SP-420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias

Launched by ABFERO PHARMACEUTICALS, INC · Jan 10, 2019

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Eligibility criteria

• 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