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Phase 2a Pilot Study of NBMI Treatment in Patients With Beta Thalassemia Major, Requiring Iron Chelation (EMERA007)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04092205
Recruitment Status : Recruiting
First Posted : September 17, 2019
Last Update Posted : December 2, 2019
Sponsor:
Information provided by (Responsible Party):
EmeraMed

Brief Summary:

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

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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)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Iron Thalassemia

Arm Intervention/treatment
Experimental: Experimental: Treatment
28 days treatment with NBMI 600 mg/day
Drug: Emeramide
Lipophilic, membrane passing Metal chelator and anti oxidant
Other Names:
  • NBMI
  • Irminix




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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)

  2. 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)

  3. 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


Other Outcome Measures:
  1. 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.

  2. 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)

  3. Malondialdehyde levels [ Time Frame: Visit 2, 4 ]
    Indicator of oxidative stress measured in μmol/l.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patient has clinically confirmed documented diagnosis of Beta Thalassemia Major, according to the current clinical criteria, and are on blood transfusions.
  2. Patient has been on a stable maintaince therapy with deferasirox for at least during last 3 months.
  3. Patients current chelation therapy is considered inadequate, meaning that there is evidence from clinical monitoring that chronic iron overload is present (e.g. serum ferritin >1,000 µg/l), for at least during the last 3 months.
  4. The ferritin level has been stable with max 10% difference between max-to-low, and the iron chelator treatment dose has been unchanged during the last 3 months
  5. Patient is aged 18 years or older at screening.
  6. Female patients are only eligible for the study if they are either surgically sterile or at least 2 years postmenopausal, or have a negative result of serum hCG test at screening and if willing to use acceptable, effective methods of contraception during the trial and for three month after the end of trial participation as defined inpoint 7.7. of this the protocol.
  7. Male patients must either be surgically sterile or he and his female spouse/partner who is of childbearing potential must be willing to use highly effective methods of contraception consisting of 2 forms of birth control (1 of which must be a barrier method) starting at screening and continuing throughout the study.
  8. Patient is fluent in the local language and provides written informed consent.

Exclusion Criteria:

  1. Known history or presence of clinically significant other, hematologic, endocrine, oncologic, pulmonary, immunologic, genitourinary, psychiatric, or cardiovascular disease or any other condition which, in the opinion of the Investigator, would jeopardize the safety of the subject or impact the validity of the study results.
  2. Known or suspected allergy hypersensitivity or idiosyncratic reaction to NBMI or any other drug substances with similar activity.
  3. History of drug or alcohol addiction requiring treatment.
  4. History of malabsorption within the last year or presence of clinically significant gastrointestinal disease or surgery that may affect drug bioavailability, including but not limited to cholecystectomy.
  5. Presence of hepatic or renal dysfunction. (SGOT and SGPT and bilirubin > X3 (3 fold) UNL. creatinine > 1.5mg/dl).
  6. Female patient who is pregnant (serum hCG level consistent with pregnancy diagnosis); or breastfeeding.
  7. Participation in a clinical trial that involved administration of an investigational medicinal product within 90 days prior to drug administration, or recent participation in a clinical investigation that, in the opinion of the Investigator, would jeopardize subject safety or the integrity of the study results.
  8. Have clinically significant abnormal laboratory values (e.g. liver enzymes).
  9. Have clinically significant findings from a physical examination (e.g. fever).
  10. Patient has Inflammatory disorders, liver disease such as hepatitis, malignancy or other condition that could influence ferritine levels and therefore validity of study results.

Information from the National Library of Medicine

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


Contacts
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Contact: Tanja Turk, M. Pharm. +386 1 5442501 tanja.turk@crs.si

Locations
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Albania
University Hospital Center "Mother Theresa" Tirana Recruiting
Tirana, Albania, 1006
Contact: Manika Kreka, Associate Professor    +355692108850    mkreka@cvbf.net   
Contact: Ornelia Cullufe       ocullufe@cvbf.net   
Sponsors and Collaborators
EmeraMed

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Responsible Party: EmeraMed
ClinicalTrials.gov Identifier: NCT04092205    
Other Study ID Numbers: EMERA007
First Posted: September 17, 2019    Key Record Dates
Last Update Posted: December 2, 2019
Last Verified: November 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
N,N'-Bis(2-mercaptoethyl)isophthalamide
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action