Malignant Neoplasms of Mesothelial and Soft TissuePhase 2NCT02949947
Efficacy of Ferric Carboxymaltose in Gastrointestinal Stromal Tumor (GIST) Patients With Iron Deficiency Anemia (IDA) Receiving Systemic Therapy
A Phase 2 study testing Ferric Carboxymaltose, Iron Supplements and Questionnaire in people with Malignant Neoplasms of Mesothelial and Soft Tissue and Gastrointestinal Stromal Tumor With Neurogenic Differentiation, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
The goal of this clinical research study is to compare Injectafer® (ferric carboxymaltose) with an iron supplement to learn which may be more effective in improving red blood cell counts in patients who have iron-deficiency anemia (a low red blood cell count) because of a gastrointestinal stromal tumor (GIST) and/or… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Malignant Neoplasms of Mesothelial and Soft Tissue, Gastrointestinal Stromal Tumor With Neurogenic Differentiation
- What is being tested?
- Ferric Carboxymaltose, Iron Supplements and Questionnaire
- Type of treatment
- Pill or oral medicine, Injection or infusion, Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Dec 2017to 6 Mar 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- GIST patients with IDA planned to start or are receiving systemic therapy with TKIs.
- Evidence of iron deficiency anemia including, Hgb < 11 g/dL, but > 8 g/dL; and transferrin saturation (TSAT) < 20%.
- No H/O allergic reaction to iron therapy.
- No clinical signs active of bleeding.
- Adequate hematologic (ANC > 1500/mm^3, platelet count > 100,000/mm^3), renal (serum creatinine < 1.5mg/dL), and hepatic (serum bilirubin count < 1.5 x normal and serum glutamic-oxaloacetic transaminase (SGOT) or serum glutamic pyruvic transaminase (SGPT) < 3 x normal) functions.
- Patients must have Eastern Cooperative Oncology Group (ECOG) performance status of 0 - 2.
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- Signed informed consent to the study.
- Male and Females of child bearing potential must use acceptable methods of birth control which include oral contraceptives, spermicide with either a condom, diaphragm or cervical cap, use of an intrauterine device (IUD) or abstinence.
- Patients are required to read and understand English to comply with protocol requirements.
- Age >=18 years old.
- Life expectancy of at least 6 months.
Who cannot join
- Pregnant or lactating women.
- Patients with any co-morbid condition which renders patients at high risk of treatment complication.
- Patient has uncontrolled angina, congestive heart failure (New York Heart Association > class II or known ejection fraction < 40%), uncontrolled cardiac arrhythmia or hypertension, or acute myocardial infarction within 3 months.
- Patient has an active seizure disorder. (Patients with a previous history of seizure disorders will be eligible for the study, if they have had no evidence of seizure activity, and they have been free of antiseizure medication for the previous 5 years).
- Psychological, social, familial, or geographical reasons that would prevent scheduled visits and follow-up.
- Prior surgery or radiotherapy (RT) within 2 weeks of study entry.
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- Known hypersensitivity reaction to any component of ferric carboxymaltose.
- Any anemia treatment within 4 weeks before inclusion (oral iron, IV iron, or erythropoiesis-stimulating agents), or transfusion of PRBCs in 2 weeks.
- Hemochromatosis or other iron storage disorders.
- Known positive hepatitis with evidence of active disease.
- Patients with overt bleeding.
- Ferritin >/= 800 ng/mL.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
About this study
The goal of this clinical research study is to compare Injectafer® (ferric carboxymaltose) with an iron supplement to learn which may be more effective in improving red blood cell counts in patients who have iron-deficiency anemia (a low red blood cell count) because of a gastrointestinal stromal tumor (GIST) and/or systemic therapy.
The safety of ferric carboxymaltose will also be studied.
Read more
This is an investigational study. Ferric carboxymaltose is FDA approved and commercially available to treat iron deficiency anemia; however, it is considered investigational to use in patients who have cancer-related or systemic therapy-related anemia.
Up to 50 participants will take part in this study. All will be enrolled at MD Anderson.
Read the detailed description
Study Groups and Study Drug Administration:
If you are found to be eligible to take part in this study, you will be randomly assigned (as in the flip of a coin) to 1 of 2 study groups. This is done because no one knows if one group is better, the same, or worse than the other.
- If you are in Group A, you will receive ferric carboxymaltose injection by vein over about 15 minutes. You will receive 2 injections about 7 days apart (for example, on Days 0 [the day you are assigned to a study group] and 7).
- If you are in Group B, you will take iron supplements by mouth every day. This is considered standard of care for iron deficiency anemia and the study staff will discuss with you which iron supplements you will take and their risks.
You and the study staff will know to which group you are assigned.
