Contents

• 2. SYNOPSIS
• 4. GLOSSARY OF ABBREVIATIONS AND TERMS
• 5. ETHICS
  • 5.1. Institutional Review Board (IRB)
  • 5.2. Ethical Conduct of the Study
  • 5.3. Participant Information, Consent, and Assent
• 6. INVESTIGATORS AND STUDY ADMINISTRATIVE STRUCTURE
  • 6.1. Principal Investigators
  • 6.2. Administrative Structure
• 7. INTRODUCTION
  • 7.1. Background Information
  • 7.2. Scientific Rationale
  • 7.3. Potential Risks and Benefits
  • 7.4. Best Pharmaceuticals for Children Act
  • 7.5. Data Sources
  • 7.6. Structure for this Clinical Study Report
• 8. OVERALL OBJECTIVES
  • 8.1. Primary Aim
  • 8.2. Secondary Aims
• 9. INVESTIGATIONAL PLAN
  • 9.1. Overall Design and Plan Description
  • 9.2. Discussion of Study Design
  • 9.3. Selection of Study Population
  • 9.4. Treatments
  • 9.5. Efficacy and Safety Variables
  • 9.7. Statistical Methods and Determination of Sample Size
  • 9.8. Changes in the Conduct of the Study or the Planned Analyses
• 10. STUDY PARTICIPANTS
  • 10.1. Disposition of Participants
  • 10.2. Protocol Deviations
• 11. PHARMACOKINETIC EVALUATION
  • 11.1. Dosing and PK Sampling
  • 11.2. Data Sets Analyzed
  • 11.3. Demographic and Other Baseline Characteristics
  • 11.4. Measurement of Treatment Compliance
  • 11.5. Pharmacokinetic Analysis and Results
  • 11.6. Other Analyses
  • 11.7. Conclusions
• 12. SAFETY EVALUATION
  • 12.1. Demographic and Other Baseline Characteristics
  • 12.2. Extent of Exposure
  • 12.3. Adverse Events
  • 12.4. Deaths, Other Serious Adverse Events, and Other Significant Adverse Events
  • 12.5. Clinical Laboratory Evaluation
  • 12.6. Other Observations Related to Safety
  • 12.7. Safety Conclusions
• 13. DISCUSSION AND OVERALL CONCLUSIONS
  • 13.1. Primary Endpoint: PK
  • 13.2. Secondary Endpoint: Safety
  • 13.3. Overall Conclusions
• 14. TABLES, FIGURES, AND GRAPHS REFERRED TO BUT NOT INCLUDED IN THE TEXT
  • 14.1. Study Participants
  • 14.2. Dosing and Pharmacokinetic Data
  • 14.3. Safety Data
  • 14.4. Figures
  • 14.5. SAE Narrative
• 15. REFERENCE LIST
• 16. APPENDICES (list of contents)

Study data is available on the DASH repository at hhttps://dash.nichd.nih.gov/study/228258

This study was registered on ClinicalTrails.gov at https://clinicaltrials.gov/ct2/show/NCT01744730

This study was conducted in compliance with Good Clinical Practices (GCP) as outlined in the International Conference on Harmonization (ICH), including the archiving of essential documents.

All unpublished information contained within this report is confidential and the sole property of the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).