Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia

NCT01709032 | Completed Phase 1 Results

• 1 other identifier: 12-009449
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 2

Brief Summary

We hypothesize that the combination treatment with deferasirox and deferiprone will be well tolerated and will result in significant improvement in cardiac and liver iron levels.

Conditions

Thalassemia Major With Severe Transfusional Iron Overload

Keywords

Thalassemia; Chelation; Transfusion; Safety

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Improvement in Liver Iron Concentration

  Determine the safety of the combination of Deferasirox and Deferiprone for the treatment of subjects with Thalassemia Major and Severe Iron Overload by assessing change in liver iron concentration from baseline to follow-up

  12 months

Secondary Outcomes (1)

• Number of Participants With Improvement in Cardiac T2* MRI

  12 months

Study Arms (1)

Deferasirox and deferiprone

EXPERIMENTAL

Drug: Deferasirox and deferiprone

Interventions

Deferasirox and deferiprone DRUG

Also known as: Exjade, Ferriprox

Deferasirox and deferiprone

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Alpha or beta thalassemia
• Receiving chronic transfusions (at least 20 transfusions in lifetime) with iron overload requiring treatment with chelation
• Serum ferritin \>500 ng/ml
• Liver iron concentration equal to or greater than 10 mg/g dw (by R2 MRI) or 7 to 10 mg/g dw (by R2 MRI) and not improving OR cardiac T2\* between 6 and \<20 ms
• Women of childbearing age must have a negative pregnancy test
• Agree to use approved method of contraception for the duration of the study
• Subjects must have a good understanding of the study and be willing to comply with study procedures

You may not qualify if:

• Subjects with past history of unexplained neutropenia (ANC \< 1500/mcL), clinically significant renal disease (creatinine above the upper limit of normal), proteinuria \>300 mg/L, clinically significant liver disease (ALT \> 5x upper limit of normal), pulmonary or cardiovascular disease
• History of other clinically relevant oral, endocrine, neurologic, psychiatric, immunologic, bone marrow or skin disorder that contraindicates dosing with deferasirox or deferiprone
• History of adverse reaction or known allergy to either deferasirox or deferiprone necessitating drug discontinuation
• Currently receiving treatment for active hepatitis
• Use of any investigational agent in the past 30 days
• Cardiac T2\* \<6 ms, left ventricular ejection fraction \< 56%, and/or arrhythmia (certain subjects may be eligible if they have already had a trial of deferoxamine and deferiprone). Subjects who refuse to use deferoxamine after extensive consultation with at least 2 health care providers will also be allowed to participate.
• Pregnant or breastfeeding females
• Unwilling or unable to comply with study related procedures

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead
• The Cooley's Anemia Foundation,: collaborator
• Ann & Robert H Lurie Children's Hospital of Chicago: collaborator

Study Sites (2)

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Thalassemia

Interventions

Deferasirox; Deferiprone

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

Intervention Hierarchy (Ancestors)

Benzoates; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Triazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Pyridones; Pyridines

Results Point of Contact

• Title: Janet Kwiatkowski, MD
• Organization: Children's Hospital of Philadelphia

kwiatkowski@email.chop.edu

215-590-3437

Study Officials

• Janet L Kwiatkowski, MD, MSCE

  Children's Hospital of Philadlephia

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 16, 2012
• First Posted: October 17, 2012
• Study Start: September 1, 2012
• Primary Completion: August 1, 2015
• Study Completion: August 1, 2017
• Last Updated: March 6, 2019
• Results First Posted: March 6, 2019

Record last verified: 2019-02

Locations

US (2)