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

Primary Purpose

Iron Overload, Beta-Thalassemia

Status
Terminated
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
SP-420
Sponsored by
Sideris Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Iron Overload focused on measuring Chelation, Iron Overload, Thalassemia, Transfusion, Iron Chelation

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Sideris Investigative Site
  • Sideris Investigative Site
  • Sideris Investigative Site
  • Sideris Investigative Site
  • Sideris Investigative Site
  • Sideris Investigative Site
  • Sideris Investigative Site

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm 6

Arm Type

Experimental

Experimental

Experimental

Experimental

Experimental

Experimental

Arm Label

1.5 mg/kg

3 mg/kg

6 mg/kg

12 mg/kg

24 mg/kg

9 mg/kg

Arm Description

1.5 mg/kg SP-420 once daily for 14 days

3 mg/kg SP-420 once daily for 14 days

6 mg/kg SP-420 once daily for 14 days

12 mg/kg SP-420 once daily for 14 days

24 mg/kg SP-420 once daily for 28 days

9 mg/kg SP-420 twice daily for 28 days

Outcomes

Primary Outcome Measures

Number of Participants with Adverse Events

Secondary Outcome Measures

Peak Plasma Concentration (Cmax) of SP-420
Area under the plasma concentration versus time curve (AUC) of SP-420

Full Information

First Posted
October 21, 2014
Last Updated
September 28, 2015
Sponsor
Sideris Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT02274233
Brief Title
Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia
Official Title
Multicenter, Open-label, Dose-escalation Study of SP-420 in Subjects With Transfusion-dependent β-Thalassemia
Study Type
Interventional

2. Study Status

Record Verification Date
September 2015
Overall Recruitment Status
Terminated
Why Stopped
Changes in renal function parameters.
Study Start Date
October 2014 (undefined)
Primary Completion Date
September 2015 (Actual)
Study Completion Date
September 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sideris Pharmaceuticals

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Iron Overload, Beta-Thalassemia
Keywords
Chelation, Iron Overload, Thalassemia, Transfusion, Iron Chelation

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
24 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1.5 mg/kg
Arm Type
Experimental
Arm Description
1.5 mg/kg SP-420 once daily for 14 days
Arm Title
3 mg/kg
Arm Type
Experimental
Arm Description
3 mg/kg SP-420 once daily for 14 days
Arm Title
6 mg/kg
Arm Type
Experimental
Arm Description
6 mg/kg SP-420 once daily for 14 days
Arm Title
12 mg/kg
Arm Type
Experimental
Arm Description
12 mg/kg SP-420 once daily for 14 days
Arm Title
24 mg/kg
Arm Type
Experimental
Arm Description
24 mg/kg SP-420 once daily for 28 days
Arm Title
9 mg/kg
Arm Type
Experimental
Arm Description
9 mg/kg SP-420 twice daily for 28 days
Intervention Type
Drug
Intervention Name(s)
SP-420
Primary Outcome Measure Information:
Title
Number of Participants with Adverse Events
Time Frame
Up to 35 days
Secondary Outcome Measure Information:
Title
Peak Plasma Concentration (Cmax) of SP-420
Time Frame
Day 7
Title
Area under the plasma concentration versus time curve (AUC) of SP-420
Time Frame
Day 7

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Sideris Pharmaceuticals, Inc. Sideris Pharmaceuticals, Inc.
Organizational Affiliation
Sideris Pharmaceuticals, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Sideris Investigative Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Sideris Investigative Site
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Facility Name
Sideris Investigative Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Sideris Investigative Site
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 2N2
Country
Canada
Facility Name
Sideris Investigative Site
City
Beirut
Country
Lebanon
Facility Name
Sideris Investigative Site
City
Bangkok
ZIP/Postal Code
10700
Country
Thailand
Facility Name
Sideris Investigative Site
City
Izmir
ZIP/Postal Code
35100
Country
Turkey

12. IPD Sharing Statement

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

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