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Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02274233
Recruitment Status : Terminated (Changes in renal function parameters.)
First Posted : October 24, 2014
Last Update Posted : September 29, 2015
Sponsor:
Information provided by (Responsible Party):
Sideris Pharmaceuticals

Study Description
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Brief Summary:
The purpose of this study is to assess safety and amount of the study drug in the blood after increasing doses of SP-420. The study will be conducted in patients with β-thalassemia.

Condition or disease Intervention/treatment Phase
Iron Overload Beta-Thalassemia Drug: SP-420 Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 24 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Multicenter, Open-label, Dose-escalation Study of SP-420 in Subjects With Transfusion-dependent β-Thalassemia
Study Start Date : October 2014
Actual Primary Completion Date : September 2015
Actual Study Completion Date : September 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Iron Thalassemia

Arms and Interventions
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Arm Intervention/treatment
Experimental: 1.5 mg/kg
1.5 mg/kg SP-420 once daily for 14 days
 Drug: SP-420
Experimental: 3 mg/kg
3 mg/kg SP-420 once daily for 14 days
 Drug: SP-420
Experimental: 6 mg/kg
6 mg/kg SP-420 once daily for 14 days
 Drug: SP-420
Experimental: 12 mg/kg
12 mg/kg SP-420 once daily for 14 days
 Drug: SP-420
Experimental: 24 mg/kg
24 mg/kg SP-420 once daily for 28 days
 Drug: SP-420
Experimental: 9 mg/kg
9 mg/kg SP-420 twice daily for 28 days
 Drug: SP-420


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants with Adverse Events [ Time Frame: Up to 35 days ]

Secondary Outcome Measures :
  1. Peak Plasma Concentration (Cmax) of SP-420 [ Time Frame: Day 7 ]
  2. Area under the plasma concentration versus time curve (AUC) of SP-420 [ Time Frame: Day 7 ]

Eligibility Criteria
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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:

  • Subject has iron-overload secondary to β-thalassemia requiring chronic red blood cell transfusions and iron chelation therapy
  • Subject weighs ≥35 kg
  • Subject is willing to discontinue current iron chelation therapy at least 7 days prior to the first dose of SP-420 and for the duration of the study
  • Serum ferritin ≥700 ng/mL and iron saturation ≥70% within 3 weeks before Baseline (Day 1)
  • Cardiac T2* score >20 msec within 6 months before Baseline (Day 1)
  • Willing to use contraception during the study

Exclusion Criteria:

  • Pregnant or breast-feeding
  • Serum creatinine greater than the upper limit of normal
  • Platelet count <100 × 10^9/L
  • Use of another investigational drug within the last 30 days
  • Significant cardiac, renal, hepatic dysfunction or other clinically significant conditions that, in the opinion of the Investigator, would exclude the subject
Contacts and Locations
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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): NCT02274233


Locations
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United States, Massachusetts
Sideris Investigative Site
Boston, Massachusetts, United States, 02115
United States, New York
Sideris Investigative Site
New York, New York, United States, 10065
United States, Pennsylvania
Sideris Investigative Site
Philadelphia, Pennsylvania, United States, 19104
Canada, Ontario
Sideris Investigative Site
Toronto, Ontario, Canada, M5G 2N2
Lebanon
Sideris Investigative Site
Beirut, Lebanon
Thailand
Sideris Investigative Site
Bangkok, Thailand, 10700
Turkey
Sideris Investigative Site
Izmir, Turkey, 35100
Sponsors and Collaborators
Sideris Pharmaceuticals
Investigators
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Study Director: Sideris Pharmaceuticals, Inc. Sideris Pharmaceuticals, Inc. Sideris Pharmaceuticals, Inc.
More Information
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Responsible Party: Sideris Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02274233    
Other Study ID Numbers: SP-420-702
First Posted: October 24, 2014    Key Record Dates
Last Update Posted: September 29, 2015
Last Verified: September 2015
Keywords provided by Sideris Pharmaceuticals:
Chelation
Iron Overload
Thalassemia
Transfusion
Iron Chelation
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Iron Overload
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Iron Metabolism Disorders
Metabolic Diseases