Trial Outcomes & Findings for A Trial Comparing Ferumoxytol With Placebo for the Treatment of Iron Deficiency Anemia (NCT NCT01114139)
NCT ID: NCT01114139
Last Updated: 2022-04-21
Results Overview
Participants who achieved a ≥2.0 g/dL increase in hemoglobin at any time from Baseline up to Week 5 are presented. Increase in hemoglobin at any time from Baseline up to Week 5 was calculated for each participant based on: Hemoglobin Change = Hemoglobin (Week X) - Hemoglobin (Baseline), where Week X was any post-Baseline visit up to and including Week 5. Baseline was defined as the Day 1 value (prior to injection of study drug). The screening or most recent value prior to Day 1 was used for any participant with missing Day 1 information. Participants with no post-Baseline hemoglobin values were classified as not achieving a ≥2.0 g/dL increase. Statistical analysis was performed for data up to Week 5 only.
COMPLETED
PHASE3
812 participants
Baseline (Day 1) through Week 5
2022-04-21
Participant Flow
The study was open to enrollment for adult participants with iron deficiency anemia (IDA), defined as hemoglobin \<10.0 gram (g)/deciliter (dL) and transferrin saturation (TSAT) \<20%, and a history of unsatisfactory oral iron therapy or in whom oral iron could not be used.
Participant milestones
| Measure |
Ferumoxytol
Participants received a total of 2 doses of intravenous (IV) ferumoxytol 510 milligrams (mg) (17 milliliters \[mL\]). The first IV 510 mg dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose, for a total cumulative dose of 1.02 g.
|
Placebo
Participants received a total of 2 doses of IV saline (17 mL). The first IV dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose.
|
|---|---|---|
|
Overall Study
STARTED
|
609
|
203
|
|
Overall Study
Received at Least 1 Dose of Study Drug
|
608
|
200
|
|
Overall Study
COMPLETED
|
569
|
187
|
|
Overall Study
NOT COMPLETED
|
40
|
16
|
Reasons for withdrawal
| Measure |
Ferumoxytol
Participants received a total of 2 doses of intravenous (IV) ferumoxytol 510 milligrams (mg) (17 milliliters \[mL\]). The first IV 510 mg dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose, for a total cumulative dose of 1.02 g.
|
Placebo
Participants received a total of 2 doses of IV saline (17 mL). The first IV dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose.
|
|---|---|---|
|
Overall Study
Adverse Event
|
3
|
2
|
|
Overall Study
Lost to Follow-up
|
15
|
3
|
|
Overall Study
Withdrawal by Subject
|
17
|
6
|
|
Overall Study
Other-Missed 1 visit
|
1
|
0
|
|
Overall Study
Other-Protocol Violation
|
1
|
0
|
|
Overall Study
Other-Out of window for study drug dose
|
1
|
0
|
|
Overall Study
Other-Participant falsified records
|
0
|
1
|
|
Overall Study
Other-Participant went on hospice
|
0
|
1
|
|
Overall Study
Other-Physician changed treatment
|
1
|
0
|
|
Overall Study
Other-Withdrew prior to receiving dose
|
1
|
3
|
Baseline Characteristics
A Trial Comparing Ferumoxytol With Placebo for the Treatment of Iron Deficiency Anemia
Baseline characteristics by cohort
| Measure |
Ferumoxytol
n=608 Participants
Participants received a total of 2 doses of IV ferumoxytol 510 mg (17 mL). The first IV 510 mg dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose, for a total cumulative dose of 1.02 g.
|
Placebo
n=200 Participants
Participants received a total of 2 doses of IV saline (17 mL). The first IV dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose.
|
Total
n=808 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
44.8 years
STANDARD_DEVIATION 13.82 • n=5 Participants
|
46.0 years
STANDARD_DEVIATION 13.58 • n=7 Participants
|
45.1 years
STANDARD_DEVIATION 13.76 • n=5 Participants
|
|
Sex: Female, Male
Female
|
542 Participants
n=5 Participants
|
178 Participants
n=7 Participants
|
720 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
66 Participants
n=5 Participants
|
22 Participants
n=7 Participants
|
88 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline (Day 1) through Week 5Population: Intent-To-Treat (ITT) Population: Any randomized participant who had any exposure to study drug (ferumoxytol or placebo) and was based upon randomized treatment assignment.
