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

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.

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