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Treatment of severe acute GVHD after allogeneic hematopoietic stem cell transplantation with steroids versus MSC and steroids.
A prospective double-blind placebo-controlled randomized phase III trial

To improve the response rate to treatment of severe acute GVHD (grade II-IV with gut involvement ) by adding infusion of Mesenchymal Stroma Cells to standard high dose prednisolone.

Treatment of severe steroid-refractory acute GvHD with mesenchymal stromal cells.
A phase III randomized double-blind multi-center HOVON study.

Primary objective* To improve the response rate to treatment of severe steroid-refractory acute GvHD grade II-IV (with gut and/or liver involvement) by early addition of MSC to standardized second line treatmentSecondary objectives* To study the…

A multi-center, randomized, double-blind, placebo-controlled clinical trial of deferasirox in patients with myelodysplastic syndromes (low/int-1 risk) and transfusional iron overload (TELESTO)

Use of iron chelation therapy has demonstrated benefits in terms of morbidity and mortality for chronically-transfused thalassemia patients with iron overload. Recent retrospective data (Leitch 2007, Rose 2010, Sanz 2008) suggest that overall…

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

The primary objective of this study is to confirm the efficacy and safety of Genz 112638 after 39 weeks of treatment in patients with Gaucher disease type 1.The secondary objective of this study is to determine the long term efficacy, safety, and…

A Three-part Study of Eltrombopag in Thrombocytopenic Subjects with Myelodysplastic Syndromes or Acute Myeloid Leukemia (Part 1: open-label, Part 2: randomized, double-blind, Part 3: extension).;ASPIRE: A Study of EltromboPag In Myelodysplastic SyndRomes and AcutE Myeloid Leukemia

Part 1 (open-label, 8 week): safety and tolerability of eltrombopag, optimal dose escalation scheme for use in Part 2, PK.Part 2: Primary: the reduction in the number of clinically relevant thrombocytopenic events (*Grade 3 hemorrhagic adverse…

A Phase 3 Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)

Primary:* To demonstrate the clinical efficacy of rozanolixizumab in maintenance treatment in study participants with primary ITPSecondary:* To assess the safety and tolerability of rozanolixizumabExploratory:* To evaluate the clinical efficacy as…

A 3-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Indolent and Smoldering Systemic Mastocytosis with Symptoms Inadequately Controlled with Standard Therapy

This study has been transitioned to CTIS with ID 2024-512585-34-00 check the CTIS register for the current data. Primary: Part 1 • To determine the RP2D of avapritinib in patients with ISM for use in Part 2 and Part 3 of the study.Part 2 • To…

A Randomized, Double-blind, Multicenter Study Comparing Magrolimab in Combination with Azacitidine versus Azacitidine Plus Placebo in Treatment-naïve Patients with Higher Risk Myelodysplastic Syndrome

Primary (Efficacy):To evaluate the efficacy of magrolimab + azacitidine compared with that of azacitidine + placebo in previously untreated participants with intermediate/high/very high risk MDS by IPSS-R as measured by CR rateTo evaluate the…

A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia

* The primary objective of this study is to compare the proportion of warm antibody autoimmune hemolytic anemia (wAIHA) subjects whoachieve a durable hemoglobin response between the fostamatinib and placebo groups.* The secondary objectives of this…

A 24-week with possible extension, prospective, multicenter, randomized, double blind, placebo-controlled, 2-parallel group with a randomization 1:1, phase III study to compare efficacy and safety of oral masitinib to placebo in treatment of patients with Smouldering or Indolent Severe Systemic mastocytosis with handicap, unresponsive to optimal symptomatic treatment

To evaluate the efficacy and safety of oral masitinib versus placebo in the treatment of patients suffering from Smouldering or Indolent SevereSystemic mastocytosis with handicap unresponsive to optimal symptomatic treatment.

GRAVITAS-301: A Randomized, Double-Blind, Placebo Controlled Phase 3 Study of Itacitinib or Placebo in Combination With Corticosteroids for the Treatment of First-Line Acute Graft Versus Host Disease

Primary objective: Compare the efficacy of itacitinib in combination with corticosteroids versus placebo in combination with corticosteroids in terms of overall response rate (ORR) at Day 28 in subjects with aGVHD.Secondary objectives:- Compare the…