Length of Study:
You may receive up to 2 injections of ferric carboxymaltose (if you are in Group A) or up to 3 months of oral iron supplements (if you are in Group B). You will no longer be able to take the study drug if intolerable side effects occur or if you are unable to follow study directions.
Your participation on the study will be over after you have completed the Week 24 visit.
Study Visits:
Baseline (within 1 week after you have been assigned to a study group):
- You will have a physical exam.
- You will complete a questionnaire about your health. It should take about 5 minutes to complete.
- Blood (about 1 tablespoon) will be drawn for routine tests and to test the level of iron in your blood.
One (1) time every week during Months 1-3, blood (about 1 tablespoon) will be drawn for routine tests.
At about Weeks 4, 8, 12, and 24 (the end-of-study visit):
- You will have a physical exam (Weeks 12 and 24 only).
- You will complete the same questionnaire you did at baseline.
- Blood (about 1 tablespoon) will be drawn for routine tests and to test the level of iron in your blood.
During Weeks 16 and 20, blood (less than 1 tablespoon) will be drawn to test the level of iron in your blood.
If you leave the study before Week 24, you will have the Week 24 study visits as soon as possible after you leave the study.
- Conditions
- Malignant Neoplasms of Mesothelial and Soft Tissue, Gastrointestinal Stromal Tumor With Neurogenic Differentiation
- Being tested
- Ferric Carboxymaltose, Iron Supplements, Questionnaire
- Keywords
- Malignant Neoplasms of Mesothelial and Soft Tissue, Gastrointestinal Stromal Tumor, GIST, Iron-deficiency anemia, Ferric Carboxymaltose, Injectafer, Iron supplement, Health Questionnaire, Survey
- Medical subject headings
- Plexosarcoma, Gastrointestinal Stromal Tumors, Anemia, Iron-Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ferric CarboxymaltoseDrug
Also called Injectafer
15 mg/kg by vein (up to 750 mg) over 15 min infusion. Dose repeated 1 week later.
Iron SupplementsDietary supplement
Participants take iron supplements by mouth every day for up to 3 months.
QuestionnaireBehavioural
Also called Survey
Health questionnaire completed at Baseline and at Weeks 4, 8, 12, and 24.
| Study group | Role | What they receive |
|---|---|---|
| Group A - Ferric Carboxymaltose | Experimental | 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. Receives: Drug: Ferric Carboxymaltose; Behavioral: Questionnaire |
| Group B - Iron Supplement | Active comparator | 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. Receives: Dietary Supplement: Iron Supplements; Behavioral: Questionnaire |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete Response Rate in Hemoglobin (HGB)
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.
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Oct 2016 (estimated)
- Study start18 Dec 2017
- Primary completion6 Mar 2019
- Study completion6 Mar 2019
Why it stopped: Terminated per PI's request
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Malignant Neoplasms of Mesothelial and Soft TissueStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- American Regent, Inc.
- Responsible party
- Sponsor
- Investigators
- Saroj Vadhan-Raj, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02949947
- Sponsor's protocol ID
- 2015-0588
- Other IDs
- NCI-2016-01925
- Status last verified
- Mar 2020
- First submitted
- 27 Oct 2016
- Last updated
- 6 Apr 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Ferric Carboxymaltose in Gastrointestinal Stromal Tumor (GIST) Patients With Iron Deficiency Anemia (IDA) Receiving Systemic Therapy
- ClinicalTrials.gov ID
- NCT02949947
- Acronym / sponsor ID
- 2015-0588
- Conditions
- Malignant Neoplasms of Mesothelial and Soft Tissue, Gastrointestinal Stromal Tumor With Neurogenic Differentiation
- Keywords
- Malignant Neoplasms of Mesothelial and Soft Tissue, Gastrointestinal Stromal Tumor, GIST, Iron-deficiency anemia, Ferric Carboxymaltose, Injectafer, Iron supplement, Health Questionnaire
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Terminated per PI's request
- Enrolment
- 3 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- American Regent, Inc.
- Study start
- 18 Dec 2017 (actual)
- Primary completion
- 6 Mar 2019 (actual)
- Study completion
- 6 Mar 2019 (actual)
- First posted
- 31 Oct 2016 (estimated)
- Results
- Posted 6 Apr 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical research study is to compare Injectafer® (ferric carboxymaltose) with an iron supplement to learn which may be more effective in improving red blood cell counts in patients who have iron-deficiency anemia (a low red blood cell count) because of a gastrointestinal stromal tumor (GIST) and/or systemic therapy.
The safety of ferric carboxymaltose will also be studied.
This is an investigational study. Ferric carboxymaltose is FDA approved and commercially available to treat iron deficiency anemia; however, it is considered investigational to use in patients who have cancer-related or systemic therapy-related anemia.