Participants who achieved a ≥2.0 g/dL increase in hemoglobin at any time from Baseline up to Week 5 are presented. Increase in hemoglobin at any time from Baseline up to Week 5 was calculated for each participant based on: Hemoglobin Change = Hemoglobin (Week X) - Hemoglobin (Baseline), where Week X was any post-Baseline visit up to and including Week 5. Baseline was defined as the Day 1 value (prior to injection of study drug). The screening or most recent value prior to Day 1 was used for any participant with missing Day 1 information. Participants with no post-Baseline hemoglobin values were classified as not achieving a ≥2.0 g/dL increase. Statistical analysis was performed for data up to Week 5 only.
Outcome measures
| Measure |
Ferumoxytol
n=608 Participants
Participants received a total of 2 doses of IV ferumoxytol 510 mg (17 mL). The first IV 510 mg dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose, for a total cumulative dose of 1.02 g.
|
Placebo
n=200 Participants
Participants received a total of 2 doses of IV saline (17 mL). The first IV dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose.
|
|---|---|---|
|
Participants Who Achieved A ≥2.0 g/dL Increase In Hemoglobin At Any Time From Baseline To Week 5
Up to Week 2
|
252 Participants
|
5 Participants
|
|
Participants Who Achieved A ≥2.0 g/dL Increase In Hemoglobin At Any Time From Baseline To Week 5
Up to Week 3
|
386 Participants
|
7 Participants
|
|
Participants Who Achieved A ≥2.0 g/dL Increase In Hemoglobin At Any Time From Baseline To Week 5
Up to Week 4
|
460 Participants
|
8 Participants
|
|
Participants Who Achieved A ≥2.0 g/dL Increase In Hemoglobin At Any Time From Baseline To Week 5
Up to Week 5
|
493 Participants
|
11 Participants
|
SECONDARY outcome
Timeframe: Baseline (Day 1), Week 5Population: Intent-To-Treat (ITT) Population: Any randomized participant who had any exposure to study drug (ferumoxytol or placebo) and was based upon randomized treatment assignment.
Mean change in hemoglobin from Baseline to Week 5 was calculated for each participant as: Hemoglobin Change = Hemoglobin (Week X) - Hemoglobin (Baseline), where Week X was any post-Baseline visit up to and including Week 5. Baseline was defined as the Day 1 value (prior to injection of study drug). The screening or most recent value prior to Day 1 was used for any participant with missing Day 1 information. If the Week 5 hemoglobin value was missing, the change from Baseline was imputed to be zero. Participants without any post-Baseline hemoglobin values were treated as non-responders.
Outcome measures
| Measure |
Ferumoxytol
n=608 Participants
Participants received a total of 2 doses of IV ferumoxytol 510 mg (17 mL). The first IV 510 mg dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose, for a total cumulative dose of 1.02 g.
|
Placebo
n=200 Participants
Participants received a total of 2 doses of IV saline (17 mL). The first IV dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose.
|
|---|---|---|
|
Mean Change In Hemoglobin From Baseline To Week 5
|
2.6 g/dL
Standard Deviation 1.52
|
0.1 g/dL
Standard Deviation 0.89
|
SECONDARY outcome
Timeframe: Baseline (Day 1) through Week 5Population: ITT Population: Any randomized participant who had any exposure to study drug (ferumoxytol or placebo) and was based upon randomized treatment assignment.
Participants who achieved a ≥12.0 g/dL hemoglobin level at any time from Baseline up to Week 5 are presented. Increase in hemoglobin at any time from Baseline up to Week 5 was calculated for each participant based on: Hemoglobin Change = Hemoglobin (Week X) - Hemoglobin (Baseline), where Week X was any post-Baseline visit up to and including Week 5. Baseline was defined as the Day 1 value (prior to injection of study drug). The screening or most recent value prior to Day 1 was used for any participant with missing Day 1 information. Participants without any post-Baseline hemoglobin values were treated as non-responders.
