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

• Status: Withdrawn
• Trial end date: 2023-01-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Abfero Pharmaceuticals, Inc

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