CTI is a leader in rare disease and orphan disease research

More than 3/4 of our active prospective studies are in these therapeutic areas

Successful rare disease drug/device development programs require experience, expertise, and dedication. They are often fraught with challenges from designing an executable protocol to patient and investigator identification.  At CTI, we have the experience required and the resources necessary to support rare disease drug/device development from the regulatory planning stage through to marketing approval.  We have developed a unique feasibility process that includes peer-to-peer interaction to support protocol development, site/investigator identification, and can provide information critical to building customized enrollment strategies.  In addition to offering full clinical operations services, we have the ability to provide strategic regulatory consulting, work with patient support and advocacy groups, and develop/manage patient registries. 

We offer our sponsors:

  • Regulatory experts with significant experience in pathway negotiations including Fast Track, Orphan, and Breakthrough Designations
  • Insight into clinical program planning, trial design/execution, and development of creative solutions for patient identification, recruitment, and retention challenges
  • An employee turnover rate less than half of the reported industry average, resulting in team continuity
  • Experience with first-in-human, first-in-class, and first-in-patient trials
  • Personal relationships with key investigators and site personnel
  • Executive management involvement on every project
  • CTI studies regularly exceed industry standards for enrollment, despite challenging patient populations
  • A proven track record of success with more than 100 drug and device approvals

Some of our adult and pediatric experience includes:

  • Adenovirus
  • Alport syndrome
  • Antibody mediated rejection (AMR)
  • Atypical hemolytic-uremic syndrome (aHUS)
  • Autoimmune hepatitis
  • BK virus
  • Βeta-Thalassemia
  • Bronchiolitis obliterans syndrome (BOS)
  • Bronchopulmonary dysplasia
  • Cerebrotendinous xanthomatosis (CTX)
  • Choroideremia
  • Childhood cerebral adrenoleukodystrophy (CCALD)
  • Cystinosis
  • Cytomegalovirus (CMV)
  • Distal renal tubular acidosis (dRTA)
  • Delayed graft function (DGF)
  • Facioscapulohumeral muscular dystrophy (FSHD)
  • Farber Disease
  • Focal segmental glomerulosclerosis (FSGS)
  • Glioblastoma
  • Glomerulopathy (C3G)
  • Graft versus host disease (GVHD)
  • Heparin-induced thrombocytopenia (HIT)
  • Hereditary orotic aciduria
  • Hurler Syndrome (mucopolysaccharidosis I, MPS I)
  • Hunter’s Syndrome (mucopolysaccharidosis II, MPS II)
  • Ischemia reperfusion injury (IRI)
  • Lymphangioleiomyomatosis (LAM)
  • Methotrexate-induced toxicity
  • Mitochondrial depletion syndrome (T2K deficiency)
  • Multiple myeloma
  • Muscular dystrophy
  • Narcolepsy
  • Niemann-Pick Type C
  • Pantothenate kinase-associated neurodegeneration (PKAN)
  • Paroxysmal nocturnal hemoglobinuria
  • Phenylketonuria
  • Polycythemia vera
  • Polyoma virus
  • Pompe disease
  • Sanfilippo A Syndrome (mucopolysaccharidosis IIIA, MPS IIIA)
  • Sanfilippo B Syndrome (mucopolysaccharidosis IIIB, MPS IIIB)
  • Sickle cell disease
  • Tuberous sclerosis
  • Urea cycle disorders
  • West syndrome
  • Wolman's disease (lysosomal acid lipase deficiency)
  • X-linked creatine transport deficiency (CTD)
  • X-linked hypohidrotic ectodermal dysplasia (XLHED)