Iron OverloadPhase 2NCT03801889
SP-420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias
A Phase 2 study testing SP-420 in people with Iron Overload and Beta-Thalassemia, run by Abfero Pharmaceuticals, Inc at 4 sites in 4 countries.
Trial summary
The purpose of this study is to test the safety and tolerability of SP-420 and it's efficacy in terms of lowering iron in subjects with Beta-thalassemia or other rare anemias who need regular blood transfusions. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Iron Overload, Beta-Thalassemia
- What is being tested?
- SP-420
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 4 sitesin 4 countries
- Study dates
- Aug 2020to Jan 2023
- 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
- At least 18 years old
- Iron-overload secondary to β-thalassemia (homozygote or compound heterozygote) or other rare anemias (e.g., aplastic anemia, pure red-cell dysplasia ) requiring chronic RBC transfusions and iron chelation therapy
- On a stable dose of iron chelation for at least 4 weeks prior to screening visit
- Weight ≥35 kg at screening
- Willing to discontinue current iron chelation therapy 7 days (± 3 days) prior to the first dose of SP-420 and for the duration of the current study
- LIC ≥5 and ≤25 mg/g dry weight on the R2-MRI obtained within 2 weeks prior to the baseline visit
- Cardiac T2* score > 12 msec obtained on the MRI obtained within 2 weeks prior to the baseline visit
Who cannot join
- Pregnant or breast-feeding
- Current malignancy with the exceptions of localized basal cell or squamous cell skin cancer or localized prostate cancer or is receiving immunotherapy, chemotherapy or radiation therapy for a malignancy
- Current myelodysplastic syndrome
- Alanine aminotransferase (ALT) >4 times the upper limit of normal, decompensated cirrhosis, or ascites at screening
- Past history of clinically significant kidney disease (per the Principal Investigator)
- Serum creatinine greater than the upper limit of normal during screening
Show all 11Show fewer
- Urine protein to creatinine ratio > 0.5 mg/mg during screening
- Ongoing symptoms of cardiac dysfunction or failure
- Ongoing symptoms of neuropathy, including peripheral sensory neuropathy, peripheral motor neuropathy, or paresthesia at screening
- Received another investigational drug within 30 days or investigational antibody within 90 days of Day 1 of the study
- Other condition that, in the opinion of the PI, would interfere with the conduct of the study
Trial locations and contacts
4 sites in 4 countries.
4 sites in 4 countries
About this study
Official title: Multicenter, Open-label, 52 Week, Dose-escalation Study of SP 420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias
The purpose of this study is to test the safety and tolerability of SP-420 and it's efficacy in terms of lowering iron in subjects with Beta-thalassemia or other rare anemias who need regular blood transfusions.
- Conditions
- Iron Overload, Beta-Thalassemia
- Being tested
- SP-420
- Keywords
- chelator
- Medical subject headings
- Iron Overload, beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SP-420Drug
Self-administered by mouth
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | SP-420 initially at 28 mg/kg Receives: Drug: SP-420 |
| Cohort 2 | Experimental | SP -20 initially at 56 mg/kg Receives: Drug: SP-420 |
| Cohort 3 | Experimental | SP-420 initially at 84 mg/kg Receives: Drug: SP-420 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The incidence of treatment-emergent Adverse Events (AEs)
Measured: Week 24
The incidence of treatment-emergent Adverse Events (AEs)
Measured: Week 52
Secondary outcomes (6)
Change in liver iron concentration (LIC) on R2-MRI from baseline
Measured: Week 24
Change in liver iron concentration (LIC) on R2-MRI from baseline
Measured: Week 52
Change in cardiac iron content (CIC) on T2*-MRI from baseline
Measured: Week 24
Change in cardiac iron content (CIC) on T2*-MRI from baseline
Measured: Week 52
Total iron removed by chelator (in mg) from baseline
Measured: Week 24
Total iron removed by chelator (in mg) from baseline
Measured: Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2019
- Study startAug 2020 (estimated)
- Primary completionSep 2022 (estimated)
- Study completionJan 2023 (estimated)
Why it stopped: Protocol suspended prior to patient enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Iron OverloadStudies 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
- Non-randomised
- Intervention model
- Sequential
- Model description
- This is a multi-center, open-label, dose-escalation study designed to evaluate the safety, tolerability, and iron clearing efficacy of SP 420 administered three-times per week in subjects with transfusion-dependent beta-thalassemia or other rare anemias. Approximately 24 subjects are to be enrolled in 3 cohorts (doses of 28 mg/kg, 56 mg/kg and 84 mg/kg) of approximately 8 subjects each. Based upon the results from lower dose cohorts, if needed the size of latter cohorts may be increased to improve the power of the study to detect efficacy to approximately 74 subjects in total.
