SP-420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias
Study Details
Study Description
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 transfusions.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Cohort 1 SP-420 initially at 28 mg/kg |
Drug: SP-420
Self-administered by mouth
|
| Experimental: Cohort 2 SP -20 initially at 56 mg/kg |
Drug: SP-420
Self-administered by mouth
|
| Experimental: Cohort 3 SP-420 initially at 84 mg/kg |
Drug: SP-420
Self-administered by mouth
|
Outcome Measures
Primary Outcome Measures
- The incidence of treatment-emergent Adverse Events (AEs) [Week 24]
- The incidence of treatment-emergent Adverse Events (AEs) [Week 52]
Secondary Outcome Measures
- Change in liver iron concentration (LIC) on R2-MRI from baseline [Week 24]
- Change in liver iron concentration (LIC) on R2-MRI from baseline [Week 52]
- Change in cardiac iron content (CIC) on T2*-MRI from baseline [Week 24]
- Change in cardiac iron content (CIC) on T2*-MRI from baseline [Week 52]
- Total iron removed by chelator (in mg) from baseline [Week 24]
- Total iron removed by chelator (in mg) from baseline [Week 52]
Eligibility Criteria
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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 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 | İzmir | Turkey |
Sponsors and Collaborators
- Abfero Pharmaceuticals, Inc
Investigators
- Principal Investigator: Ali Taher, MD, PhD, American University of Beirut Medical Center
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- SP-420-705