From True Blue Tribune
Otsuka Pharmaceutical Initiates Phase 3 Clinical Trial Of Tolvaptan In Patients With Autosomal Dominant Polycystic Kidney Disease
Otsuka Pharmaceutical Development & Commercialization, Inc., announced today that patient enrollment has begun for a new Phase 3b study of tolvaptan for adult patients with autosomal dominant polycystic kidney disease (ADPKD), the most common, life-threatening, inherited genetic kidney disorder.1 The company reached an agreement with the United States Food & Drug Administration (FDA) on a Special Protocol Assessment (SPA) for the design and planned analysis of this multi-center, placebo-controlled, double-blind, parallel-group trial designed to compare the efficacy and safety of tolvaptan (45 to 120 mg per day split-dose) in subjects with chronic kidney disease between late stage 2 and early stage 4 due to ADPKD.
“Otsuka is pleased that the FDA has agreed to the design for this study and we are happy to report that the first patient has been enrolled into the trial,” said William H. Carson, M.D., president and CEO of Otsuka Pharmaceutical Development & Commercialization, Inc. “This study represents our ongoing commitment to patients with this rare genetic condition to provide them and their physicians with a novel treatment option.”
The trial, which will take place at approximately 220 sites worldwide, is seeking to enroll approximately 1,300 tolvaptan-naïve adult (male and female) patients with ADPKD between the ages of 18-66, depending on their estimated glomerular filtration rate (eGFR), a renal function test, at time of enrollment.
Total study duration is about 15 months, and the treatment period will last for approximately 13 ½ months. The primary endpoint will compare the efficacy of tolvaptan treatment in reducing the change in eGFR, a renal function test, from baseline to post-treatment follow-up as compared to placebo in patients with ADPKD who tolerate tolvaptan during an initial run-in period.
The trial will also compare overall safety and specifically hepatic safety and compare incidence of ADPKD complications during the trial.
For more information on the trial, please visit: www.clinicaltrials.gov
The FDA issued a Complete Response Letter for tolvaptan to treat ADPKD in August 2013 and requested further information to evaluate the efficacy and safety of the drug.
About Tolvaptan for ADPKD
Tolvaptan is under investigation as a treatment to slow the progression of kidney disease in patients at risk of rapidly progressing ADPKD.2 Tolvaptan was studied in patients with enlarged kidneys who were in chronic kidney disease (CKD) stages 1-3 at initiation of treatment.2 Results were published in the New England Journal of Medicine in December 2012.2
Cyst formation in the kidney has been shown to be dependent on binding of a hormone, arginine vasopressin, to the V2 receptor.2,3 Vasopressin stimulates V2 receptors and activates the cyclic AMP pathway causing acceleration of cyst proliferation and fluid secretion, leading to enlarged, dysfunctional kidneys. 2,3 Tolvaptan is believed to inhibit cyst formation, proliferation and growth as a selective V2 receptor antagonist to block the effects of vasopressin.2 [Read more]
PKD Awareness
From Authint Mail, Kashmir
Kidney disease which was once more common in people aged 65 and above is currently attacking young people. Most individuals are unaware that kidney disease is a silent killer as it show no symptoms until the condition becomes grave. It is very important to detect the symptoms in its initial stages to prevent the situation from becoming critical. Chronic and polycystic kidney disease is often undiagnosed due to unclear signs and symptoms allowing the condition to aggravate further.
Dr Avinash Ignatius, senior consultant nephrologist DaVita, Pune Region, says ‘Frequent urination at night is one of the most common and early symptom of chronic kidney disease and it should not be ignored even though it is harmless. While other symptoms of the disease usually develop at later stage of the disease after the kidney has lost approximately 80% of its function.’
There are some key symptoms of acute and chronic kidney disease that are listed below. One should immediately consult their doctor in case they experience any of these symptoms to avoid disease severity.
