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FDA Stem Cell Guide

How Legitimate Stem Cell Clinical Trials Work

Legitimate stem cell research follows a specific structure — one that provides real oversight, real informed consent, and real data collection.

The Investigational New Drug application

Before a stem cell product can be given to humans in the United States, the sponsor (usually a manufacturer or academic institution) must submit an Investigational New Drug (IND) application to the FDA. The IND summarizes preclinical safety data, describes the manufacturing process, and lays out the exact clinical trial protocol. The FDA has 30 days to place the IND on clinical hold or allow it to proceed.

The phases

  1. Phase 1 — a small number of participants, focused on safety, dose, and side effects.
  2. Phase 2 — a larger group, focused on preliminary evidence of effectiveness and further safety information.
  3. Phase 3 — larger, often randomized and controlled trials designed to confirm effectiveness and monitor rarer side effects. Data from adequate and well-controlled Phase 3 trials typically forms the basis of a Biologics License Application.
  4. Phase 4 — post-approval studies after the product is on the market.

Institutional Review Board oversight

Every trial must be reviewed and approved by an Institutional Review Board (IRB). The IRB is an independent committee responsible for protecting the rights and welfare of human research subjects. It reviews the protocol, the informed consent document, and ongoing safety data.

Informed consent

Legitimate trials require participants to sign a detailed informed consent document that describes the purpose of the study, what will happen, the known risks, the unknowns, alternatives to participating, and the participant's right to withdraw at any time without penalty.

Registration on ClinicalTrials.gov

Most trials in the United States are required to be registered on ClinicalTrials.gov. Each trial has a unique identifier (NCT number), a listed sponsor, inclusion and exclusion criteria, contact information for study coordinators, and, once the trial concludes, summary results.

What legitimate trials generally do not do

  • Charge participants thousands of dollars for the investigational product itself.
  • Advertise through late-night infomercials or celebrity endorsements.
  • Claim to treat many unrelated conditions in the same study.
  • Skip informed consent or IRB review.

How to find real trials

Search ClinicalTrials.gov by condition and location. Bring the NCT number to your physician for review. Academic medical centers and major hospital systems can often help identify whether a specific trial is a fit for your situation.