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A Phase II, double-blind, placebo-controlled, randomized, cross-over, dose-ranging study of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema due to C1-inhibitor deficiency type I and II

The purpose of this study is to find out how safe and effective the new drug PHA-022121 is for the treatment of Hereditary Angioedema. The main purpose of this research study is to test the effectiveness of 3 different doses of PHA-022121 in…

A Phase 2, Single-Arm Study of the Biomarker Effects of ALZ-801 in Subjects with Early Alzheimer*s Disease Who Are Carriers of the *4 Variant of the Apolipoprotein E Gene (APOE4/4 or APOE3/4)

This study has been transitioned to CTIS with ID 2024-515858-25-00 check the CTIS register for the current data. Objectives - Core StudyPrimary Objectives• To evaluate the effects of oral ALZ-801 in subjects with Early AD who have the APOE4/4 or…

N- acetylcysteine treatment for skin picking in children and young adults with PWS: A randomized, doubleblind, placebo-controlled, cross-over trial.

To evaluate the effects of N-acetylcysteine (NAC; Fluimucil® in Dutch) on skin picking behaviour in children and young adults with PWS.

A Phase 2 Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of AL002 in Participants with Early Alzheimer*s Disease

To evaluate the efficacy of AL002 in participants with Early AD in delaying disease progression compared to standard of care

A randomized, double-blind, dose-ranging, placebo-controlled, Phase 2a evaluation of the safety, tolerability, and pharmacokinetics of PLN-74809 in participants with primary sclerosing cholangitis (PSC) and suspected liver fibrosis (INTEGRIS-PSC)

To assess the safety and tolerability of PLN 74809 in participants with PSC and suspected liver fibrosis

Treatment of Skin Severity in Ichthyosis with Hyperbaric Oxygen Therapy

To assess clinical improvement of skin severity in patients with ichthyosis

A Pivotal Phase 1/2, Single-Arm, Open-label Study to Evaluate the Safety and Efficacy of Ponatinib With Chemotherapy in Pediatric Patients With Philadelphia Chromosome-Positive (Ph+) Acute Lymphoblastic Leukemia (ALL) Who Have Relapsed or Are Resistant or Intolerant to a Prior Tyrosine Kinase Inhibitor-Containing Therapy, or Who Have the T315I Mutation

Primary Objectives:Phase 1 Primary Objective** To determine the RP2D of ponatinib (tablet and AAF) in combination with chemotherapy.Phase 2 Primary Objective* * To determine the efficacy of ponatinib in combination with chemotherapy as measured by…

Autoimmune epilepsy Modulated by IVIg - effects on Cortical Excitability, the AMICE study

1. Assessing the efficacy of IVIg in autoimmune epilepsy, both clinically and serologically.2. Identifying an objective marker of therapy response in epilepsy, measuring cortical excitability by TMS-EEG/EMG, in vivo.3. Providing evidence that…

Primary percutaneous placement of metal stents for palliative biliary drainage in patients with a primary malignant perihilar stricture

To proof feasibility and safety of direct SEMS placement for palliative treatment of perihilar cholangiocarcinoma.