Stem Cell Transplant and Cellular Therapy Program Clinical Trials

When you become a patient in our Stem Cell Transplant and Cellular Therapy Program, our physicians may consider you for one of our many clinical trials. Below we have provided you with some information regarding clinical trials so that you and your loved ones can make informed decisions about whether participation in a trial is right for you.

Our clinical trial availability changes frequently as trials close to enrollment or new ones are added. We suggest that you talk to your Stem Cell Transplant and Cellular Therapy Program physician about current clinical trials for your disease, or you may go to the National Institutes of Health database of clinical trials and follow the search instructions to find cancer trials recruiting at the University of Iowa.

Clinical trial participation is completely voluntary! Your Stem Cell Transplant and Cellular Therapy Program physician will review all standard and clinical trial treatment options available.

What are clinical trials?

Clinical trials are closely monitored studies designed to find new ways to prevent, diagnose, or treat diseases. Nearly all standard cancer treatments widely used today were once part of clinical trials.

What are some risks and benefits of participating in a clinical trial?


  • Access to treatment that would otherwise not be available to you
  • Close monitoring and follow-up of your disease
  • Contributing to the advancement of medical science


  • Side effects that may range from mild to severe
  • Possible unknown health effects

What are the different phases of drug trials?

Phase I:

Phase I trials provide early evaluation of a treatment that researchers believe will be beneficial based on results in the lab. They may be designed to establish drug side effects, or the best dose and dosing schedule at which to administer a drug or combination of drugs. They may also provide early results regarding the effectiveness of the treatment being evaluated.

Phase II:

Once information regarding side effects and drug dosing is available, a phase II trial evaluates treatment effectiveness. Usually the number of patients enrolled in phase I and II trials are relatively small (less than 100).

Phase III:

Phase III trials test treatments that were proven safe and effective in Phase I and II trials. Phase III trials are almost always randomized trials in which the new treatment is compared to a standard treatment option. Randomization means that a patient is selected by chance to be in a treatment group. Phase III trials usually enroll hundreds of patients.

Expanded Access/Compassionate Use:

Expanded Access studies, sometimes also referred to as Compassionate Use programs, are a way in which drug manufacturers provide investigational drugs to patients who have a serious disease but cannot participate in a controlled clinical trial. The primary purpose of an expanded access program is to treat a patient’s condition, and not to collect data about the study drug(s).

What are common misconceptions about clinical trials?

"The clinical trial will cover all costs."

Clinical trials usually cover only the costs of items considered research-related, but you and your insurance provider will be responsible for covering the costs of all standard tests, drugs, and clinic appointments. Please refer to your study consent form for a list of costs covered by the study.

"I will be a guinea pig for unproven treatments."

Your physician will discuss available standard treatments and clinical trial options. If your physician presents a clinical trial to you, it is because he/she believes that you will be a good candidate for the study, and that it could provide you with a potentially beneficial treatment that may be better than standard treatment. However, participation in clinical trials is completely voluntary, and you will never be pressured into participating.

"I might receive a placebo instead of treatment."

Cancer patients will never, ever receive placebos instead of treatment. This would be highly unethical. If you choose to participate in a phase III clinical trial, it is possible that you may be randomized to standard treatment rather than the investigational treatment arm, but you will always receive treatment.

Adult Clinical Trials

  • Single Autologous Transplant Followed by Consolidation and Maintenance for Participants Diagnosed With Multiple Myeloma or a Related Plasma Cell Malignancy. (Single-center, investigator initiated); IRB 201208775
  • Impact of Donor Statin Use on Graft-versus-Host Disease after Unrelated Donor Hematopoietic Cell Transplantation: NMDP IRB-2011-0269. (Multi-center); IRB 201311794
  • A Multi-center Phase II Trial Randomizing Novel Approaches for Graft-versus-Host- CTN#1203. (Multi-center); IRB 201403761
  • A Pilot Study of CDX-301 (rhuFlt3L) with or without Plerixafor for the Mobilization and Transplantation of Allogeneic Blood Cell Grafts in HLA-Matched Donor/Recipient Sibling Pairs (pharmaceutical), IRB 201506507
  • A Phase III Randomized, Placebo-controlled Clinical Trial to Evaluate the Safety and Efficacy of MK-8228 (Letermovir) for the Prevention of Clinically Significant Human Cytomegalovirus (CMV) Infection in Adult, CMV-Seropositive Allogeneic Hematopoietic Stem Cell Transplant Recipients (pharmaceutical), IRB 201504507 (Pending, not yet open)
  • A Phase 2b, Randomized, Double-Blind, Placebo-Controlled Multi-Center Study Evaluating Antiviral Effects, Pharmacokinetics, Safety, and Tolerability of GS-5806 in Hematopoietic Cell Transplant (HCT) Recipients with Respiratory Syncytial Virus (RSV) Infection of the Lower Respiratory Tract (pharmaceutical), pending

Pediatric Clinical Trials

  • Defibrotide for Patients with Hepatic Veno-Occlusive Disease (VOD): A Treatment IND Study (Under CFR 312.34); (Pharmaceutical); IRB 201003719
  • A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) for transplantation in pediatric and adult patients with hematologic malignancies and other indications. (Multi-center); IRB 201107716
  • A Research Database for Hematopoietic Stem Cell Transplantation. (Multi-Center); IRB 200306043.
  • A Research Sample Repository for Allogeneic Unrelated Hematogoietic Stem Cell Transplantation. (Multi-Center); IRB 200306044
  • A Randomized, Multi-Center, Phase III Trial of Calcineurin Inhibitor-Free Interventions for Prevention of Graft-versus Host-Disease CTN 1301 (multicenter); IRB 201411790
  • A Phase 3, Open-label, Multicenter Study of the Safety/Tolerability and Efficacy of Brincidofovir (CMX001) for the Prevention of Adenovirus (AdV) Disease in Subjects with Asymptomatic AdV Infection at Risk of Progression and for the Treatment of Subjects with Localized or Disseminated AdV Disease, (pharmaceutical), IRB 201408501
  • A Phase II, Randomized, Double-blind, Placebo-controlled study to examine the effects of DAS181 in immunocompromised subjects with Lower Respiratory Tract Parainfluenza Infection on Supplemental Oxygen (pharmaceutical), IRB 201411501