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

Status: Recruiting
Phase:
DFCI Protocol ID:

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.

Conducting Institutions:

Children's Hospital Boston, Dana-Farber Cancer Institute

Overall PI:

Ellis Neufeld, MD, PhD, Brigham and Women's Hospital

Site-responsible Investigators:

Contacts:

Dana-Farber Cancer Institute: Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP, ctip@partners.org

Eligibility 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

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