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

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ClinicalTrials.gov Identifier: NCT03801889
Recruitment Status : Withdrawn (Protocol suspended prior to patient enrollment)
First Posted : January 14, 2019
Last Update Posted : October 5, 2020
Sponsor:
Information provided by (Responsible Party):
Abfero Pharmaceuticals, Inc

Tracking Information
First Submitted Date  ICMJE January 8, 2019
First Posted Date  ICMJE January 14, 2019
Last Update Posted Date October 5, 2020
Estimated Study Start Date  ICMJE August 2020
Estimated Primary Completion Date September 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 10, 2019)
  • The incidence of treatment-emergent Adverse Events (AEs) [ Time Frame: Week 24 ]
  • The incidence of treatment-emergent Adverse Events (AEs) [ Time Frame: Week 52 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 10, 2019)
  • Change in liver iron concentration (LIC) on R2-MRI from baseline [ Time Frame: Week 24 ]
  • Change in liver iron concentration (LIC) on R2-MRI from baseline [ Time Frame: Week 52 ]
  • Change in cardiac iron content (CIC) on T2*-MRI from baseline [ Time Frame: Week 24 ]
  • Change in cardiac iron content (CIC) on T2*-MRI from baseline [ Time Frame: Week 52 ]
  • Total iron removed by chelator (in mg) from baseline [ Time Frame: Week 24 ]
  • Total iron removed by chelator (in mg) from baseline [ Time Frame: Week 52 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE SP-420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias
Official Title  ICMJE Multicenter, Open-label, 52 Week, Dose-escalation Study of SP 420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias
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 Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention Model Description:
This is a multi-center, open-label, dose-escalation study designed to evaluate the safety, tolerability, and iron clearing efficacy of SP 420 administered three-times per week in subjects with transfusion-dependent beta-thalassemia or other rare anemias. Approximately 24 subjects are to be enrolled in 3 cohorts (doses of 28 mg/kg, 56 mg/kg and 84 mg/kg) of approximately 8 subjects each. Based upon the results from lower dose cohorts, if needed the size of latter cohorts may be increased to improve the power of the study to detect efficacy to approximately 74 subjects in total.
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Iron Overload
  • Beta-Thalassemia
Intervention  ICMJE Drug: SP-420
Self-administered by mouth
Study Arms  ICMJE
  • Experimental: Cohort 1
    SP-420 initially at 28 mg/kg
    Intervention: Drug: SP-420
  • Experimental: Cohort 2
    SP -20 initially at 56 mg/kg
    Intervention: Drug: SP-420
  • Experimental: Cohort 3
    SP-420 initially at 84 mg/kg
    Intervention: Drug: SP-420
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Withdrawn
Actual Enrollment  ICMJE
 (submitted: September 30, 2020)
0
Original Estimated Enrollment  ICMJE
 (submitted: January 10, 2019)
74
Estimated Study Completion Date  ICMJE January 2023
Estimated Primary Completion Date September 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   Lebanon,   Thailand,   Turkey
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03801889
Other Study ID Numbers  ICMJE SP-420-705
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Abfero Pharmaceuticals, Inc
Study Sponsor  ICMJE Abfero Pharmaceuticals, Inc
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Ali Taher, MD, PhD American University of Beirut Medical Center
PRS Account Abfero Pharmaceuticals, Inc
Verification Date September 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP