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Randomized Pilot Study for the Treatment of Cutaneous Leiomyomas With Botulinum Toxin

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ClinicalTrials.gov Identifier: NCT00971620
Recruitment Status : Completed
First Posted : September 4, 2009
Results First Posted : January 6, 2015
Last Update Posted : April 7, 2017
Sponsor:
Information provided by (Responsible Party):
Edward Cowen, M.D., National Institutes of Health Clinical Center (CC)

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Single Group Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Conditions Cutaneous Leiomyomas
Hereditary Leiomyomatosis and Renal Cell Cancer
Interventions Biological: Botulinum toxin type A
Other: Placebo
Enrollment 18
Recruitment Details  
Pre-assignment Details  
Arm/Group Title BTX-A Placebo/Saline
Hide Arm/Group Description BTX-A intralesional injection Saline intralesional injection
Period Title: Overall Study
Started 9 9
Completed 9 9
Not Completed 0 0
Arm/Group Title BTX-A Placebo/Saline Total
Hide Arm/Group Description BTX-A intralesional injection Saline intralesional injection Total of all reporting groups
Overall Number of Baseline Participants 9 9 18
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants 9 participants 18 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
8
  88.9%
9
 100.0%
17
  94.4%
>=65 years
1
  11.1%
0
   0.0%
1
   5.6%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 9 participants 9 participants 18 participants
48.4  (15.6) 45.5  (11.4) 47.0  (13.8)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants 9 participants 18 participants
Female
8
  88.9%
6
  66.7%
14
  77.8%
Male
1
  11.1%
3
  33.3%
4
  22.2%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants 9 participants 18 participants
Hispanic or Latino
0
   0.0%
0
   0.0%
0
   0.0%
Not Hispanic or Latino
9
 100.0%
9
 100.0%
18
 100.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants 9 participants 18 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
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
9
 100.0%
9
 100.0%
18
 100.0%
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%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 9 participants 9 participants 18 participants
9
 100.0%
9
 100.0%
18
 100.0%
Leiomyoma Distribution  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants 9 participants 18 participants
Single
1
  11.1%
3
  33.3%
4
  22.2%
Segmental
7
  77.8%
6
  66.7%
13
  72.2%
Disseminated
1
  11.1%
0
   0.0%
1
   5.6%
Location of Lesions  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants 9 participants 18 participants
Neck
1
  11.1%
0
   0.0%
1
   5.6%
Torso
6
  66.7%
4
  44.4%
10
  55.6%
Extremity
2
  22.2%
5
  55.6%
7
  38.9%
Volume of Drug Administered  
Mean (Standard Deviation)
Unit of measure:  mL
Number Analyzed 9 participants 9 participants 18 participants
2.41  (0.70) 1.12  (0.38) 1.76  (.54)
1.Primary Outcome
Title Change in Worst Lesional Pain in the Past Week Based on Brief Pain Inventory
Hide Description Change in worst lesional pain in the past week based on Brief Pain Inventory (BPI) from Week 0 to Week 4 in treated patients versus controls. The BPI uses an arbitrary units on a 0-10 scale. For the purposes of the statistical calculation, a difference of 1 standard deviation between groups at baseline vs. week 4 was considered significant. Any BPI value above zero (no pain) is abnormal. The mean change indicates mean change in pain score.
Time Frame Between week 0 and week 4
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title BTX-A Placebo/Saline
Hide Arm/Group Description:
BTX-A intralesional injection
Saline intralesional injection
Overall Number of Participants Analyzed 9 9
Mean (Standard Error)
Unit of Measure: units on a scale
-2.50  (0.46) -1.26  (0.53)
2.Primary Outcome
Title Median Change in Average Pain Between Two Arms
Hide Description Change in average pain was assessed by the Brief Pain Inventory (BPI). The BPI is a validated pain assessment tool that assesses severity of pain, location of pain, impact of pain on daily functions, pain medications, and amount of pain relief in the past 24 hours or past week (e.g. scale of 0-10 (worst pain)).
