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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03801889
Recruitment Status : Not yet recruiting
First Posted : January 14, 2019
Last Update Posted : January 14, 2019
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Iron Overload Beta-Thalassemia Drug: SP-420 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 74 participants
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
Official Title: Multicenter, Open-label, 52 Week, Dose-escalation Study of SP 420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias
Estimated Study Start Date : May 2019
Estimated Primary Completion Date : September 2021
Estimated Study Completion Date : January 2022


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




Primary Outcome Measures :
  1. The incidence of treatment-emergent Adverse Events (AEs) [ Time Frame: Week 24 ]
  2. The incidence of treatment-emergent Adverse Events (AEs) [ Time Frame: Week 52 ]

Secondary Outcome Measures :
  1. Change in liver iron concentration (LIC) on R2-MRI from baseline [ Time Frame: Week 24 ]
  2. Change in liver iron concentration (LIC) on R2-MRI from baseline [ Time Frame: Week 52 ]
  3. Change in cardiac iron content (CIC) on T2*-MRI from baseline [ Time Frame: Week 24 ]
  4. Change in cardiac iron content (CIC) on T2*-MRI from baseline [ Time Frame: Week 52 ]
  5. Total iron removed by chelator (in mg) from baseline [ Time Frame: Week 24 ]
  6. Total iron removed by chelator (in mg) from baseline [ Time Frame: Week 52 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03801889


Contacts
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Contact: Thomas Neenan, PhD +17812667297 tneenan@abfero.com
Contact: Ajay Duggal, MD +447715366179 aduggal@abfero.com

Locations
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Canada
University of Toronto- University Health Network
Toronto, Canada
Contact: Kevin Kuo, MD         
Lebanon
American University of Beirut Medical Center
Beirut, Lebanon
Contact: Ali Taher, MD         
Thailand
Siriraj Hospital
Bangkok, Thailand
Contact: Vip Viprakasit, MD         
Turkey
Ege University Hospital
İzmir, Turkey
Contact: Yesim Aydinok, MD         
Sponsors and Collaborators
Abfero Pharmaceuticals, Inc
Investigators
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Principal Investigator: Ali Taher, MD, PhD American University of Beirut Medical Center

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Responsible Party: Abfero Pharmaceuticals, Inc
ClinicalTrials.gov Identifier: NCT03801889    
Other Study ID Numbers: SP-420-705
First Posted: January 14, 2019    Key Record Dates
Last Update Posted: January 14, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Abfero Pharmaceuticals, Inc:
chelator
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Iron Overload
Anemia
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn
Iron Metabolism Disorders
Metabolic Diseases