DE

Rare Disease Clinical Trials

We deal with the challenges, so you can focus on the opportunities

Not only due to a limited number of patients and qualified study sites, orphan disorders require unique approaches in study conduct. Knowing the exceptional challenges, we handle these projects with profound understanding of Orphan Drug Development.
 

Our core competencies:

  • Thorough protocol design taking into consideration a potential lack of literature
  • Searching and choosing motivated and experienced sites
  • Efficient patient recruitment
  • Acting as sponsor for EU Orphan Drug Designations

 

Therapeutic approaches include:

  • Cystic fibrosis
  • Systemic lupus erythematosus (SLE)
  • Limbal stem cell deficiency (LSCD)
  • Glioblastoma
  • Neuroblastoma
  • Hepatocellular carcinoma
  • Fabry disease
  • Primary progressive aphasia
  • Haemophilia B
  • Head and neck carcinoma
  • Huntington’s disease
  • Advanced renal cell carcinoma
  • Malignant ascites
  • Primary sclerosing cholangitis
  • Acute-on-chronic liver failure (ACLF)
  • Multiple myeloma
  • Eosinophilic esophagitis
  • Ehlers-Danlos syndrome
  • Indolent systemic mastocytosis
  • Granulomatosis with polyangiitis (Wegener`s)
  • Acute myeloid leukemia
  • Friedreich’s ataxia
  • Schnitzler’s syndrome

Pediatric Studies in Orphan Diseases

  • Neuroblastoma
  • Limbal stem cell deficiency (LSCD)
  • Systemic lupus erythematosus (SLE)
  • Childhood cerebral adrenoleukodystrophy (CCALD)

If you’re interested in our broad experience in overcoming the hurdles of rare disease trials,  get in touch with us.

  • Prof. Michael Hudecek

    Chair of Cellular Immunotherapy from University Hospital Wuerzburg

    "Since 2018, FGK has been an invaluable partner in our clinical early-phase CAR-T cell projects. Their extensive experience in advanced new therapies, like ATMP and cell therapy projects, has provided us with the expertise and support needed to navigate this complex field. Their commitment to high-quality research and their deep understanding of innovative therapies make them a trusted and essential partner in our development efforts. We look forward to continuing this successful collaboration."