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 2
  • Study Type

  • Status

  • Intervention/Treatment

  • Study Participants

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.
Study Started
Aug 31
Primary Completion
Sep 30
Study Completion
Jan 31
Last Update
Oct 05

Drug SP-420

Self-administered by mouth

Cohort 1 Experimental

SP-420 initially at 28 mg/kg

Cohort 2 Experimental

SP -20 initially at 56 mg/kg

Cohort 3 Experimental

SP-420 initially at 84 mg/kg


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
No Results Posted