بسم الله الرحمن الرحيم جامعة أم درمان الإسلامية كلية الطب و العلوم الصحية - قسم طب المجتمع مساق البحث العلمي / الدفعة 21 Basics of Clinical Trials

A clinical trial is a research study in human volunteers to answer specific health questions. Carefully conducted clinical trials are the safest and fastest scientific way to: – Test new treatments; – Test new combinations of drugs; – Test new approaches to surgical procedure or radiation, medical device, or of nutrition or behavioral changes in people; In order to find out if is safe and effective for people to use to improve health.

Some studies are done in different populations, such as: In children, in whom the treatment was not previously tested; Or to compare already existing treatments to determine which is better; Or to study different ways to use the standard treatments (=current, approved treatments) So, they will be more effective, easier to use, and/ or to decrease side effects; The overall purpose of a clinical trial is to learn, not to treat patients.

Clinical Trials Are conducted according to a plan called a protocol. The protocol describes: what types of patients may enter the study, schedules of tests and procedures, drugs, dosages, and length of study, as well as the outcomes that will be measured. Each person participating in the study must agree to the rules set out by the protocol.

Kinds of clinical trials 1- Prevention trials: look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vitamins, vaccines, minerals, or lifestyle changes. 2- Diagnostic trials: (for new techniques) to find better tests for diagnosing a particular disease.

3- Screening trials: (for new techniques) to test the best way to detect certain diseases or health conditions; 4- Treatments trials: to test new treatments or new ways to use existing treatments; 5- Quality of Life trials (or Supportive Care trials): options for improving the quality of life for individuals with a chronic illness.

Who can contribute in the clinical trials? It is important to test drugs and medical products in: 1- People whom are meant to be helped. 2- Ensure that a variety of people of different ages, races, ethnic groups, and genders are included in clinical trials (as people may respond differently). 3- People who have used standard (approved) treatment options; but either did not work for them, or unable to tolerate certain side effects. So clinical trials may provide another option when standard therapy has failed. 4- People who want to contribute to the advancement of medical knowledge.

Possible Risks There are generally known & unknown risks associated with clinical trials, such as: 1- There may be unpleasant, serious, or life- threatening side effects resulting from the treatment; 2- The treatment may not be effective for some participants; 3- The protocol may require more of the participant's time and attention than a standard treatment. (To visit the study site on a regular basis, be subjected to additional tests, get more treatments than are normally necessary, stay in the hospital or follow complex dosage requirements, etc....)

Phases of Clinical Trials After a treatment is tested in the laboratory, it can go to human testing. There are four phases of human testing: Phase I: Researchers test a new treatment in a small group of healthy volunteers (20-80) for the first time to test its safety, identify the maximum tolerated dose, find a safe dosage range and identify side effects. Phase II: The treatment is given to a larger group of patients ( ) with disease who might benefit from the drug, to see if it is effective, to evaluate its safety and to gather information regarding safe dose range.

Phase III: The treatment is given to large groups of patients (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments and collect information that will allow the drug or treatment to be used safely. Phase IV: Trials are carried out in the general population after the new drug has been approved for use, investigators are looking for additional information, including the treatment's risks, benefits, and optimal use. Phase V: Trials may be conducted to determine better dosing guidelines, new formulations, effects on different populations or new indications.

What is informed consent? Informed consent: is the process of learning the key fact about the clinical trial, it gives complete information about why the trial is being done, process and how long will it take, benefits and risks of a study, and is written clearly, so participants can understand it.

Methodology 1. Ethical approval and informed consent. 2. Inclusion and exclusion criteria: clinical trials have guidelines about who will participate, using Inclusion and exclusion criteria. Inclusion criteria: are the factors that allow one to participate in the trial; Exclusion criteria: are the factors that disallow one to participate in the trial; These criteria are based on factors such as: age, gender, type and stage of diseases, previous treatment & other medical conditions; These criteria help to reduce the amount of variation in the study, without threatening the scientific integrity of the trial, by removing medical variations that might complicate analyzing the results.

3- Placebo: The control group receives dummy treatment which is indistinguishable from the treatment under study. A low-dose group: for the situation when a placebo group can not be used because of ethical issues.

4- Methods of Blinding a- In a single-blinded trial, only the patient is unaware of the treatment; b- Double-blinded means that neither the patients nor the investigator is aware of the treatment. (to prevent assessment and response biases). However, in several fields, such as surgery, it may be impossible to run a double-blinded study. c- In a triple-blinded trial, patient, investigator and data-monitoring body are unaware of the treatment group; While in open trials, the investigators and patients know what treatment each patient takes.

5- Evaluation and endpoint measurements. 6- Protocol deviations.

Protocol of a clinical trial includes the following:- Protocol of a clinical trial includes the following: 1- Definition 2- Importance 3- Format: (a) Title page;(b) Background and hypothesis; (c) Study design;(d) Materials and methodology; (e) Data collection and handling;(f) Statistical analysis; (g) References;(h) Summary.

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