Outcome measures
| Measure |
Ferumoxytol
n=608 Participants
Participants received a total of 2 doses of IV ferumoxytol 510 mg (17 mL). The first IV 510 mg dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose, for a total cumulative dose of 1.02 g.
|
Placebo
n=200 Participants
Participants received a total of 2 doses of IV saline (17 mL). The first IV dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose.
|
|---|---|---|
|
Participants Achieving A Hemoglobin Level ≥12.0 g/dL At Any Time From Baseline To Week 5
Up to Week 2
|
33 Participants
|
3 Participants
|
|
Participants Achieving A Hemoglobin Level ≥12.0 g/dL At Any Time From Baseline To Week 5
Up to Week 3
|
131 Participants
|
5 Participants
|
|
Participants Achieving A Hemoglobin Level ≥12.0 g/dL At Any Time From Baseline To Week 5
Up to Week 4
|
240 Participants
|
5 Participants
|
|
Participants Achieving A Hemoglobin Level ≥12.0 g/dL At Any Time From Baseline To Week 5
Up to Week 5
|
307 Participants
|
6 Participants
|
SECONDARY outcome
Timeframe: Baseline (Day 1), Week 5Population: Intent-To-Treat (ITT) Population: Any randomized participant who had any exposure to study drug (ferumoxytol or placebo) and was based upon randomized treatment assignment.
Mean change in TSAT from Baseline to Week 5 was calculated for each participant as: TSAT Change = TSAT (Week 5) - TSAT (Baseline). TSAT, measured as a percentage, was part of the iron panel laboratory evaluations. Of the transferrin available to bind iron, this value indicates how much serum iron is bound. For example, a value of 20% means that 20% of iron-binding sites of transferrin are being occupied by iron. Baseline was defined as the Day 1 value (prior to injection of study drug). The screening or most recent value prior to Day 1 was used for any participant with missing Day 1 information. If the Week 5 TSAT value was missing, the change from Baseline was imputed to be zero.
Outcome measures
| Measure |
Ferumoxytol
n=608 Participants
Participants received a total of 2 doses of IV ferumoxytol 510 mg (17 mL). The first IV 510 mg dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose, for a total cumulative dose of 1.02 g.
|
Placebo
n=200 Participants
Participants received a total of 2 doses of IV saline (17 mL). The first IV dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose.
|
|---|---|---|
|
Mean Change In TSAT From Baseline To Week 5
|
11.4 percentage of saturation
Standard Deviation 15.06
|
0.4 percentage of saturation
Standard Deviation 5.81
|
SECONDARY outcome
Timeframe: Baseline (Day 1), Week 5Population: Intent-To-Treat (ITT) Population: Any randomized participant who had any exposure to study drug (ferumoxytol or placebo) and was based upon randomized treatment assignment.
The FACIT-Fatigue questionnaire is a 13-item questionnaire designed and validated to specifically assess the presence and impact of treatment on fatigue and related symptoms, such as tiredness, on health-related quality of life in anemic participants with cancer. The questionnaire has 13 items, each measured on a 4-point Likert scale. Scoring ranges from 0 (the most fatigued) to 52 (the least fatigued) points, with higher scores representing better functioning or less fatigue. Mean change in FACIT-Fatigue Score from Baseline to Week 5 was calculated for each participant as: FACIT-Fatigue Score Change = FACIT-Fatigue Score (Week 5) - FACIT-Fatigue Score (Baseline). Baseline was defined as the Day 1 value (prior to first dose of study drug).The screening or most recent value prior to Day 1 was used for any participant with missing Day 1 information. If the Week 5 FACIT-Fatigue Score value was missing, the change from Baseline was imputed to be zero.
