By Elissa Ritt, MAS
In a prior blog, we discussed how participating in clinical trials can be a great way to expose patients to cutting-edge treatments that might not be approved by the U.S. Food and Drug Administration (FDA) to treat certain diseases, but could help to further research so that future patients may benefit. One such study is Polyneuropathy and Treatment with Hizentra (PATH), which is being conducted by CSL Behring to evaluate the efficacy, safety and tolerability of treating Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) patients with subcutaneous immune globulin (SCIG).
CIDP is a rare disease that usually results in progressive weakness in the legs and sometimes the arms. The early diagnosis and treatment of CIDP can help to prevent disability in patients. Steroids, plasmapheresis and intravenous IG (IVIG) are commonly used to treat CIDP. IVIG is FDA-approved for the treatment of CIDP; however, SCIG is not.
IVIG is a purified solution of human antibodies that is administered through an intravenous infusion, or through a needle that is inserted into a vein. SCIG is similar in composition to IVIG, but it is delivered via fine needles subcutaneously, or under the skin. Although IVIG is generally well-tolerated, SCIG may result in fewer side effects for some patients. Additionally, because SCIG can be administered by the patient at any time or place, it may be more convenient than IVIG, which is usually administered in an infusion center or in the patient’s home over many hours.
The PATH study will evaluate whether SCIG is more effective than a placebo in treating CIDP. Patients may be eligible to enroll if they have been diagnosed with CIDP, have had more than four IVIG infusions in the prior nine months, and are over 18 years of age. In the study, patients stabilized on IVIG will be randomized to receive weekly infusions of one of two Hizentra doses (0.2 or 0.4 g/kg body weight) or a placebo for 24 weeks. The study will measure the proportion of patients who experience a relapse in their CIDP over a period of 52 weeks. If during the study a patient’s symptoms get worse, that patient will be given IVIG and will exit the study at that time.
Enrollment information and more details on the PATH study can be found on www.clinicaltrials.gov. Simply copy and paste this code into the search engine on the website homepage: NCT01545076. Patients interested who meet the enrollment criteria could help researchers develop a new treatment for patients who suffer from CIDP.