Efficacy Study in Removing Excess Iron From the Heart

NCT00105495 | Completed Phase 4

• 1 other identifier: LA16-0102
• Study Type: interventional
• Target: 60
• Locations: 2 countries
• Sites: 4

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.

Conditions

Thalassemia Major; Hemosiderosis

Keywords

Iron Overload; Thalassemia; Haemosiderosis; Cardiac; Deferiprone; Chelation

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

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

Interventions

Ferriprox (deferiprone) DRUG

Desferal (deferoxamine) DRUG

Eligibility Criteria

• Age: 18 Years - 36 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• ApoPharma: lead

Study Sites (4)

1st Department of Pediatrics, Athens University, Aghia Sophia Children's Hospital

Athens, 11527, Greece

Location

Aghia Sophia Children's Hospital

Athens, 11527, Greece

Location

Ospedale Regionale Microcitemie, Dipartimento di Scienze

Cagliari, Sardinia, 09100, Italy

Location

Dipartimento di Scienze e Dell' Adolescenza, University of Turin

Turin, 10126, Italy

Location

MeSH Terms

Conditions

beta-Thalassemia; Hemosiderosis; Iron Overload; Thalassemia; Hemochromatosis

Interventions

Deferiprone; Deferoxamine

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Metal Metabolism, Inborn Errors; Metabolism, Inborn Errors

Intervention Hierarchy (Ancestors)

Pyridones; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Hydroxamic Acids; Hydroxylamines; Amines; Organic Chemicals; Hydroxy Acids; Carboxylic Acids

Study Officials

• Renzo Galanello, M.D.

  Ospedale Regionale Microcitemie, Cagliari, Italy

  PRINCIPAL INVESTIGATOR

• Antonio Piga, M.D.

  Dipartimento di Scienze Pediatriche e Dell'Adolescenza, University of Turin, Turin, Italy

  PRINCIPAL INVESTIGATOR

• Markissia Karagiorga, M.D.

  Aghia Sophia Children's Hospital, Athens, Greece

  PRINCIPAL INVESTIGATOR

• Vassilis Ladis, M.D.

  1st Department of Pediatrics, Athens University, Aghia Sophia Children's Hospital, Athens, Greece

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: March 15, 2005
• First Posted: March 16, 2005
• Study Start: December 1, 2002
• Study Completion: October 1, 2004
• Last Updated: January 5, 2016

Record last verified: 2006-10

Locations

IT (2); GR (2)