Outcome measures
| Measure |
Ferumoxytol
n=608 Participants
Participants received a total of 2 doses of IV ferumoxytol 510 mg (17 mL). The first IV 510 mg dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose, for a total cumulative dose of 1.02 g.
|
Placebo
n=200 Participants
Participants received a total of 2 doses of IV saline (17 mL). The first IV dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose.
|
|---|---|---|
|
Mean Change In Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Score From Baseline To Week 5
|
11.7 units on a scale
Standard Deviation 11.73
|
6.8 units on a scale
Standard Deviation 9.51
|
SECONDARY outcome
Timeframe: From Baseline (Day 1) up to Week 5Population: Intent-To-Treat (ITT) Population: Any randomized participant who had any exposure to study drug (ferumoxytol or placebo) and was based upon randomized treatment assignment.
The time to hemoglobin increase of ≥2.0 g/dL or hemoglobin value of ≥12.0 g/dL was defined as the days from Baseline (Day 1) to the first time the participant had an increase in hemoglobin of ≥2.0 g/dL or hemoglobin value of ≥12.0 g/dL, and was calculated using a Kaplan-Meier curve. Participants who did not have a hemoglobin increase of ≥2.0 g/dL or to a hemoglobin level ≥12.0 g/dL were censored at their last visit day. Participants without any post-Baseline study visits were not included.
Outcome measures
| Measure |
Ferumoxytol
n=608 Participants
Participants received a total of 2 doses of IV ferumoxytol 510 mg (17 mL). The first IV 510 mg dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose, for a total cumulative dose of 1.02 g.
|
Placebo
n=200 Participants
Participants received a total of 2 doses of IV saline (17 mL). The first IV dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose.
|
|---|---|---|
|
Time To Hemoglobin Increase Of ≥2.0 g/dL Or A Hemoglobin Value Of ≥12.0 g/dL From Baseline
|
23.5 Days
Interval 15.0 to 29.0
|
42.5 Days
Interval 44.0 to
Insufficient number of participants with events.
|
Adverse Events
Ferumoxytol
Placebo
Serious adverse events
| Measure |
Ferumoxytol
n=608 participants at risk
Participants received a total of 2 doses of IV ferumoxytol 510 mg (17 mL). The first IV 510 mg dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose, for a total cumulative dose of 1.02 g.
|
Placebo
n=200 participants at risk
Participants received a total of 2 doses of IV saline (17 mL). The first IV dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose.
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
0.16%
1/608
|
1.5%
3/200
|
|
Cardiac disorders
Supraventricular Tachycardia
|
0.16%
1/608
|
0.00%
0/200
|
|
Gastrointestinal disorders
Colitis Ulcerative
|
0.16%
1/608
|
0.00%
0/200
|
|
Gastrointestinal disorders
Gastrointestinal Haemorrhage
|
0.16%
1/608
|
0.00%
0/200
|
|
Gastrointestinal disorders
Vomiting
|
0.16%
1/608
|
0.00%
0/200
|
|
General disorders
Chest Pain
|
0.16%
1/608
|
0.00%
0/200
|
|
General disorders
Disease Progression
|
0.16%
1/608
|
0.00%
0/200
|
|
Immune system disorders
Anaphylactic Reaction
|
0.16%
1/608
|
0.00%
0/200
|
|
Immune system disorders
Hypersensitivity
|
0.49%
3/608
|
0.00%
0/200
|
|
Infections and infestations
Gastroenteritis
|
0.16%
1/608
|
0.00%
0/200
|
|
Infections and infestations
Gastroenteritis Viral
|
0.16%
1/608
|
0.00%
0/200
|
|
Infections and infestations
Lobar Pneumonia
|
0.16%
1/608
|
0.00%
0/200
|
|
Infections and infestations
Septic Shock
|
0.16%
1/608
|
0.00%
0/200
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
0.16%
1/608
|
0.00%
0/200
|
|
Musculoskeletal and connective tissue disorders
Intervertebral Disc Protrusion
|
0.16%
1/608
|
0.00%
0/200
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Lung Neoplasm Malignant
|
0.00%
0/608
|
0.50%
1/200
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Small Intestine Carcinoma
|
0.16%
1/608
|
0.00%
0/200
|
|
Nervous system disorders
Convulsion
|
0.00%
0/608
|
0.50%
1/200
|
|
Nervous system disorders
Hypoaesthesia
|
0.16%
1/608
|
0.00%
0/200
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.16%
1/608
|
0.00%
0/200
|
|
Reproductive system and breast disorders
Dysfunctional Uterine Bleeding
|
0.00%
0/542
|
0.56%
1/178
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.16%
1/608
|
0.00%
0/200
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.16%
1/608
|
0.00%
0/200
|
Other adverse events
| Measure |
Ferumoxytol
n=608 participants at risk
Participants received a total of 2 doses of IV ferumoxytol 510 mg (17 mL). The first IV 510 mg dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose, for a total cumulative dose of 1.02 g.
