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Phases of a Clinical Trial

Clinical trial treatments typically consist of several phases, with each phase focused on a different purpose. Most of the time, when you take part in a clinical trial, you will only be in that one phase of the study. Treatments move through the phases, but patients do not.

  • Phase I: Testing if a new treatment is safe. Doctors look for the best way to administer the treatment and for signs that cancer responds to the treatment 
  • Phase II: Testing if a specific type of cancer responds to the new treatment
  • Phase III: Testing if a new treatment is better than a standard cancer treatment
  • Phase IV: Examining long-term benefits and side effects

Phase I Trials

The goal of a Phase I trial is to determine the safety of the new treatment, learn the best way to administer the treatment (orally, intravenously, etc.) and to see whether or not the cancer responds.

Phase I trials usually include 15 to 30 patients, divided into small groups called cohorts. When the first cohort receives a dose of the new drug, doctors may collect blood or urine samples to measure the drug levels.

If no severe side effects present themselves, the next cohort will receive a higher dose of the same drug. This continues with each new cohort until the best dose is determined. With each increasing dose, doctors test each patient to see if he or she is responding to the protocol treatment. If the doctors find that the treatment is safe, then the trial will move forward to be studied in a Phase II trial.

The average length of a Phase I clinical trial can be relatively short due to it being an introductory phase.

Phase II Trials

Typically, a Phase II trial has less than 100 patients. While the main goal is to see if the treatment works, doctors still closely watch for side effects. If the new treatment works, doctors may go on to study it in a Phase III trial.

The average length of a Phase II clinical trial can range from less than a year to up to two years.

Phase III Trials

Phase III trials typically include a high number of patients. Hundreds or even thousands of patients around the country or world may participate. Patients enrolled in a Phase III clinical trial will be placed into one of the following groups:

  • Control group: this group receives the standard treatment
  • Study group: this group receives the new treatment being tested

Common questions patients have include:

How Are Patients Put Into Groups?

A computer program will randomly assign patients into groups. This helps to avoid bias in the clinical trial. (Bias happens when human choices affect a study’s results.)

Will My Doctor Know Which Group I’m In?

It depends on the trial. For a single blind study, patients do not know which group they are in, but their doctor does. For a double-blind study, neither the patient nor their doctors know which group the patient is in. (In case of an emergency, however, doctors can receive this information.)

Could I be Given a Placebo?

A placebo is something that looks like medicine but isn’t. If a placebo is used, it is administered along with the best standard treatment. Using a placebo allows doctors to compare standard treatment alone to standard treatment with a new drug. If there isn’t a standard treatment, then the placebo may be given alone, but this is uncommon.

After the Phase III trial, the FDA reviews the clinical trial results to make sure the treatment is safe and effective for people to use. The FDA decides whether to approve the treatment so that it is available for all patients.

The average length of a Phase III clinical trial can vary, ranging from one to four years or longer.

Phase IV Trials

In Phase IV trials, doctors study treatments that the FDA has already approved. The goal of Phase IV trials is to continue studying any side effects of a new treatment.

The average length of a Phase IV clinical trial is usually shorter than Phase III so typically fewer than four years.

TAILOR RT: A Randomized Trial of Regional Radiotherapy in Biomarker Low-Risk Node Positive and T3N0 Breast Cancer (NCT03488693)
Principal Investigator: Supriya Jain
Locations: Banner MD Anderson Cancer Center at McKee Medical Center and Banner MD Anderson Cancer Center at North Colorado Medical Center

T-DM1 and Tucatinib Compared With T-DM1 Alone in Preventing Relapses in People With High Risk HER2-Positive Breast Cancer, the CompassHER2 RD Trial (NCT04457596
Principal Investigator: Shakeela Bahadur (AZ), Esther Mondo (NoCo) 
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical CenterBanner MD Anderson Cancer Center at McKee Medical Center and Banner MD Anderson Cancer Center at North Colorado Medical Center

