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

Sponsor

Sideris Pharmaceuticals (Industry)

Overall Status

Terminated

CT.gov ID

NCT02274233

Collaborator

(none)

Enrollment

Locations

Arms

Duration (Months)

3.4

Patients Per Site

0.3

Patients Per Site Per Month

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Iron Overload Beta-Thalassemia	Drug: SP-420	Phase 1

Study Design

Study Type:

Interventional

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 :

Oct 1, 2014

Actual Primary Completion Date :

Sep 1, 2015

Actual Study Completion Date :

Sep 1, 2015

Arms and Interventions

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

Primary Outcome Measures

Number of Participants with Adverse Events [Up to 35 days]

Secondary Outcome Measures

Peak Plasma Concentration (Cmax) of SP-420 [Day 7]
Area under the plasma concentration versus time curve (AUC) of SP-420 [Day 7]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts 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

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

Locations

	Site	City	State	Country	Postal Code
1	Sideris Investigative Site	Boston	Massachusetts	United States	02115
2	Sideris Investigative Site	New York	New York	United States	10065
3	Sideris Investigative Site	Philadelphia	Pennsylvania	United States	19104
4	Sideris Investigative Site	Toronto	Ontario	Canada	M5G 2N2
5	Sideris Investigative Site	Beirut		Lebanon
6	Sideris Investigative Site	Bangkok		Thailand	10700
7	Sideris Investigative Site	Izmir		Turkey	35100