Beta Thalassemia MajorPhase 2EMERA007NCT04092205
Phase 2a Pilot Study of NBMI Treatment in Patients With Beta Thalassemia Major, Requiring Iron Chelation
A Phase 2 study testing Emeramide in people with Beta Thalassemia Major, run by EmeraMed at 1 site in Albania.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Beta Thalassemia Major
- What is being tested?
- Emeramide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteAlbania
- Study dates
- 21 Nov 2019to 30 Aug 2020
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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
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
Read the detailed description
Patients with iron overload, not controlled with current therapy will be enrolled to the study. After initial treatment with standard chelation therapy (deferasirox), patients will receive 600 mg daily dose of emeramide (NBMI) for 28 days. After that follow up period on standard treatment with deferasirox shall follow.
- Conditions
- Beta Thalassemia Major
- Being tested
- Emeramide
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EmeramideDrug
Also called NBMI, Irminix
Lipophilic, membrane passing Metal chelator and anti oxidant
| Study group | Role | What they receive |
|---|---|---|
| Experimental: Treatment | Experimental | 28 days treatment with NBMI 600 mg/day Receives: Drug: Emeramide |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 28 days
Secondary outcomes (3)
Change in serum ferritin levels
Change in serum ferritin levels measured in µg/l (V2 level compared to V4 level and V5 level compared to V2 level)
Measured: 28 days
Change in iron load in the liver and heart
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)
Measured: 28 days
Percentage of NBMI-treated patients who will develop a response
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
Measured: 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Sep 2019
- Study start21 Nov 2019
- Primary completion30 Jul 2020
- Study completion30 Aug 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta Thalassemia MajorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- An open label, active-treatment controlled once daily oral administration for 28 days and 28 days follow up
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- EmeraMed
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04092205
- Sponsor's protocol ID
- EMERA007
- Acronym
- EMERA007
- Status last verified
- Aug 2021
- First submitted
- 29 Jul 2019
- Last updated
- 26 Aug 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04092205
- Acronym / sponsor ID
- EMERA007 / EMERA007
- Conditions
- Beta Thalassemia Major
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- EmeraMedIndustry (pharma, biotech, devices)
- Study start
- 21 Nov 2019 (actual)
- Primary completion
- 30 Jul 2020 (actual)
- Study completion
- 30 Aug 2020 (actual)
- First posted
- 17 Sep 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Detailed description
Patients with iron overload, not controlled with current therapy will be enrolled to the study. After initial treatment with standard chelation therapy (deferasirox), patients will receive 600 mg daily dose of emeramide (NBMI) for 28 days. After that follow up period on standard treatment with deferasirox shall follow.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- An open label, active-treatment controlled once daily oral administration for 28 days and 28 days follow up
| Arm | Type | Receives |
|---|---|---|
| Experimental: Treatment | Experimental | Drug: Emeramide28 days treatment with NBMI 600 mg/day |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (10 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EmeramideAlso: NBMI, IrminixLipophilic, membrane passing Metal chelator and anti oxidant | Drug | Experimental: Treatment |
Primary outcome
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.
3 secondary outcomes
Change in serum ferritin levels
Time frame: 28 days
Change in iron load in the liver and heart
Time frame: 28 days
Percentage of NBMI-treated patients who will develop a response
Time frame: 28 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Emeramide in people with Beta Thalassemia Major, run by EmeraMed at 1 site in Albania.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Albania. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events (time frame: 28 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Aug 2021.