Combination Therapy Compared With Single-Drug Therapy in Patients With Cardiac Diseases

Study Results

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2005-06-30

Study Completion Date

2009-04-30

Brief Summary

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The purpose of this study is to determine whether left ventricular function improves more rapidly with deferoxamine (DFO) and deferiprone (L1) combination therapy than with DFO monotherapy in patients with thalassemia and decreased ejection fractions. Secondary aims include evaluating changes in myocardial iron burden using T2\* and estimating the relative incidence and severity of chelator-induced toxicity.

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Detailed Description

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DESIGN NARRATIVE:

Participants will be randomized to 1 year of treatment with L1/DFO combination therapy or DFO monotherapy. At baseline, 6 months, and 1 year on therapy, cardiac function will be assessed by MRI measurement of left ventricular ejection fraction (LVEF), T2\*, Holter monitoring, and electrocardiography. Additional monitoring for safety includes weekly blood testing, monthly visits, and periodic eye and ear exams.

Conditions

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Cardiovascular Diseases Heart Diseases Beta-Thalassemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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L1/DFO

Deferoxamine (DFO) and deferiprone (L1) combination therapy

Group Type EXPERIMENTAL

Deferoxamine

Intervention Type DRUG

Deferoxamine will be given daily for 12-24h/day 7 days a week either subcutaneous or intravenous at up to 50-60 mg/kg/day.

Deferiprone (L1)

Intervention Type DRUG

The dose of L1, 75mg/kg in three divided oral doses, is the maximum dose at which toxicity has been tested in prospective trials

DFO

Deferoxamine (DFO) monotherapy

Group Type ACTIVE_COMPARATOR

Deferoxamine

Intervention Type DRUG

Deferoxamine will be given daily for 12-24h/day 7 days a week either subcutaneous or intravenous at up to 50-60 mg/kg/day.

Interventions

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Deferoxamine

Deferoxamine will be given daily for 12-24h/day 7 days a week either subcutaneous or intravenous at up to 50-60 mg/kg/day.

Intervention Type DRUG

Deferiprone (L1)

The dose of L1, 75mg/kg in three divided oral doses, is the maximum dose at which toxicity has been tested in prospective trials

Intervention Type DRUG

Other Intervention Names

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DFO L1

Eligibility Criteria

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Inclusion Criteria

* Transfusion-dependent beta-thalassemia (eight or more transfusion episodes in the previous year)
* Left ventricular ejection fraction by MRI less than or equal to 56% by balanced steady-state free precession (SSFP) or 63% by spoiled gradient recalled echo (SPGR)
* Currently on treatment with subcutaneous or intravenous DFO; participants must be willing and able to chelate 7 days per week 12 - 24 hours per day
* Serum ferritin greater than 1000 µg/L or ferritin between 500 µg/L and 1000 µg/L and cardiac T2\* less than 20 ms

Exclusion Criteria

* Pacemaker, severe claustrophobia, or other contraindications to MRI; severe congestive heart failure (New York Heart Association Classification IV); congenital or acquired valvular heart disease significant enough to require surgery or medications
* Currently receiving treatment for hepatitis; renal insufficiency defined by a clinically significant abnormal serum creatinine with a calculated creatinine clearance of less than 50 ml/min according to the Cockroft formula
* A neutrophil count less than 1.5 x 109/L on two or more occasions at least 4 weeks apart within the past year and not associated with an acute viral illness or a platelet count less than 80 x 109/L on two or more occasions at least 4 weeks apart within the past year
* Treatment with L1 or Exjade during the previous 2 weeks or previous adverse experience to L1 requiring suspension
* Infection with HIV
* Active participation in other investigational drug or device studies
* Unwilling to consider treatment with DFO at a dose of 50-60 mg/kg 12-24 hours per day 7 days per week
* Women who are pregnant or breast feeding
* Systemic infection or cardiovascular, hepatic, renal, pulmonary, or gastrointestinal disease that would prevent patients from undergoing any of the study-required treatments or procedures or requires treatment with any contraindicated medication(s)
* Presence of any other condition that, in the opinion of the investigator, would make the patient unsuitable for enrollment or could interfere with the patient's compliance with the protocol; may include but is not limited to alcohol or drug abuse
* For women of child-bearing potential, an inability or unwillingness to use a highly effective method of contraception (e.g., implants, injectables, combined oral contraceptives, or some intrauterine devices)

Minimum Eligible Age

18 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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John Porter, MD

Role: PRINCIPAL_INVESTIGATOR

University College, London

Patricia J. Giardina, MD

Role: STUDY_CHAIR

Weill Medical College of Cornell University

Ellis J. Neufeld, MD

Role: STUDY_CHAIR

Boston Children's Hospital

Elliott P, Vichinsky, MD

Role: STUDY_CHAIR

Children's Hospital and Research Institute, Oakland

Sonja McKinlay, Ph.D.

Role: STUDY_CHAIR

New England Research Institutes, Inc.

Locations

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Children's Hospital of Los Angeles

Los Angeles, California, United States

Site Status

Children's Hospital

Oakland, California, United States

Site Status

Children's Memorial Hospital

Chicago, Illinois, United States

Site Status

Children's Hospital

Boston, Massachusetts, United States

Site Status

Weill Medical College of Cornell University

New York, New York, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Countries

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United States

References

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Padhani ZA, Gangwani MK, Sadaf A, Hasan B, Colan S, Alvi N, Das JK. Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with transfusion-dependent beta thalassaemia. Cochrane Database Syst Rev. 2023 Nov 17;11(11):CD011626. doi: 10.1002/14651858.CD011626.pub3.

Reference Type DERIVED

PMID: 37975597 (View on PubMed)