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
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
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
Generally, but not always,
eligibility in a phase I trial is limited to patients with
very advanced disease that is resistant to standard
All clinical trials involving
human subjects are vetted by the FDA,
other scientists, institutional review boards, which often have
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.
perspective on phase I trials:
My assumption is that most
patients enroll in phase I trials with a hope to benefit
... 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
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.
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.
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
will be emailed to
Support@lymphomation.org with your answer in the subject
I would enroll in a phase
I trial ONLY if there was a chance to benefit
dosing be considered and often used in phase I studies?
Do you want to be
informed about new phase I trials?
Use of this
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.