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

Iron Overload Clinical Trial

Official title:

Multicenter, Open-label, 52 Week, Dose-escalation Study of SP 420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT03801889
• Other study ID #: SP-420-705
• Status: Withdrawn
• Phase: Phase 2
• Start date: August 2020
• Est. completion date: January 2023

Study information

• Verified date: September 2020
• Source: Abfero Pharmaceuticals, Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: January 2023
• Est. primary completion date: September 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• At least 18 years old

• Iron-overload secondary to ß-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

Related Conditions & MeSH terms

• Beta-Thalassemia
• Iron Overload
• Thalassemia

Intervention

Drug:
• SP-420
Self-administered by mouth

Locations

Country	Name	City	State
Canada	University of Toronto- University Health Network	Toronto
Lebanon	American University of Beirut Medical Center	Beirut
Thailand	Siriraj Hospital	Bangkok
Turkey	Ege University Hospital	Izmir

Sponsors (1)

Lead Sponsor	Collaborator
Abfero Pharmaceuticals, Inc

Countries where clinical trial is conducted

Canada,  Lebanon,  Thailand,  Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The incidence of treatment-emergent Adverse Events (AEs)		Week 24
Primary	The incidence of treatment-emergent Adverse Events (AEs)		Week 52
Secondary	Change in liver iron concentration (LIC) on R2-MRI from baseline		Week 24
Secondary	Change in liver iron concentration (LIC) on R2-MRI from baseline		Week 52
Secondary	Change in cardiac iron content (CIC) on T2*-MRI from baseline		Week 24
Secondary	Change in cardiac iron content (CIC) on T2*-MRI from baseline		Week 52
Secondary	Total iron removed by chelator (in mg) from baseline		Week 24
Secondary	Total iron removed by chelator (in mg) from baseline		Week 52