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

Iron Overload Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02274233
• Other study ID #: SP-420-702
• Status: Terminated
• Phase: Phase 1
• First received: October 21, 2014
• Last updated: September 28, 2015
• Start date: October 2014
• Est. completion date: September 2015

Study information

• Verified date: September 2015
• Source: Sideris Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationLebanon: Ministry of Public HealthThailand: Food and Drug AdministrationCanada: Health CanadaTurkey: Ministry of Health
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 24
• Est. completion date: September 2015
• Est. primary completion date: September 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Subject has iron-overload secondary to ß-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 Design

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Beta-Thalassemia
• Iron Overload
• Thalassemia

Intervention

Drug:
• SP-420

Locations

Country	Name	City	State
Canada	Sideris Investigative Site	Toronto	Ontario
Lebanon	Sideris Investigative Site	Beirut
Thailand	Sideris Investigative Site	Bangkok
Turkey	Sideris Investigative Site	Izmir
United States	Sideris Investigative Site	Boston	Massachusetts
United States	Sideris Investigative Site	New York	New York
United States	Sideris Investigative Site	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Sideris Pharmaceuticals

Countries where clinical trial is conducted

United States,  Canada,  Lebanon,  Thailand,  Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants with Adverse Events		Up to 35 days	Yes
Secondary	Peak Plasma Concentration (Cmax) of SP-420		Day 7	No
Secondary	Area under the plasma concentration versus time curve (AUC) of SP-420		Day 7	No