solution will consist of 100 units of BOTOX® in 10 cc of preservative free normal saline

*Crossover subjects were added to Experimental Group for analysis*

Normal saline Injection solution will consist of 10 cc preservative free normal saline.

Optional 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.

Total of all reporting groups
Overall Number of Baseline Participants 6 6 12
Hide Baseline Analysis Population Description
Per the study protocol, two arms of the study are analyzed; the experimental arm and the placebo arm *4 subjects cross over from ARM 2 to ARM 1after 16 weeks. Crossover subjects were added to Experimental Group for analysis with Adverse Events, International Index of Erectile Function, Penile Curvature, and Reduction in Plaque Size*
Age, Categorical   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 6 participants 12 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
6
 100.0%
6
 100.0%
12
 100.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
[1]
Measure Analysis Population Description: Per the study protocol, two arms of the study are analyzed; the experimental arm and the placebo arm
Age, Continuous   [1] 
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 6 participants 6 participants 12 participants
58.86  (4.741) 57.83  (3.710) 58.38  (4.154)
[1]
Measure Analysis Population Description: Per the study protocol, two arms of the study are analyzed; the experimental arm and the placebo arm
Sex: Female, Male   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 6 participants 12 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
6
 100.0%
6
 100.0%
12
 100.0%
[1]
Measure Analysis Population Description: Per the study protocol, two arms of the study are analyzed; the experimental arm and the placebo arm
Ethnicity (NIH/OMB)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 6 participants 12 participants
Hispanic or Latino
0
   0.0%
0
   0.0%
0
   0.0%
Not Hispanic or Latino
6
 100.0%
6
 100.0%
12
 100.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
[1]
Measure Analysis Population Description: Per the study protocol, two arms of the study are analyzed; the experimental arm and the placebo arm
Race (NIH/OMB)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 6 participants 12 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
1
  16.7%
1
   8.3%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
6
 100.0%
5
  83.3%
11
  91.7%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
[1]
Measure Analysis Population Description: Per the study protocol, two arms of the study are analyzed; the experimental arm and the placebo arm
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 6 participants 6 participants 12 participants
6 6 12
1.Primary Outcome
Title Average Percent Change of Penile Curvature in Degrees
Hide Description

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

Time Frame Baseline (Pre-Treatment Screening Visit) to end of treatment at week 16
Hide Outcome Measure Data
Hide Analysis Population Description
*Crossover subjects were added to Experimental Group for analysis*
Arm/Group Title Experimental Placebo Comparator
Hide Arm/Group Description:
100 units of Botulinum Toxin Type A Injection solution will consist of 100 units of BOTOX® in 10 cc of preservative free normal saline
Normal saline Injection solution will consist of 10 cc preservative free normal saline
Overall Number of Participants Analyzed 10 6
Mean (Standard Deviation)
Unit of Measure: percent change
-21.73  (57.69) 2.429  (81.59)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Experimental, Placebo Comparator
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5154
Comments This will be a pilot study; it was assessed that 10 subjects in each arm, for a total of 20 subjects, will allow us to determine if the therapy is effective. To allow for screen failures, 45 subjects are planned for screening.
Method t-test, 2 sided
Comments [Not Specified]
2.Secondary Outcome
Title Change in Penile Blood Flow for Peak Systolic Velocity (PSV) and End-diastolic Velocity (EDV)
Hide Description

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

Time Frame Baseline (Pre-Treatment Screening Visit) to end of treatment at week 16
Hide Outcome Measure Data
Hide Analysis Population Description
*Penile Doppler ultrasound was not performed on optional crossover patients. Data reflects scores derived from Experimental and Placebo group analysis.
Arm/Group Title 100 Units of Botulinum Toxin Type A Normal Saline
Hide Arm/Group Description:

Injection solution will consist of 100 units of BOTOX® in 10 cc of preservative free normal saline

100 units of Botulinum Toxin Type A: Approximately 20 to 30 injections of 100 units of BOTOX® given with a 20 gage needle directly into the penile plaque

Injection solution will consist of 10 cc preservative free normal saline

Preservative free normal saline: Approximately 20 to 30 injections of 10cc of preservative free normal saline given with a 20 gage needle directly into the penile plaque

