H-22411: BOTOX® for 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).

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.

Eligibility Criteria:
Inclusion Criteria:

* Subjects with stable Peyronie's plaques.
* Males at least 18 years of age
* Must give informed consent.

Exclusion Criteria:

* 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.
* 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.