Download What Are Clinical Trials?

Cancer Clinical Trials:
The Basics
What Are Cancer Clinical Trials?
• Research studies
involving people
• Try to answer
scientific questions and
find better ways to
prevent, diagnose, or
treat cancer
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Why Are Cancer Clinical Trials
Important?
• Cancer affects all of us
• Each year in the U.S.A:
– More than half a million
people are expected to die of
cancer — more than 1,500
people a day
– 1 of 4 deaths is from cancer
– More than 1 million new
cancer cases are expected to
be diagnosed
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Why Are Cancer Clinical Trials
Important?
• Clinical trials translate
results of basic scientific
research into better ways to
prevent, diagnose, or treat
cancer
• The more people that take part, the faster we can:
– Answer critical research questions
– Find better treatments and ways to prevent cancer
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Do Many People Participate in
Cancer Clinical Trials?
• Only 3 percent of U.S.
adults with cancer
participate in clinical
trials
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Types of Cancer Clinical Trials
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Treatment trials
Prevention trials
Early-detection trials/screening trials
Diagnostic trials
Quality-of-life studies/supportive
care studies
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Clinical Trial Phases
Phase 1 trials
• How does the agent affect the human body?
• What dosage is safe?
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Clinical Trial Phases
Phase 2 trials
• Does the agent or intervention have an effect
on the cancer?
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Clinical Trial Phases
Phase 3 trials
• Is the new agent or intervention (or new use of
a treatment) better than the standard?
• Participants have an equal chance to be
assigned to one of two or more groups
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Randomized Trials
Participants have an equal chance to be assigned
to one of two or more groups:
• One gets the most widely accepted treatment
(standard treatment)
• The other gets the new treatment being tested,
which researchers hope and have reason to
believe will be better than standard treatment
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Randomization
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Why Is Randomization
Important?
• So all groups are as alike as possible
• Provides the best way to prove the
effectiveness of a new agent or intervention
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Cancer Treatment Trials
• What new treatments
can help people who
have cancer?
• What is the most
effective treatment for
people who have
cancer?
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Cancer Treatment Trials
Placebos are almost never used:
• Placebos are used only when no standard
treatment exists
• Patients are told of this possibility before
deciding to take part
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Cancer Prevention Trials
• Evaluate the
effectiveness of ways
to reduce the risk of
cancer
• Enroll healthy people
at high risk for
developing cancer
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Cancer Prevention Trials
• Action studies
(“doing something”)
• Agent studies
(“taking something”)—
also called
“chemoprevention
studies”
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Chemoprevention Trials
• Phase 3 chemoprevention trials compare a
promising new agent with either a:
– Standard agent
– Placebo
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Clinical Trial Protocol
• A recipe or blueprint
• Strict scientific guidelines:
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Purpose of study
How many people will participate
Who is eligible to participate
How the study will be carried out
What information will be gathered about participants
Endpoints
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Benefits of Participation
Possible benefits:
• Patients will receive, at a minimum, the best
standard treatment
• If the new treatment or intervention is
proven to work, patients may be among the
first to benefit
• Patients have a chance to help others and
improve cancer care
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Risks of Participation
Possible risks:
• New treatments or interventions under study
are not always better than, or even as good
as, standard care
• Even if a new treatment has benefits, it may
not work for every patient
• Health insurance and managed care
providers do not always cover clinical trials
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Patient Protection
• There have, unfortunately,
been past abuses in patient
protection
• Federal regulations ensure
that people are told about
the benefits, risks, and
purpose of research before
they agree to participate
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How Are Patients’ Rights
Protected?
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Informed consent
Scientific review
Institutional review boards (IRBs)
Data safety and monitoring boards
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How Are Patients’ Rights
Protected?
Informed consent:
• Purpose
• Procedures
• Risks and potential
benefits
• Individual rights
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How Are Patients’ Rights
Protected?
• Scientific review
• Institutional review boards (IRBs) are required
by Federal law for trials that are:
– Federally funded
– Subject to FDA regulation
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How Are Patients’ Rights
Protected?
Data and safety monitoring boards:
• Ensure that risks are minimized
• Ensure data integrity
• Stop a trial if safety concerns arise or
objectives have been met
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Why Do So Few Cancer Patients
Participate in Clinical Trials?
Sometimes patients:
• Don’t know about clinical trials
• Don’t have access to trials
• May be afraid or suspicious of research
• Can’t afford to participate
• May not want to go against physician’s wishes
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Why Do So Few Cancer Patients
Participate in Clinical Trials?
