This trial is evaluating whether Treatment will improve 1 primary outcome, 12 secondary outcomes, and 2 other outcomes in patients with Charcot-Marie-Tooth Disease, Type IA. Measurement will happen over the course of at month 6 and 9.
This trial requires 187 total participants across 2 different treatment groups
This trial involves 2 different treatments. Treatment is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 3 and have had some early promising results.
"The type iii HMSN may present with a variety of symptoms. The common treatments for types ii and iii HMSN may include surgical, medical and/or neuropsychological intervention. Neurodevelopmental treatment could benefit some patients. Copyright © 2015 John Wiley and Sons, Ltd." - Anonymous Online Contributor
"There is a high incidence of hmsn type iii in a subset of patients with BAV. The high concentration of Hmsn-III in all BAV patients and in controls suggests that hmsn type iii may be a BAV-specific marker." - Anonymous Online Contributor
"HMSN type iii is the commonest subtype of HMSN. Although the exact cause is not known, hmsn type iii is associated with an increased risk of developing prostate cancer." - Anonymous Online Contributor
"There is limited evidence of efficacy with anti-HV envelope peptides or anti-HIV envelope vaccines, but clinical workability and other considerations still need to be addressed before hmsn type iii will become a therapeutic intervention for HIV-associated malignancies like pancreatic cancer." - Anonymous Online Contributor
"The first sign of an hmsn type iii-associated disease is usually pain in the lower back and/or lower back pain. The second sign of an hmsn type iii-associated disease may be the swelling of the lymph nodes surrounding the hmsn type iii-associated disease. The third sign of an hmsn type iii-associated disease is usually the enlargement of the lymph nodes surrounding the hmsn type iii-associated disease, which may look similar to the disease of the hmsn type iii cells in size." - Anonymous Online Contributor
"Patients with hmsn type iii will experience a progressive decline in exercise capacity which may be related to atherosclerotic changes or an increased incidence of cardiovascular events associated with lipid metabolism abnormalities. Data from a recent study underscore the need for efficient treatment of hmsn type iii to halt its deteriorating course and minimize disability and morbidity." - Anonymous Online Contributor
"Although Hmsn type iii is not a single gene, we have demonstrated that Hmsn type iii mutations are found in many individuals from families. Recent findings suggest that this gene is responsible for at least some cases with an autosomal recessive inheritance." - Anonymous Online Contributor
"A large number of people are treated for HCV and HIV infection every day in the United Kingdom. Although this is a high risk group for a range of diseases, no studies have been published on the safety of antiviral therapies. The National Hospital Safety Audit Study will assess the overall risks of antiviral therapy by surveying all the patients treated during the year immediately preceding the study commences, and comparing to the controls in the same cohort that did not receive antiviral therapy." - Anonymous Online Contributor
"Data from a recent study, we only reported 1 case of severe HRS and did not include severe HRS type iii. More information on severe HRS type iii is needed, including clinical and survival outcomes." - Anonymous Online Contributor
"More research is needed to determine if specific treatments have any effect on various stages of this disease; however, a few treatments are starting to show positive outcomes. If you are looking for a similar clinical trial for promising new drugs, use the Power website to learn more." - Anonymous Online Contributor
"Despite early detection, the average age of death due to hmsn type iii remains around 15 years. The age of the person at death has increased over time, but with a smaller overall increase in age of death than would be expected for the increase in age of detection of this syndrome." - Anonymous Online Contributor
"In general, the most common side effects include fatigue, nausea and weight loss with only 9% of patients experiencing diarrhea, constipation and vomiting. If treatment includes chemotherapy, [prognosis is excellent] if not treated, it is fatal. Other common side effects include allergic reactions to chemotherapy, prolonged febrile neutropenia, and shortness of breath. There is also the concern of the potential for CLL patients to develop an allergy to abacavir, an antiretroviral medication used for the treatment of HIV/AIDS. Please check if [medication lists] have been uploaded. Patients should also be aware that some side effects will only become evident many months to years after treatment." - Anonymous Online Contributor