Overview

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Withdrawn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