NCT02274233

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.

Trial Health

63
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
24

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Oct 2014

Geographic Reach
5 countries

7 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2014

Completed
20 days until next milestone

First Submitted

Initial submission to the registry

October 21, 2014

Completed
3 days until next milestone

First Posted

Study publicly available on registry

October 24, 2014

Completed
10 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2015

Completed
Last Updated

September 29, 2015

Status Verified

September 1, 2015

Enrollment Period

11 months

First QC Date

October 21, 2014

Last Update Submit

September 28, 2015

Conditions

Iron OverloadBeta-Thalassemia

Keywords

ChelationIron OverloadThalassemiaTransfusionIron Chelation

Outcome Measures

Primary Outcomes (1)

  • Number of Participants with Adverse Events

    Up to 35 days

Secondary Outcomes (2)

  • Peak Plasma Concentration (Cmax) of SP-420

    Day 7

  • Area under the plasma concentration versus time curve (AUC) of SP-420

    Day 7

Study Arms (6)

1.5 mg/kg

EXPERIMENTAL

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

Drug: SP-420

3 mg/kg

EXPERIMENTAL

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

Drug: SP-420

6 mg/kg

EXPERIMENTAL

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

Drug: SP-420

12 mg/kg

EXPERIMENTAL

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

Drug: SP-420

24 mg/kg

EXPERIMENTAL

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

Drug: SP-420

9 mg/kg

EXPERIMENTAL

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

Drug: SP-420

Interventions

SP-420DRUG
1.5 mg/kg12 mg/kg24 mg/kg3 mg/kg6 mg/kg9 mg/kg

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

Sideris Investigative Site

Boston, Massachusetts, 02115, United States

Location

Sideris Investigative Site

New York, New York, 10065, United States

Location

Sideris Investigative Site

Philadelphia, Pennsylvania, 19104, United States

Location

Sideris Investigative Site

Toronto, Ontario, M5G 2N2, Canada

Location

Sideris Investigative Site

Beirut, Lebanon

Location

Sideris Investigative Site

Bangkok, 10700, Thailand

Location

Sideris Investigative Site

Izmir, 35100, Turkey (Türkiye)

Location

MeSH Terms

Conditions

Iron Overloadbeta-ThalassemiaThalassemia

Interventions

SP-420

Condition Hierarchy (Ancestors)

Iron Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesAnemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Sideris Pharmaceuticals, Inc. Sideris Pharmaceuticals, Inc.

    Sideris Pharmaceuticals, Inc.

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 21, 2014

First Posted

October 24, 2014

Study Start

October 1, 2014

Primary Completion

September 1, 2015

Study Completion

September 1, 2015

Last Updated

September 29, 2015

Record last verified: 2015-09

Locations

TU(1)CA(1)TH(1)LE(1)US(3)