From PKD Foundation
Clinical Studies
For drugs and therapies to be developed for PKD, clinical studies must be conducted. Patients play a key part. Learn how you can help unlock the secrets of PKD and find a treatment by participating in a study.
Participate in PKD Clinical Studies to Discover Treatments and Improve Your Health
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Types of Clinical Studies
The two types of studies currently underway for PKD patients include observational studies and clinical trials.
Observational studies monitor and record various clinical and/or subjective factors over a period oftime, like blood pressure or quality of life, to better understand how the disease progresses. Involvement includes activities such as having conversations with researchers and physicians, or recording daily events in journals. No drugs are given or other interventions are made to alter the course of the disease. An example of an observational study is the Consortiumfor Radiologic Imaging Studies of Polycystic Kidney Disease (CRISP).
Clinical trials monitor and record various clinical and/or subjective parameters by testing experimental treatments, devices or combinations of drugs that may alter the course of the disease. Participants receive specific interventions which may be medical products such as drugs or devices; procedures; orchanges to participants’ behavior such as diet. The purpose of a clinical trial is to test a new drug or other intervention for safety and effectiveness in treating the disease in question before it can be prescribed for patients. An example of a clinical trial is theTolvaptan TEMPO ¾ Trial.
According to clinicaltrials.gov, an intervention is a processor action that is the focus of a clinical study. This can include giving participants drugs, medical devices, procedures,vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches such as surveys, education and interviews.
Phases of a Clinical Trial
A new drug to treat PKD must move through each stage before it can be reviewed for approval by the FDA.
Phase I – The new drug is tested for safety and side effects in a small number of healthy volunteers withPKD.
Phase II – The new drug is tested for safety, doseranging and preliminary effectiveness in a small number of volunteers.
Phase III – The new drug is tested in a large number of volunteers with PKD to establish effectiveness, monitor side effects and compare results with currenttreatments. The data collected during the clinical trial is analyzed and then submitted to the FDA for regulatory review, which can take one to three years. If there is an intent to apply for an NDA (new drug application), it is communicated to the FDA and shapes the design of the study. Once it is approved, the new drug can be prescribed by physicians to treat PKD.
Phase IV – These studies are done after the drug has been approved by the FDA and it is in use (considered a post-marketing study).Clinical Trials Awareness Program (CTAP)
Perhaps the single greatest impediment to clinical trial patient participation/recruitment begins with awareness. We believe it is our goal to address this challenge and launched the Clinical Trials Awareness Program, an aggressive program to help create awareness amongst patients and families and speed up clinical trial recruitment. The program is focused on educating patients about current studies so they can make educated decisions about participating. The goal is to simplify the process of finding clinical studies for PKD patients in their geographic area by sending Accelerating Clinical Trials (ACT) Alert emails about studies that are being conducted.
Participate in a Clinical Trial
To participate in a clinical trial, you will need to have a formal diagnosis of PKD made by a physician. Learn more about the implications of a PKD diagnosis for yourself or your children.
By participating in a clinical trial, you can:
Play a more active role in your own health care.
Have access to new treatments before they are available to the public.
Contribute to the development of treatments for PKD.
Make things better for the next generation of those with PKD.
PKD Research Funding
From Targeted News Service, by MARY GRACE DEJUCOS
To participate in a clinical trial, you will need to have a formal diagnosis of PKD made by a physician. Learn more about the implications of a PKD diagnosis for yourself or your children.
By participating in a clinical trial, you can:
Play a more active role in your own health care.
Have access to new treatments before they are available to the public.
Contribute to the development of treatments for PKD.
Make things better for the next generation of those with PKD.
PKD Research Funding
From Targeted News Service, by MARY GRACE DEJUCOS
WASHINGTON, July 22 -- The U.S. National Institutes of Health has announced that it expects to award a maximum of four discretionary grants to support research for polycystic kidney disease.
