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.

Dermatology