Time Frame Between weeks 0 and week 4
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title BTX-A Placebo/Saline
Hide Arm/Group Description:
BTX-A intralesional injection
Saline intralesional injection
Overall Number of Participants Analyzed 9 9
Median (Full Range)
Unit of Measure: Score
0.00
(-6.00 to 2.00)
0.00
(-5.00 to 3.00)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection BTX-A, Placebo/Saline
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .70
Comments [Not Specified]
Method Wilcoxon rank sum test
Comments [Not Specified]
Method of Estimation Estimation Parameter Hodges-Lehmann
Estimated Value 0.00
Confidence Interval (2-Sided) 95%
-2.00 to 4.00
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Number of Participants With Adverse Events
Hide Description Here is the number of participants with adverse events. For a detailed list of adverse events, see the adverse event module.
Time Frame 37 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title BTX-A Placebo/Saline
Hide Arm/Group Description:
BTX-A intralesional injection
Saline intralesional injection
Overall Number of Participants Analyzed 9 9
Measure Type: Number
Unit of Measure: participants
4 5
4.Secondary Outcome
Title Visual Analog Scale (VAS) of Patient Perceived Pain at Leiomyoma Site Prior to Ice Provocation at Week 0 vs. Week 4
Hide Description The VAS is a commonly used validated tool for assessment of pain. For this measure, a 10-cm VAS was used to assess current patient pain/discomfort before application of ice to study lesions at week 0 and week 4. A clinically meaningful change in chronic pain intensity using the VAS has been determined as a reduction of 2 points or 30%. Range is 0-10; 0 is no pain and 10 is worst possible pain.
Time Frame Week 0 vs. week 4
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title BTX-A Placebo/Saline
Hide Arm/Group Description:
BTX-A intralesional injection
Saline intralesional injection
Overall Number of Participants Analyzed 9 9
Median (Full Range)
Unit of Measure: Score
0.00
(-3.30 to 0.70)
0.40
(-1.30 to 1.50)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection BTX-A, Placebo/Saline
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .06
Comments [Not Specified]
Method Wilcoxon rank sum test
Comments [Not Specified]
5.Secondary Outcome
Title Comparison of Change in Skin Related Quality of Life by Total Dermatology Life Quality Index (DLQI) at Week 0 vs. Week 4
Hide Description The DLQI is a 10-question quality of life survey which has been extensively validated and frequently used in dermatologic disorders such as atopic dermatitis, acne, and psoriasis. Score is 0-30 based on 10 questions. The higher the score, the more quality of life is impaired.
Time Frame Week 0 vs. week 4
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title BTX-A Placebo/Saline
Hide Arm/Group Description:
BTX-A intralesional injection
Saline intralesional injection
Overall Number of Participants Analyzed 9 9
Median (Full Range)
Unit of Measure: Units on a scale
-4.00
(-8.00 to 2.00)
0.00
(-1.00 to 4.00)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection BTX-A, Placebo/Saline
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .007
Comments [Not Specified]
Method WIlcoxon rank sum test
Comments [Not Specified]
6.Secondary Outcome
Title Specific Skin Pain-Related Question on the Dermatology Life Quality Index
Hide Description The DLQI is a 10-question quality of life survey which has been extensively validated and frequently used in dermatologic disorders such as atopic dermatitis, acne, and psoriasis. Participants response to the question "Over the last week, how itchy, sore, painful or stinging has your skin been?" was assessed by the Dermatology Life Quality Index. This outcome refers to a single specific question on the DLQI, so the range for this outcome is 0-3. Lower values in the DLQI indicate less impairment (or greater improvement) in life quality from the skin disease.
Time Frame Week 0 vs. week 4
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title BTX-A Placebo/Saline
Hide Arm/Group Description:
BTX-A intralesional injection
Saline intralesional injection
Overall Number of Participants Analyzed 9 9
Median (Full Range)
Unit of Measure: Units on a scale
-1.00
(-2.00 to 1.00)
0.00
(-1.00 to 0.00)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection BTX-A, Placebo/Saline
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.48
Comments [Not Specified]
Method Wilcoxon rank sum test
Comments [Not Specified]
7.Secondary Outcome
Title Change in Post-Ice Provocation Visual Analog Score (VAS) Between Week 12 and Week 24
Hide Description The VAS is a commonly used validated tool for assessment of pain. The 10-cm VAS was used to assess current patient pain/discomfort before and after application of ice to study lesions. A clinically meaningful change in chronic pain intensity using the VAS has been determined as a reduction of 2 points or 30%. Scale is 0-10. 10 denotes worse pain than 0.
