Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia

• Sponsor: Genzyme, a Sanofi Company (Industry)
• Overall Status: Completed
• CT.gov ID: NCT00069862
• Collaborator: (none)
• Enrollment: 25
• Location: 1
• Duration (Months): 26
• Patients Per Site Per Month: 1

Study Details

Study Description

Brief Summary

A clinical trial designed to compare the safety and iron excretion properties of desferoxamine (DFO) and deferitrin (GT56-252), an experimental oral iron chelator.

Condition or Disease	Intervention/Treatment	Phase
Beta-Thalassemia	Drug: Deferitrin (GT56-252); Drug: desferoxamine (DFO)	Phase 1/Phase 2

Study Design

• Study Type: Interventional
• Allocation: Non-Randomized
• Intervention Model: Crossover Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia
• Study Start Date: Sep 1, 2003
• Actual Study Completion Date: Nov 1, 2005

Outcome Measures

Primary Outcome Measures

1. safety and tolerability []

2. iron excretion in urine and stool []

3. pharmacokinetic measurements []

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Beta-thalassemia patients, 18 years of age or older, currently undergoing chronic blood transfusion therapy and iron chelation therapy who weigh more than 40 kg.

• No clinically significant findings on physical exam, medical history, or screening laboratories.

• Serum ferritin greater than 500 ng/mL, serum creatine creatinine within the normal range and platelet count greater than 100,000/mm3.

• Willing and able to discontinue DFO or L1 for the period of study.

• Woman of child-bearing potential must have a negative serum pregnancy test at screening and use a medically acceptable form of birth control during the study and for 1 month afterward. Male patients must also use barrier contraceptives during the study and for 1 month afterward.

• Have a level of understanding and willingness to cooperate with the confinement and all procedures. Able to provide voluntary signed/dated written informed consent.

Exclusion Criteria:

• Serious medical condition unrelated to Beta-Thalassemia.

• Participation in a previous investigational drug study within 30 days preceding screening.

• Patients with a known allergy to DFO that prevents chronic administration.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	The New York Presbyterian Hospital-Weill Medical College of Cornell University	New York	New York	United States	10021

Sponsors and Collaborators

• Genzyme, a Sanofi Company

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• ClinicalTrials.gov Identifier: NCT00069862
• Other Study ID Numbers: GTC-134-102
• First Posted: Oct 3, 2003
• Last Update Posted: Mar 5, 2015
• Last Verified: Mar 1, 2015

• Keywords provided by , ,: Beta Thalassemia
• Additional relevant MeSH terms: Thalassemia; beta-Thalassemia; Iron Overload

Study Results

No Results Posted as of Mar 5, 2015