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

September 28, 2015 updated by: Sideris Pharmaceuticals

Multicenter, Open-label, Dose-escalation Study of SP-420 in Subjects With Transfusion-dependent β-Thalassemia

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

24

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5G 2N2
        • Sideris Investigative Site
  • Lebanon
      • Beirut, Lebanon
        • Sideris Investigative Site
  • Thailand
      • Bangkok, Thailand, 10700
        • Sideris Investigative Site
  • Turkey
      • Izmir, Turkey, 35100
        • Sideris Investigative Site
  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Sideris Investigative Site
    • New York
      • New York, New York, United States, 10065
        • Sideris Investigative Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Sideris Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

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:

  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of Participants with Adverse Events
Time Frame: Up to 35 days
Up to 35 days

Secondary Outcome Measures

Outcome Measure
Time Frame
Peak Plasma Concentration (Cmax) of SP-420
Time Frame: Day 7
Day 7
Area under the plasma concentration versus time curve (AUC) of SP-420
Time Frame: Day 7
Day 7

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sideris Pharmaceuticals

Investigators

  • Study Director: Sideris Pharmaceuticals, Inc. Sideris Pharmaceuticals, Inc., Sideris Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

October 1, 2014

Primary Completion (Actual)

September 1, 2015

Study Completion (Actual)

September 1, 2015

Study Registration Dates

First Submitted

October 21, 2014

First Submitted That Met QC Criteria

October 22, 2014

First Posted (Estimate)

October 24, 2014

Study Record Updates

Last Update Posted (Estimate)

September 29, 2015

Last Update Submitted That Met QC Criteria

September 28, 2015

Last Verified

September 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SP-420-702

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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