Clinical trial NCT01110356: Ferric Carboxymaltose Treatment to Improve Fatigue Symptoms in Iron-deficient Non-anaemic Women of Child Bearing Age
Official title: A Multicentre Randomised Placebo-controlled Study to Assess the Efficacy and Safety of a Single Administration of Ferric Carboxymaltose in Improving Fatigue Symptoms in Iron-deficient Non-anaemic Women of Child Bearing Age
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Iron Deficiency
Lead sponsor: Vifor Pharma
Sponsor class: INDUSTRY
Collaborators: name: SGS S.A.
First posted: 2010-04-26
Enrollment: 294
Sex: FEMALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: research study of Ferric carboxymaltose to treat fatigue/exhaustion symptoms, believed to be due to iron deficiency.


Eligibility criteria: Inclusion Criteria:

* Signed informed consent prior to study specific procedures.
* Premenopausal, regularly menstruating women.
* Age ≥18 years.
* Body weight between 50 and 90 kg.
* Haemoglobin ≥115 g/L.
* Iron deficiency at screening defined as follows:
* S-ferritin level \<50 ng/mL, AND, TfS \<20%, OR,
* S-ferritin level \<15 ng/mL.
* Serum C-reactive protein:
* \<5 mg/L if not on oral contraception, OR,
* \<20 mg/L if use of oral contraception.
* Minimum total score of 5 on the Piper Fatigue Scale (PFS) (mean of items 2 to 23).
* Negative pregnancy test (serum human chorionic gonadotropin (hCG) at screening.
* Normal levels of vitamin B12 and folic acid at screening.
* Adequate contraception during the study period and for 1 month following study completion.
* Availability and willingness to complete all study visits and procedures per protocol.

Exclusion Criteria:

* Haemoglobin level \<115 g/L.
* Haemoglobinopathy.
* Haemochromatose.
* Major depressive disorder based on Patient Health Questionnaire (PHQ-9) (5 items with scores ≥2; one of which corresponds to question number 1 or 2).
* Any active or unstable concurrent medical condition (e.g., cancer, renal dysfunction, liver dysfunction (aspartate aminotransferase (AST); alanine aminotransferase (ALT) \>3-fold upper limit), angina (Class IV).
* Known human immunodeficiency virus/acquired immunodeficiency syndrome, hepatitis B virus or hepatitis C virus infection.
* Chronic inflammatory disease (e.g., rheumatoid arthritis; inflammatory bowel disease).
* Documented history of clinically significant level of sleep apnoea defined as 5 or more episodes per hour of any type of apnoea.
* Intake of concurrent medications that could interfere with physical or mental performance (e.g., antidepressive, antihistamines, narcotic or any chemotherapeutic agents known to cause drowsiness).
* Important recent weight loss (\>10% within the past month).
* Body weight \<50 kg or \>90 kg.
* Thyroid dysfunction, thyroid stimulating hormone \>4 μU/mL.
* Intake of iron preparations 4 weeks prior to screening.
* Use of gestagens e.g., Implanon, Mirena, Depo-Provera for menstruation repression (see Section 7.7, Prohibited Therapy or Concomitant Treatment, page 35).
* Known hypersensitivity to FCM or to any other iron preparation.
* Pregnancy (positive hCG test at screening) or breast feeding.
* Participation in any other interventional trial within 4 weeks prior to screening.
* Inability to fully comprehend and/or perform study procedures or provide written consent in the Investigator's opinion.
* Subject is not using adequate contraceptive precautions during the study and for up to 1 month after the last dose of the study medication. A highly effective method of birth control is defined as those which result in a low failure rate (i.e., less than 1% per year) when used consistently and correctly such as implants, injectables, combined oral contraceptives, some intra-uterine devices, sexual abstinence or vasectomised partner.
* Subject previously has entered this study.
* Subject will not be available for follow-up assessments.

Primary outcomes:
measure: To assess the efficacy of a single intravenous (IV) administration of FCM (1,000 mg) compared with placebo in improving fatigue symptoms in IDNA women of child bearing age.
timeFrame: Day 56
Secondary outcomes:
measure: To compare efficacy of a single IV application of FCM with that of placebo on change of iron status on Day 56 (i.e., proportion of subjects with haemoglobin (Hb) ≥12 g/dL; serum-ferritin (s-ferritin) ≥50 ng/mL; transferrin saturation (TfS) >20%).
timeFrame: Day 56
measure: To determine the relationship between change in iron status (s-ferritin and TfS) and improvement of fatigue symptoms.
timeFrame: Day 56
Other outcomes:
Locations:
city: Vienna
country: Austria
facility: Universitätsklinik für Frauenheilkunde
Study officials:
affiliation: Quartier UNIL-CHUV
name: Bernard Favrat
role: PRINCIPAL_INVESTIGATOR
References:
citation: Favrat B, Balck K, Breymann C, Hedenus M, Keller T, Mezzacasa A, Gasche C. Evaluation of a single dose of ferric carboxymaltose in fatigued, iron-deficient women--PREFER a randomized, placebo-controlled study. PLoS One. 2014 Apr 21;9(4):e94217. doi: 10.1371/journal.pone.0094217. eCollection 2014.