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Abfero Pharmaceuticals, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ali Taher, MD, PhD, Principal investigator, American University of Beirut Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03801889
- Sponsor's protocol ID
- SP-420-705
- Status last verified
- Sep 2020
- First submitted
- 8 Jan 2019
- Last updated
- 5 Oct 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter, Open-label, 52 Week, Dose-escalation Study of SP 420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias
- ClinicalTrials.gov ID
- NCT03801889
- Acronym / sponsor ID
- SP-420-705
- Conditions
- Iron Overload, Beta-Thalassemia
- Keywords
- chelator
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Protocol suspended prior to patient enrollment
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Abfero Pharmaceuticals, IncIndustry (pharma, biotech, devices)
- Study start
- Aug 2020 (estimated)
- Primary completion
- Sep 2022 (estimated)
- Study completion
- Jan 2023 (estimated)
- First posted
- 14 Jan 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.
The purpose of this study is to test the safety and tolerability of SP-420 and it's efficacy in terms of lowering iron in subjects with Beta-thalassemia or other rare anemias who need regular blood transfusions.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- This is a multi-center, open-label, dose-escalation study designed to evaluate the safety, tolerability, and iron clearing efficacy of SP 420 administered three-times per week in subjects with transfusion-dependent beta-thalassemia or other rare anemias. Approximately 24 subjects are to be enrolled in 3 cohorts (doses of 28 mg/kg, 56 mg/kg and 84 mg/kg) of approximately 8 subjects each. Based upon the results from lower dose cohorts, if needed the size of latter cohorts may be increased to improve the power of the study to detect efficacy to approximately 74 subjects in total.
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: SP-420SP-420 initially at 28 mg/kg |
| Cohort 2 | Experimental | Drug: SP-420SP -20 initially at 56 mg/kg |
| Cohort 3 | Experimental | Drug: SP-420SP-420 initially at 84 mg/kg |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- At least 18 years old
- Iron-overload secondary to β-thalassemia (homozygote or compound heterozygote) or other rare anemias (e.g., aplastic anemia, pure red-cell dysplasia ) requiring chronic RBC transfusions and iron chelation therapy
- On a stable dose of iron chelation for at least 4 weeks prior to screening visit
- Weight ≥35 kg at screening
- Willing to discontinue current iron chelation therapy 7 days (± 3 days) prior to the first dose of SP-420 and for the duration of the current study
Key exclusion criteria (11 listed)
- Pregnant or breast-feeding
- Current malignancy with the exceptions of localized basal cell or squamous cell skin cancer or localized prostate cancer or is receiving immunotherapy, chemotherapy or radiation therapy for a malignancy
- Current myelodysplastic syndrome
- Alanine aminotransferase (ALT) >4 times the upper limit of normal, decompensated cirrhosis, or ascites at screening
- Past history of clinically significant kidney disease (per the Principal Investigator)
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 |
|---|---|---|
| SP-420Self-administered by mouth | Drug | Cohort 1, Cohort 2, Cohort 3 |
Primary outcome
The incidence of treatment-emergent Adverse Events (AEs)
Time frame: Week 24
The incidence of treatment-emergent Adverse Events (AEs)
Time frame: Week 52
6 secondary outcomes
Change in liver iron concentration (LIC) on R2-MRI from baseline
Time frame: Week 24
Change in liver iron concentration (LIC) on R2-MRI from baseline
Time frame: Week 52
Change in cardiac iron content (CIC) on T2*-MRI from baseline
Time frame: Week 24
Change in cardiac iron content (CIC) on T2*-MRI from baseline
Time frame: Week 52
Total iron removed by chelator (in mg) from baseline
Time frame: Week 24
Total iron removed by chelator (in mg) from baseline
Time frame: Week 52
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Oct 2020.
- 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.
Ali Taher, MD, PhD principal investigator
American University of Beirut Medical Center
4 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing SP-420 in people with Iron Overload and Beta-Thalassemia, run by Abfero Pharmaceuticals, Inc at 4 sites in 4 countries.
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 7 inclusion and 11 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: withdrawn, as of the sponsor's last update on 5 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Canada, Lebanon, Thailand, Turkey (Türkiye). 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 was withdrawn before enrolling participants, so there are no results.
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?
The incidence of treatment-emergent Adverse Events (AEs) (time frame: Week 24). The study lists 2 primary outcomes in all. 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 5 Oct 2020.