EMBER-3: A Phase 3, Randomized, Open-Label Study of Imlunestrant, Investigator's Choice of Endocrine Therapy, and Imlunestrant Plus Abemaciclib in Patients With Estrogen Receptor Positive, HER2 Negative Locally Advanced or Metastatic Breast Cancer Previously Treated With Endocrine Therapy (NCT04975308)
Principal Investigators: Shakeela Bahadur (AZ), Esther Mondo (NoCo)
Locations: Banner MD Anderson Cancer Center at Banner Gateway Medical Center, Banner MD Anderson Cancer Center at McKee Medical Center and Banner MD Anderson Cancer Center at North Colorado Medical Center

Inflammatory Breast Cancer (IBC) Registry (NCT00477100
Principal Investigators: Shakeela Bahadur AZ), Esther Mondo (NoCo)
Locations: Banner MD Anderson Cancer Center at Banner Gateway Medical CenterBanner MD Anderson Cancer Center at Banner Boswell Medical CenterBanner MD Anderson Cancer Center at McKee Medical Center and Banner MD Anderson Cancer Center at North Colorado Medical Center

Potential Impact of the COVID -19 Pandemic on Financial Toxicity in Breast Cancer Surgical Patients: The Impact on Out of Pocket Costs, Lost Wages and Economic Strain (NCT04169542
Principal Investigators: Molly Decker (NoCo)
Locations: Banner MD Anderson Cancer Center at McKee Medical Center and Banner MD Anderson Cancer Center at North Colorado Medical Center

Prospective comparison of MRI versus contrast enhanced mammography prior to surgery in breast cancer patients: a randomized controlled trial (NCT04770714
Principal Investigator: Michael Morris
Locations: Banner MD Anderson Cancer Center at Banner Gateway Medical Center and Banner MD Anderson Cancer Center at Banner - University Medical Center Phoenix

Phase I, Open-label Study to Evaluate Safety, Tolerability and Preliminary Efficacy of Modified Salmonella Typhimurium SGN1 in Patients With Advanced Solid Tumor (NCT05038150
Principal Investigator: Madappa Kundranda
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

Shortening Adjuvant Photon Irradiation to Reduce Edema (SAPHIRE): A Randomized Trial of Hypofractionated Versus Conventionally Fractionated Regional Nodal Irradiation for Invasive Breast Cancer (NCT02912312
Principal Investigators: Emily Grade (AZ), Supriya Jain (NoCo)
Locations: Banner MD Anderson Cancer Center at Banner Gateway Medical CenterBanner MD Anderson Cancer Center at McKee Medical Center and Banner MD Anderson Cancer Center at North Colorado Medical Center 

Phase 1/2 Dose-Escalation and Expansion Study of FLX475 Alone and in Combination With Pembrolizumab in Advanced Cancer (NCT03674567
Principal Investigator: Tomislav Dragovich
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

Colorectal Cancer

Minimal Residual Disease Assessment in Colorectal Cancer (MiRDA-C) (NCT04739072)
Principal Investigator: Tomislav Dragovich
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center 

A Randomized Phase 3 Study of MRTX849 in Combination With Cetuximab Versus Chemotherapy in Patients With Advanced Colorectal Cancer With KRAS G12C Mutation With Disease Progression On or After Standard First-Line Therapy (NCT04793958
Principal Investigator: Tomislav Dragovich
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center 

A Phase 2/3, Randomized, Open-Label Study of Maintenance GRT-C901/GRT-R902, A Neoantigen Vaccine, in Combination With Immune Checkpoint Blockade for Patients With Metastatic Colorectal Cancer (NCT05141721
Principal Investigator: Madappa Kundranda 
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

Colorectal, Hepatocellular Carcinoma

Intravenous T3011 Given as a Single Agent and in Combination With Other Therapy in Subjects With Advanced Solid Tumors (NCT04780217)
Principal Investigator: Jiaxin Niu
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

Gastric/GEJ

Randomized, Double-Blind, Placebo-Controlled, Phase 2 Trial to Evaluate the Safety and Efficacy of TU-100 as an Adjunct to an Enhanced Recovery After Surgery (ERAS) Protocol in Subjects Undergoing Bowel Resection (NCT04742907
Principal Investigator: Michael Choti
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

