Patients in Clinical Trials: Peggy Shares Her Experience
Peggy Kindler has lived with ulcerative colitis for 28 years. A 48-year old social worker from Pittsburgh, Peggy was diagnosed with ulcerative colitis when she was in college. "I took sulfasalazine at first, then switched to mesalamine." She did well until 1992, when she came down with a viral infection that caused a flare of her colitis. Prednisone brought this attack under control, and Peggy did well on Asacol® (mesalamine) for the next 10 years. But her most severe bout with the disease was still to come.
In 2002, a severe flare of ulcerative colitis sent Peggy from the doctor's office right to the hospital. She was admitted and treated immediately with intravenous prednisone. "I was so sick I was glad to be in the hospital," Peggy recalls. "I told myself they'll be able to do something for me now." But her symptoms failed to respond to the prednisone and her condition worsened. "I had 10 or more bowel movements a day," she remembers. "I couldn't eat much. And I was worried about having surgery." She was running out of options. As her pain and debilitating diarrhea increased, Peggy was facing the treatment of last resort for severe ulcerative colitis -- colectomy (surgical removal of the colon). But she was willing to try different options to avoid surgery — and one of those options happened to be a clinical trial.
"Patients like Peggy are the sickest of the sick," says Dr. Scott Plevy. "They literally have one foot on the operating table." Dr. Plevy is Associate Professor of Medicine and Co-Director of the Inflammatory Bowel Disease Center at the University of Pittsburgh's School of Medicine. Although surgery can be a cure for ulcerative colitis in most patients, it does have some risks, such as infection or bowel obstruction.
Dr. Plevy stopped by Peggy's room and told her about a clinical trial for a new drug that might offer her an alternative to surgery. This alternative, known as Nuvion® (visilizumab), is an investigational drug currently under development by California-based biotechnology company Protein Design Labs, Inc. Visilizumab belongs to a class of biologic drugs known as monoclonal antibodies. Visilizumab targets T cells, which play a role in the immune response that results in the intestinal inflammation in ulcerative colitis and Crohn's disease.
"He told me we could always do something else if it didn't work," Peggy says. So she agreed to enroll in the clinical trial.
Peggy Kindler was the first ulcerative colitis patient in the United States to receive visilizumab. Before the drug could be administered, Peggy met with a clinical nurse specialist, another member of the clinical trial team, who explained what the new drug was, what it was supposed to do, how it would be administered, potential side effects, and what would be required of Peggy if she agreed to participate in the trial.
"They did some blood work. And I had lots of consent forms to fill out," Peggy recalls. Once the forms were signed, she was given the first of two doses of the drug, which was administered on two consecutive days. She remained in the hospital so that her condition and response to the drug could be monitored. "After the first dose, I had chills and vomiting and generally felt sick," Peggy says. "But after the second dose the next day, I felt fine." The clinical trial medical staff monitored her carefully, and Dr. Plevy visited every day to chat and see how she was progressing. Five days after the second dose, she went home from the hospital.
Clinical trial enrollment is a careful process for the patient. The patient should discuss participation with his or her physician, who should be kept updated about the patient's progress in the trial. The Food and Drug Administration (FDA), the government agency responsible for approving new drugs, protects patient rights by requiring that people enrolled in trials sign an informed consent form. The consent procedure mandates that researchers provide adequate information about the study and respond fully to participants' questions. The investigators must be certain that the patient understands all risks and responsibilities and is aware of other treatment options. Patients enrolling in a trial are closely monitored throughout by the clinical investigators. If a patient qualifies for participation in a clinical trial, and is comfortable with the risks involved, the experience can be worthwhile, as Peggy discovered.
As part of the trial's protocol, nine follow-up visits to monitor safety and efficacy were required periodically, up to one year after the last dose of visilizumab. Peggy underwent a flexible sigmoidoscopy at intervals over the next 90 days to check the condition of her colon. She also kept a record of the number of bowel movements she had each day, and if she experienced any soiling, bleeding or other symptoms. "I kept the diary in the bathroom so I would remember to fill it out," she notes. "But none of this was inconvenient."
What Happens Next?
Peggy suffered no side effects from doses of visilizumab, except for the flu-like symptoms she experienced after the first dose. Within a few days after the treatments were completed, she noticed a dramatic decrease in her bowel movements. "A month after treatment, I felt like I was never sick," she says. Mesalamine was prescribed as maintenance medication to prevent another flare.
For Peggy, being in the clinical trial was an interesting and rewarding experience. "I liked having the opportunity to contribute to medical knowledge. I wasn't scared to try Nuvion. I was so sick I figured if this will make me better then let's try it." She believes her recovery proves that the drug may work. "I felt like it was a wonder drug for me. And I'm glad I could help get a drug on the market that may benefit others."
Visilizumab is currently in Phase I/II trials. Phase I trials test potential new therapies in humans to demonstrate safety at the projected dose range and to determine how the drug is broken down and used by the body. Phase II trials are the pilot trials, testing a dose or two in patients with the proposed disease. If these are promising, then Phase III trials are conducted to demonstrate safety and effectiveness in larger trials involving hundreds of patients. If you are interested in enrolling in a clinical trial, either for visilizumab or for another investigational treatment, visit www.crohnscolitisfoundation.org/trials to see what's available in your area.
Karen McNally Bensing, M.S.L.S. is a freelance writer and librarian from Cleveland, Ohio.
For further information, call Crohn's & Colitis Foundation's IBD Help Center: 888.MY.GUT.PAIN (888.694.8872).
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Published: May 1, 2012