What is a clinical trial?

A clinical trial is a research study involving human participants designed to evaluate medical interventions — such as drugs, devices, or behavioral strategies. Trials follow a structured protocol and are registered on ClinicalTrials.gov. They progress through phases: Phase 1 (safety), Phase 2 (efficacy), Phase 3 (large-scale comparison), and Phase 4 (post-market surveillance).

How do I find clinical trials I'm eligible for?

You can search ClinicalTrials.gov or use ClinicalMetric to filter by condition, phase, or location. Each trial listing includes eligibility criteria such as age range, sex, diagnosis, and prior treatment history. Contact the study team directly or ask your physician to refer you to a relevant trial.

Are clinical trials safe to participate in?

Clinical trials are conducted under strict ethical and regulatory oversight, including IRB approval and FDA regulation in the US. All participants must give informed consent after reviewing potential risks and benefits. Phase 1 trials carry more uncertainty, while Phase 3 trials involve interventions with an established safety profile. Participation is always voluntary and you may withdraw at any time.

What are the phases of clinical trials?

Clinical trials progress through four main phases. Phase 1 tests safety and dosing in a small group (20–80 people). Phase 2 evaluates efficacy and side effects in a larger group (100–300). Phase 3 compares the intervention against standard treatments in thousands of participants. Phase 4 occurs after approval and monitors long-term effects in the general population.

Do participants get paid for joining clinical trials?

Many clinical trials offer compensation for time and travel expenses, though payment structures vary widely by study. Compensation is not intended to be coercive. Some trials also cover treatment costs for participants. Always review the consent form carefully and ask the study coordinator about any financial considerations before enrolling.

Phase 1 Clinical Trials 2026: First-in-Human Studies and Early Drug Development

Medical Notice

This article is for informational purposes only and does not constitute medical advice. Clinical trial eligibility and availability vary. Always consult a qualified healthcare professional before making any medical decisions or considering participation in a clinical trial.

Summary

Phase 1 clinical trials — "first-in-human" studies — are the earliest stage of testing a new drug or treatment in people. Their primary goal is not to prove the treatment works, but to determine whether it is safe, how the body processes it, and what dose can be given without unacceptable side effects. In 2026, Phase 1 oncology trials often show early signs of efficacy that make them attractive options for patients with advanced cancer who have exhausted standard options. This guide explains what Phase 1 trials involve, who participates, what the experience is like, and how to find these studies.

What Phase 1 Trials Are Testing

Before any new drug can enter a clinical trial, it must have substantial preclinical evidence — laboratory studies and animal studies demonstrating the mechanism of action, preliminary efficacy, and toxicology data. The FDA requires an Investigational New Drug (IND) application to be approved before Phase 1 can begin, reviewed by the Center for Drug Evaluation and Research (CDER) or Center for Biologics Evaluation and Research (CBER) depending on the drug type. Phase 1 studies then address four fundamental questions: Is the drug safe at the doses being tested? What side effects occur? How does the body absorb, distribute, metabolize, and excrete the drug (pharmacokinetics, PK)? What dose reaches the target tissues in sufficient concentrations to be pharmacodynamically active (PD)?

In oncology, Phase 1 trials follow a dose escalation design — starting at a fraction (commonly 1/10th) of the maximum tolerated dose in the most sensitive animal species and escalating in cohorts of 3–6 patients until the maximum tolerated dose (MTD) or recommended Phase 2 dose (RP2D) is established. For most modern targeted agents and immunotherapies, PK/PD modeling guides dose selection rather than MTD — because many targeted agents (kinase inhibitors, monoclonal antibodies) don't have clear toxicity dose-response curves. Modern "3+3" designs are increasingly replaced by model-based designs (mTPI, BOIN, CRM) that use Bayesian statistical updating to more efficiently find the optimal dose with fewer patients.

Who Participates in Phase 1 Trials

Phase 1 oncology trials typically enroll patients with advanced (metastatic or unresectable) cancer who have exhausted standard treatment options. Unlike healthy volunteer Phase 1 trials (common for non-oncology drugs), cancer Phase 1 studies enroll patients because the risk/benefit calculation differs — patients with advanced cancer may have limited other options, while exposing healthy people to an experimental drug with unknown human safety is not ethically justified for cancer drugs. A significant proportion of Phase 1 oncology participants receive clinical benefit — response rates of 10–20% are common even in true Phase 1 studies, and for targeted agents selected by biomarker, early efficacy can be remarkable.

Non-oncology Phase 1 trials (for cardiovascular drugs, neurological agents, antibiotics, vaccines) typically enroll healthy volunteers who receive compensation for their time and willingness to take early safety risk. Healthy volunteer Phase 1 studies are conducted at specialized Phase 1 clinical pharmacology units — dedicated facilities with intensive monitoring, frequent blood draws for PK, and 24-hour observation capability. The BIA 10-2474 TeGenero disaster (2006, where 6 healthy volunteers experienced life-threatening cytokine storm from a CD28 superagonist) and other early safety incidents have led to much more stringent starting dose selection and monitoring protocols for immunomodulatory first-in-human studies.