Phase 1/2 Dose-Escalation and Expansion Study of FLX475 Alone and in Combination With Pembrolizumab in Advanced Cancer (NCT03674567)
Principal Investigator: Jiaxin Niu
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

HCC

Solid Tumor 

Phase 2b Study of GC4711 in Combination With SBRT for Nonmetastatic Pancreatic Cancer (NCT04698915
Principal Investigator: Gary Walker 
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center 

A Phase I/IIa Clinical Trial to Evaluate the Safety, Tolerability and Pharmacokinetics of TST001 Administered as Monotherapy or in Combination With Nivolumab in Patients With Locally Advanced or Metastatic Solid Tumors (NCT04396821)
Principal Investigator: Madappa Kundranda
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

A Phase I Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Biologic Effect of VG2025 in Subjects With Advanced Malignant Solid Tumors (NCT05266612
Principal Investigator: Madappa Kundranda 
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center 

A Phase 2 Study of Olaparib in Combination with Pembrolizumab in Participants with Previously Treated, Homologous Recombination Repair Mutation (HRRm) and/or Homologous Recombination Deficiency (HRD)-Positive Advanced Cancer (NCT04123366)
Principal Investigator: Sheakeela Bahadur
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

TiNivo-2: A Phase 3, Randomized, Controlled, Multicenter, Open-label Study to Compare Tivozanib in Combination With Nivolumab to Tivozanib Monotherapy in Subjects With Renal Cell Carcinoma Who Have Progressed Following One or Two Lines of Therapy Where One Line Has an Immune Checkpoint Inhibitor (NCT04987203)
Principal Investigator: Alex Bowman
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

Bladder Urothelial

A Phase 1/1b Dose Escalation/Expansion Study of NGM831 as Monotherapy and in Combination With Pembrolizumab in Advanced or Metastatic Solid Tumors (NCT05215574)
Principal Investigator: Madappa Kundranda
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center 

Bladder Cancer

Phase III Randomized Trial of Concurrent Chemoradiotherapy With or Without Atezolizumab in Localized Muscle Invasive Bladder Cancer (NCT03775265)
Principal Investigator: Eric Anderson
Location: 
Banner MD Anderson Cancer Center at McKee Medical Center and Banner MD Anderson Cancer Center at North Colorado Medical Center

A Randomized Phase III Trial of Intravesical BCG veRsus Intravesical Docetaxel and GEmcitabine Treatment in BCG Naïve High Grade Non-Muscle Invasive Bladder Cancer (BRIDGE) (NCT05538663)
Principal Investigator: Chinedu Mmeje
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center 

A Phase 2 Multi-Cohort, Open-Label, Multi-Center Clinical Study Evaluating the Efficacy and Safety of Disitamab Vedotin (RC48-ADC) Alone and in Combination With Pembrolizumab in Subjects With Locally-Advanced Unresectable or Metastatic Urothelial Carcinoma That Expresses HER2 (NCT04879329)
Principal Investigator: Alex Bowman
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center 

Germ Cell Tumor

A Prospective Observational Cohort Study to Assess miRNA 371 for Outcome Prediction in Patients With Newly Diagnosed Germ Cell Tumors (NCT04435756)
Principal Investigator: Alex Bowman
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center 

Metastatic Renal Cell Carcinoma

Phase III Trial of Immunotherapy-Based Combination Therapy With or Without Cytoreductive Nephrectomy for Metastatic Renal Cell Carcinoma (PROBE Trial) (NCT04510597)
Principal Investigator: Chinedu Mmeje
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center 

Prostate Cancer

Randomized Phase II Trial of Niraparib with Standard Combination Radiotherapy and Androgen Deprivation Therapy (ADT) in High Risk Prostate Cancer (with Initial Phase I)(NCT04037254)
Principal Investigator: Alex Bowman
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center 

Parallel Phase III Randomized Trials for High Risk Prostate Cancer Evaluating De-Intensification for Lower Genomic Risk and Intensification of Concurrent Therapy for Higher Genomic Risk With Radiation (PREDICT-RT*) (NCT04513717)
Principal Investigator: Eric Anderson
Location: 
Banner MD Anderson Cancer Center at McKee Medical Center and Banner MD Anderson Cancer Center at North Colorado Medical Center

