Iron Deficiency Anemia
Conditions
Keywords
Iron deficiency anemia, Feraheme, ferumoxytol, chronic kidney disease, CKD, pediatric, dialysis-dependent, nondialysis-dependent
Brief summary
Study AMAG-FER-CKD-253 is an extension study of the combined AMAG-FER-CKD-251 (NCT01155375) and AMAG-FER-CKD-252 (NCT01155388) studies to evaluate the efficacy and safety of episodic treatment of iron deficiency anemia (IDA) with ferumoxytol.
Detailed description
Study AMAG-FER-CKD-251 was a study evaluating the efficacy and safety of intravenous (IV) ferumoxytol in pediatric participants with dialysis-dependent chronic kidney disease (CKD). Study AMAG-FER-CKD-252 was a study evaluating the efficacy and safety of IV ferumoxytol in pediatric participants with nondialysis-dependent chronic kidney disease. Due to significant challenges with enrollment for both studies, Study AMAG-FER-CKD-252 was combined with Study AMAG-FER-CKD-251 and enrollment continued under Study AMAG-FER-CKD-251. Participants were enrolled by age cohorts in a stepwise manner following a safety review by the Data Safety Monitoring Board of 1 age cohort prior to enrollment of a subsequent age cohort, with progression from oldest to youngest: Randomization was stratified by the following age cohorts: 12 to \<18 years, 6 to \<12 years, 2 to \<6 years, and 6 months to \<2 years. Participants who completed the combined AMAG-FER-CKD-251 and AMAG-FER-CKD-252 studies had the option of participating in this extension study.
Interventions
DRUGFerumoxytol
IV Ferumoxytol
Sponsors
AMAG Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Months to 17 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria include: 1. Participants who had completed participation in the combined AMAG-FER-CKD-251 and AMAG-FER-CKD-252 studies within 4 weeks of screening 2. Female participants of childbearing potential who are sexually active must be on an effective method of birth control and agree to remain on birth control until completion of participation in the study 3. Participant and/or legal guardian is capable of understanding and complying with the protocol requirements and is available for the duration of the study Key
Exclusion criteria
include: 1. Experienced a serious adverse event related to IV iron therapy in the combined AMAG-FER-CKD-251 and AMAG-FER- CKD-252 studies 2. Hemoglobin level ≤7 g/dL
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Mean Change In Hemoglobin From Baseline To Week 5 | Baseline, Week 5 | Mean changes in hemoglobin following the first course of ferumoxytol from Baseline to Week 5 were to be presented. Despite efforts to complete the studies as designed, several factors contributed to significant challenges in enrollment and led the Sponsor to discontinue the combined AMAG-FER-CKD-251 and AMAG-FER-CKD-252 studies, and the AMAG-FER-CKD-253 study. Blood samples were collected, but not run through an analysis to obtain outcome measure data. As such, the data set for this primary outcome measure cannot be summarized nor can the statistical analysis, as described in the protocol, be provided in a way that will provide any significant data based upon the limited study datasets. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Proportion Of Participants With An Increase In Hemoglobin From Baseline To Week 5 And To Week 7 | Baseline, Week 5 and Week 7 | The proportion of participants with an increase hemoglobin to ≥1.0 g/dL or to ≥12.0 g/dL during the period from Baseline to Week 5 and to Week 7 following each course of ferumoxytol was to be presented. However, despite efforts to complete the studies as designed, several factors contributed to significant challenges in enrollment and led the Sponsor to discontinue the combined AMAG-FER-CKD-251 and AMAG-FER-CKD-252 studies, and the AMAG-FER-CKD-253 study. Blood samples were collected, but not run through an analysis to obtain outcome measure data. As such, the data set for this secondary outcome measure cannot be summarized in a way that will provide any significant data based upon the limited study datasets. |
