FERRIC CARBOXYMALTOSE for Pulmonary hypertension | Iron deficiency

Inclusion criteria

• {"criterion_text":"- ≥18 years of age\n- WHO functional class II – IV\n- Iron deficiency defined as TSAT <21% (no more than ≥3 months old at randomization)\n- PH defined by echocardiography and/or right heart catheterization (RHC) according to the following WHO groups: -\tGroup 1 PH: •\tPatients 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. •\tEchocardiographic evidence of a high or intermediate probability for PH as per 2022 ESC PH guidelines. -\tGroup 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.: •\tEchocardiography (<6mo before randomization): -\tPresence of LVH or LA-enlargement -\tE/e’ >15 (at rest or exercise) -\tTRVmax >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. •\tRHC (<6mo before randomization) -\tmPAP > 20 mmHg -\tPCWP > 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 -\tGroup 4 PH: •\tInoperable CTEPH •\tpersistent/recurrent CTEPH (> 1 year after endarterectomy or > 6 months after balloon pulmonary angioplasty) ineligible for balloon pulmonary angioplasty. •\tEchocardiographic evidence of a high or intermediate probability for PH as per 2022 ESC PH guidelines."}

Exclusion criteria

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

Primary endpoints

• {"endpoint_text":"- Change in 6MWD from baseline to 24 weeks follow-up","definition_or_measurement_approach":"Change in 6 minute walking distance (6MWD) from baseline to 24 weeks of follow-up."}

Secondary endpoints

• {"endpoint_text":"- Change in EQ5D from baseline to 24 weeks follow-up","definition_or_measurement_approach":"Change from baseline to 24 weeks in EQ-5D (disease-specific health status / health related QoL)."}
• {"endpoint_text":"- Change in MLHFQ from baseline to 24 weeks follow-up","definition_or_measurement_approach":"Change from baseline to 24 weeks in Minnesota Living with Heart Failure Questionnaire (MLHFQ)."}
• {"endpoint_text":"- Change in FSS from baseline to 24 weeks follow-up","definition_or_measurement_approach":"Change from baseline to 24 weeks in Fatigue Severity Scale (FSS)."}
• {"endpoint_text":"- 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)","definition_or_measurement_approach":"Time-to-event analysis comparing hazard ratios between treatment arms for a composite clinical worsening event from first patient visit to last patient visit."}

Methods

• Use of recruitment materials (posters and flyers) — K2_Recruitment material poster and K2_Recruitment material flyer available in English, Dutch and French (documents listed for Belgium).

Belgium

Earliest CTIS Part Ii Submission Date

13-11-2025

Latest Decision Or Authorization Date

11-05-2026

Processing Time Days

179

Number Of Sites

7

Number Of Participants

306

Primary sponsor

Full Name

Ziekenhuis Oost Limburg

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Belgium

Third parties

• {"country":"Belgium","full_name":"KCE (Belgian Health Care Knowledge Centre)","duties_or_roles":"Source of monetary support / funding","organisation_type":""}