Trial Outcomes & Findings for A Trial of Ferumoxytol for the Episodic Treatment of Iron Deficiency Anemia in Pediatric Participants With Chronic Kidney Disease (NCT NCT01264679)

Last Updated: 2022-04-28

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

8 participants

Primary outcome timeframe

Baseline, Week 5

Results posted on

2022-04-28

Participant Flow

Participant milestones

Participant milestones
Measure	Ferumoxytol When a participant had persistent or recurrent iron deficiency anemia (IDA) (defined as hemoglobin \<12.0 grams \[g\]/deciliter \[dL\] and with either transferrin saturation \[TSAT\] \<40% or ferritin \<100 nanograms \[ng\]/milliliter \[mL\]), the participant began a 7-week treatment period. Participants received 2 intravenous (IV) injections of ferumoxytol 7.0 milligrams \[mg\] iron (Fe)/kilogram (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.
Overall Study STARTED	8
Overall Study Received at Least 1 Dose of Study Drug	7
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Reasons for withdrawal
Measure	Ferumoxytol When a participant had persistent or recurrent iron deficiency anemia (IDA) (defined as hemoglobin \<12.0 grams \[g\]/deciliter \[dL\] and with either transferrin saturation \[TSAT\] \<40% or ferritin \<100 nanograms \[ng\]/milliliter \[mL\]), the participant began a 7-week treatment period. Participants received 2 intravenous (IV) injections of ferumoxytol 7.0 milligrams \[mg\] iron (Fe)/kilogram (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.
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

The birth date is missing for 1 of the 7 pediatric participants. Therefore, mean and standard deviation for Age, Continuous is based on 6 participants.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ferumoxytol n=7 Participants 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.
Age, Categorical <=18 years	7 Participants n=7 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=7 Participants
Age, Categorical >=65 years	0 Participants n=7 Participants
Age, Continuous	15.3 years STANDARD_DEVIATION 1.51 • n=6 Participants • The birth date is missing for 1 of the 7 pediatric participants. Therefore, mean and standard deviation for Age, Continuous is based on 6 participants.
Sex: Female, Male Female	4 Participants n=7 Participants
Sex: Female, Male Male	3 Participants n=7 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=7 Participants
Race (NIH/OMB) Asian	1 Participants n=7 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=7 Participants
Race (NIH/OMB) Black or African American	0 Participants n=7 Participants
Race (NIH/OMB) White	2 Participants n=7 Participants
Race (NIH/OMB) More than one race	4 Participants n=7 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=7 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome data not reported

Adverse Events

Ferumoxytol

Serious events: 3 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Ferumoxytol n=7 participants at risk 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.
Cardiac disorders Cardiac failure congestive	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
General disorders Device breakage	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Injury, poisoning and procedural complications Ligament rupture	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Renal and urinary disorders Renal failure chronic	14.3% 1/7 • Number of events 2 • Randomization up to 24 months

Other adverse events

Other adverse events
Measure	Ferumoxytol n=7 participants at risk 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.
Blood and lymphatic system disorders Nephrogenic anaemia	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Cardiac disorders Congestive cardiomyopathy	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Cardiac disorders Ventricular flutter	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Ear and labyrinth disorders Vertigo	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Gastrointestinal disorders Diarrhoea	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Gastrointestinal disorders Gastritis	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Gastrointestinal disorders Nausea	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Gastrointestinal disorders Peritoneal lesion	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
General disorders Oedema peripheral	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Infections and infestations Device related infection	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Infections and infestations Influenza	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Infections and infestations Nasopharyngitis	28.6% 2/7 • Number of events 8 • Randomization up to 24 months
Infections and infestations Oral herpes	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Infections and infestations Pharyngitis	28.6% 2/7 • Number of events 2 • Randomization up to 24 months
Infections and infestations Urinary tract infection	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Injury, poisoning and procedural complications Procedural nausea	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Injury, poisoning and procedural complications Procedural vomiting	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Injury, poisoning and procedural complications Ureteric anastomosis complication	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Metabolism and nutrition disorders Hyperkalaemia	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Metabolism and nutrition disorders Hypocalcaemia	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Musculoskeletal and connective tissue disorders Joint swelling	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Psychiatric disorders Depression	28.6% 2/7 • Number of events 3 • Randomization up to 24 months
Renal and urinary disorders Anuria	28.6% 2/7 • Number of events 2 • Randomization up to 24 months
Renal and urinary disorders Bladder spasm	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Renal and urinary disorders Renal colic	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Vascular disorders Air embolism	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Vascular disorders Hypertension	14.3% 1/7 • Number of events 1 • Randomization up to 24 months
Gastrointestinal disorders Dental caries	14.3% 1/7 • Number of events 1 • Randomization up to 24 months

Additional Information

Medical Information

AMAG Pharmaceuticals, Inc.

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee If there is no multi-site publication within 18 months after the Study has been completed or terminated at all Study sites, and all data has been received by Sponsor, the Site and SMO shall have the right to publish its results from the Study for non-commercial purposes, if submitted to Sponsor for review 60 days prior to submission of publication. Publication must remove all confidential information and may be delayed by up to 120 days to allow Sponsor to protect its interests.
Publication restrictions are in place

Restriction type: OTHER