| Mean Change In TSAT From Baseline To Week 5 And To Week 7 | Baseline, Week 5 and Week 7 | Mean changes in TSAT following the first course of ferumoxytol from Baseline to Week 5 and to Week 7 were to be presented. However, despite efforts to complete the studies as designed, several factors contributed to significant challenges in enrollment and led the Sponsor to discontinue the combined AMAG-FER-CKD-251 and AMAG-FER-CKD-252 studies, and the AMAG-FER-CKD-253 study. Blood samples were collected, but not run through an analysis to obtain outcome measure data. As such, the data set for this secondary outcome measure cannot be summarized in a way that will provide any significant data based upon the limited study datasets. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Ferumoxytol When a participant had persistent or recurrent IDA (defined as hemoglobin \<12.0 g/dL and with either TSAT ≤40% or ferritin \<100 ng/mL), the participant began a 7-week treatment period. Participants received 2 IV injections of ferumoxytol 7.0 mg Fe/kg (maximum of 510 mg/dose), the first dose administered on Day 1 and the second on Days 3 through 9 of the Treatment Period. | 7 |
| Total | 7 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Participation in Another Clinical Trial | 1 |
| Overall Study | Physician Decision | 1 |
| Overall Study | Sponsor's Suspension of Dosing | 2 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Ferumoxytol |
|---|---|
| Age, Categorical <=18 years | 7 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Age, Continuous | 15.3 years STANDARD_DEVIATION 1.51 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 4 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 2 Participants |
| Sex: Female, Male Female | 4 Participants |
| Sex: Female, Male Male | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 7 |
| other Total, other adverse events | 7 / 7 |
| serious Total, serious adverse events | 3 / 7 |
Outcome results
Primary
Mean Change In Hemoglobin From Baseline To Week 5
Mean changes in hemoglobin following the first course of ferumoxytol from Baseline to Week 5 were to be presented. Despite efforts to complete the studies as designed, several factors contributed to significant challenges in enrollment and led the Sponsor to discontinue the combined AMAG-FER-CKD-251 and AMAG-FER-CKD-252 studies, and the AMAG-FER-CKD-253 study. Blood samples were collected, but not run through an analysis to obtain outcome measure data. As such, the data set for this primary outcome measure cannot be summarized nor can the statistical analysis, as described in the protocol, be provided in a way that will provide any significant data based upon the limited study datasets.
Time frame: Baseline, Week 5
Population: ITT Population included all randomized participants who had received at least 1 dose of study drug. Sample data were collected, but not run through any analysis to obtain outcome measure data. As such, summary of the data set is not possible.
Secondary
Mean Change In TSAT From Baseline To Week 5 And To Week 7
Mean changes in TSAT following the first course of ferumoxytol from Baseline to Week 5 and to Week 7 were to be presented. However, despite efforts to complete the studies as designed, several factors contributed to significant challenges in enrollment and led the Sponsor to discontinue the combined AMAG-FER-CKD-251 and AMAG-FER-CKD-252 studies, and the AMAG-FER-CKD-253 study. Blood samples were collected, but not run through an analysis to obtain outcome measure data. As such, the data set for this secondary outcome measure cannot be summarized in a way that will provide any significant data based upon the limited study datasets.
Time frame: Baseline, Week 5 and Week 7
Population: ITT Population included all randomized participants who had received at least 1 dose of study drug. Sample data were collected, but not run through any analysis to obtain outcome measure data. As such, summary of the data set is not possible.
Secondary
Proportion Of Participants With An Increase In Hemoglobin From Baseline To Week 5 And To Week 7
The proportion of participants with an increase hemoglobin to ≥1.0 g/dL or to ≥12.0 g/dL during the period from Baseline to Week 5 and to Week 7 following each course of ferumoxytol was to be presented. However, despite efforts to complete the studies as designed, several factors contributed to significant challenges in enrollment and led the Sponsor to discontinue the combined AMAG-FER-CKD-251 and AMAG-FER-CKD-252 studies, and the AMAG-FER-CKD-253 study. Blood samples were collected, but not run through an analysis to obtain outcome measure data. As such, the data set for this secondary outcome measure cannot be summarized in a way that will provide any significant data based upon the limited study datasets.
Time frame: Baseline, Week 5 and Week 7
Population: ITT Population included all randomized participants who had received at least 1 dose of study drug. Sample data were collected, but not run through any analysis to obtain outcome measure data. As such, summary of the data set is not possible.