Trial Outcomes & Findings for Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia (NCT NCT01709032)

Results Overview

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

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

9 participants

Primary outcome timeframe

12 months

Results posted on

2019-03-06

Participant Flow

Participant milestones

Participant milestones
Measure	Deferasirox and Deferiprone Deferasirox and deferiprone
Overall Study STARTED	9
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Deferasirox and Deferiprone n=9 Participants Deferasirox and deferiprone
Age, Continuous	28.9 years STANDARD_DEVIATION 5.6 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Region of Enrollment United States	9 Participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months

Population: Number with 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

Outcome measures

Outcome measures
Measure	Deferasirox and Deferiprone n=6 Participants Deferasirox and deferiprone
Number of Participants With Improvement in Liver Iron Concentration	3 Participants

SECONDARY outcome

Timeframe: 12 months

Population: Number of participants with improvement

Improvement in Cardiac T2\* MRI from baseline to determine if there is a reduction of cardiac iron burden.

Outcome measures

Outcome measures
Measure	Deferasirox and Deferiprone n=6 Participants Deferasirox and deferiprone
Number of Participants With Improvement in Cardiac T2* MRI	3 Participants

Adverse Events

Deferasirox and Deferiprone

Serious events: 3 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Deferasirox and Deferiprone n=9 participants at risk Deferasirox and deferiprone
General disorders viral syndrome	11.1% 1/9 • Number of events 1
Infections and infestations perirectal abscess	11.1% 1/9 • Number of events 2
Gastrointestinal disorders Rectal bleeding from hemorrhoid	11.1% 1/9 • Number of events 1

Other adverse events

Other adverse events
Measure	Deferasirox and Deferiprone n=9 participants at risk Deferasirox and deferiprone
Musculoskeletal and connective tissue disorders Arthralgia	44.4% 4/9 • Number of events 7
Gastrointestinal disorders Diarrhea	44.4% 4/9 • Number of events 4
Gastrointestinal disorders Nausea/vomiting	33.3% 3/9 • Number of events 3
Gastrointestinal disorders Abdominal pain	22.2% 2/9 • Number of events 3
Ear and labyrinth disorders Tinnitus	11.1% 1/9 • Number of events 1
Hepatobiliary disorders ALT elevated	11.1% 1/9 • Number of events 1

Additional Information

Janet Kwiatkowski, MD

Children's Hospital of Philadelphia

Phone: 215-590-3437

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place