Designed novel endpoint strategies with multidomain endpoints evaluated with the Global Statistical Test to assure convergence at reasonable sample sizes
- Lysosomal Storage Diseases:
- Pompe disease
- Successfully designed three-part randomized control, sequential-cohort, adaptive design study in patients with Pompe Disease to assess safety and efficacy of ex vivo lentiviral vector-mediated HSC gene therapy.
- Achieved global regulatory approvals for Master Protocol registrational trial design with multidomain responder index (multicomponent primary endpoint).
- Gaucher’s disease
- Mentored client executive leadership on navigating complex global regulatory challenges
- Designed registrational clinical trial in Gaucher Type 3 patients (neuronopathic Gaucher’s) to assess safety and efficacy of autologous hematopoietic stem cell (HSC)-based gene therapy.
- Synthesized multicomponent endpoints to address disease heterogeneity and achieved regulatory body acceptance of pivotal study design aligned with PFDD guidance.
- Acquired global regulatory alignment on biostatistical framework integrating trial simulations for sample size, I/E and endpoint optimization with disease progression modeling.
- Pompe disease
Biotechnology Leadership
Mentored client executive leadership on navigating complex global regulatory challenges
Identify Treatment Populations
Designed registrational clinical trial in Gaucher Type 3 patients (neuronopathic Gaucher’s) to assess safety and efficacy of autologous hematopoietic stem cell (HSC)-based gene therapy.
Trial Design
Synthesized multicomponent endpoints to address disease heterogeneity and achieved regulatory body acceptance of pivotal study design aligned with PFDD guidance.