18 and older, any sex, with Cutaneous Leiomyomas or Hereditary Leiomyomatosis and Renal Cell Cancer. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Change in Worst Lesional Pain in the Past Week Based on Brief Pain InventoryPrimary· Between week 0 and week 4
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.
Group
Value
95% CI
BTX-A
-2.50
± 0.46
Placebo/Saline
-1.26
± 0.53
Median Change in Average Pain Between Two ArmsPrimary· Between weeks 0 and week 4
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)).
Group
Value
95% CI
BTX-A
0.00
-6.00 – 2.00
Placebo/Saline
0.00
-5.00 – 3.00
Number of Participants With Adverse EventsSecondary· 37 months
Here is the number of participants with adverse events. For a detailed list of adverse events, see the adverse event module.
Group
Value
95% CI
BTX-A
4
Placebo/Saline
5
Visual Analog Scale (VAS) of Patient Perceived Pain at Leiomyoma Site Prior to Ice Provocation at Week 0 vs. Week 4Secondary· Week 0 vs. week 4
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.
Group
Value
95% CI
BTX-A
0.00
-3.30 – 0.70
Placebo/Saline
0.40
-1.30 – 1.50
Comparison of Change in Skin Related Quality of Life by Total Dermatology Life Quality Index (DLQI) at Week 0 vs. Week 4Secondary· Week 0 vs. week 4
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.
Group
Value
95% CI
BTX-A
-4.00
-8.00 – 2.00
Placebo/Saline
0.00
-1.00 – 4.00
Specific Skin Pain-Related Question on the Dermatology Life Quality IndexSecondary· Week 0 vs. week 4
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.
Group
Value
95% CI
BTX-A
-1.00
-2.00 – 1.00
Placebo/Saline
0.00
-1.00 – 0.00
Change in Post-Ice Provocation Visual Analog Score (VAS) Between Week 12 and Week 24Secondary· Between week 12 and 24
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.
Group
Value
95% CI
BTX-A
4.10
2.50 – 5.90
Placebo/Saline
-0.30
-8.50 – 1.10
Immunohistochemical Staining of Cutaneous Leiomyomas for Acetylcholinesterase (AchE) Before (i.e.,Week 0) and 12 Weeks After Botulinum Toxin AdministrationSecondary· Week 0 vs. week 12
AchE staining was scored as 0 (none), 1 (rare), 2 (scattered), or 3 (focal or greater).
Group
Value
95% CI
BTX-A
1.00
0.00 – 2.00
Placebo/Saline
0.00
-1.00 – 0.00
Immunohistochemical Staining of Cutaneous Leiomyomas for C-fos Before (i.e., Week 0) and 12 Weeks After Botulinum Toxin AdministrationSecondary· Week 0 vs. week 12
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).
Group
Value
95% CI
BTX-A
-1.00
-1.00 – 0.00
Placebo/Saline
0.00
0.00 – 1.00
Percentage of Patients With a Change in Average Pain ScoreSecondary· Week 0 score vs. week 4 score
Average pain was determined from a 0-10 scale question on the Brief Pain Inventory (BPI). 10 denotes worse pain.
>50% pain reduction
Group
Value
95% CI
BTX-A
44
Placebo/Saline
22
≤50% pain reduction
Group
Value
95% CI
BTX-A
0
Placebo/Saline
11
No change in pain
Group
Value
95% CI
BTX-A
22
Placebo/Saline
33
Increased pain
Group
Value
95% CI
BTX-A
22
Placebo/Saline
22
Missing
Group
Value
95% CI
BTX-A
11
Placebo/Saline
11
Percentage of Patients With a Change in Pre-Ice Visual Analog Score (VAS) Between Week 0 and Week 4Secondary· Week 0 vs. week 4
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.
>50% pain reduction
Group
Value
95% CI
BTX-A
22
Placebo/Saline
11
≤50% pain reduction
Group
Value
95% CI
BTX-A
33
Placebo/Saline
0
No change in pain
Group
Value
95% CI
BTX-A
0
Placebo/Saline
11
Increased pain
Group
Value
95% CI
BTX-A
11
Placebo/Saline
55
Missing
Group
Value
95% CI
BTX-A
33
Placebo/Saline
22
Percentage of Patients With a Change in Post-Ice Visual Analog Score (VAS) Between Week 0 and Week 4Secondary· Week 0 vs. week 4
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.
>50% pain reduction
Group
Value
95% CI
BTX-A
33
Placebo/Saline
33
≤50% pain reduction
Group
Value
95% CI
BTX-A
44
Placebo/Saline
44
No change in pain
Group
Value
95% CI
BTX-A
0
Placebo/Saline
0
Increased pain
Group
Value
95% CI
BTX-A
22
Placebo/Saline
22
Missing
Group
Value
95% CI
BTX-A
0
Placebo/Saline
0
Adverse events — posted to ClinicalTrials.gov
Time frame: 37 months.
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Cutaneous leiomyomas are benign tumors of smooth muscle origin. They can be very painful, and current treatments for the tumors and for the associated pain do not produce satisfactory results. One potential treatment for localized severe muscle pain involves injections with botulinum toxin A. This study will investigate the effectiveness, side effects, and dosage of botulinum toxin A (BOTOX) as a treatment for patients with pain associated with cutaneous leiomyomas.
This study will include 18 subjects, all of whom will be 18 years of age and older, who have pain associated with cutaneous leiomyomas.
For the 24-week study, patients will be randomly assigned to one of two treatment groups. Neither the study team nor the patient will know to which group patients have been assigned. Before the study begins, all participants must provide a full medical history for research and evaluation purposes, fill out pain and quality-of-life questionnaires, and undergo an ice test in which researchers will apply ice to the site of the cutaneous leiomyomas and ask participants to evaluate the level of pain before and after ice application. Both groups will be required to keep a pain diary throughout the study to record their level of pain on a daily basis, and will be asked to avoid or restrict the use of specific medications or other remedies to treat the pain.
At the first visit (Week 0), one group will receive a prescribed dose of botulinum toxin A, which will be administered as an injection into the leiomyoma, and the other (control) group will receive a placebo injection of a saline solution. Patients will return 4 weeks later, at which time they will undergo a medical examination, and the ice test, and complete questionnaires to assess responses and level of pain. Patients will return in Week 12, at which time the group assignment will be revealed (un-blinded) to investigators and patients. Patients who received placebo injections will be offered the opportunity to receive injection of botulinum toxin A into their leiomyomas. All patients will undergo a medical examination, the ice test, complete questionnaires, and continue completing their daily pain diaries at home. The final visit, in Week 24, will follow the same procedure as the Week 4 visit.
At the end of the study, patients may be eligible to have one or more of the painful cutaneous leiomyomas surgically removed if the researchers believe that the skin lesions can be removed with a reasonable cosmetic result.
Publications & conference data
1 peer-reviewed publication reference this trial (live from Europe PMC):
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
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Sponsor: as reported to ClinicalTrials.gov by National Cancer Institute (NCI)
Last refreshed: 7 April 2017
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00971620.