Rare Diseases

LIST OF CLINICAL TRIALS BY THERAPEUTIC AREA

  • CARDIOLOGY

  • ENDOCRINOLOGY

  • MULTIPLE ESCLEROSIS

  • RARE DISEASES

RARE DISEASES

Drug: Eliglustat

  1. Manufacturer: Genzyme
  2. Indication: Type 1 Gaucher disease
  3. Studies:
    • A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic Goals with Enzyme Replacement Therapy (ENCORE)
    • A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 (ENGAGE)
    • A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Once Daily versus Twice Daily Dosing of Genz-112638 in Patients with Gaucher Disease Type 1 who have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638
  4. Role: Clinical operations: feasibility, site selection, site management in Latin America
  5. Status: published

 

Drug: Mipomersen

  1. Manufacturer: IONES and Genzyme
  2. Indication: Severe Heterozygous Familial Hypercholesterolemia Study: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients with Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol
    • Role Clinical operations: feasibility, site selection, site management in Latin America
    • Status: published

 

Drug: Agalsidase beta

  1. Manufacturer: Genzyme
  2. Indication: Fabry Disease
  3. Study: A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe
  4. Role: Clinical operations: feasibility, site selection, site management in Latin America
  5. Status: published

 

Drug: Recombinant Human Acid Sphingomyelinase (rhASM)

  1. Manufacturer: Genzyme
  2. Indication: Niemann-Pick disease (NPD)
  3. Study: A Phase 2, Multi-Center, Randomized, Open-Label, Repeat Dose, Dose-Comparison Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Human Acid Sphingomyelinase (rhASM) in Patients With Acid Sphingomyelinase Deficiency (ASMD)
  4. Role: Clinical operations: feasibility, site selection, site management in Latin America
  5. Status: published