Up to 50 participants will take part in this study. All will be enrolled at MD Anderson.
Detailed description
Study Groups and Study Drug Administration:
If you are found to be eligible to take part in this study, you will be randomly assigned (as in the flip of a coin) to 1 of 2 study groups. This is done because no one knows if one group is better, the same, or worse than the other.
- If you are in Group A, you will receive ferric carboxymaltose injection by vein over about 15 minutes. You will receive 2 injections about 7 days apart (for example, on Days 0 [the day you are assigned to a study group] and 7).
- If you are in Group B, you will take iron supplements by mouth every day. This is considered standard of care for iron deficiency anemia and the study staff will discuss with you which iron supplements you will take and their risks.
You and the study staff will know to which group you are assigned.
Length of Study:
You may receive up to 2 injections of ferric carboxymaltose (if you are in Group A) or up to 3 months of oral iron supplements (if you are in Group B). You will no longer be able to take the study drug if intolerable side effects occur or if you are unable to follow study directions.
Your participation on the study will be over after you have completed the Week 24 visit.
Study Visits:
Baseline (within 1 week after you have been assigned to a study group):
- You will have a physical exam.
- You will complete a questionnaire about your health. It should take about 5 minutes to complete.
- Blood (about 1 tablespoon) will be drawn for routine tests and to test the level of iron in your blood.
One (1) time every week during Months 1-3, blood (about 1 tablespoon) will be drawn for routine tests.
At about Weeks 4, 8, 12, and 24 (the end-of-study visit):
- You will have a physical exam (Weeks 12 and 24 only).
- You will complete the same questionnaire you did at baseline.
- Blood (about 1 tablespoon) will be drawn for routine tests and to test the level of iron in your blood.
During Weeks 16 and 20, blood (less than 1 tablespoon) will be drawn to test the level of iron in your blood.
If you leave the study before Week 24, you will have the Week 24 study visits as soon as possible after you leave the study.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group A - Ferric Carboxymaltose | Experimental | Drug: Ferric Carboxymaltose, Behavioral: QuestionnaireParticipants 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. |
| Group B - Iron Supplement | Active comparator | Dietary Supplement: Iron Supplements, Behavioral: QuestionnaireParticipants 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- GIST patients with IDA planned to start or are receiving systemic therapy with TKIs.
- Evidence of iron deficiency anemia including, Hgb < 11 g/dL, but > 8 g/dL; and transferrin saturation (TSAT) < 20%.
- No H/O allergic reaction to iron therapy.
- No clinical signs active of bleeding.
- Adequate hematologic (ANC > 1500/mm^3, platelet count > 100,000/mm^3), renal (serum creatinine < 1.5mg/dL), and hepatic (serum bilirubin count < 1.5 x normal and serum glutamic-oxaloacetic transaminase (SGOT) or serum glutamic pyruvic transaminase (SGPT) < 3 x normal) functions.
Key exclusion criteria (12 listed)
- Pregnant or lactating women.
- Patients with any co-morbid condition which renders patients at high risk of treatment complication.
- Patient has uncontrolled angina, congestive heart failure (New York Heart Association > class II or known ejection fraction < 40%), uncontrolled cardiac arrhythmia or hypertension, or acute myocardial infarction within 3 months.
- Patient has an active seizure disorder. (Patients with a previous history of seizure disorders will be eligible for the study, if they have had no evidence of seizure activity, and they have been free of antiseizure medication for the previous 5 years).
- Psychological, social, familial, or geographical reasons that would prevent scheduled visits and follow-up.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ferric CarboxymaltoseAlso: Injectafer15 mg/kg by vein (up to 750 mg) over 15 min infusion. Dose repeated 1 week later. | Drug | Group A - Ferric Carboxymaltose |
| Iron SupplementsParticipants take iron supplements by mouth every day for up to 3 months. | Dietary supplement | Group B - Iron Supplement |
| QuestionnaireAlso: SurveyHealth questionnaire completed at Baseline and at Weeks 4, 8, 12, and 24. | Behavioural | Group A - Ferric Carboxymaltose, Group B - Iron Supplement |
Primary outcome
Complete Response Rate in Hemoglobin (HGB)
Time frame: 3 months
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Apr 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- University of Texas MD Anderson Cancer Center WebsiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Saroj Vadhan-Raj, MD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ferric Carboxymaltose, Iron Supplements and Questionnaire in people with Malignant Neoplasms of Mesothelial and Soft Tissue and Gastrointestinal Stromal Tumor With Neurogenic Differentiation, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 6 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 6 Apr 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Complete Response Rate in Hemoglobin (HGB) (time frame: 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Apr 2020.