Time Frame Between week 12 and 24
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title BTX-A Placebo/Saline
Hide Arm/Group Description:
BTX-A intralesional injection
Saline intralesional injection
Overall Number of Participants Analyzed 9 9
Median (Full Range)
Unit of Measure: Score
4.10
(2.50 to 5.90)
-0.30
(-8.50 to 1.10)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection BTX-A, Placebo/Saline
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .04
Comments [Not Specified]
Method Wilcoxon rank sum test
Comments [Not Specified]
8.Secondary Outcome
Title Immunohistochemical Staining of Cutaneous Leiomyomas for Acetylcholinesterase (AchE) Before (i.e.,Week 0) and 12 Weeks After Botulinum Toxin Administration
Hide Description AchE staining was scored as 0 (none), 1 (rare), 2 (scattered), or 3 (focal or greater).
Time Frame Week 0 vs. week 12
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title BTX-A Placebo/Saline
Hide Arm/Group Description:
BTX-A intralesional injection
Saline intralesional injection
Overall Number of Participants Analyzed 9 9
Median (Full Range)
Unit of Measure: Score
1.00
(0.00 to 2.00)
0.00
(-1.00 to 0.00)
9.Secondary Outcome
Title Immunohistochemical Staining of Cutaneous Leiomyomas for C-fos Before (i.e., Week 0) and 12 Weeks After Botulinum Toxin Administration
Hide Description c-fos, a marker of neuronal activation after pain stimulation, was scored as 0 (none), 1 (scattered), 2 (<66% of tumor cells), or 3 (≥66% of tumor cells).
Time Frame Week 0 vs. week 12
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title BTX-A Placebo/Saline
Hide Arm/Group Description:
BTX-A intralesional injection
Saline intralesional injection
Overall Number of Participants Analyzed 9 9
Median (Full Range)
Unit of Measure: Score
-1.00
(-1.00 to 0.00)
0.00
(0.00 to 1.00)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection BTX-A, Placebo/Saline
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .07
Comments [Not Specified]
Method Wilcoxon rank sum test
Comments [Not Specified]
10.Secondary Outcome
Title Percentage of Patients With a Change in Average Pain Score
Hide Description Average pain was determined from a 0-10 scale question on the Brief Pain Inventory (BPI). 10 denotes worse pain.
Time Frame Week 0 score vs. week 4 score
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title BTX-A Placebo/Saline
Hide Arm/Group Description:
BTX-A intralesional injection
Saline intralesional injection
Overall Number of Participants Analyzed 9 9
Measure Type: Number
Unit of Measure: percentage of patients
>50% pain reduction 44 22
≤50% pain reduction 0 11
No change in pain 22 33
Increased pain 22 22
Missing 11 11
11.Secondary Outcome
Title Percentage of Patients With a Change in Pre-Ice Visual Analog Score (VAS) Between Week 0 and Week 4
Hide Description The VAS is a commonly used validated tool for assessment of pain. The 10-cm VAS was used to assess current patient pain/discomfort before and after application of ice to study lesions. A clinically meaningful change in chronic pain intensity using the VAS has been determined as a reduction of 2 points or 30%. Scale of 0-10. 10 = worse pain.
Time Frame Week 0 vs. week 4
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title BTX-A Placebo/Saline
Hide Arm/Group Description:
BTX-A intralesional injection
Saline intralesional injection
Overall Number of Participants Analyzed 9 9
Measure Type: Number
Unit of Measure: percentage of patients
>50% pain reduction 22 11
≤50% pain reduction 33 0
No change in pain 0 11
Increased pain 11 55
Missing 33 22
12.Secondary Outcome
Title Percentage of Patients With a Change in Post-Ice Visual Analog Score (VAS) Between Week 0 and Week 4
Hide Description The VAS is a commonly used validated tool for assessment of pain. The 10-cm VAS was used to assess current patient pain/discomfort before and after application of ice to study lesions. A clinically meaningful change in chronic pain intensity using the VAS has been determined as a reduction of 2 points or 30%. Scale is 0-10. 10 = worse pain.
