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Rare Metabolic Disease Clinical Trial Solutions

Rare Metabolic Research Experience

With enterprise-wide rare metabolic disease clinical capabilities and experience, PPD is at the forefront of clinical research in rare metabolic disorders.

We attack rare metabolic disorder trial challenges head-on by embedding specialized and flexible development strategies that include a rigorous assessment of feasibility, robust training and supportive relationships through site network collaborations with patient advocacy groups, patient concierge services and more.

PPD has extensive experience in many rare metabolic indications, including:

  • Mucopolysaccharidosis (I, II, III)
  • Hyponatremia
  • Hypoparathyroidism
  • Lysosomal acid lipase deficiency
  • Gaucher disease
  • Glycogen storage disease type I/II
  • Ornithine transcarbamylase deficiency
  • Fabry disease
  • Inborn errors of metabolism
40+ rare metabolic and endocrine studies
Rare disease and pediatric expertise
Gene therapy experience

On Target: Complex Glycogen Storage Disease Study Enrollment and Retention Success Case Study

Despite a limited patient population, a study with a complex schedule of assessment (requiring both overnight stays and home health care visits), and implementation of CGM and lab testing to confirm eligibility, PPD developed and executed a strategy for a Phase I-II Glycogen Storage Disease Type IA trial that:

  • Enhanced CGM compliance
  • Kept enrollment on track
  • Maintained 100% retention in the study

What Can You Expect? Agile, Novel Solutions for Site and Patient Access

  • Seamless collaboration with PPD pediatricians, geneticists and rare disease experts to understand unique and specific medical or logistical issues that may arise in patients with each diagnosis and trial
  • Close collaboration with patient advocacy groups to represent the patient perspective in protocols, enhance awareness in the community, assist in patient outreach and provide connections to key opinion leaders
  • Removal of barriers to participation for hard-to-find patients by maximizing study decentralization through remote data collection and procedures and support for patient cross-border travel
  • Teams experienced with cutting-edge gene therapies, including clinical execution and site/team training, regulatory strategy and evidence planning
  • Utilization of feasibility tools that harness an extensive database to determine patient location, numbers, types of providers and your competitive landscape
  • Passionate teams dedicated to individual patients as well as their caregivers and advocacy groups