Patient participation in clinical trials

Clinical trials are crucial to help doctors and scientists understand how to safely treat a particular condition, to evaluate new treatments and to test drug safety and efficacy. They have an important role in every step of managing a condition with different clinical trials helping with prevention, diagnosis, treatments and follow-up support.

However, accessing and participating in clinical trials can feel like an enormous and stressful task for patients and caregivers. Clinical trials are still poorly understood by the public and often have a negative connotation possibly due to patients’ misconceptions. There are numerous resources to help with the search for clinical trials, however, sorting through them and trying to understand the science jargon can be overwhelming. Patients must often conduct their own research between medical appointments. Initiating a search on the clinical trials raises questions for patients: How should you search for your condition? What are the appropriate search terms? Do you miss anything in the results if you choose one term over another? There is also concern that the clinical trial information available for patients is not clear and fit-for-purpose. Patients also frequently experience fear, stigma and worry about the burden that may be associated with participating in a clinical trial.

A strong patient education agenda should be a priority in every pharmaceutical product development plan. Doctors should be able to explain and discuss clinical trials with their patients and patients should always consult their doctors about participating in a clinical trial. It is essential that a more transparent view of clinical trials, how they work and how they are carefully designed is put in place worldwide to encourage patients to become willing trial participants with full understanding of the advantages and drawbacks associated. A recent study by Moorcraft et al. (2016) showed that 48–50 % of patients approached for a clinical trial would have liked more detailed information on the study procedures/drugs than was provided.

Rare diseases, such as Birt-Hogg-Dube (BHD) Syndrome, face the additional major challenge of recruiting enough suitable patients because there are so few patients that recruitment must occur from a wide geographical area.

At Clinicaltrials.gov there are currently four BHD Syndrome-related open studies, three in the US and one in Europe. These studies are currently recruiting participants:

  • Everolimus Therapy in People with  BHD-Associated Kidney Cancer or Sporadic Chromophobe Renal Cancer – aiming to assess if everolimus is safe and effective in people with BHD-associated kidney cancer or sporadic chromophobe renal cancer.
    • United States, NCI, NIH in Maryland
    • Eligibility: People ages 18 and over with BHD-associated kidney cancer or advanced sporadic chromophobe renal cancer.
  • Genetic Analysis of BHD Syndrome and Characterization of Predisposition to Kidney Cancer – aiming to investigate the genetic cause ofBHD syndrome and the relationship of this disorder to kidney cancer.
    • United States, NCI, NIH in Maryland
    • Eligibility: Patients with known or suspected BHD Syndrome and their family members of any age will be recruited worldwide. Patients with at least one histologically confirmed fibrofolliculomas; or with clinical evidence of multiple skin papules and a personal or family history of spontaneous pneumothorax or kidney cancer; or with spontaneous pneumothorax and skin papules or kidney cancer and a positive family history of spontaneous pneumothorax, skin papules or kidney cancer; or a relative with a confirmed diagnosis of BHD, or renal tumour histology consistent with BHD.
  • Prevalence of Spontaneous Pneumothorax in BHD – aiming to assess the prevalence of BHD among patients with spontaneous pneumothorax.
    • Netherlands, Rijnstate
    • Eligibility: People ages 18 and over treated in Rijnstate hospital for primary spontaneous pneumothorax and with informed consent.
  • Clinical Manifestations and Molecular Bases of Heritable Urologic Malignant Disorders – aiming to investigate the clinical manifestations and molecular genetic defects of heritable urologic malignant disorders.
    • United States, NCI NIH in Maryland
    • Eligibility: Individuals and family members all ages with a suspected or an established diagnosis of Von Hippel-Lindau (VHL) syndrome or hereditary papillary renal carcinoma (HPRC), Type I; or of an inherited urologic malignancy in which the disease gene is not yet known, specifically hereditary forms of Type II papillary renal cancer, clear cell renal carcinoma, renal oncocytoma, chromophobe renal carcinoma or BHD syndrome; or individuals and family members who have urologic malignant diseases of suspected, but not proven genetic etiology.

Let us know what is your opinion on clinical trials. Are you currently enrolled on any BHD clinical trial? What are your major concerns? You can find more information about the process of clinical trials here.

  •  Moorcraft SY, Marriott C, Peckitt C, Cunningham D, Chau I, Starling N, Watkins D, & Rao S (2016). Patients’ willingness to participate in clinical trials and their views on aspects of cancer research: results of a prospective patient survey. Trials, 17 PMID: 26745891

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