Selumetinib for Watson Syndrome
This study is evaluating whether selumetinib is effective in treating people with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas.
See full descriptionAbout the trial for Watson Syndrome
Treatment Groups
This trial involves 2 different treatments. Selumetinib is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 3 and have had some early promising results.
About The Treatment
Side Effect Profile for AZD6244 (Selumetinib) Treatment
Eligibility
This trial is for patients born any sex aged 18 and older. There are 6 eligibility criteria to participate in this trial as listed below.
Approximate Timelines
Measurement Requirements
This trial is evaluating whether Selumetinib will improve 2 primary outcomes, 8 secondary outcomes, and 2 other outcomes in patients with Watson Syndrome. Measurement will happen over the course of Approximately 3 years.
Patient Q & A Section
How many people get watson syndrome a year in the United States?
The incidence of Watson syndrome (WAS) in the US population has increased substantially since at least 1985. Especially among adults 20 years and older, the prevalence of WAS was found to be higher than previously estimated.
Does watson syndrome run in families?
Individuals with Watson syndrome have a family history of the disorder. Further studies are needed to determine whether genes predispose to this condition or environmental factors influence the development of the syndrome.
What is the primary cause of watson syndrome?
The main cause of Watson syndrome is not congenital toxoplasmosis or malformations of the heart, but rather the presence of a muscular dystrophy. The clinical presentation of Watson Syndrome is similar to Duchenne muscular dystrophy, however, there are many differences including severity of muscle weakness and the development of some cardiac features such as pulmonary hypertension. Watson Syndrome is named after Dr. James Watson who was one of the first to describe this disorder in 1959.
Is selumetinib safe for people?
Selumetinib is well tolerated in patients with advanced non-small cell lung cancer (NSCLC) who have previously received platinum-based chemotherapy. The safety profile in these patients appears similar to that of previous studies involving patients treated with EGFR inhibitors alone or in combination with platinum therapy. ClinicalTrials.gov identifier: NCT00716365.
What are the signs of watson syndrome?
Watson's syndrome has a number of possible manifestations. The most common presenting symptom is non-restorative sleep. However, patients can have other simple symptoms like headache and nausea. Other characteristic features include severe chest wall weakness, stridor, short stature, and delayed motor development. Although this syndrome was first described by Watson in 1910, it was only recently recognized as a separate entity. Watson's Syndrome is named after Dr. William Watson who first collected the facts of the disorder at the Mayo Clinic in Rochester, Minnesota. The combination of these facts led him to suspect that a connective tissue disorder (CTD) might be present. In addition to Watson himself, his research team also included several other physicians who were involved in researching the disorder.
Have there been other clinical trials involving selumetinib?
No previous clinical trials of selumetinib are available. However, selumetinib showed promising efficacy in preclinical studies in non-small cell lung cancer. Further clinical trials of selumetinib in solid tumors are warranted.
What are the latest developments in selumetinib for therapeutic use?
Selumetinib (AZD6244) has been approved by the Food and Drug Administration (FDA) as a second-line treatment for metastatic renal cell carcinoma (mRCC) after failure of prior therapies or intolerance to prior therapy. It has also received approval from the European Commission for mRCC treatment following progression through or intolerance to platinum-containing chemotherapy. In May 2013, selumetinib was approved as a first-line treatment for patients with unresectable locally advanced or metastatic RCC who have received prior systemic therapy. In November 2014, selumetinib was approved by the FDA to treat adult patients who have received prior therapy for metastatic papillary thyroid cancer.
What causes watson syndrome?
The cause of Watson Syndrome remains unknown. A small number of cases have been reported but the epidemiology is still not understood. Watson syndrome occurs more frequently in women than men (approximately one in 1000 female births compared to one in 2000 male births); this is true at every age group. The reasons why only some women develop Watson Syndrome remain unclear. There is no evidence that it has any hereditary component. It is conceivable that women are predisposed to developing Watson Syndrome as a result of estrogen deficiency. Watson Syndrome should be considered in the differential of all unexplained death of young children.
How does selumetinib work?
Selumetinib may induce tumor cell death through inhibition of PI3K signaling. Inhibition of AKT activity may also contribute to the antitumor effects seen with selumetinib.
What is the latest research for watson syndrome?
Watson syndrome is a rare disease that affects children who are born small for gestational age and who develop lung abnormalities. Some lung problems may resolve on their own, but they need to be monitored closely. If the lung problems do not resolve, then surgical treatment may be necessary. For adults, there is no clear treatment for Watson syndrome. However, some researchers believe that lung transplantation may help patients with certain severe forms of this disorder. Although preliminary studies suggest that lung transplantation may be helpful for some patients, more research is needed to determine whether it improves patient outcomes or decreases them.
What is selumetinib?
Seldesleukin is an orally bioavailable inhibitor of JAK2 kinase. The FDA approved selumetinib (AZD1156) as a treatment for chronic myelogenous leukemia (CML) and Philadelphia chromosome positive (Ph+) acute lymphoblastic leukemia (ALL). Selumetinib has been shown to inhibit JAK2 kinase activity with IC50 values of 0.1nM at right-shifted JAK2 V617F mutant protein, 1.0nM at WT JAK2 protein and 3.0nM at WT JAK2 protein. Pharmacokinetic studies show that 50 - 90% of oral doses are absorbed from the gastrointestinal tract.