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

A Blinded, Randomized, Placebo-Controlled Study to Investigate the Efficacy and Safety of Injectafer (Ferric Carboxymaltose) in the Treatment of Iron Deficient Patients With Fibromyalgia

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02409459

Enrollment

Registered

2015-04-06

Start date

2015-03-31

Completion date

2016-03-31

Last updated

2018-02-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Iron Deficiency, Fibromyalgia

Brief summary

This study investigates the efficacy and safety of an IV iron Injectafer in the treatment of iron deficient patients with fibromyalgia. Eligible subjects will be randomized in a 1:1 ratio to receive Injectafer or Placebo on days 0 and 5. All treated subjects will be followed for efficacy and safety for 42 days.

Detailed description

This is a blinded, randomized, placebo-controlled study. All subjects who meet the inclusion requirements and no exclusion criteria will be entered into an up to 14 day screening phase. Eligible subjects will be randomized in a 1:1 ratio to receive Injectafer or Placebo on days 0 and 5. All treated subjects will be followed for efficacy and safety for 42 days. Subjects will visit the clinic on days 0 and 5 for treatment, and then on days 14, 28, and 42. The subject's participation in the study will be for approximately 42 days from day 0.

Interventions

DRUGInjectafer

DRUGPlacebo

Normal saline solution

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

DOUBLE (Subject, Investigator)

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

* Male or female subject's ≥ 18 years of age, able to give informed consent to the study. * Fibromyalgia diagnosis based on The 2011 modification of the American College of Rheumatology (ACR) 's 2010 preliminary criteria for diagnosing fibromyalgia (2011ModCr) * A baseline score ≥ 60 on the FIQR * Subject's current medications to treat fibromyalgia, including pain medicines, anti-depressants, sleep medications must be on a stable dose for at least 30 days. * Subject's current narcotic medication must be on a stable dose for at least 30 days prior to randomization * Subjects at risk for pregnancy must have a negative pregnancy test at baseline and be practicing an acceptable form of birth control: have had a hysterectomy or tubal ligation, or otherwise be incapable of pregnancy, or have practiced any of the following methods of contraception for at least one month prior to study entry: hormonal contraceptives, spermicide and barrier, intrauterine device, partner sterility.

Exclusion criteria

* Parenteral iron use within 4 weeks prior to screening. * History of \> 10 blood transfusions in the past 2 years. * Anticipated need for blood transfusion during the study. * Known hypersensitivity reaction to any component of Injectafer® (Ferric Carboxymaltose). * Current or acute or chronic infection other than viral upper respiratory tract infection * Malignancy (other than basal or squamous cell skin cancer or the subject has been cancer free for ≥ 5 years). * Active inflammatory arthritis (e.g. rheumatoid arthritis, SLE). * Pregnant or lactating women. * Severe peripheral vascular disease with significant skin changes. * Seizure disorder currently being treated with medication. * Baseline ferritin ≥ 50 ng/mL. * Baseline TSAT ≥ 20%. * History of hemochromatosis or hemosiderosis or other iron storage disorders. * Known positive hepatitis with evidence of active disease. * Hemoglobin greater than the upper limit of normal. * Calcium or phosphorous outside the normal range. * Known positive hepatitis B antigen (HBsAg) or hepatitis C viral antibody (HCV) with evidence of active hepatitis (i.e., AST/ALT greater than the upper limit of normal). * Known positive HIV-1/HIV-2 antibodies (anti-HIV) * Received an investigational drug within 30 days before randomization. * Chronic alcohol or drug abuse within the past 6 months. * Any other pre-existing laboratory abnormality, medical condition or disease which in view of the investigator participation in this study may put the subject at risk. * Subject unable to comply with the study requirements.

Design outcomes

Primary

Measure	Time frame	Description
Proportion of Patients With a ≥13 Point Improvement in FIQR Score	Day 42	The primary efficacy endpoint was the percentage of subjects who had a ≥13-point improvement in the FIQR from baseline to Day 42.

Secondary

Measure	Time frame	Description
Change in FIQR Score	Change from Baseline in FIQR score at Day 42	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.
Change in BPI, Pain Severity	Change from Baseline in Brief Pain Inventory Pain Severity Score at Day 42	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.
Change in BPI, Pain Interference	Change from Baseline in Brief Pain Inventory Pain Interference Score at Day 42	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.
Change in Iron Indices, Serum Ferritin	Change from Baseline in Iron Indices, Serum ferritin at Day 42	—
Change in Iron Indices - Transferrin Saturation	Change from baseline in Iron Indices, Transferrin saturation, at Day 42	—
Change in Fatigue Visual Numeric Scale	Change from Baseline in Fatigue Visual Numeric Scale at Day 42	Scores range from 0 to 10, with the higher score indicating more fatigue.

Countries

United States

Participant flow

Participants by arm

Arm	Count
Placebo Group B: Placebo	40
Injectafer Group A: Injectafer	41
Total	81

Baseline characteristics

Characteristic	Placebo	Injectafer	Total
Age, Continuous	44 years STANDARD_DEVIATION 10.85	41 years STANDARD_DEVIATION 11.07	43 years STANDARD_DEVIATION 10.97
History of drug allergy No	17 Participants	21 Participants	38 Participants
History of drug allergy Yes	23 Participants	20 Participants	43 Participants
History of iron intolerance No	37 Participants	35 Participants	72 Participants
History of iron intolerance Yes	3 Participants	6 Participants	9 Participants
IRLS score per Arm/Group	16.3 units on a scale STANDARD_DEVIATION 14.9	13.4 units on a scale STANDARD_DEVIATION 13.16	14.8 units on a scale STANDARD_DEVIATION 14.04
Race/Ethnicity, Customized Race African American	7 Participants	7 Participants	14 Participants
Race/Ethnicity, Customized Race Asian	0 Participants	1 Participants	1 Participants
Race/Ethnicity, Customized Race Caucasian	30 Participants	33 Participants	63 Participants
Race/Ethnicity, Customized Race Hispanic	2 Participants	0 Participants	2 Participants
Race/Ethnicity, Customized Race Other	1 Participants	0 Participants	1 Participants
Sex: Female, Male Female	40 Participants	40 Participants	80 Participants
Sex: Female, Male Male	0 Participants	1 Participants	1 Participants

Adverse events

Event type	EG000 affected / at risk	EG001 affected / at risk
deaths Total, all-cause mortality	0 / 41	0 / 40
other Total, other adverse events	16 / 41	2 / 40
serious Total, serious adverse events	0 / 41	0 / 40

Outcome results

Primary

Proportion of Patients With a ≥13 Point Improvement in FIQR Score

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

Time frame: 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.

Arm	Measure	Value (COUNT_OF_PARTICIPANTS)
Injectafer	Proportion of Patients With a ≥13 Point Improvement in FIQR Score	30 Participants
Placebo	Proportion of Patients With a ≥13 Point Improvement in FIQR Score	26 Participants

Secondary

Change in BPI, Pain Interference

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.

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