Conference Day 1
Tuesday 30th September 2025
7:30 AM Registration & Coffee
8:20 AM Chair’s Opening Remarks
Highlighting the Importance of the Patient Voice in Rare & Genetic Kidney Disease to Improve Patient Engagement & Clinical Trail Enrolment
8:30 AM Making Clinical Trials a Standard of Care in Paediatric Rare Kidney Disease to Empower Patients & Improve Enrolment
Synopsis
- Discussing successful strategies to democratize clinical trial awareness such as navigation programs and user-friendly databases that centralize trial information for clinicians and patients
- Leveraging lessons learnt from the oncology field to make trials a routine part of patients care and foster a culture where trial consideration is standard in the rare kidney disease community
- Focussing on innovative solutions to enhance clinical trial matching such as centralized platforms, longitudinal databases and the emerging ‘hub and spoke’ model to improve access and accelerate recruitment
9:00 AM Panel Discussion: Ensuring the Patient Voice is Heard to Work Towards Successful Collaboration Between Industry, Academia, Clinicians and Patients
Synopsis
- Elevating the lived experience of patients to drive relevant research and diagnosis
- Developing strategies to prioritize patient preferences in trial design, care pathways and policy advocacy Outlining the role that patient advocacy can have in articulating the needs and priorities of the rare and genetic kidney
Reaffirming Past, Present & Future Challenges in a Changing Regulatory Landscape to Promote Alignment in the Kidney Disease Community
9:30 AM Clinical Trial Considerations in Cystinuria to Support Regulatory Approvals in the United States Vs Europe
Synopsis
- Highlighting differences between the US and Europe in conducting clinical trials in rare kidney diseases
- A case study focused on Cystinuria and dRTA
- Discussing the regulatory frameworks governing development and implementation for therapeutics in rare kidney disease
10:00 AM Session reserved for MediBeacon
10:30 AM Speed Networking
Synopsis
Put a face to a name – this session is the perfect opportunity to get face-to-face time with key opinion leaders, leading companies, and innovative researchers in the rare and genetic nephrology field. Establish meaningful connections to build upon for the rest of the conference and gain individual insight beyond the papers and press releases into the pioneering research and technique applications.
11:00 AM Morning Break & Networking
11:30 AM Proteinuria: A Key Endpoint in Rare Glomerular Diseases
Synopsis
In recent years, proteinuria has become regulatory approvable endpoint for new therapeutics for rare glomerular diseases and considerations for drug developers include:
- Considerations of measuring urinary albumin vs. total protein
- Timed 24-hour proteinuria or albuminuria excretion vs. spot urinary protein-to-creatine ratio
- Implications of varying recommendations in KDIGO and other nephrology practice guidelines
12:00 PM Panel Discussion: Defining Surrogate Endpoints for Rare Kidney Disease
Synopsis
- Analyzing lessons learned regarding surrogate endpoints for rare and genetic kidney diseases
- Examining the application of PLA IIR as a potential endpoint for Membranous Nephropathy
- Outlining the unique considerations relating to demonstrating efficacy in transplant patients with disease recurrence
12:30 PM Lunch & Networking
What’s Next in the Renal Renaissance? Exploring the Latest Clinical Developments to Stay Ahead of the Curve
1:30 PM Hidden Statistical Gems: Evaluating Statistical Models to Minimize Sample Sizes in Rare Diseases
Synopsis
- Discussing flexible trial designs to reduce overall number of patients needed in rare disease trials
- Exploring statistical methods for effectively incorporating RWD to minimize placebo groups
- Delve into the application of sophisticated statistical modeling techniques such as disease progression models, PK/PD modeling, and machine learning to more precisely predict drug effects, stratify patient responses, and enable smaller, more targeted clinical trials in genetically heterogeneous rare diseases
2:00 PM Revolutionizing Clinical Trial Design for IgA Nephropathy to Overcome Challenges Associated with Slow Disease Progression
Synopsis
- Exploring surrogate endpoints beyond GFR to capture changes in disease activity earlier in progression
- Advancing innovative trial designs with flexible durations and interim analyses to solidify the evaluation of therapeutic efficacy
- Applying emerging and established biomarkers to identify a variety of patient subgroups for targeted enrolment resulting in small, efficient trials
2:30 PM Navigating the Key Complexities in Clinical Trials for Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Synopsis
- Explore the spectrum of ADPKD’s renal and extra-renal complications, alongside the unpredictable nature of disease progression, which complicates diagnosis and patient management
- Address the practical challenges in optimizing current treatments like tolvaptan, focusing on adherence, managing side effects, and patient selection for long-term benefit
- Beyond Kidney Function: Discuss the significant impact of ADPKD on patient quality of life, including pain and psychosocial burdens, underscoring the critical need for integrated, multidisciplinary care
3:00 PM Delving into Clinical Trial Design for APOL1-Mediated Kidney Disease
Synopsis
- Updates on the progression of the AZD2373 for the treatment of APOL1-Mediated kidney disease
- Delving into successful phase 2 study design in rare renal diseases
- Unpacking the mechanism of action for mineralocorticoid receptor agonists
3:30 PM Panel Discussion: Lessons Learnt from Successful Clinical Trials in IgAN & C3G to Inform Successful Clinical Design & Development in Wider Rare & Genetic Kidney Diseases
Synopsis
- Advancing understanding of disease pathophysiology and biomarker identification to guide the development of therapies with distinguished mechanism of actions
- Discussing suitable design of placebo arm and standard of care for rare diseases
- Examining the importance of integrating patient-reported outcomes, quality of like measures and comparative effectiveness research early in clinical development to demonstrate meaningful differentiation
4:00 PM Afternoon Refreshments & Poster Session
Synopsis
Immerse yourself in an engaging and informal session, join your peers in a relaxed atmosphere that encourages meaningful conversations and discussions. Explore a range of exciting poster presentations and showcase your own developments in the Rare & Genetic Kidney Therapeutics world. Don’t miss out on the chance to submit your own posters and get ready to connect, learn, and present. To submit your poster please contact info@hansonwade.com
4:30 PM Real-World Use of Lupkynis to Treat Lupus Nephritis & Real-World Clinical Practice
Synopsis
- Highlighting the fundamental differences in the standards and requirements for clinical outcome assessments used for regulatory approval versus those used in routine clinical care
- Highlighting the challenges in applying traditional COA methodologies to small, heterogenous populations seen in rare disease trials and pushing for tailored approaches
- Focusing on strategies to translate advanced academic COA methodologies into practical, regulator-accepted tools for effective drug development in rare kidney diseases
5:00 PM Panel: Discussing the Evolving Role of Biomarkers for Therapeutic Differentiation in a Crowded Landscape
Synopsis
- Exploring the increasing need for biomarkers that can meaningfully differentiate the clinical impact of emerging renal therapies for rare kidney diseases
- Discussing the repurposing of established biomarkers in new contexts to reveal subtle and clinically relevant differences between new treatments
- Addressing opportunities and challenges in utilizing novel biomarkers in rare kidney disease trials to demonstrate clinical relevance
- Understanding the validation journey needed to gain acceptance within the community and regulatory agencies