Phase 2a Pilot Study of NBMI Treatment in Patients With Beta Thalassemia Major, Requiring Iron Chelation (EMERA007)
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|ClinicalTrials.gov Identifier: NCT04092205|
Recruitment Status : Recruiting
First Posted : September 17, 2019
Last Update Posted : December 2, 2019
A pilot study to explore safety and efficacy of NBMI treatment in patients with Beta Thalassemia Major requiring iron chelation
Investigational product: NBMI (N1,N3-bis(2-mercaptoethyl) isophthalamide), INN: Emeramide
Indication: Beta Thalassemia Major
|Condition or disease||Intervention/treatment||Phase|
|Beta Thalassemia Major||Drug: Emeramide||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||An open label, active-treatment controlled once daily oral administration for 28 days and 28 days follow up|
|Masking:||None (Open Label)|
|Official Title:||EMERA007 - An Open Label, Active-treatment Controlled, Phase 2a Pilot Study to Explore Safety and Efficacy of NBMI Treatment in Patients With Beta Thalassemia Major, Requiring Iron Chelation|
|Actual Study Start Date :||November 21, 2019|
|Estimated Primary Completion Date :||March 30, 2020|
|Estimated Study Completion Date :||June 30, 2020|
Experimental: Experimental: Treatment
28 days treatment with NBMI 600 mg/day
Lipophilic, membrane passing Metal chelator and anti oxidant
- Incidence of Treatment-Emergent Adverse Events [ Time Frame: 28 days ]The safety assessment is based on the number, type and severity of adverse events. The incidence of adverse events will be summarized by organ class, severity and duration.
- Change in serum ferritin levels [ Time Frame: 28 days ]Change in serum ferritin levels measured in µg/l (V2 level compared to V4 level and V5 level compared to V2 level)
- Change in iron load in the liver and heart [ Time Frame: 28 days ]Change in iron load in the liver and heart as measured by magnetic resonance imaging (MRI) techniques for assessing tissue iron T* (V2 level compared to V4 level and V5 level compared to V2 level)
- Percentage of NBMI-treated patients who will develop a response [ Time Frame: 28 days ]Percentage of NBMI-treated patients who will develop a response to NBMI (response will be defined as lack of increase of ferritin levels ) as compared between V2 level and V4 level
- Kidney markers (serum creatinine levels, glomerular filtration calculation) [ Time Frame: 28 days ]Change from baseline after both treatments. Serum creatinine levels will be measured in μmol/l, glomerular filtration in ml/min.
- Pharmacokinetic parameters [ Time Frame: Visit 4, 5, 6, 7 ]Pharmacokinetic parameters derived from plasma concentrations of NBMI (Time of maximum analyte concentration - Tmax, Maximum drug concentration - Cmax,The terminal elimination rate constant - λz, Apparent terminal elimination half-life - t1/2, The area under the plasma concentration - AUC0-t, Area under the concentration-time curve - AUC0-∞, Area under the concentration-time curve - AUC%Extrap)
- Malondialdehyde levels [ Time Frame: Visit 2, 4 ]Indicator of oxidative stress measured in μmol/l.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04092205
|Contact: Tanja Turk, M. Pharm.||+386 1 email@example.com|