Rare Nephrology CRO Services
Accelerating Rare Renal Development Through Targeted Patient Access, Precision Diagnostics and Deep Expertise
At Worldwide, we deliver specialized rare nephrology expertise across the full spectrum of complex renal indications, from IgA nephropathy and glomerulonephritis to rare and emerging renal disease. Our renal team’s experience over the past five years spans 26 studies in 16 indications, more than 1,250 sites, and approximately 8,000 patients, including the pivotal studies behind two rare renal therapy approvals.
Why Rare Nephrology Demands Specialized Support
Thoughtful planning, deep therapeutic expertise, and operational flexibility for small patient populations and complex renal endpoints
We bring together proven, high-performing renal site networks, central pathology that protects diagnostic accuracy end to end, and deep therapeutic expertise in rare renal disease, to help our partners de-risk development and reach decisions with confidence.
De-Risking Timelines Through Proven Strategies
In rare renal diseases, early regulatory engagement is critical, as decisions regarding endpoints, biomarker validation, and designation strategies often shape the speed, efficiency, and ultimate success of the development program
DiagnosticPrecision for Endpoint Integrity
Biopsy confirmed eligibility and tight serological criteria mean diagnostic consistency is designed into the protocol and maintained globally.
Deep Therapeutic and Program Expertise for Predictable Enrolment
When the globally eligible patient population is limited and disease activity is intermittent, enrollment strategies must be designed around real-world patient presentation patterns rather than traditional Phase III recruitment models
Phases, Indications, and Modalities
Specialized Rare Nephrology CRO Across Phases, Indications & Modalities
Rare glomerular disease
- Lupus Nephritis
- IgA Nephropathy (Berger’s Disease)
- C3 Glomerulonephritis (C3G)
- IC-MPGN
- Focal Segmental Glomerulosclerosis (FSGS)
- Atypical Hemolytic Uremic Syndrome (aHUS)
Inherited and metabolic renal disease
- Polycystic Kidney Disease (PKD and ADPKD)
- Primary Hyperoxaluria Type I
- Alport Syndrome
- X-Linked Hypophosphatemia
- Renal Stones and Calculi
Chronic and transplant
- Chronic Kidney Disease
- End-Stage Kidney Disease
- Diabetic Nephropathy
- Kidney Transplant Rejection and Post-Transplant Recurrence
Renal and urogenital oncology
- Kidney Cancer and Metastatic Renal Cell Carcinoma
- Bladder Cancer, including non-muscle-invasive
Services & Capabilities
What We Bring to Rare Renal Programs
- Protocol Design & Scientific Strategy
- Renal Endpoint Management
- Central Pathology Review
- Regulatory Strategy & Designation Support
- Global Site Selection & Activation
- Patient Identification & Enrollment
- International Patient Travel & Coordination
- Biomarker Strategy & Bioanalytical Services
- Pediatric Nephrology Delivery
- Kidney Transplant Programs
- Sample Collection & Patient Logistics
Central Pathology Review
Biopsy interpretation and eligibility documentation reviewed centrally across light microscopy, electron microscopy and immunofluorescence, so diagnostic accuracy holds from trial entry through endpoint analysis.
Rare Disease Site Network & Patient Access
Renal disease team experience over the past five years spans 1,250+ sites and 16 indications, built around disease prevalence and long-standing investigator relationships that keep enrollment on target in small patient populations.
Regulatory Strategy & Scientific Partnership
Senior nephrology and translational medicine experts supporting KOL engagement, designation strategies, and regulatory meetings from protocol development through submission
Pediatric Nephrology Delivery
A dedicated pediatric nephrology network of more than 70 sites across 13 countries, with a pediatric nephrologist on the medical monitoring team.
Kidney Transplant Programs
A dedicated transplant site network and delivery experience spanning transplant rejection, post-transplant recurrence and longitudinal registry work.
Sample Collection & Patient Logistics
Non-spoken instructional videos for urine collection, male and female collection containers, cooler backpacks for confidential transport, and at-home sample pickup where it can be arranged.
