Efficacy Study in Removing Excess Iron From the Heart

Brief Title

Efficacy Study in Removing Excess Iron From the Heart

Official Title

Randomized Trial Comparing the Relative Efficacy of Deferiprone to That of Deferoxamine in Removing Excess Cardiac Iron in Thalassemia Major Patients

Brief Summary

      The purpose of this study is to determine whether deferiprone has superior efficacy in
      removing excess iron from the heart when compared with deferoxamine.
    

Detailed Description

      This study is a multi-center, randomized, open-label, controlled clinical trial. The study
      population is participants with thalassemia major who are receiving regular chelation therapy
      with deferoxamine. A total of sixty (60) participants will be enrolled among the
      investigative sites.

      The primary objective of this study is to determine whether deferiprone exhibits superior
      efficacy in removing excess iron from the heart compared to that of the standard therapy,
      deferoxamine.

      The secondary objective is to evaluate the relative efficacy of deferiprone with respect to
      that of deferoxamine as assessed by serum ferritin concentration and liver iron
      concentration.

      The primary efficacy measure in this study will be the participants' cardiac iron status, as
      determined by heart MRI T2* assessments.

      The secondary efficacy measure will be by serum ferritin concentration and liver iron
      concentration. This will be measured by the Superconducting Quantum-Interference Device
      (SQUID) BioSusceptometer.

      The duration of treatment is 12 months.
    

Study Phase

Phase 4

Study Type

Interventional


Primary Outcome

To determine whether deferiprone exhibits superior efficacy in removing excess iron from the heart compared to that of deferoxamine, as reflected by MRI T2* assessments in the heart in participants treated with either chelator

Secondary Outcome

 To evaluate the relative efficacy of deferiprone with respect to that of deferoxamine as assessed by serum ferritin concentration and liver iron concentration (LIC)

Condition

Thalassemia Major

Intervention

Ferriprox (deferiprone)


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

60

Start Date

December 2002

Completion Date

October 2004


Eligibility Criteria

        Inclusion Criteria:

          -  Diagnosis of thalassemia major as confirmed by laboratory and clinical criteria

          -  Participants who are well transfused-maintaining a mean pre-transfusion Hb
             (hemoglobin) no less than 9 g/dL.

          -  Between 18 and 36 years of age.

          -  Receiving ongoing chelation therapy with deferoxamine for at least the past five
             years. Those who have been exposed to deferiprone for

             ≤ 6 months but not within the last 2 years prior to commencement of this study will be
             considered eligible to participate.

          -  Abnormal heart MRI T2* greater than or equal to 8 ms and < 20 ms.

          -  If female, fertile, and is neither pregnant nor lactating, confirms she will use an
             effective method of contraception for the length of the trial and has a negative
             pregnancy test immediately prior to commencement of study drug OR has had a tubal
             ligation OR a hysterectomy OR is post menopausal (at least 1 year no menses prior to
             enrollment in the study) OR their only sexual partner has been sterilized (if male).

          -  If male and fertile, he confirms that he and/or his partner will use an effective
             method of contraception for the length of the trial.

          -  Provide a signed and witnessed written informed consent obtained prior to the first
             study intervention.

        Exclusion Criteria:

          -  Have anemia other than thalassemia.

          -  HIV antibody positive.

          -  Clinical evidence of cardiomyopathy as shown by LV Shortening Fraction < 30 % and/or
             CMR derived LV (left ventricular) Ejection Fraction < 56 %.

          -  Severe/significant arrhythmia, including those who have had atrial fibrillation
             (participants with occasional ectopic beats and normal echo can be included) or those
             requiring treatment.

          -  Previously discontinued therapy with deferiprone or deferoxamine because of an adverse
             drug reaction to either chelator.

          -  Have received deferiprone in the last five years. However those who have been exposed
             to deferiprone for ≤ 6 months but not within the last 2 years prior to commencement of
             this study will be considered eligible to participate.

          -  Evidence of abnormal liver function (liver enzymes > 3 times upper limit of normal -
             entry may be delayed until return to normal).

          -  Have disorders associated with neutropenia (ANC < 1.5 x 10^9/L) or thrombocytopenia
             (platelet count <50 x 10^9/L) in the twelve months prior to start of study medication,
             except for participants who have been treated with interferon and in whom the ANC has
             fully recovered. Participants with neutropenia or thrombocytopenia in the last year,
             which resolved with splenectomy, may be considered for this study.

          -  Those who refuse to participate in the screening procedures or who are unable to
             participate in screening procedures or who are unable to comply with requirements of
             the protocol.

          -  Receiving other investigational products.

          -  Those in the opinion of the Investigator, who represent poor medical, psychological or
             psychiatric risks for whom participation in an investigational trial would be unwise.

          -  Those who are pregnant, breastfeeding or planning to become pregnant during the study
             period.

          -  Metallic objects in his/her body, such as artificial joints, inner ear (cochlear)
             implants, brain aneurysm clips, pacemakers, and metallic foreign bodies in the eye or
             other body areas.

          -  History of malignancy.

          -  Participants with claustrophobia.

          -  History of alcohol or drug abuse.

          -  Participants who are, in the opinion of the Investigator, excessively obese.
      

Gender

All

Ages

18 Years - 36 Years

Accepts Healthy Volunteers

No

Contacts

Renzo Galanello, M.D., , 

Location Countries

Greece

Location Countries

Greece

Administrative Informations


NCT ID

NCT00105495

Organization ID

LA16-0102



Study Sponsor

ApoPharma


Study Sponsor

Renzo Galanello, M.D., Principal Investigator, Ospedale Regionale Microcitemie, Cagliari, Italy


Verification Date

October 2006