|
Placebo
n=200 participants at risk
Participants received a total of 2 doses of IV saline (17 mL). The first IV dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose.
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal Pain
|
1.8%
11/608
|
2.5%
5/200
|
|
Gastrointestinal disorders
Abdominal Pain Upper
|
1.2%
7/608
|
0.50%
1/200
|
|
Gastrointestinal disorders
Constipation
|
1.6%
10/608
|
1.5%
3/200
|
|
Gastrointestinal disorders
Diarrhoea
|
2.8%
17/608
|
3.0%
6/200
|
|
Gastrointestinal disorders
Nausea
|
4.6%
28/608
|
2.5%
5/200
|
|
Gastrointestinal disorders
Toothache
|
0.82%
5/608
|
1.5%
3/200
|
|
Gastrointestinal disorders
Vomiting
|
2.0%
12/608
|
1.0%
2/200
|
|
General disorders
Fatigue
|
2.0%
12/608
|
1.5%
3/200
|
|
General disorders
Oedema Peripheral
|
1.6%
10/608
|
0.50%
1/200
|
|
General disorders
Pyrexia
|
1.2%
7/608
|
0.50%
1/200
|
|
Infections and infestations
Nasopharyngitis
|
2.6%
16/608
|
2.0%
4/200
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
1.3%
8/608
|
1.0%
2/200
|
|
Infections and infestations
Urinary Tract Infection
|
2.8%
17/608
|
3.0%
6/200
|
|
Injury, poisoning and procedural complications
Skin Laceration
|
0.00%
0/608
|
1.5%
3/200
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
1.5%
9/608
|
0.00%
0/200
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
1.5%
9/608
|
2.5%
5/200
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
1.6%
10/608
|
1.0%
2/200
|
|
Musculoskeletal and connective tissue disorders
Muscle Spasms
|
1.2%
7/608
|
1.0%
2/200
|
|
Musculoskeletal and connective tissue disorders
Pain in Extremity
|
1.2%
7/608
|
0.50%
1/200
|
|
Nervous system disorders
Dizziness
|
3.9%
24/608
|
3.5%
7/200
|
|
Nervous system disorders
Dysgeusia
|
1.5%
9/608
|
0.50%
1/200
|
|
Nervous system disorders
Headache
|
5.8%
35/608
|
6.0%
12/200
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
1.2%
7/608
|
0.50%
1/200
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
1.5%
9/608
|
2.0%
4/200
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.49%
3/608
|
1.5%
3/200
|
|
Skin and subcutaneous tissue disorders
Rash
|
2.0%
12/608
|
0.00%
0/200
|
|
Vascular disorders
Hypertension
|
1.5%
9/608
|
0.00%
0/200
|
|
Vascular disorders
Hypotension
|
1.3%
8/608
|
0.50%
1/200
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee If no multi-site publication within 18 months after Study has been completed or terminated at all Study sites, and all data have been received by Sponsor, Site, and Site Management Organization (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 180 days to allow Sponsor to protect its interests.
- Publication restrictions are in place
Restriction type: OTHER