Trial Outcomes & Findings for Efficacy of Ferric Carboxymaltose in Gastrointestinal Stromal Tumor (GIST) Patients With Iron Deficiency Anemia (IDA) Receiving Systemic Therapy (NCT NCT02949947)

Last Updated: 2020-04-06

Results Overview

The primary endpoint is response (CR rate) in HGB within 3 months. Participant considered as to have a complete response (CR) if his/her HGB level increases \> 2 g/dL from baseline during 3 months following initiation of the study drug, and/or transfusion-dependent patient is transfusion free.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

3 participants

Primary outcome timeframe

3 months

Results posted on

2020-04-06

Participant Flow

Recruitment Period: 12/2017 to 1/2019

A total of 3 participants were enrolled in the study; however only 1 started on the trial since 1 participant non-compliance and 1 participant insurance changed. Due to low accrual the study terminated early.

Participant milestones

Participant milestones
Measure	Group A - Ferric Carboxymaltose Participants receive a Ferric Carboxymaltose injection by vein. Dose repeated 1 week later. Health questionnaire completed at Baseline and at Weeks 4, 8, 12, and 24. Ferric Carboxymaltose: 15 mg/kg by vein (up to 750 mg) over 15 min infusion. Dose repeated 1 week later. Questionnaire: Health questionnaire completed at Baseline and at Weeks 4, 8, 12, and 24.	Group B - Iron Supplement Participants take iron supplements by mouth every day for up to 3 months. Health questionnaire completed at Baseline and at Weeks 4, 8, 12, and 24. Iron Supplements: Participants take iron supplements by mouth every day for up to 3 months. Questionnaire: Health questionnaire completed at Baseline and at Weeks 4, 8, 12, and 24.
Overall Study STARTED	1	2
Overall Study COMPLETED	0	1
Overall Study NOT COMPLETED	1	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Group A - Ferric Carboxymaltose Participants receive a Ferric Carboxymaltose injection by vein. Dose repeated 1 week later. Health questionnaire completed at Baseline and at Weeks 4, 8, 12, and 24. Ferric Carboxymaltose: 15 mg/kg by vein (up to 750 mg) over 15 min infusion. Dose repeated 1 week later. Questionnaire: Health questionnaire completed at Baseline and at Weeks 4, 8, 12, and 24.	Group B - Iron Supplement Participants take iron supplements by mouth every day for up to 3 months. Health questionnaire completed at Baseline and at Weeks 4, 8, 12, and 24. Iron Supplements: Participants take iron supplements by mouth every day for up to 3 months. Questionnaire: Health questionnaire completed at Baseline and at Weeks 4, 8, 12, and 24.
Overall Study Non Compliance	0	1
Overall Study Insurance change	1	0

Baseline Characteristics

Efficacy of Ferric Carboxymaltose in Gastrointestinal Stromal Tumor (GIST) Patients With Iron Deficiency Anemia (IDA) Receiving Systemic Therapy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Group A - Ferric Carboxymaltose n=1 Participants Participants receive a Ferric Carboxymaltose injection by vein. Dose repeated 1 week later. Health questionnaire completed at Baseline and at Weeks 4, 8, 12, and 24. Ferric Carboxymaltose: 15 mg/kg by vein (up to 750 mg) over 15 min infusion. Dose repeated 1 week later. Questionnaire: Health questionnaire completed at Baseline and at Weeks 4, 8, 12, and 24.	Group B - Iron Supplement n=2 Participants Participants take iron supplements by mouth every day for up to 3 months. Health questionnaire completed at Baseline and at Weeks 4, 8, 12, and 24. Iron Supplements: Participants take iron supplements by mouth every day for up to 3 months. Questionnaire: Health questionnaire completed at Baseline and at Weeks 4, 8, 12, and 24.	Total n=3 Participants Total of all reporting groups
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: 3 months

Population: Due to early termination of the protocol and low accrual not enough data collected to analyze the Complete Response Rate in Hemoglobin.

Outcome measures

Outcome data not reported

Adverse Events

Group A - Ferric Carboxymaltose

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Group B - Iron Supplement

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Group A - Ferric Carboxymaltose n=1 participants at risk Participants receive a Ferric Carboxymaltose injection by vein. Dose repeated 1 week later. Health questionnaire completed at Baseline and at Weeks 4, 8, 12, and 24. Ferric Carboxymaltose: 15 mg/kg by vein (up to 750 mg) over 15 min infusion. Dose repeated 1 week later. Questionnaire: Health questionnaire completed at Baseline and at Weeks 4, 8, 12, and 24.	Group B - Iron Supplement n=2 participants at risk Participants take iron supplements by mouth every day for up to 3 months. Health questionnaire completed at Baseline and at Weeks 4, 8, 12, and 24. Iron Supplements: Participants take iron supplements by mouth every day for up to 3 months. Questionnaire: Health questionnaire completed at Baseline and at Weeks 4, 8, 12, and 24.
Gastrointestinal disorders Constipation	100.0% 1/1 • From baseline up to 3 months	0.00% 0/2 • From baseline up to 3 months

Additional Information

Saroj Vadhan,Clinical Professor, Cytokine & Supportive Oncology

UT MD Anderson Cancer Center

Phone: (713) 792-7966

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place