CLINICAL TRIALS LEAD TO CURES
A clinical trial is a research study designed to promote medical knowledge. Investigators, typically medical professionals, initiate clinical trials in order to test new therapeutic interventions for a particular disorder or condition. These interventions may involve drugs, operative procedures, or lifestyle changes. Clinical trials through the Weill Cornell/NewYork-Presbyterian Leukemia Program are primarily investigating new pharmaceutical agents. These trials offer our patients the opportunity to gain access to new treatments before they are publicly available.
To learn about current trials in the Leukemia Program, visit the following links:
- Myeloproliferative neoplasms
- Myelodysplastic syndromes
How do clinical trials work?
In order for a new drug to receive approval from the Food and Drug Administration (FDA), a federal agency of the U.S. Department of Health and Human Services, it must first go through consecutive clinical trial phases. Upon successful completion of these phases, the drug may be marketed for medical use.
- Phase 0 trials initiate the first human exposure to the drug. This is based on research in the laboratory. The drug is administered at a very low dose to a small number of subjects in order to assess how it interacts with the body.
- Phase 1 trials are intended to assess the safety of the drug at various dosages. The drug is again administered to a small group of subjects, and researchers are looking for any side effects that may emerge.
- Phase 2 trials recruit a larger group of participants in order to evaluate efficacy or effectiveness of the drug. Safety is continuously evaluated and additional side effects are identified.
- Phase 3 trials further evaluate safety and efficacy of the drug in a large subject group in order to confirm results from the previous phases and to compare the drug to existing treatments.
The duration and timeline of each phase varies according to the individual trial. It may take several years for a drug to complete all phases. It is important to note that while the drug proceeds through these phases, the participants do not. That means that each phase represents a distinct trial with its own protocol and timeline.
Clinical trials are managed by a principal investigator and his/her research team, which may include doctors, nurses, social workers, and other research and healthcare professionals. The trials are often sponsored by organizations such as pharmaceutical companies, academic medical centers, and federal agencies. Access to medical information about study participants is restricted to members of the trial research team and cannot be shared with outside parties without the participant’s consent.
Participation in a clinical trial is completely voluntary and up to the discretion of the patient and his/her family and physician. The treating physician will often be the best source of information about available trials, and may recommend participating in one in lieu of or in addition to standardized treatment, depending on the patient’s individual case.
Before enrolling in a clinical trial, patients must be assessed for specific eligibility criteria, which are outlined in the study protocol. Inclusion criteria are factors that allow participation in the trial, whereas exclusion criteria are factors that disqualify participation. The criteria are based on characteristics including, but not limited to, age, gender, medical history, treatment history, and disease type and stage.
If an individual is eligible for and wishes to enroll in a trial, he/she will be required to sign an informed consent form that details important information about participating in the trial. The informed consent process ensures that the participant understands the risks, potential benefits of, and alternatives to the study. Upon signing the form, participants remain free to withdraw from the study at any time, for any reason.
For more general information about clinical trials, visit www.clinicaltrials.gov.