IV Iron Replacement for Iron Deficiency in Idiopathic Pulmonary Arterial Hypertension (IPAH) Patients

• Status: Completed
• Trial end date: 2017-12-22
• Target enrollment: 0
• Participant gender: All

Summary

This study will establish whether intravenous iron replacement has clinical benefit in idiopathic pulmonary arterial hypertension.

A 24-week double-blind, randomised, placebo-controlled, crossover study will investigate whether a single dose of 1g of Ferinject® or CosmoFer improves cardiopulmonary haemodynamics, exercise capacity and quality of life and is well-tolerated.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Imperial College London
• Collaborator: Fu Wai Hospital, Beijing, China
• Treatments: Iron

Criteria

• Males or females aged between 16-75 years old

• PAH which is idiopathic, heritable or associated with anorexigens.

• Iron deficiency (TfR levels > 28.1 nmol/l, where sTfR analysis is available, Ferritin < 37 ug/l; transferrin saturations < 16.4%; iron < 10.3 umol/l)

• Documented diagnosis of PAH by right heart catheterisation performed at any time prior to Screening showing: resting mean pulmonary artery pressure >25mmHg, pulmonary capillary wedge pressure =/< 15 mm Hg and normal or reduced cardiac output;

• 6 minute walking distance greater than 50m at entry;

• Stable on an unchanged PAH therapeutic regime (any combination of endothelin receptor antagonist, phosphodiesterase inhibitor or prostacyclin analogue) for at least 1 month.

• Able to provide written informed consent prior to any study-mandated procedures

• Female subjects of child-bearing potential are eligible to participate if they agree to use one of the following contraception methods:

• Abstinence

• Contraceptive methods with a failure rate of < 1%:

• Oral contraceptive, either combined or progestogen alone;

• Injectable progestogen;

• Implants of levonorgestrel;

• Estrogenic vaginal ring;

• Percutaneous contraceptive patches;

• Intrauterine device (IUD) or intrauterine system (IUS) that meets the <1% failure rate as stated in the product label;

• Male partner(s) sterilization (vasectomy with documentation of azoospermia) prior to the female subject's entry into the study;

• Double barrier method: condom and occlusive cap (diaphragm or cervical/vault caps) plus vaginal spermicidal agent (foam/gel/film/cream/suppository).

Exclusion criteria

• Unable to provide informed consent.

• Clinically-significant renal disease (Creatinine clearance < 30 ml/min per 1.73 m2 calculated from CKD-Epi http://www.qxmed.com/renal/Calculate-CKD-EPI-GFR.php) or liver disease (including serum transaminases > 3 times upper limit of normal).

• Haemoglobin concentration <10 g/dl.

• Patients will be excluded if any single parameter (iron, ferritin or transferrin saturation) exceeds 1x upper limit of normal (ULN) in the local lab reference range.

• Patients with moderate to severe hypophosphatemia as defined as <0.65mmol/L

• Known to have haemoglobinopathy e.g. sickle cell disease, thalassaemia.

• Admission to hospital related to PAH or change in PAH therapy within 1 month prior to Screening.

• Evidence of left ventricular disease or significant lung disease on high-resolution CT scanning or lung function as judged by the investigator

• Acute or chronic infection or inflammation.

• Significant uncontrolled asthma as judged by the investigator, eczema or atopic allergies.

• Females who are lactating or pregnant.

• Individuals known to have HIV, Hepatitis B or C or Creutzfeld-Jakob disease.

• Known hypersensitivity to Ferinject® or any of its excipients.

• Evidence of disturbances in utilisation of iron.

• Significant blood loss (e.g. GI bleed) within the last 3 months or history of menorrhagia.

• Unable to perform a Cardiopulmonary Exercise Test i.e. due to syncope or musculoskeletal factors.

• Patients who have received an investigational medicinal product within 30 days of entering the baseline visit