Protocol Number: 09-C-0072

Title:

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

Number:

09-C-0072

Summary:

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(Trademark)) 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 divided into two groups. Before the study begins, all participants must provide a full medical history for research and evaluation purposes, fill out a pain questionnaire, 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. 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 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, and the other (control) group will receive a placebo injection of a saline solution. After the initial treatment, patients will return 4 weeks later, at which time all patients will undergo a medical examination and the ice test to assess level of pain. Patients will return in Week 12, at which time all patients in both groups will be offered the opportunity to receive an injection of botulinum toxin A, and will undergo a medical examination and the ice test. 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.

Sponsoring Institute:

National Cancer Institute (NCI)

Recruitment Detail

Type: Participants currently recruited/enrolled

Gender: Male & Female

Referral Letter Required: No

Population Exclusion(s): Children

Eligibility Criteria:

INCLUSION CRITERIA:

1. Subjects must be age greater than or equal to18 years

2. Subjects must have a prior biopsy-diagnosis of cutaneous leiomyoma.

3. Subjects must have at least 1 symptomatic leiomyomas or regions less than or equal to 60 cm(2) of leiomyomas with pain characterized as greater than 4 based on a 10-point scale, indicating pain of at least moderate severity.

4. Pain episodes must occur at least once a week.

5. Subjects must have the ability to participate fully and comply with the procedures of the protocol in the opinion of the investigator.

6. Written informed consent has been obtained including consenting to have tissue samples stored, however subjects are allowed to refuse sample storage

7. Negative urine or serum pregnancy test in females of childbearing potential.

8. Subjects who are clinically stable such that they can be expected to complete the 24-week study.

EXCLUSION CRITERIA:

1. Subjects with allergies to BTX-A.

2. Females with a positive pregnancy test, or who are breast-feeding, planning a pregnancy during the study, who think that they may be pregnant at the start of the study or females of childbearing potential who are unable or unwilling to use a reliable form of contraception during the study.

3. Subjects with neuromuscular junction disorders (ie. myasthenia gravis or Lambert-Eaton syndrome) or peripheral motor neuropathic diseases (ie. amyotrophic lateral sclerosis or motor neuropathy).

4. Subjects with infection at the intended sites of injection.

5. Subjects who have had prior Botulinum toxin product within the past 6 months.

6. Subjects with pain resulting from other disease(s).

7. Subjects taking pain medications, neuroactive agents, or other therapy directed toward treatment of cutaneous leiomyomas concurrently or within 5 days or 5 half-lives (whichever is longer) of BTX-A treatment, other than specified rescue pain medications).

8. Subjects with late-stage cancers or unstable disease (such as hemodynamic instability, i.e., systolic or diastolic blood pressure fall of 20 mm Hg or greater from the stable patient's baseline measurement).

9. A condition or situation that, in the investigator's opinion, may put the subject at significant risk or interfere significantly with the subject's participation in the study.

Special Instructions:

Currently Not Provided

Keywords:

Cutaneous Leiomyomas

Skin Pain

Botox Therapy

Hereditary Leiomyomatosis

Renal Cell Cancer

Recruitment Keyword(s):

Cutaneous Leiomyoma

Hereditary Leiomyomatosis

Renal Cell Cancer

Condition(s):

Cutaneous Leiomyomas

Hereditary Leiomyomatosis and Renal Cell Cancer

Investigational Drug(s):

None

Investigational Device(s):

None

Intervention(s):

Drug: Botulinum Toxin-A

Supporting Site:

National Cancer Institute

Contact(s):

NCI Referral Office
National Institute of Health Clinical Center (CC), 9000 Rockville Pike, Bethesda, Maryland 20892, United States: NCI Clinical Trials Referral Office
Phone: 1-888-NCI-1937
Fax: Not Listed
Electronic Address: ncicssc@mail.nih.gov

Citation(s):

Archer CB, Whittaker S, Greaves MW. Pharmacological modulation of cold-induced pain in cutaneous leiomyomata. Br J Dermatol. 1988 Feb;118(2):255-60.

Batchelor RJ, Lyon CC, Highet AS. Successful treatment of pain in two patients with cutaneous leiomyomata with the oral alpha-1 adrenoceptor antagonist, doxazosin. Br J Dermatol. 2004 Apr;150(4):775-6.

Raj S, Calonje E, Kraus M, Kavanagh G, Newman PL, Fletcher CD. Cutaneous pilar leiomyoma: clinicopathologic analysis of 53 lesions in 45 patients. Am J Dermatopathol. 1997 Feb;19(1):2-9.

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