Symptoms of Acute Kidney Failure (AKF)
The symptoms of acute kidney disease are same as observed in many other clinical conditions. These clinical signs may be the only issue in the early stage and include –
Headaches
Nausea and Fatigue
Loss of appetite
Itching and dry skin
Swelling in the feet
Generalized ill feeling
Symptoms observed in later stage are -
Bloody stools and prolonged bleeding
Mood swings (in case of elder people)
Pain in ribs and hips
Hand tremors and seizures
Elevated blood pressure
Hiccups and decreased sensation
Symptoms of Chronic Kidney Disease (CKD)
Acute kidney disease symptoms progress soon into the chronic kidney disease symptoms and are generally not observed till the kidney function is affected to a greater extent.
Urinary changes – Kidneys are responsible for formation of urine and thus, any changes in the color, frequency and appearance of urine should not be ignored. Other changes include –
Difficulty in urinating
Appearance of blood in urine
Frequently urinating at night times
Urinating in lesser or greater amounts
Swelling – When kidneys are affected to a greater extent, they are unable to remove extra wastes and fluids from the body. This can cause your face, hands, feet and ankles to swell.
Nausea – When the wastes in the body build up in the blood, it can cause vomiting.
Back or leg pain – Kidney cysts (fluid-packed sacs) from polycystic kidney disease and other kidney problems cause pain in your back, side and legs.
Itching/rash – With kidneys being unable to clear wastes from body, they build up in the blood and cause severe itching or skin rashes.
Fatigue and weakness – When your kidneys get damaged, blood levels of erythropoietin (hormone produced by kidneys that help RBCs carry blood) decrease resulting in anemia. There is low delivery of oxygen to the body cells causing fatigue and weakness.
Metallic taste – The blood levels of urea are increased in kidney disease. This causes the breakdown of urea into ammonia resulting in urine-like breath and also an unpleasant metallic taste in the mouth.
Cold feeling – With low oxygen delivery due to kidney disease and anemia, you may experience cold even when warm. Infection to kidney (pyelonephritis) is also found to cause fever with chills.
Dizziness – With kidney failure relating to anemia, there is an inadequate supply of oxygen to both the brain and the body. This is found to cause issues with concentration, memory-related problems and dizziness.
Shortness of breath – Sometimes, excess fluid can fill up your lungs. Anemia, a common side-effect [Read more]
Gift of Life
A Loveland woman has received a life-saving kidney donation. In January, 9NEWS brought you the story of Phyllis and Gary McCormack. Phyllis had polycystic kidney disease, which caused cysts to form on her kidneys.
Because PKD is genetic, her daughters couldn't donate. Gary has kidney stones and is diabetic, so he couldn't either. Phyllis needed an "altruistic" donor, someone who didn't even know her, but was willing to give her a kidney. To find that person, Gary became a walking billboard. Everywhere he went he wore a special sweatshirt, which read, "KIDNEY NEEDED Save a Live Please Call 970-667-7841."
After our story aired, the McCormack's, along with the American Transplant Foundation, received dozens of phone calls from people, who were interested in donating. Many of them were tested. A few months later, Phyllis got a phone call from University of Colorado Hospital.
"She said, "Phyllis, are you sitting down? And I said, 'no, but I will be.' And, she said, 'We have found a donor for you. Not only have we found a donor for you, its a perfect match,'" Phyllis said.
She immediately called Gary, who was at the gym.
"I said, 'I got my donor.' And he told all of his cronies there. I could hear all of the cheering going on," she said.
Several weeks later, with Gary and their daughters by her side, Phyllis underwent transplant surgery at University of Colorado Hospital. Three days later, she went home.
About a week after surgery, Phyllis met the woman, who gave her a kidney. They hugged for a long time and cried. However, the donor wants to remain publicly anonymous.
Off camera, Phyllis' donor told 9News. "I did this to help somebody, not to get on TV or have my name out there. I just wanted to help somebody," she said. "I could help her and I was the one, for whatever reason."
Phyllis' donor first thought about donating 15 years ago when someone she knew donated a kidney. While she was interested in donating, she realized it was not the right time. However, she never stopped thinking about being a donor someday. [Read more]
No comments:
Post a Comment