Beta-Thalassemia, Iron Overload
Conditions
Keywords
chelator
Brief 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.
Interventions
DRUGSP-420
Self-administered by mouth
Sponsors
Abfero Pharmaceuticals, Inc
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Intervention 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.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* 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 * 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The incidence of treatment-emergent Adverse Events (AEs) | Week 24 |
Secondary
| Measure | Time frame |
|---|---|
| Change in liver iron concentration (LIC) on R2-MRI from baseline | Week 24 |
| Change in cardiac iron content (CIC) on T2*-MRI from baseline | Week 24 |
| Total iron removed by chelator (in mg) from baseline | Week 24 |
Countries
Canada, Lebanon, Thailand, Turkey (Türkiye)
Outcome results
None listed