Posts Tagged ‘participate in a clinical trial’

Revolutionary Research Underway with First-in-Man Clinical Trial for Hunter Syndrome

Hunter SyndromeAs an academic medical center, many physicians at Emory not only take care of patients on a daily basis, but also partake in research and teaching/training the future generations of physicians.

It’s through discovery that happens during clinical trials and research that allows for new advancements in medicine to be made. Providing the standard of care of is not always enough. New treatment therapies, technology and care delivery techniques must be developed, tested and perfected in order to keep advancing health care.

Not sure what clinical trials are? Check out our blog post that explains Clinical Trails>>

 
Currently happening at Emory is the first-in-man clinical trial, testing a newly developed drug for a rare genetic condition called mucopolysaccharidosis type II (MPS II) or Hunter syndrome.

Hunter syndrome is a hereditary disease in which a critical enzyme is either missing, or there is not enough of it. Without enough of this enzyme, long chains of sugar molecules do not break down properly in the body and accumulate in the organs and tissues and become toxic.

To treat most cases of Hunter syndrome, an enzyme replacement medication is used, but this regimen does not cross into the brain to treat the most severe cases of Hunter syndrome. In this particular trial, Emory researchers are testing whether a man-made enzyme fused with an antibody and injected into the bloodstream will deliver the enzyme therapy into the brain. This is the first time this medication combination has been tested in a human.

The video below features Emory patient, Chris Dutcher. “By testing this new medication for the first time in a human, my hope is to help children with severe Hunter syndrome,” says Dutcher, whose younger brother also has the same disorder.

Chris has the mild version of Hunter syndrome, but his symptoms are still quite serious and involve the lining of the brain and spinal cord, which are not treated by existing medications. He is the first patient to receive the new treatment therapy.

More information about Hunter syndrome and this clinical trial can be found in the Emory News Center. Learn more about clinical trials at Emory Healthcare.

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Understanding Clinical Trials – Part 4: What Questions Should You Ask Before Enrolling in a Clinical Trial?

Clinical Trials QuestionsParticipation in clinical trials is 100% voluntary. While there are many benefits to participating in clinical trials, there can also be risks. If you have been offered a clinical trial, or are considering participating, it is important to talk with your physician before enrolling in one. Your health care team wants to make sure you have all the information needed to make a decision.

Do not hesitate to ask any questions or bring up any issues concerning the trial with the research team associated with the clinical trial. Tips for asking about clinical trials include:

  • Take a family member or caregiver with you for support and for help in asking questions and recording answers.
  • Plan questions ahead of time — you can still ask new questions you think of while you’re there, but it helps to be prepared ahead of time.
  • Write down your questions in advance, to make sure you remember to ask them all.
  • Write down the answers, so that you can review them whenever you want.

The National Institutes of Health (NIH) developed a list of suggestions that may be helpful to review as you think about the questions you may have:

Questions about the Study

  • What is the purpose?
  • Why do researchers think the approach may be effective?
  • Who will fund the study?
  • Who has reviewed and approved the study?
  • How are study results and safety of participants being checked?
  • How long will the study last?
  • What will my responsibilities be if I participate?

Questions about Possible Risks/Benefits

  • What are my possible short-term benefits?
  • What are my possible long-term benefits?
  • What are my short-term risks, such as side effects?
  • What are my possible long-term risks?
  • What other options do people with my disease have?
  • How do the possible risks and benefits of this trial compare with those options?

Questions about Participation and Care

  • What kinds of therapies, procedures and /or tests will I have during the trial?
  • Will they hurt, and if so, for how long?
  • How do the tests in the study compare with those I would have outside of the trial?
  • Will I be able to take my regular medications while in the clinical trial?
  • Where will I have my medical care?
  • Who will be in charge of my care?

Questions about Personal Health Issues

  • How could being in this study affect my daily life?
  • Can I talk to other people in the study?

Questions about Financial Issues

  • Will I have to pay for any part of the trial such as tests or the study drug?
  • If so, what will the charges likely be?
  • What is my health insurance likely to cover?
  • Who can help answer any questions from my insurance company or health plan?
  • Will there be any travel or child care costs that I need to consider while I am in the trial?

At Emory Healthcare, our physicians and researchers want to answer all your questions about clinical trials and research. View some of our frequently asked questions, or hear from our patients who have gone through clinical trials.

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