Why Worldwide for Rare Nephrology Trials
Proven Delivery in Ultra-Rare Renal Indications
Pivotal Phase III Delivery in Adult Lupus Nephritis
Worldwide delivered the Phase III study supporting the first FDA-approved oral therapy for adult lupus nephritis across 27 countries and 240 sites.
Ultra-Rare Enrollment Delivered to Plan
A global Phase III in C3G and IC-MPGN across 19 countries and 123 sites finished enrollment three weeks early at 130% of target, and led to FDA approval in July 2025 and European approval in 2026 for patients 12 and older.
Enrollment Designed Around Disease Activity
Urine dipsticks for early flare detection, pre-screening chart review and language-matched referral coordination keep eligible patients ready to screen. Two IgA nephropathy programs closed enrollment six weeks and six months ahead of target.
Specialized Rare Nephrology Leadership
Meet Our Rare Nephrology Experts
Our renal portfolio is co-chaired by two executive directors with a combined 16 years in rare renal disease, supported by medical monitors who practise in nephrology, including a pediatric nephrologist.
Partnership & Approach
Senior Partnership Across the Life of Your Rare Renal Program
Sponsors work directly with the senior scientific, medical and project leaders who co-chair our renal portfolio, and who have the authority to decide without routing it upward. Sponsors know their asset better than anyone; what we bring is a team that has taken rare renal programs the whole way and stays with them.
The Track Record Behind the Team
Thirty urogenital studies over the past five years, across 18 indications and 47 countries, including two rare renal therapies taken through to approval.
Partnership in first LN oral therapy
27
Countries
240
Sites
358
Patients
The clinical trial that brought the first FDA-approved oral therapy for adult lupus nephritis to market, run by Worldwide.
Five years of urogenital delivery
30
Sites
18
Indications
47
Countries
10,580
Subjects Enrolled
An ultra-rare indication taken to approval
19
Countries
123
Sites
130+
of Enrollment Target
Enrollment closed three weeks ahead of schedule on a global Phase III in C3G and IC-MPGN, leading to FDA approval in July 2025 and European approval in 2026 for patients 12 and older.
Frequently Asked Questions
Your Questions Answered
We’ve answered the questions sponsors ask most often about rare renal programs. If you have others, our team is here to help.
Our rare renal experience spans lupus nephritis, IgA nephropathy, C3 glomerulopathy and IC-MPGN, FSGS, atypical HUS, polycystic kidney disease, primary hyperoxaluria type I, Alport syndrome, X-linked hypophosphatemia, renal stones, chronic and end-stage kidney disease, diabetic nephropathy, kidney transplant rejection and post-transplant recurrence, and renal and urogenital oncology, across Phase I through approval.
Worldwide’s renal disease team experience over the past five years spans 26 studies, 16 indications, more than 1,250 sites, and approximately 8,000 patients. The relationships are built around disease prevalence and established referral pathways, which is what makes fast activation possible on an ultra-rare indication.
Central pathology review is integrated at the protocol level, covering light microscopy, electron microscopy and immunofluorescence. Biopsy interpretation and eligibility documentation are managed consistently across all sites, so the trial-entry diagnosis holds through regulatory submission. On ultra-rare indications that are often misdiagnosed on biopsy, that consistency is what protects the primary endpoint.
Our nephrology medical and scientific team tracks FDA, EMA, and MHRA guidance closely across rare renal indications. Endpoint strategy, designation opportunities including FastTrack, Breakthrough, and Orphan Drug, and KOL engagement go into protocol design from the start.
Worldwide maintains long-standing relationships with nephrology KOLs and indication-specific investigator networks across rare renal indications, alongside partnerships with patient advocacy groups for education, awareness and referral. These relationships are cultivated over time, so they are in place before a program needs them.
24-hour and first morning void collections carry most of the dropout risk in rare renal trials. We support them with non-spoken instructional videos, male and female collection containers, cooler backpacks for confidential transport, at-home pickup where it can be arranged, and home health, travel concierge and patient navigator services alongside.
Yes. We maintain a dedicated pediatric nephrology network of more than 70 sites across 13 countries, and a pediatric nephrologist sits on the medical monitoring team with 15 years in clinical practice and 20 in rare renal research.
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