Table 1 A framework of fundamental elements in rare disease N-of-1 trials
From: A framework for N-of-1 trials of individualized gene-targeted therapies for genetic diseases
Introduction | Background Information Nonclinical and clinical data Rationale Risk-Benefit Assessment |
Objectives and Endpoints | Primary Secondary Exploratory |
Study Design and Dose Rationale | Overall design Treatment goals Dose rationale End of study definition |
Study Population | Inclusion criteria Exclusion criteria |
Treatment | Study drug information Route of administration Dose escalation Dose modifications Stopping rules |
Study drug | Preparation, handling, storage, accountability Product storage and stability Preparation of study drug |
Study Procedures | Procedures for screening and enrollment - Study assessments and procedures (include all assessments with description) |
Safety | Definition of adverse events and serious adverse events Assessment of severity of adverse events Assessment of AE relationship to study drug Reporting responsibilities |
Statistical considerations | Pre-specify frequency of outcomes measures and select appropriate time-series methods Pre-design any composite outcome measures Consider combining data across similar N-of-1 trials |
Data handling and record keeping | Source documents/case reports forms Study records retention Data sharing |
