On Important Paediatric Cancer Clinical Trials

Statistically and clinically significant improvements have been achieved in all major forms of childhood cancers through conduct of well-organised single institution and cooperative group clinical trials which have resulted in sequential and steady improvement in survival rates since the 1960s when curative treatments were first devised. SEER data document that the overall childhood cancer mortality rates have consistently declined throughout the 1975-95 time period.1 Documentation of the...

Study Design For Childhood Cancer Trials

Because childhood cancer is rare and the response to conventional treatment good, most children never experience recurrent disease and are thus Table 7.3. Risk group assignments for intergroup Rhabdomyosarcoma Study Group study V Table 7.3. Risk group assignments for intergroup Rhabdomyosarcoma Study Group study V Favourable orbit eyelid, head and neck (excluding parameningeal), genitourinary (not bladder or prostate) and biliary tract. Unfavourable bladder, prostate, extremity, parameningeal,...

Of Trials In Children With Cancer

Children and adolescents constitute a special vulnerable population of research subjects, often grouped with other special classes, like the mentally retarded, mentally ill and prisoners. There are special federal protections which apply to all research involving children as subjects which are covered by Subpart D of Part 46 of Title 45 of the Code of Federal Regulations (45 CFR 46), requiring that institutional review boards (IRBS) give consideration to the degree of risk, the benefit to child...

Look Into The Future Of Childhood Cancer Research

Despite the progress of the last half century there remain a number of challenges in childhood cancer. The focus of research in certain patient subsets with very high cure rates will be on quality of life endpoints. For example, retinoblastoma is curable in nearly 100 of cases, so preservation of sight and reduction of second malignancies (not survival) are now considered to be the primary goals and endpoints, and trials avoiding enucleation and eliminating external beam therapy are now the...

Clinical Trials In Lung Cancer

Clinical trials have resulted in significant seminal trials which have led to changes in the management of these patients. Those seminal studies in screening, chemoprevention and treatment are outlined. Three US randomised screening studies failed to detect an impact of screening high-risk patients with chest radiographs or sputum cytology on mortality, although earlier stage cancers were detected in the screened groups.3-5 These studies have been criticised for a number of potential...

Defining Outcomes

For any trial, it is crucial to have an a priori hypothesis and a clinically relevant primary outcome on which the power calculation is based. This essential primary step prevents the sacrifice of quality for expediency and discourages opportunistic and inappropriate manipulation of collected data. Outcomes of choice include those that are purely clinical, as well as others which may be patient-centred or economic. The precise nature of the primary and secondary outcomes will depend on the type...

Clinical Trial Methods

Most effect measurements of the respiratory diseases discussed here are influenced to a non-negligible degree by the patients' expectations. One typical example of this is that in some double-blind, placebo-controlled single dose trials measuring bronchodilation, there is an immediate response in lung function also in the placebo group, which probably is due to (false) expectations. The classical methods to avoid expectation bias, blinding and randomisation, are therefore important. A clinical...

Large Number Of Different Skin Diseases

Unlike most other organs that usually count around 50 to 100 diseases, the skin has a complement of 1000 to 2000 conditions and over 3000 dermatological categories can be found in the International Classification for Disease version 9 (ICD-9). This is partly justified by the skin being a large and visible organ. Beside disorders primarily affecting the skin, there are cutaneous manifestations with most of the major systemic diseases (e.g., vascular and connective tissue diseases). The...

Case Study 5fu Plus Leucovorin In Colon Cancer

As is clear, the history of clinical trials in GI cancer is long and has been very successful. As an example illustrating several facets of both the past history of GI clinical trials and issues that will likely be faced again in future studies, here we present a case study of the development, establishment and replacement of what was once the US standard of care for advanced colorectal cancer and, as of 2002, remains the standard for adjuvant stage 3 colon cancer, the 'Mayo Clinic' bolus...

Gynaecology And Infertility

A potential problem that can occur with randomised controlled trials is where the intervention is not targeted at individual patients, but at groups of patients. This can happen where the intervention is an information package for the management of menorrhagia in primary care9 or a clinical guideline for the management of infertility.10 In these studies, randomising patients to receive management using the information package or clinical guidelines would have introduced contamination, since GPs...

Psychiatric Treatments And Their Evaluation The 1950s Onwards

Kopeloff and Cheney's study of removal of focal infections as a treatment for particular forms of mental illness has many aspects of a modern clinical trial, although it is missing that most essential component, random allocation. Kopeloff and Cheney decided themselves in regard to each patient whether they should be operated on or whether they should be a control. It was Fisher who recognised the need for randomisation to treatment groups in medical, biological and agricultural experiments,...

Historical Perspective On Clinical Trials In Breast Cancer

Scientific understanding of the biology of breast cancer has changed radically in the past 50 years. Results of large randomised trials have played a major role in this transition. From the nineteenth century and up into the 1970s, breast cancer was understood to be a local regional disease that spread by direct extension along lymphatic pathways to distant sites. This concept gave rise to the surgical methods promoted by W.S. Halsted14-16 around the turn of the twentieth century, i.e.,...

References

Committee for Proprietary Medicinal Products (CPMP). Clinical investigation of steroid contraceptives in women. The European Agency for the Evaluation of Medicinal Products (2000). 2. Van Look PFA, Perez-Palacios G. Contraceptive Research and Development 1984 to 1994. Oxford Oxford University Press (1994). 3. Rabe T, Runnebaum B. Fertility Control - Update and Trends. Berlin Springer-Verlag (1999). 4. Trussell J, Kost K. Contraceptive failure in the United States a critical review of the...

Clinical Trials Methods

In dental research, the clinical trials methods used mainly follow those developed and adopted in medical research. The basic design principles and considerations are very similar, thus the following discussion on clinical trials methods can be kept short and precise for general areas, with specific areas unique in dentistry discussed in more detail. A few references on clinical trial methodology and some from the dental literature are recommended for general reading.1417 As with the...

Adaptive Designs Of Clinical Trials

Adaptive designs have the dual goals of efficient learning from all relevant results and effective treatment of patients. They are more flexible than conventional designs, and have application in all phases of drug development. Such designs can be implemented using Bayesian methodology as a means to incorporate new information into the trial design. Designs of clinical trials for breast cancer are usually static in the sense that the sample size and any prescription for assigning treatment,...

Phase Iii Studies

Most drugs for obstructive airway diseases are given for maintenance treatment, and the main point to document is the level of disease control of the proposed treatment. At the same time it is important to document the adverse event profile, since most of the drugs in this therapeutic area are considered very safe, and safety must not be an issue of a new drug. Thus the pivotal confirmatory trials for the airway diseases asthma, COPD and rhinitis are parallel group, diary card studies typically...

Skin Disorders And Clinical Trial Methods Adapting Study Design To Setting And Disease

As for other disciplines, the last few decades have seen an impressive increase in the number of clinical trials carried out in dermatology. However, there are indications that the upsurge of clinical research has not been paralleled by a refinement in clinical trial methodology and the quality of randomised control trials (RCTs) in dermatology falls well below the usually accepted standards.1315 In this section we would like to mention some issues which deserve special attention when designing...