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

Phase 2

Withdrawn

• Conditions: Iron Overload; Beta-Thalassemia
• Interventions: Drug: SP-420

• Registration Number: NCT03801889
• Lead Sponsor: Abfero Pharmaceuticals, Inc

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-01-08
• Study First Submitted QC: 2019-01-10
• Study First Posted: 2019-01-14
• Study Start: 2020-08
• Status Verified: 2020-09
• Last Update Submitted: 2020-09-30
• Last Update Posted: 2020-10-05
• Primary Completion: 2022-09
• Study Completion: 2023-01

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Cohort 1	SP-420	SP-420 initially at 28 mg/kg
Cohort 2	SP-420	SP -20 initially at 56 mg/kg
Cohort 3	SP-420	SP-420 initially at 84 mg/kg

Primary Outcome Measures

Name	Time	Method
The incidence of treatment-emergent Adverse Events (AEs)	Week 52

Secondary Outcome Measures

Name	Time	Method
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 52
Total iron removed by chelator (in mg) from baseline	Week 52

Trial Locations

Locations (4)

University of Toronto- University Health Network; 🇨🇦 Toronto, Canada

American University of Beirut Medical Center; 🇱🇧 Beirut, Lebanon

Ege University Hospital; 🇹🇷 İzmir, Turkey

Siriraj Hospital; 🇹🇭 Bangkok, Thailand