Trial Outcomes & Findings for Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload (NCT NCT00901199)

Results Overview

Change in liver iron concentration from baseline to 12 months with the use of combined chelation therapy. The change was calculated as the liver iron concentration at 12 months minus the value at baseline.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

22 participants

Primary outcome timeframe

12 months

Results posted on

2021-08-12

Participant Flow

Participant milestones

Participant milestones
Measure	Deferasirox (Exjade) and Deferoxamine (DFO) Patients will receive combined chelation therapy using Deferasirox (Exjade) and Deferoxamine (DFO) with dosing based on liver iron concentration at baseline. Patients with moderate iron overload,defined as liver iron concentration of 5-15mg/g plus a documented endocrinopathy or cardiac iron (low MRI T2\*), will receive 7 days per week of Deferasirox (Exjade) 20-30 mg/kg and Deferoxamine (DFO) 50 mg/kg 3-5 days per week. Patients with high iron overload, defined as liver iron concentration over 15mg/g will receive Deferasirox (Exjade) 20-30 mg/kg 7 days per week and Deferoxamine (DFO) 50 mg/kg 5-7 days per week.
Overall Study STARTED	22
Overall Study COMPLETED	18
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Deferasirox (Exjade) and Deferoxamine (DFO) n=22 Participants Patients will receive combined chelation therapy using Deferasirox (Exjade) and Deferoxamine (DFO) with dosing based on liver iron concentration at baseline. Patients with moderate iron overload,defined as liver iron concentration of 5-15mg/g plus a documented endocrinopathy or cardiac iron (low MRI T2\*), will receive 7 days per week of Deferasirox (Exjade) 20-30 mg/kg and Deferoxamine (DFO) 50 mg/kg 3-5 days per week. Patients with high iron overload, defined as liver iron concentration over 15mg/g will receive Deferasirox (Exjade) 20-30 mg/kg 7 days per week and Deferoxamine (DFO) 50 mg/kg 5-7 days per week.
Age, Categorical Age, Categorical · <=18 years	6 Participants n=5 Participants
Age, Categorical Age, Categorical · Between 18 and 65 years	16 Participants n=5 Participants
Age, Categorical Age, Categorical · >=65 years	0 Participants n=5 Participants
Sex/Gender, Customized Sex/Gender Not Collected	22 Participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months

Population: Eighteen subjects completed one year of therapy. Four subjects did not complete the study for the following reasons: lost to follow-up (1), abdominal pain (1), death (1), and non-compliance (1).

Change in liver iron concentration from baseline to 12 months with the use of combined chelation therapy. The change was calculated as the liver iron concentration at 12 months minus the value at baseline.

Outcome measures

Outcome measures
Measure	Deferasirox (Exjade) and Desferal (DFO) n=18 Participants Subjects were treated with Deferasirox (Exjade) and Desferal (DFO) with dosing based on baseline iron overload.
Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 12 Months	-6.2 mg/g Interval -23.1 to 2.7

PRIMARY outcome

Timeframe: 12 months

Comparison of average serum creatinine over 12 months of combined chelation therapy compared with baseline serum creatinine.

Outcome measures

Outcome measures
Measure	Deferasirox (Exjade) and Desferal (DFO) n=22 Participants Subjects were treated with Deferasirox (Exjade) and Desferal (DFO) with dosing based on baseline iron overload.
Change in Serum Creatinine During 12 Months Combined Chelation Therapy	0.09 mg/dl Interval 0.05 to 0.12

Adverse Events

Deferasirox (Exjade) and Deferoxamine (DFO)

Serious events: 2 serious events

Other events: 1 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Deferasirox (Exjade) and Deferoxamine (DFO) n=22 participants at risk Treatment with Deferasirox (Exjade) and Deferoxamine (DFO) with dosing based on baseline iron overload.
Hepatobiliary disorders Cholelithiasis	4.5% 1/22 • Number of events 1 • 1 year
Infections and infestations Central venous line infection	4.5% 1/22 • Number of events 1 • 1 year
Cardiac disorders Death	4.5% 1/22 • Number of events 1 • 1 year

Other adverse events

Other adverse events
Measure	Deferasirox (Exjade) and Deferoxamine (DFO) n=22 participants at risk Treatment with Deferasirox (Exjade) and Deferoxamine (DFO) with dosing based on baseline iron overload.
Blood and lymphatic system disorders Transfusion reaction	4.5% 1/22 • Number of events 1 • 1 year

Additional Information

Ashutosh Lal, MD

Children's Hospital & Research Center at Oakland

Phone: 510-428-3172

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place