Trial Outcomes & Findings for Blinded Randomized Placebo-Controlled Study Efficacy and Safety of Injectafer in ID Patients With Fibromyalgia (NCT NCT02409459)

NCT ID: NCT02409459

Last Updated: 2018-02-20

Results Overview

The primary efficacy endpoint was the percentage of subjects who had a ≥13-point improvement in the FIQR from baseline to Day 42.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

81 participants

Primary outcome timeframe

Day 42

Results posted on

2018-02-20

Participant Flow

Participant milestones

Participant milestones
Measure
Injectafer
15 mg/kg up to 750 mg undiluted blinded dose of IV Injectafer (ferric carboxymaltose) at 100 mg/minute Injectafer
Placebo
15 cc of Normal Saline IV push at 2 ml/minute Normal Saline
Overall Study
STARTED
41
40
Overall Study
COMPLETED
40
39
Overall Study
NOT COMPLETED
1
1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Blinded Randomized Placebo-Controlled Study Efficacy and Safety of Injectafer in ID Patients With Fibromyalgia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Placebo
n=40 Participants
Group B: Placebo
Injectafer
n=41 Participants
Group A: Injectafer
Total
n=81 Participants
Total of all reporting groups
Age, Continuous
44 years
STANDARD_DEVIATION 10.85 • n=99 Participants
41 years
STANDARD_DEVIATION 11.07 • n=107 Participants
43 years
STANDARD_DEVIATION 10.97 • n=206 Participants
Sex: Female, Male
Female
40 Participants
n=99 Participants
40 Participants
n=107 Participants
80 Participants
n=206 Participants
Sex: Female, Male
Male
0 Participants
n=99 Participants
1 Participants
n=107 Participants
1 Participants
n=206 Participants
Race/Ethnicity, Customized
Race · African American
7 Participants
n=99 Participants
7 Participants
n=107 Participants
14 Participants
n=206 Participants
Race/Ethnicity, Customized
Race · Asian
0 Participants
n=99 Participants
1 Participants
n=107 Participants
1 Participants
n=206 Participants
Race/Ethnicity, Customized
Race · Caucasian
30 Participants
n=99 Participants
33 Participants
n=107 Participants
63 Participants
n=206 Participants
Race/Ethnicity, Customized
Race · Hispanic
2 Participants
n=99 Participants
0 Participants
n=107 Participants
2 Participants
n=206 Participants
Race/Ethnicity, Customized
Race · Other
1 Participants
n=99 Participants
0 Participants
n=107 Participants
1 Participants
n=206 Participants
History of iron intolerance
No
37 Participants
n=99 Participants
35 Participants
n=107 Participants
72 Participants
n=206 Participants
History of iron intolerance
Yes
3 Participants
n=99 Participants
6 Participants
n=107 Participants
9 Participants
n=206 Participants
History of drug allergy
No
17 Participants
n=99 Participants
21 Participants
n=107 Participants
38 Participants
n=206 Participants
History of drug allergy
Yes
23 Participants
n=99 Participants
20 Participants
n=107 Participants
43 Participants
n=206 Participants
IRLS score per Arm/Group
16.3 units on a scale
STANDARD_DEVIATION 14.9 • n=99 Participants
13.4 units on a scale
STANDARD_DEVIATION 13.16 • n=107 Participants
14.8 units on a scale
STANDARD_DEVIATION 14.04 • n=206 Participants

PRIMARY outcome

Timeframe: Day 42

Population: The Safety population consisted of all subjects who received at least one dose of randomized treatment. The Efficacy Evaluable Population consisted of all subjects who received at least 1 dose of randomized treatment with at least 1 completed post treatment FIQR evaluation.

The primary efficacy endpoint was the percentage of subjects who had a ≥13-point improvement in the FIQR from baseline to Day 42.

Outcome measures

Outcome measures
Measure
Injectafer
n=39 Participants
15 mg/kg up to 750 mg undiluted blinded dose of IV Injectafer (ferric carboxymaltose) at 100 mg/minute Injectafer
Placebo
n=39 Participants
15 cc of Normal Saline IV push at 2 ml/minute Normal Saline
Proportion of Patients With a ≥13 Point Improvement in FIQR Score
30 Participants
26 Participants

SECONDARY outcome

Timeframe: Change from Baseline in Brief Pain Inventory Pain Interference Score at Day 42

Population: Patients without a particular measure at a required time point were excluded from the analysis of that measure.

The short version of the Brief Pain Inventory (BPI) includes front and back body diagrams, 4 pain severity items and 7 pain interference items rated on 0-10 scales (with 0 being the best outcome and 10 being the worst outcome), and a question about percentage of pain relief by analgesics. The Pain Interference Score is calculated by adding the scores for the 7 pain interference questions and then dividing by 7 to give a score out of 10.

Outcome measures

Outcome measures
Measure
Injectafer
n=38 Participants
15 mg/kg up to 750 mg undiluted blinded dose of IV Injectafer (ferric carboxymaltose) at 100 mg/minute Injectafer
Placebo
n=39 Participants
15 cc of Normal Saline IV push at 2 ml/minute Normal Saline
Change in BPI, Pain Interference
-4.1 units on a scale
Standard Deviation 3.3
-2.3 units on a scale
Standard Deviation 2.6

SECONDARY outcome

Timeframe: Change from Baseline in FIQR score at Day 42

Population: Patients without a particular measure at a required time point were excluded from the analysis of that measure.

The FIQR has 21 individual questions. All questions are based on an 11-point numeric rating scale of 0 to 10, with 10 being 'worst' and all questions are framed in the context of the past 7 days. The summed score for function (range 0 to 90) is divided by 3, the summed score for overall impact (range 0 to 20) is not changed, and the summed score for symptoms (range 0 to 100) is divided by 2. The total FIQR is the sum of the three modified domain scores.

