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

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 Overview

• Status: Withdrawn
• Conditions: Iron Overload; Beta-Thalassemia
• Intervention / Treatment: Drug: SP-420

• Study Type: Interventional
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Toronto, Canada
    • University of Toronto- University Health Network
• Lebanon
  • Beirut, Lebanon
    • American University of Beirut Medical Center
• Thailand
  • Bangkok, Thailand
    • Siriraj Hospital
• Turkey
  • İzmir, Turkey
    • Ege University hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
The incidence of treatment-emergent Adverse Events (AEs)	Week 24
The incidence of treatment-emergent Adverse Events (AEs)	Week 52

Secondary Outcome Measures

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: Abfero Pharmaceuticals, Inc
• Investigators: Principal Investigator: Ali Taher, MD, PhD, American University of Beirut Medical Center

Study record dates

Study Major Dates

• Study Start (Anticipated): August 1, 2020
• Primary Completion (Anticipated): September 1, 2022
• Study Completion (Anticipated): January 1, 2023

Study Registration Dates

• First Submitted: January 8, 2019
• First Submitted That Met QC Criteria: January 10, 2019
• First Posted (Actual): January 14, 2019

Study Record Updates

• Last Update Posted (Actual): October 5, 2020
• Last Update Submitted That Met QC Criteria: September 30, 2020
• Last Verified: September 1, 2020

More Information

Terms related to this study

• Keywords: chelator
• Additional Relevant MeSH Terms: Metabolic Diseases; Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Iron Metabolism Disorders; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Iron Overload; Thalassemia; beta-Thalassemia
• Other Study ID Numbers: SP-420-705

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No