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Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias

Primary Purpose

Iron Overload, Sickle Cell Disease, Other Anemias

Status
Unknown status
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
Deferiprone
Sponsored by
ApoPharma
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Iron Overload focused on measuring Iron overload, Sickle cell disease, Deferiprone, Ferriprox, Iron chelation

Eligibility Criteria

3 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Completed study LA38-0411

  2. Females of childbearing potential must have a negative pregnancy test result at Visit 1. In addition, if applicable, they must:

    • Use an effective method of contraception according to local requirements, during the study and within 30 days following their last dose of study medication, OR
    • Have had a tubal ligation (supporting evidence required), OR
    • Have had a hysterectomy (supporting evidence required), OR
    • Participate in a non-heterosexual lifestyle, OR
    • Have a male sexual partner who has been sterilized (supporting evidence required)
  3. Fertile heterosexual males and/or their partners must agree to use an effective method of contraception during the study and for 30 days following the last dose of study medication
  4. All patients and/or their authorized legal representatives must provide signed and dated written informed consent prior to the first study intervention, and assent will be obtained from patients who are considered to be minors. Patients must be able to adhere to study restrictions, appointments, and evaluation schedules.

Exclusion Criteria:

  1. Plan to participate in another clinical trial at any time from the day of enrollment until 30 days post-treatment in the current study
  2. For only those patients who were treated with deferoxamine in study LA38-0411 (Group 2): Presence of any medical condition (including clinically significant laboratory abnormalities, such as ALT ≥ 5 x ULN or creatinine ≥ 2 x ULN), psychological condition, or psychiatric condition which in the opinion of the investigator would cause participation in the study to be unwise.
  3. Pregnant, breastfeeding, or planning to become pregnant during the study period.
  4. Treatment failure after 1 year on deferiprone which in the investigator's judgment indicates the need for the patient to be started on a different iron chelator

Sites / Locations

  • UCSF Benioff Children's Hospital Oakland
  • University of Michigan Comprehensive Cancer Center
  • Children's Hospital of Michigan
  • The Children's Hospital of Philadephia
  • Medical University of South Carolina
  • Hospital for Sick Kids
  • Zagazig University
  • Ain Shams University
  • Cairo University
  • Pediatric Hospital of Cairo University
  • Asser Central Hospital
  • Barts and The London
  • Evelina Children's Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Group 1: Deferiprone 3 years

Group 2: Deferiprone 2 years

Arm Description

Patients in this group are those who were randomized to the deferiprone arm in study LA38-0411, and hence will receive deferiprone for a total of 3 years (1 year in the initial study plus 2 years in the extension study)..

Patients in this group are those who were randomized to the deferoxamine arm in study LA38-0411, and hence will receive deferiprone for 2 years (both of them in the extension study).

Outcomes

Primary Outcome Measures

Number of subjects with adverse events (AEs)
Number of participants in each group with AEs, by frequency, severity, time to onset, duration, and relatedness to study product
Number of subjects with serious adverse events (SAEs)
Number of participants in each group with SAEs, by frequency, severity, time to onset, duration, and relatedness to study product
Discontinuations due to adverse events
Number of participants discontinued from the study because of adverse events

Secondary Outcome Measures

Change from baseline to end of study in liver iron concentration (LIC)
LIC will be determined by MRI
Change from baseline to end of study in cardiac MRI T2*
Myocardial MRI T2* will be used to monitor the myocardial iron load resulting from chronic transfusions. Low T2* levels reflect a high cardiac iron concentration and high T2* values reflect low cardiac iron.
Change from baseline to end of study in serum ferritin
Responder analysis
The percentage of patients who show a ≥20% decline from baseline in LIC or serum ferritin or a ≥20% increase from baseline in cardiac MRI T2*

Full Information

First Posted
May 5, 2015
Last Updated
January 10, 2019
Sponsor
ApoPharma
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1. Study Identification

Unique Protocol Identification Number
NCT02443545
Brief Title
Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias
Official Title
Long-term Safety and Efficacy Study of Ferriprox® for the Treatment of Transfusional Iron Overload in Patients With Sickle Cell Disease or Other Anemias
Study Type
Interventional

2. Study Status

Record Verification Date
January 2019
Overall Recruitment Status
Unknown status
Study Start Date
May 2015 (undefined)
Primary Completion Date
October 2021 (Anticipated)
Study Completion Date
April 2022 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
ApoPharma

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This is a long-term follow-up to an earlier study, LA38-0411. Its purpose is to gather more information about the safety and efficacy of deferiprone in patients with sickle cell disease or other anemias who suffer from iron overload caused by regular blood transfusions.
Detailed Description
Deferiprone (brand name Ferriprox®) is an iron chelator that is approved in the United States and over 60 other countries for the treatment of iron overload in patients with thalassemia, when other treatments are inadequate. This study has been designed to evaluate the long-term efficacy, safety, and tolerability of deferiprone to treat iron overload in patients who have sickle cell disease or other anemias. Only patients who have completed an earlier study, LA38-0411, are eligible to enroll in this one.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Iron Overload, Sickle Cell Disease, Other Anemias
Keywords
Iron overload, Sickle cell disease, Deferiprone, Ferriprox, Iron chelation

