Phase 2
N=56
IV Iron Replacement for Iron Deficiency in Idiopathic Pulmonary Arterial Hypertension (IPAH) Patients
Pulmonary Arterial Hypertension · Iron Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT01447628 ↗Enrolled (actual)
56
Serious AEs
5.4%
Results posted
Mar 2022
Primary outcome: Primary: Change in Exercise Capacity - Endurance — 315.44; 302.89 Seconds — p=0.6039
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Saline (Drug); Ferinject or CosmoFer (Drug)
- Age
- Pediatric, Adult, Older Adult · 16+ yrs
- Sex
- All
- Sponsor
- Imperial College London
- Primary completion
- Dec 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Exercise Capacity - Endurance |
315.44; 302.89 | 0.6039 |
| PRIMARY Change in Resting Pulmonary Vascular Resistance (PVR) |
7.69; 5.15; 11.70; 9.5 | 0.0747 |
| SECONDARY Oxygen Consumption (VO2) Level at Peak 12 Weeks After Study Treatment |
1.17; 1.13; 0.93; 0.94 | 0.6583 |
| SECONDARY Oxygen Consumption (VO2) at Metabolic Threshold |
10.01; 9.99; 10.91; 11.2 | 0.7711 |
| SECONDARY Ventilation / Volume of Exhaled Carbon Dioxide (VE/VCO2 Slope) |
41.15; 42.35; 35.06; 36.27 | 0.1788 |
| SECONDARY Oxygen Consumption (VO2) / Work Rate (WR) Slope |
8.12; 8.04; 5.97; 6.04 | 0.8688 |
| SECONDARY Peak Oxygen (O2) Pulse Rate |
13.30; 11.92; 6.55; 6.36 | 0.5465 |
| SECONDARY Oxygen Consumption (VO2) at the End of Endurance Cardio-pulmonary Exercise Test (CPET) |
4.25; 4.49 | 0.6241 |
| SECONDARY Oxygen Consumption (VO2) at 3 Minutes |
1.29; 1.14 | 0.4758 |
| SECONDARY Iron Indices: Serum Iron |
17.15; 15.48; 15.81; 14.31 | 0.2050 |
| SECONDARY Iron Indices: Transferrin Saturations |
26.2; 22; 26.81; 22.88 | 0.0610 |
| SECONDARY Iron Indices: Ferritin |
150.32; 92.74; 135.06; 65.5 | 0.0003 sig |
| SECONDARY Iron Indices: Soluble Transferrin Receptors (sTfR) |
28.37; 37.25 | <0.0001 sig |
| SECONDARY 6 Minute Walk Test: Distance Walked |
426.03; 424.3; 478; 474.19 | 0.8093 |
| SECONDARY 6 Minute Walk Test: Borg Dyspnoea Score After Test |
3.24; 3.68; 1.38; 1.28 | 0.0725 |
| SECONDARY Iron Indices: N-terminal Pro B-type Natriuretic Peptide (NT-pro-BNP) |
307.64; 394.94 | 0.1115 |
| SECONDARY Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR Questionnaire): Symptom Score |
7.94; 7.94 | 0.9790 |
| SECONDARY Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR Questionnaire): Activity Score |
9.12; 9.18 | 0.7702 |
| SECONDARY Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR Questionnaire): QoL Score |
7.58; 7 | 0.2219 |
| SECONDARY Mean Right Atrial Pressure (Cardiac Catheter) |
6.56; 10.12; 6.67; 4.5 | 0.1777 |
| SECONDARY Oxygen Consumption (VO2) Level at Peak |
15.10; 14.67; 14.53; 14.36 | 0.7625 |
| SECONDARY Oxygen Consumption (VO2) at Metabolic Threshold |
10.01; 9.99; 10.91; 11.2 | 0.7711 |
| SECONDARY Stroke Volume (Cardiac Catheter) |
77.87; 99.71; 55.11; 54.65 | 0.9504 |
| SECONDARY Cardiac Magnetic Resonance Imaging (MRI): Right Ventricular End-diastolic Volume |
201.35; 203.06 | 0.4478 |
| SECONDARY Cardiac Magnetic Resonance Imaging (MRI): Right Ventricular End Systolic Volume (RVESV) |
124.99; 126.31 | 0.4590 |
| SECONDARY Cardiac Magnetic Resonance Imaging (MRI): Right Ventricular Stroke Volume (RVSV) |
76.46; 76.84 | 0.8086 |
| SECONDARY Cardiac Magnetic Resonance Imaging (MRI): Right Ventricular Ejection Fraction (RVEF) |
39.89; 39.64 | 0.6944 |
| SECONDARY Cardiac Magnetic Resonance Imaging (MRI): Left Ventricular End Diastolic Volume (LVEDV) |
117.36; 118.33 | 0.585 |
| SECONDARY Cardiac Magnetic Resonance Imaging (MRI): Left Ventricular End Systolic Volume (LVESV) |
43.90; 44.43 | 0.4619 |
| SECONDARY Cardiac Magnetic Resonance Imaging: Left Ventricular Stroke Volume (LVSV) |
43.90; 73.96 | 0.7721 |
| SECONDARY Cardiac Magnetic Resonance Imaging (MRI): Left Ventricular Ejection Fraction (LVEF) |
63.10; 62.94 | 0.8332 |
| SECONDARY Cardiac Magnetic Resonance Imaging: Left Ventricular Mass |
72.82; 73.53 | 0.2651 |
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.
IV iron formulation used in Europe - Ferinject IV iron formulation used in China - CosmoFer
Eligibility Criteria
- Males or females aged between 16-75 years old
- Pulmonary Arterial Hypertension (PAH) which is idiopathic, heritable or associated with anorexigens.
- Iron deficiency (TfR levels > 28.1 nmol/l, where sTfR analysis is available, Ferritin 25mmHg, pulmonary capillary wedge pressure =/ 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 Computed Tomography (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 Human Immunodeficiency Virus (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. Gastro-intestinal 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
Data sourced from ClinicalTrials.gov (NCT01447628). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.