Parallel Phase III Randomized Trials of Genomic-Risk Stratified Unfavorable Intermediate Risk Prostate Cancer: De-Intensification and Intensification Clinical Trial Evaluation (GUIDANCE) (NCT05050084)
Principal Investigator: Eric Anderson
Location: 
Banner MD Anderson Cancer Center at McKee Medical Center and Banner MD Anderson Cancer Center at North Colorado Medical Center

Phase III Trial of Docetaxel vs. Docetaxel and Radium-223 for Metastatic Castration-Resistant Prostate Cancer (mCRPC) (NCT03574571)
Principal Investigator: Santosh Rao
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center 

Phase III Randomized Trial of Standard Systemic Therapy (SST) Versus Standard Systemic Therapy Plus Definitive Treatment (Surgery or Radiation) of the Primary Tumor in Metastatic Prostate Cancer (NCT03678025)
Principal Investigator: Chinedu Mmeje (AZ), Supriya Jain (NoCo)
Locations: Banner MD Anderson Cancer Center at Banner Gateway Medical CenterBanner MD Anderson Cancer Center at McKee Medical Center and Banner MD Anderson Cancer Center at North Colorado Medical Center

Solid Tumor

EXTernal beam radiation to Eliminate Nominal metastatic Disease (EXTEND): A randomized phase II basket trial assessing the efficacy of upfront local consolidative therapy (LCT) for oligometastatic disease (NCT03599765)
Principal Investigators: Gary Walker (AZ), Supriya Jain (NoCo)
Locations: Banner MD Anderson Cancer Center at Banner Gateway Medical CenterBanner MD Anderson Cancer Center at Banner Thunderbird Medical CenterBanner MD Anderson Cancer Center at McKee Medical CenterBanner MD Anderson Cancer Center at North Colorado Medical Center

A Phase 2, Randomized, Double-Blind, Placebo Controlled Trial to Evaluate the Safety and Efficacy of MIT-001 in Prevention of Oral Mucositis in Patients Receiving CCRT for Previously Untreated Locally Advanced HNSCC (NCT04651634
Principal Investigator: Gary Walker
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

Phase I, Open-label Study to Evaluate Safety, Tolerability and Preliminary Efficacy of Modified Salmonella Typhimurium SGN1 in Patients With Advanced Solid Tumor (NCT05038150
Principal Investigator: Madappa Kundranda 
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

Solid Tumor

A Phase I/II Study of HB-201 Monotherapy or in Combination with Nivolumab in Patients with Human Papillomavirus 16 Positive (HPV 16+) Confirmed Cancers (NCT04180215)
Principal Investigator: Jiaxin Niu
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

Squamous Cell Carcinoma

Phase I Trial With Expansion Cohort of DNA-PK Inhibition and IMRT in Cisplatin-Ineligible Patients With Stage 3-4 Local-Regionally Advanced Head and Neck Squamous Cell Carcinoma (HNSCC) (NCT04533750)
Principal Investigator: Michael Samuels
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

A Randomized, Placebo-Controlled, Double-Blind Study of Adjuvant Cemiplimab Versus Placebo After Surgery and Radiation Therapy in Patients With High Risk Cutaneous Squamous Cell Carcinoma (NCT03969004)
Principal Investigator: Jiaxin Niu
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

Phase 1/2 Dose-Escalation and Expansion Study of FLX475 Alone and in Combination With Pembrolizumab in Advanced Cancer (NCT03674567
Principal Investigator: Tomislav Dragovich
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

Phase 1/2 Dose-Escalation and Expansion Study of FLX475 Alone and in Combination With Pembrolizumab in Advanced Cancer (NCT03674567)
Principal Investigator: Tomislav Dragovich
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

Non-Small Cell Lung Cancer

Intravenous T3011 Given as a Single Agent and in Combination With Other Therapy in Subjects With Advanced Solid Tumors (NCT04780217)
Principal Investigator: Dr. Jiaxin Niu
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

