Usually, the primary goal of a phase I study is to test the safety of a new drug at different doses and to identify the maximum tolerated dose.
In these studies the study doctors will carefully monitor your vital organs for signs toxicity and will measure how long the new study drug remains in the blood, at what concentration.
Sometimes phase I studies are done to test an approved drug in combination other drugs for the first time - as this use can change the safety of the drug.
Generally, but not always, eligibility in a phase I trial is limited to patients with very advanced disease that is resistant to standard treatments.
All clinical trials involving human subjects are vetted by the FDA,
other scientists, institutional review boards, which often have consumer representation.
It was noted at Vail this summer by a prominent expert that the safety record
for phase I studies are better than for phase II studies.
An advocate's perspective on phase I trials:
My assumption is that most patients enroll in phase I trials with a hope to benefit clinically.
... However, there are at least 2 issues with this expectations, from the perspective of ethicists and those who develop and review informed consent documents, these being:
1) that the track record for achieving this goal is very low, and
2) these are dose-finding studies - for the purpose of defining the maximum tolerated dose.
Yet, the dose-finding study of new drugs is the only way forward and the exciting opportunities we see today for progress - particularly for lymphoma - would not be possible if patients did not enroll in such studies.
My perspective is that the chance to benefit from early phase studies (while low) is higher for lymphoma than for other advanced cancers, because blood cell cancers tend to be more treatment sensitive and can be effectively managed and sometimes cured even at advanced stages.
Advocates appreciate the need to test new drugs and that phase I dose-finding studies is the only way to do this appropriately - with relatively low risk. In phase I studies the dose of the drug starts low and is increased slowly in a step-wise fashion - most often in different groups of patients.
Intra-patient Dose Escalating Phase I Trials?
Sometimes, but not very often, the dose in a phase I study is increased within the same group of patients. This is called an intra-patient dose escalation, or a dose titration, study. This design ensures that patients who enroll will not receive sub-clinical doses of the study drug. However, increasing the dose of the study drug in the same patient also increases the risk of toxicity. The most appropriate design could depend on the mechanism of action of the drug and on the expectations of risk based on preclinical information or the toxicity in a similar class of drugs.
Each trial can be unique in its risk/benefit profile ... so it's not possible to have hard and fast principles on which approach is ethical or preferred by candidates for trials. This is why it's important that patient advocates are included (and actively involved) in the design and vetting of clinical trials - so we can inform investigators about patient preferences on such difficult questions. This is why advocates must also be active in their community support venues.
Finding Phase I Lymphoma or CLL Trials
Searching for phase I trials is a good way to keep current with the direction of research as this is where you will find what novel agents are in development.
FIND: Phase I studies for Lymphoma OR CLL: ClinicalTrials.gov
For lymphoma the good news is that this is crowded field. The bad news being ... that it's a crowded field with a limited pool of participants.
Questions for the patient community
Your reply will be emailed to with your answer in the subject line.
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I would enroll in a phase I trial ONLY if there was a chance to benefit or
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Should intra-patient dosing be considered and often used in phase I studies? or
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Do you want to be informed about new phase I trials? or
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Use of this information
Your replies will be used for no other purpose than to inform PAL about general patient preferences in respect to the study of new drugs for lymphoma. This will help us to comment with more authority regarding patient preferences in the design of NCI-funded trials.
Karl Schwartz
Patient Advocate
