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🔬 Exploratory IND Template for VPT-101 Micro-dose Study

⚠️ Important Disclaimers:
  • This is a template only - Must be customized for specific applications
  • Legal review required - Consult with regulatory experts before submission
  • FDA guidance - Follow current FDA guidelines and requirements
  • Professional assistance - Consider hiring regulatory consultants for complex submissions

📋 FDA Guidance Compliance

This template follows FDA guidance for Exploratory IND studies (14-day review timeline).

🔬 Study Design: Single IV Micro-dose (0.01 mg kg⁻¹)

Primary Objectives

  1. Vault PK Assessment: LC-MS/MS quantification of unique peptide tag
  2. Proof-of-Mechanism: Real-time Raman spectroscopy of serum PET fragments
  3. Safety Profile: Sub-pharmacologic dose safety evaluation

Study Population

  • Sample Size: 6 healthy volunteers
  • Age Range: 18-65 years
  • Health Status: No known plastic exposure, normal liver/kidney function
  • Exclusion Criteria: Pregnancy, recent plastic surgery, known PET allergies

📊 Endpoints

Primary Endpoints

  • Vault PK: Plasma concentration-time curve (0-24h)
  • PET Degradation: Raman spectroscopy at 0, 2, 4, 8, 12, 24h
  • Safety: Vital signs, CBC, chemistry panel, urinalysis

Secondary Endpoints

  • Biodistribution: Tissue sampling (optional biopsies)
  • Metabolite Analysis: LC-MS/MS of degradation products
  • Immune Response: Cytokine panel, complement activation

💉 Dosing Protocol

Day 1: Baseline measurements
Day 2: Single IV infusion (0.01 mg kg⁻¹ over 30 min)
Days 3-8: Daily monitoring
Day 14: Final safety assessment

🛡️ Safety Monitoring

  • Real-time: Continuous ECG, pulse oximetry during infusion
  • Daily: Vital signs, adverse event assessment
  • Weekly: Laboratory safety panels
  • Emergency: Immediate discontinuation protocol

📈 Statistical Analysis

  • Sample Size: 6 subjects (pilot study)
  • Primary Analysis: Descriptive statistics
  • Safety Analysis: Incidence of adverse events
  • PK Analysis: Non-compartmental modeling

⏰ Regulatory Timeline

  • IND Submission: Day 0
  • FDA Review: 14 days (Exploratory IND)
  • Study Start: Day 15 (if approved)
  • Interim Report: Day 30
  • Final Report: Day 60

📋 Required Documentation

Pre-IND Meeting Request

  • Briefing document (5 pages max)
  • Study protocol summary
  • Toxicology data summary
  • Manufacturing information

IND Application Components

  1. Form FDA 1571
  2. Form FDA 1572 (Investigator's Statement)
  3. Protocol: Complete study protocol
  4. Chemistry, Manufacturing, and Controls (CMC)
  5. Nonclinical Pharmacology/Toxicology
  6. Previous Human Experience
  7. Additional Information

CMC Requirements

  • Drug Substance: Vault nanoparticle characterization
  • Drug Product: Formulation, stability, sterility
  • Manufacturing: GMP-compliant production
  • Quality Control: Release specifications

Toxicology Package

  • Acute Toxicity: Single-dose studies (2 species)
  • Repeat-Dose: 7-day studies (1 species)
  • Genotoxicity: Ames test, micronucleus
  • Safety Pharmacology: Cardiovascular, respiratory, CNS

⚠️ Risk Mitigation

Known Risks

  • Allergic Reaction: Pre-treatment with antihistamines
  • Infusion Reaction: Slow infusion rate, monitoring
  • Off-target Effects: Negative control vaults included

Unknown Risks

  • Long-term Effects: Limited by micro-dose design
  • Immune Response: Monitoring for cytokine release
  • Biodistribution: Tissue sampling for analysis

✅ Success Criteria

  • Safety: No serious adverse events
  • PK: Detectable vault levels in plasma
  • Mechanism: PET degradation confirmed by Raman
  • Regulatory: FDA approval for full-dose study

🚀 Next Steps

  1. Pre-IND Meeting: Schedule with FDA
  2. Manufacturing: Scale-up GMP production
  3. Toxicology: Complete required studies
  4. IND Submission: Submit complete package
  5. Study Initiation: Begin recruitment

📞 Contact Information

  • Sponsor: VPT-101 Consortium
  • Principal Investigator: [To be determined]
  • Regulatory Affairs: screball7605@aol.com
  • FDA Contact: [Division of Clinical Trial Design and Analysis]
📋 Complete Template: This is a condensed version of the template. For the complete exploratory IND template with all sections and detailed guidance, download the full Markdown version.
📥 Download Complete Template (Markdown)

🔗 Additional Resources

💬 Community Support

Need help with regulatory submissions? Connect with our community:

  • Email: screball7605@aol.com
  • Matrix: #vault-detox:matrix.org
  • Documentation: VPT-101 Docs
⚠️ Disclaimer: This template is provided under CC0 1.0 Universal license. Modify as needed for your specific study requirements. Always consult with qualified regulatory professionals before submitting to any regulatory authority.