Outcome measures

Outcome measures
Measure
Injectafer
n=38 Participants
15 mg/kg up to 750 mg undiluted blinded dose of IV Injectafer (ferric carboxymaltose) at 100 mg/minute Injectafer
Placebo
n=39 Participants
15 cc of Normal Saline IV push at 2 ml/minute Normal Saline
Change in FIQR Score
-45.0 units on a scale
Standard Deviation 29.5
-29.7 units on a scale
Standard Deviation 27.0

SECONDARY outcome

Timeframe: Change from Baseline in Brief Pain Inventory Pain Severity Score at Day 42

Population: Patients without a particular measure at a required time point were excluded from the analysis of that measure.

The short version of the Brief Pain Inventory (BPI) includes front and back body diagrams, 4 pain severity items and 7 pain interference items rated on 0-10 scales (with 0 being the best outcome and 10 being the worst outcome), and a question about percentage of pain relief by analgesics. The Pain Severity Score is calculated by adding the scores for the 4 pain severity questions and then dividing by 4 to give a severity score out of 10.

Outcome measures

Outcome measures
Measure
Injectafer
n=38 Participants
15 mg/kg up to 750 mg undiluted blinded dose of IV Injectafer (ferric carboxymaltose) at 100 mg/minute Injectafer
Placebo
n=39 Participants
15 cc of Normal Saline IV push at 2 ml/minute Normal Saline
Change in BPI, Pain Severity
-3.1 units on a scale
Standard Deviation 2.7
-1.8 units on a scale
Standard Deviation 2.1

SECONDARY outcome

Timeframe: Change from Baseline in Fatigue Visual Numeric Scale at Day 42

Population: Patients without a particular measure at a required time point were excluded from the analysis of that measure.

Scores range from 0 to 10, with the higher score indicating more fatigue.

Outcome measures

Outcome measures
Measure
Injectafer
n=37 Participants
15 mg/kg up to 750 mg undiluted blinded dose of IV Injectafer (ferric carboxymaltose) at 100 mg/minute Injectafer
Placebo
n=39 Participants
15 cc of Normal Saline IV push at 2 ml/minute Normal Saline
Change in Fatigue Visual Numeric Scale
-3.7 units on a scale
Standard Deviation 3.2
-1.7 units on a scale
Standard Deviation 2.2

SECONDARY outcome

Timeframe: Change from Baseline in Iron Indices, Serum ferritin at Day 42

Population: Patients without a particular measure at a required time point were excluded from the analysis of that measure.

Outcome measures

Outcome measures
Measure
Injectafer
n=39 Participants
15 mg/kg up to 750 mg undiluted blinded dose of IV Injectafer (ferric carboxymaltose) at 100 mg/minute Injectafer
Placebo
n=38 Participants
15 cc of Normal Saline IV push at 2 ml/minute Normal Saline
Change in Iron Indices, Serum Ferritin
292.5 ug/L
Standard Deviation 145.9
2.0 ug/L
Standard Deviation 13.8

SECONDARY outcome

Timeframe: Change from baseline in Iron Indices, Transferrin saturation, at Day 42

Population: Patients without a particular measure at a required time point were excluded from the analysis of that measure.

Outcome measures

Outcome measures
Measure
Injectafer
n=39 Participants
15 mg/kg up to 750 mg undiluted blinded dose of IV Injectafer (ferric carboxymaltose) at 100 mg/minute Injectafer
Placebo
n=38 Participants
15 cc of Normal Saline IV push at 2 ml/minute Normal Saline
Change in Iron Indices - Transferrin Saturation
24.4 percent of transferrin saturation
Standard Deviation 11.4
5.8 percent of transferrin saturation
Standard Deviation 15.1

Adverse Events

Injectafer

Serious events: 0 serious events
Other events: 16 other events
Deaths: 0 deaths

Placebo

Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Injectafer
n=41 participants at risk
15 mg/kg up to 750 mg undiluted blinded dose of IV Injectafer (ferric carboxymaltose) at 100 mg/minute Injectafer
Placebo
n=40 participants at risk
15 cc of Normal Saline IV push at 2 ml/minute Placebo: Normal saline solution
Gastrointestinal disorders
Nausea
7.3%
3/41 • Number of events 3 • 7 months
0.00%
0/40 • 7 months
Infections and infestations
Oral herpes
0.00%
0/41 • 7 months
2.5%
1/40 • Number of events 1 • 7 months
Metabolism and nutrition disorders
Dehydration
0.00%
0/41 • 7 months
2.5%
1/40 • Number of events 1 • 7 months
Skin and subcutaneous tissue disorders
Dermatitis
2.4%
1/41 • Number of events 1 • 7 months
0.00%
0/40 • 7 months
Vascular disorders
Flushing
14.6%
6/41 • Number of events 6 • 7 months
0.00%
0/40 • 7 months
Ear and labyrinth disorders
Ear discomfort
2.4%
1/41 • Number of events 1 • 7 months
0.00%
0/40 • 7 months
Nervous system disorders
Dizziness
4.9%
2/41 • Number of events 2 • 7 months
0.00%
0/40 • 7 months
Nervous system disorders
Dysgeusia
2.4%
1/41 • Number of events 1 • 7 months
0.00%
0/40 • 7 months
Nervous system disorders
Headache
2.4%
1/41 • Number of events 1 • 7 months
0.00%
0/40 • 7 months
Nervous system disorders
Paraesthesia
2.4%
1/41 • Number of events 1 • 7 months
0.00%
0/40 • 7 months

Additional Information

Mark A. Falone, MD

Luitpold Pharmaceuticals, Inc.

Phone: 610.650.4200

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place