Title
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
Phase
Phase 2Lead Sponsor
Abfero Pharmaceuticals, IncStudy Type
InterventionalStatus
WithdrawnIndication/Condition
Beta-Thalassemia Iron OverloadIntervention/Treatment
SP-420Study Participants
0The 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