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
300 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Group 1: Deferiprone 3 years
Arm Type
Experimental
Arm Description
Patients in this group are those who were randomized to the deferiprone arm in study LA38-0411, and hence will receive deferiprone for a total of 3 years (1 year in the initial study plus 2 years in the extension study)..
Arm Title
Group 2: Deferiprone 2 years
Arm Type
Experimental
Arm Description
Patients in this group are those who were randomized to the deferoxamine arm in study LA38-0411, and hence will receive deferiprone for 2 years (both of them in the extension study).
Intervention Type
Drug
Intervention Name(s)
Deferiprone
Other Intervention Name(s)
Ferriprox tablets, Deferiprone oral solution
Primary Outcome Measure Information:
Title
Number of subjects with adverse events (AEs)
Description
Number of participants in each group with AEs, by frequency, severity, time to onset, duration, and relatedness to study product
Time Frame
From the first day of the study until the last study visit (Week 104 or early termination)
Title
Number of subjects with serious adverse events (SAEs)
Description
Number of participants in each group with SAEs, by frequency, severity, time to onset, duration, and relatedness to study product
Time Frame
From the first day of the study until 30 days after the last dose
Title
Discontinuations due to adverse events
Description
Number of participants discontinued from the study because of adverse events
Time Frame
From the first day of the study until Week 104
Secondary Outcome Measure Information:
Title
Change from baseline to end of study in liver iron concentration (LIC)
Description
LIC will be determined by MRI
Time Frame
Group 1: From Week 0 of study LA38-0411 until the last visit of study LA38-EXT (Week 104 or early termination). Group 2: From Week 0 of study LA38-EXT until the last visit (Week 104 or early termination).
Title
Change from baseline to end of study in cardiac MRI T2*
Description
Myocardial MRI T2* will be used to monitor the myocardial iron load resulting from chronic transfusions. Low T2* levels reflect a high cardiac iron concentration and high T2* values reflect low cardiac iron.
Time Frame
Group 1: From Week 0 of study LA38-0411 until the last visit of study LA38-EXT (Week 104 or early termination). Group 2: From Week 0 of study LA38-EXT until the last visit (Week 104 or early termination).
Title
Change from baseline to end of study in serum ferritin
Time Frame
Group 1: From Week 0 of study LA38-0411 until the last visit of study LA38-EXT (Week 104 or early termination). Group 2: From Week 0 of study LA38-EXT until the last visit (Week 104 or early termination).
Title
Responder analysis
Description
The percentage of patients who show a ≥20% decline from baseline in LIC or serum ferritin or a ≥20% increase from baseline in cardiac MRI T2*
Time Frame
Group 1: From Week 0 of study LA38-0411 to Weeks 0, 52, and 104 of LA38-EXT (1, 2, and 3 years of deferiprone treatment, respectively). Group 2: From Week 0 of study LA38-EXT to Weeks 52 and 104 (1 and 2 years of deferiprone treatment, respectively).

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Completed study LA38-0411 Females of childbearing potential must have a negative pregnancy test result at Visit 1. In addition, if applicable, they must: Use an effective method of contraception according to local requirements, during the study and within 30 days following their last dose of study medication, OR Have had a tubal ligation (supporting evidence required), OR Have had a hysterectomy (supporting evidence required), OR Participate in a non-heterosexual lifestyle, OR Have a male sexual partner who has been sterilized (supporting evidence required) Fertile heterosexual males and/or their partners must agree to use an effective method of contraception during the study and for 30 days following the last dose of study medication All patients and/or their authorized legal representatives must provide signed and dated written informed consent prior to the first study intervention, and assent will be obtained from patients who are considered to be minors. Patients must be able to adhere to study restrictions, appointments, and evaluation schedules. Exclusion Criteria: Plan to participate in another clinical trial at any time from the day of enrollment until 30 days post-treatment in the current study For only those patients who were treated with deferoxamine in study LA38-0411 (Group 2): Presence of any medical condition (including clinically significant laboratory abnormalities, such as ALT ≥ 5 x ULN or creatinine ≥ 2 x ULN), psychological condition, or psychiatric condition which in the opinion of the investigator would cause participation in the study to be unwise. Pregnant, breastfeeding, or planning to become pregnant during the study period. Treatment failure after 1 year on deferiprone which in the investigator's judgment indicates the need for the patient to be started on a different iron chelator
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Janet Kwiatkowski, MD
Organizational Affiliation
Children's Hospital of Philadelphia
Official's Role
Principal Investigator
Facility Information:
Facility Name
UCSF Benioff Children's Hospital Oakland
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
University of Michigan Comprehensive Cancer Center
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Children's Hospital of Michigan
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
The Children's Hospital of Philadephia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104-4399
Country
United States
Facility Name
Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
Hospital for Sick Kids
City
Toronto
State/Province
Ontario
Country
Canada
Facility Name
Zagazig University
City
Alexandria
Country
Egypt
Facility Name
Ain Shams University
City
Cairo
Country
Egypt
Facility Name
Cairo University
City
Cairo
Country
Egypt
Facility Name
Pediatric Hospital of Cairo University
City
Cairo
Country
Egypt
Facility Name
Asser Central Hospital
City
Abha
Country
Saudi Arabia
Facility Name
Barts and The London
City
London
Country
United Kingdom
Facility Name
Evelina Children's Hospital
City
London
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
36018224
Citation
Elalfy MS, Hamdy M, El-Beshlawy A, Ebeid FSE, Badr M, Kanter J, Inusa B, Adly AAM, Williams S, Kilinc Y, Lee D, Fradette C, Rozova A, Temin NT, Tricta F, Kwiatkowski JL. Deferiprone for transfusional iron overload in sickle cell disease and other anemias: open-label study of up to 3 years. Blood Adv. 2023 Feb 28;7(4):611-619. doi: 10.1182/bloodadvances.2021006778.
Results Reference
derived

Learn more about this trial

Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias

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