A randomized, double-blind, placebo-controlled, multicentre trial, assessing the impact of ferric carboxymaltose on exercise capacity and functional status in pulmonary hypertension (IRON-PH)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Ziekenhuis Oost Limburg

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

Trial duration

27 Jan 2026 → ongoing

Decision date (initial)

2025-12-12

Transition trial

No

Low-intervention

Yes

Rare-disease indication

No

Vulnerable population

No

Funding sources

KCE (Belgian Health Care Knowledge Centre)

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

The primary objective is to determine if in patients with pulmonary hypertension that are suffering from iron deficiency treatment with FCM with dosing defined according to the SmPC in comparison to matching placebo leads to a significant improvement in exercise capacity measured as the change in 6 minute walking distance from baseline to 24 weeks of follow-up.

Secondary objectives 1

1. To measure the change in disease specific health status and health related QoL

Conditions and MedDRA coding

Pulmonary hypertension

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. ≥18 years of age
2. WHO functional class II – IV
3. Iron deficiency defined as TSAT <21% (no more than ≥3 months old at randomization)
4. PH defined by echocardiography and/or right heart catheterization (RHC) according to the following WHO groups: - Group 1 PH: • Patients with a diagnosis of idiopathic PAH, hereditary PAH, drug induced PAH or PAH and associated with CTD or CHD (historical RHC available) on stable and optimized doses of PAH targeted therapies for at least 4 weeks before randomization. • Echocardiographic evidence of a high or intermediate probability for PH as per 2022 ESC PH guidelines. - Group 2 PH and baseline LVEF > 50% on imaging modality within last 6 months before randomization and on stable doses of loop diuretics and HFpEF therapies for 4 weeks. Group 2 PH can be included based on echocardiography or RHC.: • Echocardiography (<6mo before randomization): - Presence of LVH or LA-enlargement - E/e’ >15 (at rest or exercise) - TRVmax >2.8 m/s (at rest) or mPAP/CO>3 mHg/L/min (exercise) or echocardiographic evidence of high or intermediate probability for PH as per 2022 ESC PH guidelines. • RHC (<6mo before randomization) - mPAP > 20 mmHg - PCWP > 15 mmHg at rest or PCWP/CO-slope > 2mmHg/L/min or exercise PCWP>25mmHg, or PCWP 13-15 mmHg with elevation ≥18mmHg after 500 cc Fluid Challenge - Group 4 PH: • Inoperable CTEPH • persistent/recurrent CTEPH (> 1 year after endarterectomy or > 6 months after balloon pulmonary angioplasty) ineligible for balloon pulmonary angioplasty. • Echocardiographic evidence of a high or intermediate probability for PH as per 2022 ESC PH guidelines.

Exclusion criteria 15

1. Screening haemoglobin < 8 g/dl or >15 g/dl
2. Ferritin > 700 ng/mL
3. Known hypersensitivity reaction to any component of FCM
4. Group 1 PH associated with veno-occlusive diseases
5. Primary diagnosis of group 3 PH
6. Primary diagnosis of group 5 PH
7. Treatment with oral or other IV iron therapies at screening
8. Current or planned mechanical circulatory support or lung/heart transplantation
9. Any planned surgery or procedure leading to expected significant blood loss (defined as more than 250 ml = equal to 125mg of iron)
10. Haemodialysis or peritoneal dialysis (current or planned within the next 24 weeks)
11. Inability to return for follow up visits within the necessary windows
12. Concurrently in a study with another investigational product
13. Uncorrected moderate to severe aortic stenosis (AVA <1.5cm² and mean gradient >20 mmHg) or severe valvular regurgitation (except tricuspid regurgitation)
14. Impression by investigator that patient cannot perform a 6MWT
15. Active infection as judged by the investigator

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change in 6MWD from baseline to 24 weeks follow-up

Secondary endpoints 4

1. Change in EQ5D from baseline to 24 weeks follow-up
2. Change in MLHFQ from baseline to 24 weeks follow-up
3. Change in FSS from baseline to 24 weeks follow-up
4. The hazard ratio between treatment arms in developing a composite clinical worsening event in the overall trial population (from first patient visit to last patient visit)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Ziekenhuis Oost Limburg

Sponsor organisation

Ziekenhuis Oost Limburg

Address

Synaps Park 1

City

Genk

Postcode

3600

Country

Belgium

Scientific contact point

Organisation

Ziekenhuis Oost Limburg

Contact name

Katrien Tartaglia

Public contact point

Organisation

Ziekenhuis Oost Limburg

Contact name

Katrien Tartaglia

Locations

1 EU/EEA country · 7 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 24 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications