11.4 Clinical trial design for plasma therapies

Clinical trials for plasma therapies are crucial for evaluating safety and effectiveness. These trials progress through phases, starting with small safety tests and advancing to large-scale efficacy studies, ultimately leading to regulatory approval and real-world monitoring.

Designing plasma therapy trials requires careful consideration of randomization, blinding, and control groups. Patient selection criteria, sample size determination, and standardized treatment protocols are essential for generating reliable data and ensuring consistent application across trial sites.

Types of clinical trials

• Clinical trials in plasma medicine evaluate safety and efficacy of plasma-based therapies for various medical conditions
• Progression through trial phases ensures thorough assessment of plasma treatments before widespread clinical use

Phase I trials

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Top images from around the web for Phase I trials

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  First-in-human phase I clinical trial of RG7356, an anti-CD44 humanized antibody, in patients ... View original

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• 

  Frontiers | Correlative analysis from a phase I clinical trial of intrapleural administration of ... View original

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• 

  Tolerogenic dendritic cell-based treatment for multiple sclerosis (MS): a harmonised study ... View original

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• 

  First-in-human phase I clinical trial of RG7356, an anti-CD44 humanized antibody, in patients ... View original

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• Initial safety testing of plasma therapies in small groups of healthy volunteers (20-80 participants)
• Assess tolerability, pharmacokinetics, and pharmacodynamics of plasma treatments
• Determine safe dosage range and identify side effects
• Usually non-randomized and open-label design

Phase II trials

• Evaluate efficacy and further assess safety in larger patient groups (100-300 participants)
• Often randomized and may include control groups
• Determine optimal dosage and treatment protocols for plasma therapies
• Assess short-term side effects and potential risks

Phase III trials

• Large-scale studies to confirm efficacy and monitor long-term side effects (300-3000 participants)
• Randomized, controlled trials comparing plasma therapy to standard treatments or placebos
• Gather data on effectiveness across diverse patient populations
• Provide information for regulatory approval and clinical practice guidelines

Phase IV trials

• Post-marketing surveillance studies conducted after plasma therapy approval
• Monitor long-term safety and efficacy in real-world clinical settings
• Identify rare side effects and potential drug interactions
• Evaluate cost-effectiveness and quality of life outcomes for plasma treatments

Trial design considerations

• Proper trial design crucial for generating reliable data on plasma therapy efficacy and safety
• Design elements impact statistical power, bias reduction, and generalizability of results

Randomization methods

• Simple randomization assigns participants to groups using random number generators
• Block randomization ensures balanced group sizes throughout the trial
• Stratified randomization controls for important prognostic factors
• Adaptive randomization adjusts allocation based on interim results
  • Minimizes imbalances between treatment groups
  • Increases efficiency in identifying effective plasma therapies

Blinding techniques

• Single-blind trials conceal treatment assignment from participants
• Double-blind trials hide treatment allocation from both participants and researchers
• Triple-blind trials extend blinding to data analysts
• Challenges in blinding plasma device trials due to visible plasma generation
  • Sham devices or alternative energy sources may be used as controls

Control groups vs treatment groups

• Parallel group design compares plasma therapy to control group simultaneously
• Crossover design allows participants to receive both treatments in different periods
• Factorial design evaluates multiple interventions or plasma parameters concurrently
• Active control groups receive standard treatment for comparison
• Placebo control groups receive inert treatment to account for placebo effect

Patient selection criteria

• Careful selection of trial participants ensures appropriate target population for plasma therapy evaluation
• Balances internal validity with generalizability of results to broader patient groups

Inclusion criteria

• Define characteristics required for study participation
• Age range appropriate for the plasma therapy being tested
• Specific medical conditions or symptoms relevant to the treatment
• Ability to provide informed consent and comply with study procedures
• May include biomarker or genetic profiles for targeted therapies

Exclusion criteria

• Identify factors that preclude participation to ensure patient safety
• Comorbidities that could interfere with treatment efficacy or increase risks
• Medications or treatments that may interact with plasma therapy
• Pregnancy or breastfeeding status for certain studies
• History of hypersensitivity to plasma or related treatments

Sample size determination

• Power analysis calculates required number of participants
• Considers effect size, significance level, and desired statistical power
• Accounts for expected dropout rates and loss to follow-up
• May require larger samples for rare conditions or subtle treatment effects
  • Multicenter trials often necessary to achieve adequate sample sizes

Plasma therapy protocols

• Standardized protocols ensure consistent application of plasma treatments across trial sites
• Protocols detail specific parameters for plasma generation and application

Treatment frequency

• Determine optimal intervals between plasma therapy sessions
• Consider acute vs chronic conditions in scheduling treatments
• Evaluate single vs multiple treatment courses
• Account for potential cumulative effects or treatment resistance

Duration of therapy

• Establish appropriate length of individual plasma treatment sessions
• Define overall duration of treatment course (days, weeks, months)
• Consider disease progression and expected time to observe therapeutic effects
• Plan for long-term follow-up to assess durability of treatment outcomes

Plasma device specifications

• Define technical parameters for plasma generation (voltage, current, gas composition)
• Specify treatment area and depth of plasma penetration
• Standardize distance between plasma source and treatment site
• Establish quality control measures for consistent plasma output across devices
  • Regular calibration and maintenance protocols