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Electrosclerotherapy as a novel treatment option for capillary malformations: a pilot study

Determine the feasibility and explore the efficacy and safety of electrosclerotherapy as a novel treatment option for capillary malformations.

A phase I/II, open-label, uncontrolled, single-dose, dose-ascending, multi-centre trial investigating an adeno-associated viral vector containing a codon-optimized human factor IX gene (AAV5-hFIX) administered to adult patients with severe or moderately severe haemophilia B

The primary objective of this study is to investigate the safety of systemic administration of AAV5-hFIX, an adeno-associated viral vector containing a codon-optimized hFIX gene, to adult patients with severe or moderately severe haemophilia B.…

A Multicenter, Open-Label Study to Evaluate the Safety, Efficacy, Pharmacokinetics and
Pharmacodynamics of M281 Administered to Pregnant Women at High Risk for Early Onset Severe
Hemolytic Disease of the Fetus and Newborn (HDFN)

Primary Objectives: - To evaluate the safety in mother and neonate/infant of nipocalimab administered to pregnant women at high risk for EOS-HDFN. - To evaluate the efficacy of nipocalimab as measured by proportion of patients with live birth at or…

An Open-Label Extension Study of ISIS 721744 in Patients with Hereditary Angioedema

This study has been transitioned to CTIS with ID 2024-517249-15-00 check the CTIS register for the current data. Primary Objective:To evaluate the safety of extended dosing, and alternative dosing and/or dose frequency with ISIS 721744 in patients…

A Phase 2, Open-label, Dose Escalation and Dose Expansion Study of KER-047 for the Treatment of IRIDA

Primary Objectives: • To evaluate the safety and tolerability of ascending doses of KER 047 in participants with iron-refractory iron deficiency anemia (IRIDA)Secondary Objectives:• To evaluate the pharmacodynamic (PD) effects of KER 047 on iron…

A Phase I/IIb extension study assessing the long-term safety and efficacy of an adeno-associated viral vector containing a codon-optimized human factor IX gene (AAV5-hFIX) previously administered to adult patients with severe or moderately severe haemophilia B during the CT-AMT-060-01 Phase I/II study.

This study has been transitioned to CTIS with ID 2024-512603-39-00 check the CTIS register for the current data. Primary: To assess the long-term safety (6-10 years after dosing) of a systemic administration of AAV5-hFIX, an AAV vector containing a…

Ga-68-DOTA-(RGD)2 PET/CT: imaging angiogenesis in patients with arterio-venous malformations

The primary objective of this study is to assess the feasibility and safety to image αvβ3-integrin expression in patients with AVM using Ga-68-DOTA-(RGD)2 PET/CT.

Efficacy and Safety of human apotransferrin in patients with β-thalassemia intermedia

To study the effect of apotransferrin administration in patients with β thalassemia intermedia on erythropoiesis as reflected by hemoglobin level or transfusion dependency. Secondary objectives are the effect of apotransferrin on the…