Transforming Hope into Cure

The Clinical Trial Process

The National Institutes on Aging (NIA), a division of the US National Institutes of Health,
defines clinical trials as research studies in which medical, surgical, or behavioral interventions
are evaluated in people to determine their safety and effectiveness, serving as a primary method
for testing new treatments such as drugs, diets, and medical devices (e.g., pacemakers).
 

Guidelines for Manufacturing Investigational Products

In Phase 1 clinical trials involving investigational new drugs (IND), ensuring subject safety is
paramount through adherence to current Good Manufacturing Practices (CGMP). The Food and
Drug Administration (FDA) assesses pharmacological and toxicological findings and dosing
recommendations, contributing to this process.
 

To maintain the quality and safety of Phase 1 investigational drugs during development, effective
quality control (QC) procedures are essential. By following established QC protocols and
complying with CGMP, the production of equivalent investigational new drug products for
future clinical trials is streamlined. CGMP compliance in manufacturing investigational drugs
involves:
● Well-defined written procedures: Clear procedures promote consistency and accuracy in
manufacturing.
● Adequate equipment and manufacturing environment control: Proper control prevents
contamination and preserves product integrity.
● Accurate and consistent record-keeping: Thorough documentation, including testing,
tracks the process and upholds quality standards.
 

Manufacturers must explore suitable alternatives to meet these guidelines, employing appropriate
methods, facilities, and controls to ensure the investigational drug's safety, identity, strength,
quality, and purity. Manufacturers should carefully tailor standards, practices, and procedures to
align with cGMP for their specific product and manufacturing operations.
 

Clinical Trial Guidelines in Accordance with FDA Requirements
 

FDA regulations require that the Sponsors hold the responsibility of choosing proficient
investigators, equipping them with necessary information for conducting investigations,
guaranteeing diligent monitoring, maintaining adherence to the general investigational plan and
IND protocols, as well as upholding an effective IND for the studies. Furthermore, sponsors
must promptly communicate any notable new adverse effects or risks related to the drug to the
FDA and participating investigators. Additional sponsor responsibilities can be found under 21
CFR §312, outlining additional sponsor responsibilities including but not limited to ongoing trial
monitoring for adherence to the investigational plan, review and analysis of accumulating safety
and effectiveness data, compliance with 21 CFR §314.126 for well-controlled studies, a study
design enabling valid control comparisons for quantitative assessment, a bias-minimizing patient
assignment to treatment and control groups, defined and reliable methods for assessing subject
response, and an overall emphasis on quality/risk management.

 

Participating in a Clinical Trial
 

People choose to participate in clinical trials for various reasons, including when prior treatments
for their health condition have been ineffective. Some participants seek new treatment options,
while others, even those in good health, may aim to contribute to disease prevention research,
especially if they have a genetic predisposition. Many individuals engage in clinical trials to take
a more active role in their healthcare and to support researchers in advancing knowledge about
specific health issues.

Participating in a clinical trial makes individuals partners in scientific discovery, as major
medical breakthroughs rely on the generosity of clinical trial participants, regardless of their age.
The process of clinical trials typically follows a series of phases. Phase 0 is the initial step,
involving a small number of participants (10 – 15) and low drug doses to understand how the
medication metabolizes and its effects at minimal levels. Phase I focuses on determining a safe
dosing range for a drug with minimal side effects and is tested on a small group of 15 to 30
patients. Phase II evaluates safety and efficacy, often involving larger patient groups and
exploring drug combinations. Phase III compares the new drug with the standard of care,
enrolling 100 or more patients, and may include randomized, double-blind trials. Phase III results
are essential for FDA approval. Phase IV trials assess FDA-approved drugs on hundreds or
thousands of patients to study side effects, safety, and effectiveness, especially when used in
conjunction with other treatments. Before initiating a clinical trial, developers must submit an
Investigational New Drug (IND) application to the FDA, including data from animal studies,
manufacturing information, clinical protocols, prior human research data, and investigator
information.

New patients have multiple routes to explore clinical trials. Typically, once a diagnosis is
received, treatment options are discussed. Companies like xCure Inc., with whom CureScience
partners, utilize AI-based precision oncology platforms to provide patients with personalized,
precision medicine-focused therapy approaches. A widely recognized resource for current and
past clinical trial details across various diseases and their subcategories is ClinicalTrials.gov,
frequently visited by both physicians and patients for the latest information. Furthermore, many
individuals turn to condition-specific support groups and websites for additional guidance.
 

For more information:
1. https://www.fda.gov/patients/drug-development-process/step-3-clinical-research
2. https://www.nccn.org/patients/resources/clinical_trials/phases.aspx