How to Plan a Phase 1 Clinical Trial

Phase 1 Clinical Trial Planning

What is a Phase I Clinical Trial

A Phase 1 clinical trial aims to assess the drug’s safety and dosage and enable the design of Phase 2 and the rest of the clinical trial phases. The drug is tested first in humans, using a small number of participants (20 to 100).


What to Do Before a Phase I Clinical Trial

The study startup stage includes building a budget, designing a clinical trial protocol, and plenty of preclinical research. Before a Phase 1 clinical trial can begin, there must be a successful outcome of the preclinical research studies performed. During preclinical research, a new drug is tested in animals to ensure that it is safe to begin testing in humans. This involves several GLP-compliant studies, including an assessment of the following:


  • Toxicology (2 species)
  • Toxicokinetics (in vivo)
  • Mutagenicity (in vitro Ames test)
  • Reproductive toxicology
  • Safety pharmacology
  • Absorption, distribution, metabolism, and excretion of the drug


Once preclinical research has been performed, Phase 1 clinical trial design planning can begin. 


How to Design a Phase I Clinical Trial


Recruit Participants

A Phase 1 clinical trial is performed over several months in a small group of 20 to 100 healthy volunteers, typically men or those with the disease/condition. These participants will receive a “first in human” single dose of a new drug, usually orally or intravenously, and will be monitored for safety.


Determine the Starting Dose

The starting dose of a drug is based on the outcome of preclinical research studies in animals. The starting dose is very low and is administered once to ensure the safety of the participants.


Test for Safety

After receiving a single dose of the drug, post-dose blood samples are taken (typically processed to plasma) for bioanalysis to aid in the assessment of pharmacokinetics (PK) and pharmacodynamics (PD). Other samples are taken for safety testing. These tests help reveal any adverse side effects to the drug and determine how well the drug is being absorbed, distributed, metabolized, and excreted by the body.


Adjust Doses

Based on the results of tests performed, the dose of the drug is often adjusted. This may involve increasing the dose of the drug and/or the frequency of the dose administered. In single ascending dose (SAD) studies, volunteers will receive a single increased dose of the drug. In multiple ascending dose (MAD) studies, volunteers will receive multiple doses of the drug. After these adjustments, the safety of the volunteers will be reviewed again based on additional tests performed. If considered safe to do so, the drug dose is typically adjusted again.


Answer Key Questions

Human safety after administration of the new drug is the focus of Phase 1 clinical trials. Phase 1 clinical trial designs should answer key questions, including:


  • The safest dose of the drug
  • Any potential adverse side effects
  • Toxicity of the drug
  • The overall safety of the drug in humans


Clinical Trial Phases After Phase I

Phase 1 clinical trials are just the beginning of the journey researchers take to receive approval for a new drug and launch it to market. After Phase 1, the following clinical trial phases take place:


Phase II

According to the Food and Drug Administration (FDA), approximately 70% of new drugs progress into Phase 2 clinical trials. A Phase 2 clinical trial is typically performed from several months to two years in a larger group of human subjects (several hundred), including those with the disease or condition that the new drug is designed to treat. This phase may include several different groups: Those currently receiving the standard treatment for the disease and those receiving the standard treatment and the new medication or treatment. The goal of Phase 2 clinical trials is to continue studying the safety of the new drug while attempting to prove its efficacy.


Phase III

Phase 3 clinical trials can begin if a new drug is proven safe and effective in Phase 2. According to the FDA, approximately 33% of new drugs progress to Phase 3 clinical trials. During this phase, researchers will work to determine how the new drug compares to the standard treatment for the disease they are trying to treat. Tests will be conducted on up to several thousand (300 to 3000) human subjects with the condition in order to assess the drug’s efficacy, determine the drug half life, and monitor adverse reactions. This phase of the clinical trial process can last for several years (typically from one to four years). At the end of Phase 2, if successful, researchers submit a new drug application (NDA) to the FDA for approval to launch the drug to market. 


Phase IV

Phase 4 clinical trials are performed after the drug has been approved and launched to market for use in the general population. According to the FDA, approximately 25-30% of new drugs progress to Phase 4 clinical trials. These trials are performed using several thousands of humans with the condition to assess safety and efficacy. Phase 4 clinical trials are also performed to expand the testing of the drug in new human populations that were outside of the initial clinical trial populations.


Why Partner with ACM for Your Phase I Clinical Study

Regarding your Phase 1 clinical trial, ACM Global Laboratories understands the importance of delivering quick and high-quality results that impact the progress and decision-making of your study.


Aron Robinson, ACM’s Global Business Development Director of Bioanalytical Services, explains that “During Phase 1 clinical trials, the emphasis of bioanalysis work is not based on the analysis of a high number of pharmacokinetic (PK) samples, but on the quality and speed of reporting the concentration results in time for required safety reviews. In addition, it is vital to ensure the appropriate scheduling of bioanalysis work to be performed before samples originating from the Phase 1 clinical trial can be analyzed. Effective advanced planning of bioanalytical method development and method validation studies ensures an appropriate method is in place in time for the analysis of samples is paramount.”


Our experts at our ACM Bioanalysis Laboratory can turn around PK sample concentration results fast, aiding you in making essential safety decisions to adjust the dose of the drug within the trial. We’ll not only help with single and multiple ascending dose trials but with any Phase 1 clinical trials where the bioanalysis of PK and/or PD samples is required and in various biological matrices.


By partnering with ACM Global for your Phase 1 clinical trial safety testing and bioanalysis needs, you can help guarantee the safety of your trial participants and make critical, informed decisions about the next steps within your trial.


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Need Guidance for Your Clinical Study?

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