Overall Number of Participants Analyzed 6 6
Mean (Standard Deviation)
Unit of Measure: cm/s
Pre injection Right PSV -0.65670  (5.505) -9.0170  (11.43)
Pre injection Right EDV -2.10200  (6.798) -0.8667  (3.771)
Pre injection Left PSV -1.263  (9.032) -6.4330  (12.04)
Pre injection Left EDV -10.27  (8.261) -6.75  (11.63)
5 minutes right PSV 5.185  (28.48) 2.1330  (6.425)
5 minutes right EDV -11.05  (19.56) -0.1417  (3.944)
5 minutes left psv 2.863  (17.68) -6.6830  (22.76)
5 minutes left edv -2.4  (10.80) 3.5  (10.16)
15 minutes right PSV 6.917  (20.91) -15.52  (34.44)
15 minutes right EDV -10.22  (22.82) 1.55  (5.001)
15 minutes left PSV 7.183  (10.81) -15.10  (40.93)
15 minutes left EDV -2.267  (11.58) 3.367  (6.046)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection 100 Units of Botulinum Toxin Type A, Normal Saline
Comments

This will be a pilot study; it was assessed that 10 subjects in each arm, for a total of 20 subjects, will allow us to determine if the therapy is effective. To allow for screen failures, 45 subjects are planned for screening.

This analysis pertains to both categories

Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3009
Comments This applies to 100 units of Botulinum Toxin Type A arm vs the Normal saline arm
Method t-test, 2 sided
Comments [Not Specified]
3.Secondary Outcome
Title Change in Penile Blood Flow for Diameter
Hide Description

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

Time Frame Baseline (Pre-Treatment Screening Visit) to end of treatment at week 16
Hide Outcome Measure Data
Hide Analysis Population Description
*Penile Doppler ultrasound was not performed on optional crossover patients. Data reflects scores derived from Experimental and Placebo group analysis.
Arm/Group Title Experimental Placebo Comparator
Hide Arm/Group Description:
100 units of Botulinum Toxin Type A Injection solution will consist of 100 units of BOTOX® in 10 cc of preservative free normal saline
Normal saline Injection solution will consist of 10 cc preservative free normal saline
Overall Number of Participants Analyzed 6 6
Mean (Standard Deviation)
Unit of Measure: cm
Pre injection right Diameter -0.29  (0.8539) 0.1700  (0.7208)
Pre injection left diameter 0.04167  (0.5869) 0.3983  (0.4565)
5 minute right diameter 1.34200  (4.006) 0.2917  (0.6719)
5 minute left diameter -0.75330  (2.551) 0.0750  (0.9000)
15 minute right diameter -1.43700  (3.952) -0.3550  (0.6469)
15 minute left diameter -0.43500  (2.571) -0.1867  (1.203)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Experimental, Placebo Comparator
Comments This will be a pilot study; it was assessed that 10 subjects in each arm, for a total of 20 subjects, will allow us to determine if the therapy is effective. To allow for screen failures, 45 subjects are planned for screening.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0166
Comments This applies to 100 units of Botulinum Toxin Type A arm vs the Normal saline arm
Method t-test, 2 sided
Comments [Not Specified]
4.Secondary Outcome
Title Change in Penile Plaque Size
Hide Description

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

Time Frame Baseline (Pre-Treatment Screening Visit) to end of treatment at week 16
Hide Outcome Measure Data
Hide Analysis Population Description
*Crossover subjects were added to Experimental Group for analysis*
Arm/Group Title Experimental Placebo Comparator
Hide Arm/Group Description:
100 units of Botulinum Toxin Type A Injection solution will consist of 100 units of BOTOX® in 10 cc of preservative free normal saline
Normal saline Injection solution will consist of 10 cc preservative free normal saline
Overall Number of Participants Analyzed 10 6
Mean (Standard Deviation)
Unit of Measure: cubic millimeters
screening visit 717.5  (827.4) 485.8  (599.6)
week 16 198.9  (220.8) 319.0  (252.2)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Experimental, Placebo Comparator
Comments This will be a pilot study; it was assessed that 10 subjects in each arm, for a total of 20 subjects, will allow us to determine if the therapy is effective. To allow for screen failures, 45 subjects are planned for screening.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .8566
Comments Threshold for statistical significance was <0.05
Method t-test, 2 sided
Comments [Not Specified]
5.Secondary Outcome
Title Changes in International Index of Erectile Function Scores (IIEF)
Hide Description