Adjuvant Lung Cancer Enrichment Marker Identification and Sequencing Trial (ALCHEMIST) (NCT02194738)
Principal Investigator: Jiaxin Niu
Location: Banner MD Anderson Cancer Center at Banner Boswell Medical Center

Phase I/II Study of SBRT and GC4711 for Centrally Located or Large NSCLC (GRECO-1) (NCT04476797)
Principal Investigator: Supriya Jain
Location: Banner MD Anderson Cancer Center at McKee Medical Center and Banner MD Anderson Cancer Center at North Colorado Medical Center

A Randomized Phase III Trial of Induction/Consolidation Atezolizumab (NSC #783608) + SBRT Versus SBRT Alone in High Risk, Early Stage NSCLC (NCT04214262)
Principal Investigator: Supriya Jain
Location: Banner MD Anderson Cancer Center at McKee Medical Center and Banner MD Anderson Cancer Center at North Colorado Medical Center  

Non-Small Cell Lung Cancer and Small Cell Lung Cancer

Phase I, Open-label Study to Evaluate Safety, Tolerability and Preliminary Efficacy of Modified Salmonella Typhimurium SGN1 in Patients With Advanced Solid Tumor (NCT05038150
Principal Investigator: Madappa Kundranda
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

Small Cell Lung Cancer

MRI Brain Surveillance Alone Versus MRI Surveillance and Prophylactic Cranial Irradiation (PCI): A Randomized Phase III Trial in Small-Cell Lung Cancer (MAVERICK) (NCT04155034)
Principal Investigator: Supriya Jain
Location: Banner MD Anderson Cancer Center at McKee Medical Center and Banner MD Anderson Cancer Center at North Colorado Medical Center

Solid Tumor

An Open-Label, Multicenter, Phase 1/2 Study of RP1 as a Single Agent and in Combination With PD1 Blockade in Patients With Solid Tumors (NCT03767348)
Principal Investigator: Dr. Jiaxin Niu

"A Phase 2 Study of Olaparib in Combination with Pembrolizumab in Participants with Previously Treated, Homologous Recombination Repair Mutation (HRRm) and/or Homologous Recombination Deficiency (HRD)-Positive Advanced Cancer" (NCT04123366)
Principal Investigator: Sheakeela Bahadur
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

A Phase 1 Open-label, Multicenter Study Evaluating the Safety and Efficacy of KITE-363, an Autologous Anti-CD19/CD20 CAR T-cell Therapy, in Subjects With Relapsed and/or Refractory B-cell Lymphoma (NCT04989803
Principal Investigator: Matthew Ulrickson
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

A Multi-center Single Arm Phase II Study to Evaluate the Safety and Efficacy of Genetically Engineered Autologous Cells Expressing Anti-CD20 and Anti-CD19 Specific Chimeric Antigen Receptor in Subjects With Relapsed and/or Refractory Diffuse Large B Cell Lymphoma (NCT04792489
Principal Investigator: Matthew Ulrickson
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

Principal Investigator: Dr. Tomislav Dragovich
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

ALLO-501A-201: A SINGLE-ARM, OPEN-LABEL, PHASE 1/2 STUDY EVALUATING THE SAFETY, EFFICACY, AND CELLULAR KINETICS/PHARMACODYNAMICS OF ALLO--501A, AN ANTI-CD19 ALLOGENEIC CAR T CELL THERAPY, AND ALLO-647, AN ANTI-CD52 MONOCLONAL ANTIBODY, IN SUBJECTS WITH RELAPSED/REFRACTORY LARGE B-CELL LYMPHOMA (LBCL) (NCT04416984
Principal Investigator: Rajneesh Nath
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center

Phase I, Open-label Study to Evaluate Safety, Tolerability and Preliminary Efficacy of Modified Salmonella Typhimurium SGN1 in Patients With Advanced Solid Tumor (NCT05038150
Principal Investigator: Madappa Kundranda
Location: Banner MD Anderson Cancer Center at Banner Gateway Medical Center