Selection, Collection & Reporting of Clinical Trial Outcomes
Module Code 32203
Assessment Brief 2024/2025 – Protocol Sections
Submission format:
Please create a Microsoft Word document using font size 11 or 12. If you are unable to create a Word document, please create a pdf file.
The name of your document should use the following format ‘SCRO STUDENT-ID’, so if your student ID was 1234567, your filename would be ‘SCRO 1234567’.
Include your Student ID on each page but DO NOT include your name.
Word limit: 2500 words
Assessment weighting: 100% of module mark
Submission deadline: 10th February 2025, 12:00 (midday)
Submission is via CANVAS (further details will be provided on CANVAS)
Task: To write expanded protocol sections relevant to the selection, collection and reporting of clinical trial outcomes.
Topic: Treatment for adolescent idiopathic scoliosis.
Scoliosis is an abnormal sideways curvature of the spine, creating an S-shaped or C-shaped spine. Patients are diagnosed with scoliosis if they have one or more angles of lateral curvature (Cobb angle) of greater than 10° (see figure below).
Approximately 80% of patients with scoliosis have idiopathic scoliosis, where the cause is unknown. Most idiopathic scoliosis cases develop and are diagnosed amongst adolescents (10–18 years) and until the skeleton stops growing, the curvature may become worse.
Scoliosis causes changes in posture, muscle weakness, reduced mobility and in more severe cases, chronic pain and problems with breathing. For many individuals, these symptoms lead to reduced physical function and poor quality of life. It can also have a significant psychological impact, especially amongst adolescents, affecting an individual’s body image, self-esteem and confidence.
Conservative treatment options aim to slow or prevent progression and avoid the need for spinal surgery. Treatment options throughout adolescent development, include monitoring (with regular x-rays every six months or yearly, depending on severity), bracing (wearing a rigid plastic brace around the torso) and physiotherapy (low impact strengthening and stretching exercises).
Whilst there is some evidence of a benefit of bracing in terms of progression and the need for surgery, bracing requires patients to wear their (individually adapted and curve specific) brace for 23 hours per day, until skeletal maturity and consequently, adherence is often poor reducing any potential beneficial effects. Poor adherence is likely to be due to physical restriction and comfort and negative psychological impact from the brace, particularly related to the school environment.
Scoliosis specific exercises are individually adapted and curve specific and delivered by a physiotherapist. Once patients are familiar with the exercises they can also be performed at home. There is some evidence that performing such exercises regularly throughout adolescent development, can improve pain and muscle strength and may help reduce any back pain. It is not yet clear, however, whether scoliosis specific exercises can prevent progression or improve scoliosis, and they are not recommended by all specialists.
A research team plan to conduct a definitive randomised controlled trial to determine the effectiveness of regular scoliosis specific exercises compared to bracing in adolescents (10-15), diagnosed with mild to moderate scoliosis. All patients will have an x-ray and meet with a consultant every six months until skeletal maturity, as part of their usual clinical care. Those allocated to the brace will also be seen by an orthotic expert every 3 months for any adjustments that need to be made to the brace. Those allocated to the exercise group will attend weekly physiotherapy appointments for eight weeks, then once a month for a further four months. After the initial six months of treatment, they will attend a physiotherapy appointment every six months to ensure they are still completing the exercises correctly and to re-tailor the exercises if necessary. Patients will be required to perform. 20 minutes of exercises at home 4-5 times for the duration of the study or until skeletal maturity.
As an outcome expert, the team have asked you to help write a protocol for the proposed trial.
Reporting structure and allocation of marks:
The assessment should be written in the style. of an essay, using the sub-headings below. Please ensure that you write the requested detail in the correct sections. Take note of the allocation of marks below, to guide you as to how much to write in each section.
Title: Descriptive title identifying the study design, population, and interventions.
Objectives: In addition to listing the trial’s objectives, you should provide a brief, but clear rationale for your objectives and any hypotheses you are testing. If you draw information or evidence from additional resources, you should cite your sources appropriately. However, the information included in this assessment brief should provide you with sufficient rationale.
Methods – Population and Interventions: Describe your target population for your research question (detailed clinical inclusion/exclusion criteria are not required). For the intervention and comparator, we do not require any additional information than that included in the information above.
Methods - Outcomes: This section should include a full description of the chosen primary, secondary and any other trial outcomes. It is very important to explain the rationale for the choice of each outcome, with justification drawn from the wider literature, and reference to resources such as the COMET Initiative database (make sure that any additional resources are properly referenced). Where several potential outcomes are considered (for example, for the primary outcome), there should be critical discussion around strengths and weaknesses of each, with clear illustration of which elements underpinned the final decision.
For each outcome, include a definition (if needed), the analysis metric (e.g., final value, change from baseline, time to event), and the time point(s) for each outcome with justification.
The choice of outcome measurement instruments is NOT required in this section.
If relevant, you should discuss any potential issues that the chosen outcomes may have on the validity, generalisability, or interpretation of the trial (e.g., composite or surrogate outcomes).
Methods - Data Collection Methods: This section should succinctly present plans for assessment and collection of the trial outcome data, including a full description of outcome measurement instruments (e.g., questionnaires, laboratory tests) along with their reliability and validity, if known. Who are the outcome assessors? (E.g., patient-reported, clinician-reported etc.), are they blind to treatment allocation and how will the data be collected? The section should also include any related processes to promote data quality (e.g., duplicate measurements, training of assessors).
Again, there should be critical discussion and justification around the chosen methods (with mention of other measurement instrument tools that were considered) and any potential impact of your choices on the conduct, validity, and interpretability of the trial (e.g., subjective outcome measures, burden on patients).
Analyses and Reporting: You are not required to include specific analytical methods to be used, but should include, for each outcome, the method of aggregation (that is, how the data will be summarised in each group e.g., mean, proportion), and your estimate of treatment effect(s) to be reported (e.g., difference in means, risk ratio). You should also include a minimal clinically important difference (MCID) for your primary outcome, with justification. If an MCID is not available, you should suggest a sensible estimate and describe how this would be confirmed before the start of the trial.
Please also provide information of how results will be reported to ensure transparency and wide dissemination.
You should refer to the SPIRIT Statement, SPIRIT-Outcomes and SPIRIT-PRO guidelines of what detail should be included throughout.
The assessment will be marked out of a possible 100 marks. Marks will be awarded in the following proportions:
Title, objectives, population, interventions 8 marks
Trial Outcomes 38 marks
Data Collection Methods 32 marks
Analyses & Reporting 12 marks
Overall Presentation 10 marks
(including logical flow, correct use of
grammar/spelling and referencing)
Maximum marks will be given based on the following criteria:
(1) Clear description and definition of the outcomes of the trial
(2) Justification and rationale for each chosen outcome, supported by a critique of the wider literature. Consideration of the strengths and weaknesses of each outcome considered/selected. There should be critical discussion and justification around the chosen outcomes drawing upon appropriate literature.
(3) Identification of, and justification for appropriate data collection approaches and statistical/reporting considerations. Consideration of approaches that may improve trial conduct, optimize data quality and interpretation of trial findings. There should be critical discussion and justification around the chosen outcome measurement instruments drawing upon appropriate literature.