Clinical Trial Results:
An Open-Label Extension Study of the Safety and Efficacy of Ferumoxytol for the Episodic Treatment of Iron Deficiency Anemia in Pediatric Subjects with Chronic Kidney Disease
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Summary
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EudraCT number |
2010-019550-40 |
Trial protocol |
DE GB HU ES RO LT BG |
Global end of trial date |
24 Apr 2015
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Results information
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Results version number |
v1(current) |
This version publication date |
13 Dec 2017
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First version publication date |
13 Dec 2017
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Other versions |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
AMAG-FER-CKD-253
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT01264679 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
AMAG Pharmaceuticals, Inc.
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Sponsor organisation address |
1100 Winter Street, Waltham , United States, 02451
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Public contact |
Medical Information, AMAG Pharmaceuticals, Inc., +1 877-411-2510, amag@druginfo.com
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Scientific contact |
Medical Information, AMAG Pharmaceuticals, Inc., +1 877-411-2510, amag@druginfo.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
Yes
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EMA paediatric investigation plan number(s) |
EMEA-000373-PIP02-09 | ||
Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
Yes
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
24 Apr 2015
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
24 Apr 2015
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Global end of trial reached? |
Yes
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Global end of trial date |
24 Apr 2015
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Was the trial ended prematurely? |
Yes
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General information about the trial
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Main objective of the trial |
Study AMAG-FER-CKD-253 is an extension study of the combined AMAG-FER-CKD-251 (2010-019387) and AMAG-FER-CKD-252 (2010-019388) studies to evaluate the efficacy and safety of episodic treatment of iron deficiency anemia (IDA) with ferumoxytol.
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 and then led the Sponsor to discontinue the combined AMAG-FER-CKD-251 and AMAG-FER-CKD-252 studies and the AMAG-FER-CKD-253 study as designed.
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Protection of trial subjects |
This study was conducted according to international standards of Good Clinical Practice, International Conference on Harmonization (ICH), United States Food and Drug Administration regulations, applicable government regulations, and institutional research policies and procedures. AMAG will also continue to support the principles of the Declaration of Helsinki.
All participants were to remain in the clinic for 1 hour following each IV injection of ferumoxytol, with frequent monitoring of vital signs and close observation for adverse events.
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Background therapy |
There was no background therapy administered across all participant groups. | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
27 Dec 2011
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
Yes
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
United States: 1
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Country: Number of subjects enrolled |
Mexico: 1
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Country: Number of subjects enrolled |
Peru: 3
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Country: Number of subjects enrolled |
Poland: 1
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Country: Number of subjects enrolled |
Spain: 1
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Country: Number of subjects enrolled |
Germany: 1
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Worldwide total number of subjects |
8
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EEA total number of subjects |
3
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
8
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Adults (18-64 years) |
0
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
The study population will consist of pediatric participants (6 months to <18 years of age) with IDA defined as hemoglobin <12.0 grams/deciliter (g/dL) and with either transferrin saturation (TSAT) ≤40% or ferritin <100 nanograms/milliliter (ng/mL) and CKD. | ||||||||||||||||
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Pre-assignment
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Screening details |
Screening was to take place within 2 weeks of the start of the study. Screening assessments included review of inclusion/exclusion criteria, signature of informed consent, medical history, vital signs measurement, physical examination, clinical laboratory assessments including iron panel, and start of adverse events capture. | ||||||||||||||||
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Period 1
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Period 1 title |
Overall period
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Is this the baseline period? |
Yes | ||||||||||||||||
Allocation method |
Non-randomised - controlled
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Blinding used |
Not blinded | ||||||||||||||||
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Arms
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Arm title
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Ferumoxytol | ||||||||||||||||
Arm description |
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. Participants received 2 IV injections of ferumoxytol 7.0 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. | ||||||||||||||||
Arm type |
Experimental | ||||||||||||||||
Investigational medicinal product name |
Ferumoxytol
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Investigational medicinal product code |
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Other name |
Feraheme
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Pharmaceutical forms |
Solution for injection/infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
Ferumoxytol for IV injection: Each 20 mL single-use vial contains 17 mL of ferumoxytol that consists of iron, at a concentration of 30 mg Fe/mL, and mannitol, at a concentration of 44 mg/mL, in a black to reddish brown sterile, aqueous, colloidal, isotonic solution. The product contains no preservatives. Osmolality: 270-330 milliosmoles/kg; pH: 6-8.
Administration was 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.
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| Notes [1] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same. Justification: One participant was enrolled in the study, but withdrew consent prior to receiving any study drug. |
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Baseline characteristics reporting groups
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Reporting group title |
Ferumoxytol
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Reporting group description |
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. Participants received 2 IV injections of ferumoxytol 7.0 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. | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Ferumoxytol
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Reporting group description |
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. Participants received 2 IV injections of ferumoxytol 7.0 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. | ||
Subject analysis set title |
Intent-to-Treat (ITT)
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Subject analysis set type |
Intention-to-treat | ||
Subject analysis set description |
The 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 end point data. As such, summary of the data set is not possible.
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End point title |
Mean Change in Hemoglobin From Baseline to Week 5 [1] | ||||||
End point description |
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 end point data. As such, the data set for this primary end point 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.
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End point type |
Primary
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End point timeframe |
Baseline, Week 5
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Sample data were collected, but not run through any analysis to obtain end point data. As such, summary and statistical analysis of the data set is not possible. |
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| Notes [2] - Sample data were collected, but not run through any analysis to obtain end point data. |
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| No statistical analyses for this end point | |||||||
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End point title |
Proportion Of Participants With An Increase In Hemoglobin From Baseline To Week 5 And To Week 7 | ||||||
End point description |
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 end point data. As such, the data set for this secondary end point cannot be summarized in a way that will provide any significant data based upon the limited study datasets.
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End point type |
Secondary
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End point timeframe |
Baseline, Week 5 and Week 7
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| Notes [3] - As only 7 participants received study drug, efficacy parameters were not analyzed. |
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| No statistical analyses for this end point | |||||||
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End point title |
Mean Change In TSAT From Baseline To Week 5 And To Week 7 | ||||||||
End point description |
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 end point data. As such, the data set for this secondary end point cannot be summarized in a way that will provide any significant data based upon the limited study datasets.
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End point type |
Secondary
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End point timeframe |
Baseline, Week 5 and Week 7
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| Notes [4] - As only 7 participants received study drug, efficacy parameters were not analyzed. |
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| No statistical analyses for this end point | |||||||||
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Adverse events information
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Timeframe for reporting adverse events |
Randomization up to 24 months
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
13.0
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Reporting groups
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Reporting group title |
Ferumoxytol
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Reporting group description |
When a participant has persistent or recurrent iron deficiency anemia (IDA) (defined as hemoglobin <12.0 g/dL and with either TSAT ≤40% or ferritin <100 ng/mL), the participant will begin a 7-week treatment. Participants will receive 2 IV injections of ferumoxytol 7.0 mg iron (Fe) per 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. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Frequency threshold for reporting non-serious adverse events: 1% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| While sample data were collected, it was not run through any analysis to obtain the necessary end point data. As such, summary of the data set is not possible. | |||
