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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

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

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

NCT ID

NCT02274233

First Posted

October 21, 2014

Last Updated

September 28, 2015

Sponsor

Sideris Pharmaceuticals

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

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

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

Learn more about this trial

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

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