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

NCT04092205 | Completed Phase 2

• 1 other identifier: EMERA007
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

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

Conditions

Beta Thalassemia Major

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-Emergent Adverse Events

  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.

  28 days

Secondary Outcomes (3)

• Change in serum ferritin levels

  28 days

• Change in iron load in the liver and heart

  28 days

• Percentage of NBMI-treated patients who will develop a response

  28 days

Other Outcomes (3)

• Kidney markers (serum creatinine levels, glomerular filtration calculation)

  28 days

• Pharmacokinetic parameters

  Visit 4, 5, 6, 7

• Malondialdehyde levels

  Visit 2, 4

Study Arms (1)

Experimental: Treatment

EXPERIMENTAL

28 days treatment with NBMI 600 mg/day

Drug: Emeramide

Interventions

Emeramide DRUG

Lipophilic, membrane passing Metal chelator and anti oxidant

Also known as: NBMI, Irminix

Experimental: Treatment

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient has clinically confirmed documented diagnosis of Beta Thalassemia Major, according to the current clinical criteria, and are on blood transfusions.
• Patient has been on a stable maintaince therapy with deferasirox for at least during last 3 months.
• 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.
• 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
• Patient is aged 18 years or older at screening.
• 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.
• 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.
• Patient is fluent in the local language and provides written informed consent.

You may not qualify if:

• 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.
• Known or suspected allergy hypersensitivity or idiosyncratic reaction to NBMI or any other drug substances with similar activity.
• History of drug or alcohol addiction requiring treatment.
• 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.
• Presence of hepatic or renal dysfunction. (SGOT and SGPT and bilirubin \> X3 (3 fold) UNL. creatinine \> 1.5mg/dl).
• Female patient who is pregnant (serum hCG level consistent with pregnancy diagnosis); or breastfeeding.
• 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.
• Have clinically significant abnormal laboratory values (e.g. liver enzymes).
• Have clinically significant findings from a physical examination (e.g. fever).
• Patient has Inflammatory disorders, liver disease such as hepatitis, malignancy or other condition that could influence ferritine levels and therefore validity of study results.

Sponsors & Collaborators

• EmeraMed: lead

Study Sites (1)

University Hospital Center "Mother Theresa" Tirana

Tirana, 1006, Albania

Location

MeSH Terms

Conditions

beta-Thalassemia

Interventions

N,N'-Bis(2-mercaptoethyl)isophthalamide

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: An open label, active-treatment controlled once daily oral administration for 28 days and 28 days follow up

Study Record Dates

• First Submitted: July 29, 2019
• First Posted: September 17, 2019
• Study Start: November 21, 2019
• Primary Completion: July 30, 2020
• Study Completion: August 30, 2020
• Last Updated: August 26, 2021

Record last verified: 2021-08

Data Sharing

• IPD Sharing: Will not share

Locations

AL (1)