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 :

  1. Orgasmic Function (Questions 9, 10); Maximum score = 10, Minimum score = 0
  2. Sexual Desire (Questions 11, 12); Maximum score = 10, Minimum score = 0
  3. Intercourse Satisfaction (Questions 6, 7, 8); Maximum score = 15, Minimum score = 0
  4. 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*

Time Frame Baseline (Pre-Treatment Screening Visit) to end of treatment at week 16
Hide Outcome Measure Data
Hide Analysis Population Description
*Crossover subjects were added to Experimental Group for analysis*
Arm/Group Title Experimental Placebo Comparator
Hide Arm/Group Description:
100 units of Botulinum Toxin Type A Injection solution will consist of 100 units of BOTOX® in 10 cc of preservative free normal saline
Normal saline Injection solution will consist of 10 cc preservative free normal saline
Overall Number of Participants Analyzed 10 6
Mean (Standard Deviation)
Unit of Measure: score on a scale
Screening Visit 15.60  (11.75) 21  (12.13)
Week 16 17.20  (11.61) 21.50  (11.74)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Experimental, Placebo Comparator
Comments This will be a pilot study; it was assessed that 10 subjects in each arm, for a total of 20 subjects, will allow us to determine if the therapy is effective. To allow for screen failures, 45 subjects are planned for screening.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0286
Comments Threshold for statistical significance is <0.05
Method t-test, 2 sided
Comments [Not Specified]
Time Frame 2 years, 8 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title 100 Units of Botulinum Toxin Type A Normal Saline
Hide Arm/Group Description

Injection solution will consist of 100 units of BOTOX® in 10 cc of preservative free normal saline

100 units of Botulinum Toxin Type A: Approximately 20 to 30 injections of 100 units of BOTOX® given with a 20 gage needle directly into the penile plaque

Injection solution will consist of 10 cc preservative free normal saline

Preservative free normal saline: Approximately 20 to 30 injections of 10cc of preservative free normal saline given with a 20 gage needle directly into the penile plaque

All-Cause Mortality
100 Units of Botulinum Toxin Type A Normal Saline
Affected / at Risk (%) Affected / at Risk (%)
Total   0/10 (0.00%)      0/6 (0.00%)    
Hide Serious Adverse Events
100 Units of Botulinum Toxin Type A Normal Saline
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/10 (0.00%)      0/6 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
100 Units of Botulinum Toxin Type A Normal Saline
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   5/10 (50.00%)      0/6 (0.00%)    
Gastrointestinal disorders     
small bowel obstruction * [1]  1/10 (10.00%)  1 0/6 (0.00%)  0
Immune system disorders     
Cold * [2]  2/10 (20.00%)  2 0/6 (0.00%)  0
Musculoskeletal and connective tissue disorders     
back pain * [3]  1/10 (10.00%)  1 0/6 (0.00%)  0
Foot pain * [3]  1/10 (10.00%)  1 0/6 (0.00%)  0
Renal and urinary disorders     
increased frequent urination * [4]  1/10 (10.00%)  1 0/6 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
seasonal allergies * [5]  1/10 (10.00%)  1 0/6 (0.00%)  0
Vascular disorders     
Bruising * [3]  2/10 (20.00%)  2 0/6 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
[1]
1, small bowel obstruction; pain pressure in his abdomen
[2]
Cold - no treatment provided
[3]
No treatment was provided
[4]
increased frequent urination
[5]
seasonal allergies
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Mohit Khera, MD
Organization: Baylor College of Medicine
Phone: 713-798-6593
EMail: mkhera@bcm.edu
Layout table for additonal information
Responsible Party: Mohit Khera, Baylor College of Medicine
ClinicalTrials.gov Identifier: NCT00812838    
Other Study ID Numbers: 11-07-40-04
First Submitted: December 18, 2008
First Posted: December 22, 2008
Results First Submitted: January 13, 2020
Results First Posted: February 17, 2020
Last Update Posted: February 17, 2020