For more information, contact NIH OER Webmaster, FBOWebmaster@od.nih.gov. NewsFocusCode FedGrants NWNAgrants federalgrants TNSFG140721
PKD Fundraising
From Topeka Capital Journal, Topeka, Kansas
Bass tourney to raise funds for kidney transplant
A catch-and-release bass tournament and chili cookoff will be from 8 a.m. to 3 p.m. Aug. 2 in the Coeur d’ Alene Shelter at Melvern Lake. Other activities include face painting and a bake sale.
Proceeds will benefit the Midwest/West Kidney Transplant Fund in honor of Tom Thurston, who has suffered from polycystic kidney disease since birth. Over the past few years, his kidneys have become increasingly enlarged and recently began to shut down. He is awaiting a kidney transplant.
Even with insurance, Thurston’s out-of-pocket costs will be about $2,000 a month for the rest of his life. He is working with the nonprofit HelpHOPELive to raise funds to cover uninsured medical costs associated with the transplant.
Registration fees are $50 for the bass tournament and $20 for the chili cookoff. The event is free for spectators.
Donations to Thurston’s fundraising campaign can be made online at helphopelive.org or by calling (800) 642-8399. Checks can be mailed in honor of Thurston to HelpHOPELive, 2 Radnor Corporate Center, 100 Matsonford Road, Suite 100, Radnor, Pa. 19087.
From TribLive, Fayette, PA
The fourth annual Walk for PKD in Memory of Chuck Bryner will take place at Yough River Park on Aug. 2. Polycystic Kidney Disease is one of the most common life-threatening genetic diseases. There is no treatment or cure. The Walk for PKD is the PKD Foundation's largest annual fundraising event; these funds make up a third of the group's annual budget for research. Join the walk as a participant, volunteer or donor. Questions or comments can be directed to Janet Bryner at 724-434-4821.
From abc30 News, Fresno, CA
NEW HOPE FOR DIALYSIS: BIOENGINEERED BLOOD VESSELS
Nearly 400,000 Americans are on dialysis for kidney failure. The treatment uses a special machine to filter toxins from the blood and often requires a graft to connect an artery to a vein to speed blood flow. But in many patients, synthetic grafts lead to infection and frequent hospitalizations. Now a first-of-its-kind bioengineered blood vessel is changing that.
William Alexander has suffered with kidney failure for 15 years. Dialysis keeps him alive.
"(It's not) like you can't do it," Alexander told ABC30. "You've got to have dialysis to live."
However, his arm tells the story of failed blood vessel grafts used to help clean his blood.
"It's disfiguring," Jeffrey Lawson, MD, PhD, Professor of Vascular Surgery and Pathology at Duke University Medical Center, Durham, N.C., told ABC30.
It's a reality Dr. Lawson says most patients face.
"I tell many of my patients they can expect to have a procedure related to dialysis at least once a year," Dr. Lawson explained.
Now, a new bioengineered blood vessel using donated human cells could change that.
"We'll be able to reduce the number of interventions they have to have," Dr. Lawson said.
At the lab, Dr. Shannon Dahl says donated cells are placed in a bioreactor and cultured for two months.
"So we're growing the cells and we're putting the bioreactor parts together," Shannon L.M. Dahl, PhD, Vice-President, Scientific Operations, cofounder, Humacyte, Inc., Durham, N.C., told ABC30.
Once the vessel is formed, it's cleansed of the donor cells, leaving a collagen structure that the body readily accepts as its own.
"It then becomes your blood vessel as your body grows into it, which is very, very exciting," Dr. Dahl explained.
Alexander had the bioengineered vessel placed in his right arm eight months ago.
"I don't have any trouble and it's doing good, and I'm glad it's doing good," Alexander said.
If you would like more information, please contact:
Sarah Avery
Duke Medicine News and Communications
Office: 919-660-1306
Cell: 919-724-5343
sarah.avery@duke.edu
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