Status:
WITHDRAWN
SP-420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias
Lead Sponsor:
Abfero Pharmaceuticals, Inc
Conditions:
Iron Overload
Beta-Thalassemia
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief 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 tr...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
August 1 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2023
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT03801889
Start Date
August 1 2020
End Date
January 1 2023
Last Update
October 5 2020
Active Locations (4)
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1
University of Toronto- University Health Network
Toronto, Canada
2
American University of Beirut Medical Center
Beirut, Lebanon
3
Siriraj Hospital
Bangkok, Thailand
4
Ege University Hospital
Izmir, Turkey (Türkiye)