Time Frame Week 0 vs. week 4
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title BTX-A Placebo/Saline
Hide Arm/Group Description:
BTX-A intralesional injection
Saline intralesional injection
Overall Number of Participants Analyzed 9 9
Measure Type: Number
Unit of Measure: percentage of patients
>50% pain reduction 33 33
≤50% pain reduction 44 44
No change in pain 0 0
Increased pain 22 22
Missing 0 0
13.Secondary Outcome
Title Worst Pain Severity
Hide Description Pain severity was assessed by the Brief Pain Inventory (BPI). The BPI is a validated pain assessment tool that assesses severity of pain, location of pain, impact of pain on daily functions, pain medications, and amount of pain relief in the past 24 hours or past week (e.g. scale of 0-10 (worst pain)). This outcome was based on a single 0-10 question on the BPI.
Time Frame Week 0 vs. week 4
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title BTX-A Placebo/Saline
Hide Arm/Group Description:
BTX-A intralesional injection
Saline intralesional injection
Overall Number of Participants Analyzed 9 9
Median (Full Range)
Unit of Measure: Score
-2.25
(-5.00 to 0.25)
-0.75
(-3.75 to 0.50)
Time Frame 37 months
Adverse Event Reporting Description There are no unexpected adverse events at grade 2 or higher.
 
Arm/Group Title BTX-A Placebo/Saline
Hide Arm/Group Description BTX-A intralesional injection Saline intralesional injection
All-Cause Mortality
BTX-A Placebo/Saline
Affected / at Risk (%) Affected / at Risk (%)
Total   0/9 (0.00%)      0/9 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
BTX-A Placebo/Saline
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/9 (0.00%)      0/9 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
BTX-A Placebo/Saline
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   4/9 (44.44%)      5/9 (55.56%)    
Cardiac disorders     
Hypertension  1  1/9 (11.11%)  1 1/9 (11.11%)  1
Gastrointestinal disorders     
Nausea  1  1/9 (11.11%)  1 0/9 (0.00%) 
Vomiting  1  1/9 (11.11%)  1 0/9 (0.00%) 
General disorders     
Constitutional Symptoms - Other (Specify, __)  1 [1]  1/9 (11.11%)  1 1/9 (11.11%)  1
Pain::Pain NOS  1 [2]  1/9 (11.11%)  1 1/9 (11.11%)  1
Immune system disorders     
Allergic rhinitis (including sneezing, nasal stuffiness, postnasal drip)  1  0/9 (0.00%)  0 1/9 (11.11%)  1
Infections and infestations     
Infection with normal ANC or Grade 1 or 2 neutrophils::Bronchus  1  1/9 (11.11%)  1 0/9 (0.00%) 
Infection with normal ANC or Grade 1 or 2 neutrophils::Sinus  1  0/9 (0.00%)  1/9 (11.11%)  1
Infection with unknown ANC::Upper airway NOS  1  1/9 (11.11%)  1 0/9 (0.00%) 
Musculoskeletal and connective tissue disorders     
Fracture  1  0/9 (0.00%)  1/9 (11.11%)  1
Pain - Other (Specify, __)  1 [3]  2/9 (22.22%)  2 2/9 (22.22%)  2
Pain::Back  1  0/9 (0.00%)  1/9 (11.11%)  1
Pain::Bone  1  0/9 (0.00%)  1/9 (11.11%)  1
Nervous system disorders     
Pain::Head/headache  1  1/9 (11.11%)  1 0/9 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Nasal cavity/paranasal sinus reactions  1  0/9 (0.00%)  1/9 (11.11%)  1
1
Term from vocabulary, CTCAE (3.0)
Indicates events were collected by systematic assessment
[1]
BTX-A: Sinus congestion; Placebo: Sore throat
[2]
BTX-A: Skin pain. Placebo: Pain of skin r/t biopsy
[3]
BTX-A: Pain of skin; Skin pain. Placebo: Pain of skin; Stiff neck.
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
Name/Title: Dr. Edward Cowen
Organization: National Cancer Institute
Phone: 301-496-4299
Responsible Party: Edward Cowen, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier: NCT00971620     History of Changes
Obsolete Identifiers: NCT00842725
Other Study ID Numbers: 090072
09-C-0072
First Submitted: September 3, 2009
First Posted: September 4, 2009
Results First Submitted: December 11, 2014
Results First Posted: January 6, 2015
Last Update Posted: April 7, 2017