Study for Transfusionally Iron Overloaded Children, Adolescents and Adults Using FBS0701 (SSP-004184)

This study has suspended participant recruitment.
(Study activities have been suspended while sponsor evaluates nonclinical (rat) findings.The potential relevance of these findings to humans, if any, is unknown.)
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT01671111
First received: August 16, 2012
Last updated: March 18, 2014
Last verified: March 2014
  Purpose

The purpose of this extension study is to evaluate SSP-004184AQ in patients with transfusional iron overload and to provide data on long term safety and efficacy.

SSP-004184AQ is an iron chelator under development for chronic daily oral administration to patients with transfusional iron overload


Condition Intervention Phase
Iron Overload Due to Repeated Red Blood Cell Transfusions
Drug: SSP-004184AQ
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 2, Open-label, Multicentre, Extension Safety and Tolerability Study for Transfusionally Iron Overloaded Children, Adolescents and Adults Using SSP-004184 (SPD602)

Resource links provided by NLM:


Further study details as provided by Shire:

Primary Outcome Measures:
  • Change in liver iron concentration as determined by MRI [ Time Frame: up to 5 years ] [ Designated as safety issue: Yes ]
  • Change in cardiac iron concentration as determined by MRI [ Time Frame: up to 5 years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Change in serum ferritin levels [ Time Frame: up to 5 years ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 1500
Study Start Date: August 2012
Estimated Study Completion Date: June 2023
Estimated Primary Completion Date: May 2023 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: SSP-004814AQ Drug: SSP-004184AQ
Long term extension study of 8-75 mg/kg/d of SSP-004184AQ (equivalent to 7-68 mg/kg/d free acid or "active" form)
Other Name: SPD602

  Eligibility

Ages Eligible for Study:   6 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Subjects and/or parents willing and able to sign the approved informed consent/and assent (based on institutional guidelines).
  • An understanding, ability, and willingness to fully comply with study procedures and restrictions.
  • Has either completed a previous SSP-004184AQ study or prematurely discontinued from (with agreement from the Investigator and Shire physician) from one of the SSP-004184AQ studies.
  • If applicable, female subjects should be either:

    1. Post-menopausal (12 consecutive months of spontaneous amenorrhea), or
    2. Surgically sterile, or
    3. Females of child-bearing potential must have a negative urine pregnancy test at the Qualification and Enrollment Visit prior to dosing on Day 1. Females of child-bearing potential must agree to abstain from sexual activity that could result in pregnancy or agree to use acceptable methods of contraception.

      For subjects that did not transition directly from their feeder protocol and have initiated treatment with a chelator other than SSP-004184AQ:

  • Willing to discontinue all existing iron chelation therapies for a minimum period of 1 to 5 days prior to first dose of SSP-004184AQ.

(Note: This inclusion criterion is also applicable for subjects that were receiving chelators other than SSP-004184AQ in their feeder protocol.)

  • Serum ferritin greater than 500 ng/mL at the Qualification and Enrollment Visit.
  • Liver iron concentration (LIC) greater than or equal to 2.0 mg iron per g (equivalent dry weight, liver) determined by FerriScan® R2 MRI at the Qualification and Enrollment Visit (Day -28 to Day -8).

(Note: Younger subjects for whom MRI is not feasible will be considered iron overloaded on the basis of serum ferritin only.) - Mean of the previous 3 pre-transfusion hemoglobin concentrations greater than or equal to 7.5 g/dL.

Exclusion:

  • Unwilling to remain off all other existing chelation therapies during SSP-004184AQ dosing and for up to 24 hours from last dose.
  • A history of non-compliance in a prior FerroKin/Shire-sponsored SSP-004184AQ study (excluding dose suspensions that were medically warranted).
  • Cardiac MRI T2* less than 10.0 milliseconds at the Qualification and Enrollment Visit.
  • Cardiac left ventricular ejection fraction less than 50% at the Qualification and Enrollment Visit by MRI or below the locally determined normal range by echocardiography if MRI information is not available.
  • Evidence of clinically relevant oral, cardiovascular, gastrointestinal, endocrine, pulmonary, neurologic, psychiatric, immunologic, bone marrow, dermatologic, hepatic, biliary, or renal dysfunction where, in the opinion of the Investigator or the Shire Study Physician, treatment with SSP-004184AQ is relatively contraindicated.
  • Platelet count below 100,000/µL or absolute neutrophil count less than 1500/mm3 at the Qualification and Enrollment Visit.
  • Known sensitivity to any ingredient in the SSP-004184AQ formulation.
  • Pregnant or lactating females.

For subjects that did not transition directly from their feeder protocol and have initiated treatment with a chelator other than SSP-004184AQ:

  • In younger subjects for whom MRI is not feasible, evidence of severe cardiac dysfunction, as assessed by the Investigator.
  • Non-elective hospitalization within the 30 days prior to receiving the first dose of SSP 004184AQ.
  • For subjects greater than or equal to 18 years old: ALT greater than 200 IU/L at the Qualification and Enrollment Visit.

OR For subjects less than 18 years old: ALT greater than 180 IU/L at the Qualification and Enrollment Visit.

- For subjects greater than or equal to 18 years old: Evidence of severe renal insufficiency, eg, serum creatinine 1.5X above the upper limit of normal or proteinuria greater than 1 gm per day or a calculated glomerular filtration rate less than 40 mL/min.

OR For subjects less than 18 years old: Evidence of significant renal insufficiency, eg, serum creatinine above the upper limit of normal or proteinuria greater than 1 gm per day.

  • Use of any investigational agent within the 30 days prior to receiving the first dose of SSP-004184AQ.
  • Cardiac left ventricular ejection fraction below the locally determined normal range in the 12 months prior to Screening by echocardiography or MRI or less than 50% at Baseline testing by MRI (Echocardiograph is acceptable for LVEF if MRI information is not available).
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01671111

Locations
United States, California
Children's Hospital Oakland
Oakland, California, United States, 94609
United States, Massachusetts
Children's Hospital of Boston
Boston, Massachusetts, United States, 02115
Canada, Ontario
Toronto General Hospital
Toronto, Ontario, Canada, M5G 2C4
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Italy
Ospedale Regionale Microcitemie
Cagliari, Italy, 09121
Ospedale Galliera
Genova, Italy, 16128
San Luigi Hospital Thalassemia Centre
Orbassano (Torino), Italy, 10043
Thailand
Siriraj Hospital, Mahidol University
Bangkok, Thailand, 10700
United Kingdom
Whittington Hospital
London, United Kingdom, N19 5NF
Sponsors and Collaborators
Shire
Investigators
Principal Investigator: Ellis Neufeld, MD Boston Children's Hospital
  More Information

No publications provided

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01671111     History of Changes
Other Study ID Numbers: SPD602-301
Study First Received: August 16, 2012
Last Updated: March 18, 2014
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Iron Overload
Iron Metabolism Disorders
Metabolic Diseases

ClinicalTrials.gov processed this record on April 17, 2014