Clinical Data Management and Statistical Programming Support for a Phase 2b Oncology Trial

Case Study Introduction

Quanticate supported a biotechnology sponsor by helping them generate decision-ready evidence for their Phase 2b Oncology clinical trial. The study was evaluating an investigational mitogen-activated protein kinase 1/2 inhibitor (MEK1/2 inhibitor) in adult and pediatric patients with Neurofibromatosis type 1 (NF1)-associated inoperable plexiform neurofibromas (PNs).

The sponsor was short on internal resources and biometric expertise, and therefore came to Quanticate to be their in-house function for Clinical Data Management and Statistical Programming.

Objectives and Key Challenges

The sponsor has several objectives for the study:

Quantify tumour response using sensitive, reproducible imaging-based measures suitable for irregular, complex tumours.
Evaluate efficacy and safety across pediatric and adult cohorts within one coherent analytical framework.
Integrate patient-reported outcomes (PROs) to complement imaging findings with real-world symptom impact.
Produce clear, regulator-ready and publication-quality outputs, suitable for confident decision-making.
Produce CDISC submission package for regulatory approval requests and integration into ISS/ISE.

The sponsor was facing several challenges:

- Measuring response reliably: Traditional criteria, such as Response Evaluation Criteria in Solid Tumors (RECIST), can be poorly suited to irregular, diffuse PNs, increasing the risk of inconsistent assessments and therefore requiring careful presentation in statistical analysis.
- Heterogeneity in a rare disease: NF1-associated PNs vary by age, tumour location, baseline severity, and growth patterns, creating analytical complexity and subgroup interpretation risk.

Long-term safety considerations: Chronic conditions may require prolonged treatment, heightening the importance of ongoing safety surveillance and clear tolerability characterisation.
Capturing meaningful benefit: Tumour shrinkage is important, but sponsors also need to understand changes relate to symptom relief and daily function.
Missing data and attrition: Longer trials and rare disease populations increase the likelihood of incomplete follow-up, which can bias results if not handled carefully.

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Strategic Approach and Tailored Solutions

Quanticate partnered closely with the sponsor’s clinical and operational teams, as well as acted as their clinical data management and statistical programming team to design an analytical strategy aligned to study objectives and delivery timelines.

Endpoint strategy built for PNs: Quanticate supported definition and implementation of objective response metrics aligned to volumetric tumour assessment, including an objective response rate (ORR) threshold based on percent reduction in tumour volume.
Advanced imaging analytics: The trial used volumetric magnetic resonance imaging (MRI) to quantify tumour volume changes. Quanticate ensured the downstream programming and analysis approach could accommodate longitudinal volumetric data and support interpretation alongside clinical outcomes.
Cohort-aware efficacy and safety analytics: Quanticate implemented cohort-specific analyses and integrated views to help the sponsor compare patterns across age groups without overgeneralising. Safety outputs were structured to support routine monitoring and clear communication of tolerability trends.
Patient-reported outcomes integration: Quanticate supported analysis of validated patient-reported outcome (PRO) measures tailored to the NF1 population, including instruments capturing pain interference and quality-of-life impact.
Handling variability with appropriate models: Quanticate applied approaches suitable for longitudinal trial data, including generalized linear models (GLMs), mixed-effects models, and time-to-event methods such as Kaplan–Meier analyses, to describe timing and durability of response.
Missing data strategy and robustness checks: Quanticate implemented multiple imputation and sensitivity analyses to strengthen confidence in conclusions despite incomplete follow-up.
Transparent, audit-ready delivery: Quanticate provided statistical programming and quality-controlled deliverables with clear traceability and consistent documentation to support governance and external reporting.

Successful Client Outcomes

With Quanticate’s support, the sponsor moved from uncertainty to evidence with a clearer view of benefit and risk in NF1-associated inoperable PNs.

Produced imaging-driven tumour response analyses suited to complex tumour morphology.
Delivered clear efficacy and safety interpretation across pediatric and adult cohorts, supporting internal decision-making.
Enabled a more patient-centred understanding of outcomes by linking clinical measures to symptom impact.
Strengthened confidence in results through fit-for-purpose modelling and structured sensitivity testing.
Improved the usability of outputs for leadership updates, scientific dissemination, and onward development planning.

Clinical Data Management and Statistical Programming Support for a Phase 2b Oncology Trial

Case Study Introduction

Objectives and Key Challenges

Looking for clinical trial reporting support in rare oncology studies?

Strategic Approach and Tailored Solutions

Successful Client Outcomes

Conclusion

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