What Participants Experience in Phase 1 Studies

The intensity of a Phase 1 participation experience depends significantly on the drug type, route of administration, and whether it is a single ascending dose (SAD) study, multiple ascending dose (MAD) study, or food effect/drug-drug interaction sub-study. Single dose studies may require 1–3 overnight stays at the Phase 1 unit with frequent blood draws over 24–48 hours post-dose; multiple dose studies require repeat dosing visits over weeks or months. Patients in Phase 1 cancer trials typically receive treatment on a cycle schedule (e.g., IV infusion every 3 weeks) and have more frequent assessment visits than Phase 3 participants — additional PK blood draws, pharmacodynamic biomarker biopsies, and more frequent imaging.

Dose limiting toxicities (DLTs) — prespecified adverse events of sufficient severity to limit dose escalation — are monitored during the first treatment cycle and trigger DSMB review before the next dose cohort proceeds. Participants are never exposed to doses the DSMB determines are unsafe. Phase 1 participants receive detailed informed consent describing the investigational nature of the treatment, the specific known and unknown risks, the compensation structure, and their absolute right to withdraw. Compensation for healthy volunteer Phase 1 studies is typically $200–$400/day for the duration of the confinement period; cancer patient Phase 1 studies may offer travel reimbursement but typically do not provide additional compensation for participation.

Phase 1 in 2026: The Most Active Categories

Oncology accounts for approximately 60% of all Phase 1 activity. The most active Phase 1 categories in 2026 include: antibody-drug conjugates (ADCs, the fastest growing oncology drug class with 100+ Phase 1 ADC studies currently recruiting), bispecific T cell engaging antibodies (bispecific antibodies that simultaneously bind CD3 on T cells and a tumor antigen — tarlatamab for SCLC, mosunetuzumab for lymphoma have advanced to approval, with 50+ novel bispecifics in Phase 1), CAR-T cell therapy for solid tumors (the longstanding challenge of extending CAR-T success from liquid to solid tumors, with 60+ Phase 1 CAR-T solid tumor trials), and RNA-targeting medicines (mRNA therapies, siRNA, and ASOs across multiple disease areas leveraging the validated LNP delivery platform from COVID mRNA vaccines).

CRISPR and base editing in vivo gene editing trials are among the most scientifically novel Phase 1 studies in 2026 — Intellia's NTLA-2001 (TTR knockout for ATTR amyloidosis), NTLA-2002 (KLKB1 knockout for hereditary angioedema), and Beam Therapeutics BEAM-101 (base editing of BCL11A enhancer for sickle cell disease) are in Phase 1/2, demonstrating durable in vivo gene editing from a single treatment. Each of these programs required novel safety monitoring protocols for editing efficiency, off-target editing events, and immune response to viral vectors or LNP delivery systems.

How to Find Phase 1 Clinical Trials

ClinicalTrials.gov allows filtering by "Phase 1" in the study phase field, combined with disease condition and recruiting status. Major academic cancer centers — MD Anderson, Memorial Sloan Kettering, Dana-Farber, Johns Hopkins, Penn Medicine, Stanford — have dedicated Phase 1 programs with clinical pharmacologists who specialize in early drug development and can review a patient's history against multiple concurrent Phase 1 protocols to identify the best match. The NCI Cancer Therapy Evaluation Program (CTEP) maintains a searchable database of NCI-sponsored Phase 1 trials.

For patients with advanced cancer who have exhausted standard options, the NCCN guidelines explicitly recommend clinical trial participation as a category 1 recommendation, and most comprehensive cancer centers have trial matching services. Patient advocacy organizations for specific cancer types often maintain Phase 1 trial databases and can connect patients to investigative sites. Second opinions at major cancer centers specifically focused on trial access are often covered by insurance under "clinical decision making" visit codes.

Key Takeaways

Phase 1 trials test safety and pharmacokinetics first — not efficacy — but modern targeted oncology Phase 1 studies frequently show early clinical benefit in 10–20%+ of participants, making them legitimate treatment options for patients with advanced cancer.
Modern Bayesian dose escalation designs (BOIN, mTPI, CRM) are replacing "3+3" designs, finding optimal doses more efficiently and with fewer patients exposed to sub-therapeutic or excessive doses.
The most active Phase 1 categories in 2026 are ADCs (100+ Phase 1 studies), bispecific T cell engagers (50+), CAR-T for solid tumors (60+), and in vivo CRISPR/base editing programs seeking durable genetic correction from a single treatment.
Phase 1 healthy volunteer studies at specialized clinical pharmacology units offer compensation of ~$200–$400/day and are most accessible for young healthy adults; Phase 1 cancer trials are for patients with advanced malignancy who have progressed on standard treatments.
Dedicated Phase 1 programs at major cancer centers have clinical pharmacologists who review multiple concurrent protocols to identify the optimal match — a second opinion focused on trial access is worthwhile for patients with advanced cancer.