Clinical Trials and Evidence-Based Oncology Practice Flashcards

In a Phase I clinical trial for a new oncology drug, the primary objective is to determine:

Answer: Maximum tolerated dose (MTD) and dose-limiting toxicities (DLTs)

Phase I trials are primarily safety/dose-finding studies. The primary objectives are to establish the maximum tolerated dose (MTD), characterize dose-limiting toxicities (DLTs), determine the recommended Phase II dose (RP2D), and describe the pharmacokinetic profile. Efficacy is a secondary/exploratory endpoint.

The principle of equipoise in clinical trial ethics means:

Answer: There is genuine uncertainty about which treatment arm is superior, justifying randomization

Clinical equipoise means there is genuine scientific uncertainty in the expert medical community about which treatment is superior. Equipoise is the ethical justification for randomizing patients — if one treatment is clearly superior, randomization would be unethical. When evidence tips decisively in one direction, trials are stopped by data safety monitoring boards.

Informed consent for clinical trial participation must include which elements specific to research, beyond standard clinical care consent?

Answer: Voluntary nature of participation, right to withdraw, foreseeable risks/benefits, and availability of alternative treatments

Research informed consent must include (per 45 CFR 46 and ICH-GCP): a statement that the study involves research, foreseeable risks and benefits, alternative procedures, confidentiality protections, compensation for injury, contact information, and explicit statement that participation is voluntary with the right to withdraw without penalty.

A randomized controlled trial (RCT) in oncology is considered the gold standard for evidence because:

Answer: Randomization controls for confounding variables and reduces selection bias, allowing causal inference

The RCT is the gold standard because random allocation to treatment and control groups balances known and unknown confounders, minimizing selection bias. This allows valid causal inference about treatment effects. Blinding further controls for performance and detection bias.

Which term describes a trial design where a new agent is tested across multiple cancer types based on a shared molecular target (biomarker) rather than tumor histology?

Answer: Basket trial

Basket trials (also called histology-agnostic or tumor-agnostic trials) enroll patients across different tumor types that share a specific molecular alteration (e.g., NTRK fusion, MSI-H, BRAF V600E), testing whether a targeted therapy works regardless of tissue of origin. Examples: larotrectinib (NTRK), pembrolizumab (MSI-H).

The ONCO NP role in clinical trials includes which key responsibility?

Answer: Ensuring protocol adherence, obtaining informed consent, monitoring for adverse events, and educating patients about trial participation

Advanced practice oncology nurses in clinical research play critical roles: patient education about trial procedures and expectations, obtaining/witnessing informed consent, accurate data collection, toxicity monitoring per protocol, dose modification implementation, adverse event reporting, and serving as patient advocates and primary contact throughout the trial.