Doctors might:
• Lack awareness of appropriate clinical trials
• Be unwilling to “lose control” of a person’s
care
• Believe that standard therapy is best
• Be concerned that clinical trials add
administrative burdens
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NCI Information Resources
• NCI Web site
www.cancer.gov
• Cancer Information Service
– 1-800-4-CANCER
– TTY- 1-800-332-8615
– www.cancer.gov/cis
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IOWA ONCOLOGY
RESEARCH ASSOCIATION
Iowa Oncology
Research Association
• Established in 1977 as CGOP
• Became a member of NCCTG in 1978
• Designated a CCOP in 1983
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Affiliate Sites
• Mason City
• Ottumwa
• Ames
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Clinical Trials
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Chemotherapy
Radiation Therapy
Immunotherapy
Surgery
Correlative Studies
Cancer Control
Cancer Prevention
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Cooperative Group
Memberships
• North Central Cancer Treatment Group
• Eastern Cooperative Oncology Group
• National Surgical Adjuvant
Breast & Bowel Project
• Southwestern Oncology Group
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Protocol Development
• Protocol concept developed by cooperative
group committees
• Often distributed to participating investigators,
CRAs, nurses for peer review
• Submitted to NCI for review
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Implementation Of Study
• Distributed to affiliate sites via weekly emails
• Optional participation
• Factors affecting local participation
– Eligibility criteria
– Competing studies
– Test requirements
• Local IRB review
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Patient Recruitment
• Treatment and control studies – Oncology
patients referred by local physicians
to IORA
• Cancer prevention studies – Various
recruitment efforts utilized
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Drug Procurement
• Drugs ordered from sponsoring cooperative
group
or
• Drugs ordered from Pharmaceutical
Management Branch (PMB) of NCI
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Randomization /
Registration
• Fax
• Online
All randoms / registrations require at minimum a
completion of eligibility criteria checklist
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Quality Assurance
• Pathology review
• Radiation therapy review
• Surgery review
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Quality Assurance
Continued
• Data & Safety Monitoring Policies
• Data & Safety Monitoring Board (DSMB)
• Site Audits
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Adverse Event Reporting
• Common Toxicity Criteria (CTC)
• Adverse Event Expedited Reporting
System (AdEERS)
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BRAIN TUMORS
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RECURRENT BRAIN
TUMORS
• NCCTG N0272 Phase II Trial of STI=571 in
Treatment of Recurrent Oligodendroglioma & Mixed
Oligoastrocytoma
• NCCTG N0572 Phase I/II Trial of Sorafenib & CCI779 in Recurrent Glioblastoma
• NCCTG N0779 Phase II Study of Vorinostat
(SAHA) in Combination with Bortezomib (PS-341)
in Patients with Recurrent Glioblastoma Multiforme
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METASTATIC BRAIN
TUMORS
• NCCTG N0574 Phase III Randomized Trial of
the Role of Whole Brain Radiation Therapy in
Addition to Radiosurgery in Patients with 1-3
Cerebral Metastases
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BREAST CANCER
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ADJUVANT TREATMENT
• CALGB C40101 Phase III, Randomized, 2 x 2
Factorial Study of Cyclophosphamide & Doxorubicin
(CA x 4 Cycles) vs Paclitaxel (4 Cycles) as Adjuvant
Therapy for Breast Cancer in Women with 0-3
Positive Axillary Nodes
• ECOG E5103 A Double-Blind, Phase III Trial of
Doxorubicin & Cyclophosphamide Followed by
Paclitaxel with Bevacizumab or Placebo in Patients
with Lymph Node Positive & High-Risk Lymph
Node Negative Breast Cancer
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ADJUVANT TREATMENT
• ECOG PACCT-1 Program for the Assessment of
Clinical Cancer Tests: Trial Assigning
Individualized Options for Treatment
• SWOG S0221 Phase III Trial of Continuous
Schedule of AC + G vs Every 2 Week Schedule AC,
Followed by Paclitaxel Given Either Every 2 Weeks
or Weekly for 12 Weeks as Post-Operative Adjuvant
Treatment in High Risk Node Negative or Node
Positive Breast Cancer
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ER / PR POSITIVE
• NSABP B-42 A Clinical Trial to Determine the Efficacy of 5
Years of Letrozole Compared to Placebo in Patients
Completing 5 Years of Hormonal Therapy Consisting of an
AI or Tamoxifen Followed by an AI in Prolonging DiseaseFree Survival in Postmenopausal Women with Hormone
Receptor Positive Cancer
• CALGB C40503 Endocrine Therapy in Combination with
Anti-VEGF Therapy: Randomized, Double-Blind, PlaceboControlled Endocrine Therapy Plus Bevacizumab for Women
with Hormone Receptor Positive Advanced Breast Cancer
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WHOLE VS PARTIAL
BREAST IRRADIATION
• NSABP B-39 Randomized Phase III Study of
Conventional WBI vs PBI for Women with
Stage 0, I or II Breast Cancer
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HER2 Positive
• NCCTG N063D ALTTO: Adjuvant Lapatinib
and/or Trastuzumab Treatment Optimization
Study: Randomized, Open-Label, Phase III
Study of Adjuvant Lapatinib, Trastuzumab,
Their Sequence & Their Combination in
Patients with HER2 Positive Primary Breast
Cancer
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METASTATIC /
RECURRENT
• ECOG E1104 Phase I/II Study of SAHA in
Combination with Herceptin in Patients with
Advanced Metastatic and/or Local Chest Wall
Recurrent HER2 Amplified Breast Cancer
• ECOG E1105 Randomized Phase III Double-Blind,
Placebo-Controlled Trial of First-Line Chemo &
Trastuzumab With or Without Bevacizumab for
Patients with HER2 Overexpressing Metastatic
Breast Cancer
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METASTATIC /
RECURRENT
• NCCTG N0539 Phase II Trial of Fulvestrant &
Bevacizumab in Patients with Metastatic Breast
Cancer Previously Treated with an Aromatase
Inhibitor
• SWOG S0500 Randomized Phase III Trial to Test the
Strategy of Changing Therapy vs Maintaining
Therapy for Metastatic Breast Cancer Patients Who
Have Elevated Circulating Tumor Cell Levels at First
Follow-up Assessment
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OVARIAN FUNCTION
SUPPRESSION
• IBCSG 2402 Ovarian Function Suppression:
Exemestane as Adjuvant Treatment for
Premenopausal Women with Endocrine Responsive
Breast Cancer
• CALGB C40302 Endocrine Therapy With or
Without Inhibition of EGF & HER2 Growth Factor
Receptors: Randomized, Double-Blind, PlaceboControlled Phase III Trial of Fulvestrant With or
Without Lapatinib for Post-Menopausal Women With
Hormone-Receptor Positive Advanced Breast Cancer
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