Search for a trial

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…

Study on Transfusion Effects in Preterm infants

The first objective is to explore whether epo regulation and expression, at the age of two weeks after birth and three to six months post-term, are associated with the course of Hb levels. Furthermore, we will assess if epo regulation and expression…

Characterization of buffy-coat-derived granulocytes for clinical use: - identifying clinical and laboratory parameters for decision making

• To find surrogate markers in plasma in order to identify patients, which may benefit from granulocyte transfusions and which are at risk for transfusion complications.• To determine - in combination with the outcomes of the NEPTUNIS study -…

A phase II, open-label, non-controlled, intra-patient dose-escalation study to characterize the pharmacokinetics after oral administration of eltrombopag in pediatric patients with refractory, relapsed or treatment naïve severe aplastic anemia or recurrent aplastic anemia (CETB115E2201)

To characterize the PK of eltrombopag at steady state after oral administration in pediatric patients with SAA.Secondary (key only, see protocol page 40-41 for all objectives): Safety and tolerability. Efficacy (overall response rate ORR).

A Randomized, Double-blind, Active-controlled, Phase 3 study evaluating the Efficacy and Safety of ABP 959 compared with Eculizumab in Adult Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Primary Objective: The primary objective for this study is to evaluate the efficacy of ABP 959 compared with that of eculizumab based on control of intravascular hemolysis.Secondary Objective: The secondary objective is to assess the safety,…

Multicenter randomized controlled trial comparing Ferric(III)carboxymaltose infusion with oral iron suppletion in the treatment of perioperative anemia in patients with a colorectal carcinoma.

The aim of this trial is to investigate which treatment option is superior in the treatment of anemia in patients undergoing surgery in terms of hemoglobin increase, postoperative morbidity, amount of bloodtransfusions needed, length of stay,…

Randomized controlled trial on the use of EPO to reduce top-up transfusions in neonates with red blood cell alloimmunization treated with intrauterine transfusions.

The primary objective of this study is to investigate whether darbepoetin alfa is effective in reducing the incidence of late anemia in infants with HDN treated with IUT and therefore in decreasing the number of top-up transfusion required per…

Disease activity and iron status in children with inflammatory bowel disease

Primary Objective: To investigate the relationship between disease activity and iron deficiency (i.e. absolute and functional ID combined) in children with IBD. Secondary Objectives: 1.To establish the prevalence of iron deficiency (both absolute…

A prospective, lead-in study to collect bleeding episodes, Factor VIII (FVIII) infusions, and patient-reported outcomes in patients with hemophilia A

This is a so-called *lead-in study* in which patients receive their standard treatment and no study treatment. The researchers will collect dataabout the patients* bleeding episodes, FVIII containing treatments used to prevent bleeding episodes and…

Differences in coagulation between fresh frozen plasma and Solvent-detergent plasma in pediatric congenital heart surgery.

This study has been transitioned to CTIS with ID 2024-514073-22-01 check the CTIS register for the current data. To investigate differences in coagulation between (Omniplasma) and FFP in paediatric cardiac patients, who are undergoing cardiac…

COBRA-KAI study: COVID-19 vaccination in patients with reduced B-cell and T-cell immunity: response after vaccination of a kaleidoscopic group hematological patients, what*s the impact?

1.To investigate COVID-19 vaccine responses of the standard schedule of 2 vaccinations with the Moderna mRNA-1273 vaccine (primary schedule) in patients with hematologic diseases, in particular those patients who are considered immunocompromised,…

The efficacy of intermittent versus daily oral iron supplementation in anaemic pregnant women.

To compare the efficacy, side effects and therapy compliance of intermittent (three times a week) versus daily oral iron supplementation for anaemia in pregnancy attributed to iron deficiency.

Sports Participation and Injuries in Patients with Haemophilia

Primary objectives:1. To quantify physical activity, sports participation and exposure according to age, haemophilia severity and joint status;2. To assess the incidence of sports-related injuries in those who participate in sports at least once…

Convalescent Antibody-Mediated Treatment of COVID-19 Infections in Patients with B-cell dysfunction, a Randomized Trial (COVID-Compromise Study).

There have been reports of a more serious course of COVID-19 infections in patients with a reduced immune system, such as patients with congenital immune disorders, (haematological) malignancies or patients taking medications that suppress the…

A Phase 2, Multicohort Study of Daratumumab-Based Therapies in Participants with
Amyloid Light Chain (AL) Amyloidosis

This study has been transitioned to CTIS with ID 2023-507069-25-00 check the CTIS register for the current data. The primary objectives for the study are: Cohort 1, to characterize cardiac safety of different Daratumumab, cyclophosphamide,…

A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

The primary objective of this study is to assess the noninferiority of ALXN1210 compared to eculizumab in adult patients with PNH who have never been treated with a complement inhibitor. Noninferiority will be claimed if after 26 weeks of treatment…

The pharmacokinetics and pharmacodynamics of eculizumab in patients with paroxysmal nocturnal hemoglobinuria.

To describe the real-world population pharmacokinetics and pharmacodynamics of eculizumab in unselected PNH patients

A Multicenter, Open-Label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Emicizumab in Patients with Mild or Moderate Hemophilia A without FVIII Inhibitors

This study will evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in patients of all ages with mild (FVIII level between > 5% and < 40%) or moderate hemophilia A (FVIII level between >= 1% and…

Phase 3, prospective, multi-center, open label study to investigate
safety, immunogenicity, and hemostatic efficacy of PEGylated Factor VIII
(BAX 855) in previously untreated patients (PUPs) < 6 years with
severe hemophilia A (FVIII < 1%)

Primary Objective:The primary objective of the study is to determine the safety including immunogenicity of BAX 855 based on the incidence of inhibitor development to FVIII (* 0.6 Bethesda unit (BU)/mL using the Nijmegen modification of the Bethesda…

Desmopressin and FVIII concentrate combination treatment in non-severe hemophilia A patients undergoing minor interventions

To show a reduction in FVIII-concentrate consumption with perioperative desmopressin and FVIII concentrate combination treatment compared to FVIII concentrate monotherapy, without decreasing the effectivity of treatment.