Table 1 Summary of the 6 academic-sponsored multinational RCTs for rare diseases selected as use cases to describe operational hurdles
Project | Trial/ClinicalTrials.gov (NCT) | Protocol/results |
|---|---|---|
HCQ4SurfDefect | Hydroxychloroquine (HCQ on Paediatric Interstitial Lung Disease (ILD) (HCQ-chILD-EU) NCT02615938 | Prospective evaluation of hydroxychloroquine in pediatric interstitial lung diseases: Study protocol for an investigator-initiated, randomized controlled, parallel-group clinical trial [7] Randomized controlled phase 2 trial of hydroxychloroquine in childhood interstitial lung disease [8] |
Redox | A trial of Doxycycline vs. Standard Supportive Therapy in Newly-diagnosed Cardiac AL Amyloidosis Patients Undergoing Bortezomib-based Therapy NCT03474458 | Protocol not published |
ROPROP | Oral propranolol for prevention of threshold retinopathy of prematurity (ROPROP) NCT03083431 | Oral propranolol for prevention of threshold retinopathy of prematurity (ROPROP): protocol of a randomized controlled trial [9] |
ROCK ALS | Inhibition of Rho Kinase (ROCK) with fasudil as disease-modifying treatment for ALS (ROCK-ALS) NCT03792490 | ROCK-ALS: Protocol for a Randomized, placebo-controlled, double-blind Phase IIa trial of safety, tolerability and efficacy of the Rho kinase (ROCK) inhibitor fasudil in Amyotrophic Lateral Sclerosis [10] |
TAM DMD | Tamoxifen in Duchenne Muscular Dystrophy (TAMDMD) NCT03354039 | Tamoxifen in Duchenne muscular dystrophy (TAMDMD): study protocol for a multicenter, randomized, placebo-controlled, double-blind phase 3 trial [11] |
DevelopAKUre Program SONIA 1 | Dose response study of nitisinone in Alkaptonuria (SONIA 1) NCT01828463 | Suitability of nitisinone in alkaptonuria 1 (SONIA 1): an international, multicenter, randomized, open-label, no-treatment controlled, parallel-group, dose-response study to investigate the effect of once daily nitisinone on 24-h urinary homogentisic acid excretion in patients with alkaptonuria after 4 weeks of treatment [12] |
DevelopAKUre Program SONIA2 | Suitability of nitisinone in alkaptonuria 2 (SONIA 2) NCT01916382 | Efficacy and safety of once-daily nitisinone for patients with alkaptonuria (SONIA 2): an international, multicenter, open-label, randomized controlled trial [13] |
DevelopAKUre Program SOFIA | SOFIA | Subclinical ochronosis features in Alkaptonuria: A cross-sectional study [14] |