NCT07229235

Brief Summary

This study evaluates iptacopan effectiveness and safety in routine clinical practice, with a focus on hematological response, transfusion avoidance, and patient-reported outcomes. The primary objective of the REAL-CARE study is to is to assess the long-term hematological response following iptacopan initiation. This will be assessed through the absolute change in hemoglobin (Hb) levels at 12 months post-initiation, and the proportion of patients who remain free from red blood cell (RBC) transfusions, prescribed as per local requirement and based on Investigator's judgment, from Day 14 through Month 12 after starting iptacopan.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
50

participants targeted

Target at P25-P50 for all trials

Timeline
28mo left

Started Jan 2026

Typical duration for all trials

Geographic Reach
1 country

7 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress12%
Jan 2026Aug 2028

First Submitted

Initial submission to the registry

October 30, 2025

Completed
15 days until next milestone

First Posted

Study publicly available on registry

November 14, 2025

Completed
2 months until next milestone

Study Start

First participant enrolled

January 13, 2026

Completed
2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 31, 2028

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 31, 2028

Last Updated

April 16, 2026

Status Verified

April 1, 2026

Enrollment Period

2.6 years

First QC Date

October 30, 2025

Last Update Submit

April 13, 2026

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Keywords

REAL-CAREptacopanparoxysmal nocturnal hemoglobinuriaPNHNIS

Outcome Measures

Primary Outcomes (2)

  • absolute change in Hb level post iptacopan initiation

    12-month absolute change in hemoglobin (Hb) level post iptacopan initiation

    12 months

  • Proportion of patients with no RBC transfusions

    12-month proportion of patients with no Red Blood Cells (RBC) transfusions, prescribed as per local requirement and based on Investigator's judgment, after Day 14.

    12 months

Secondary Outcomes (19)

  • Socio-demographic and clinical variables of Paroxysmal Nocturnal Hemoglobinuria population

    date of iptacopan start, day 0

  • Absolute change in Hb level post iptacopan initiation

    date of iptacopan start (day 0), 24 months

  • Proportion of patients who didn't need RBC transfusion

    24 months

  • Proportion of patients with Hb levels ≥ 12 g/dL AND no RBC transfusion

    12 month, 24 month

  • Proportion of patients with change in Hb levels ≥ 2 g/dL post iptacopan initiation AND no RBC transfusion

    12 month, 24 month

  • +14 more secondary outcomes

Study Arms (1)

Iptacopan

Treatment with iptacopan

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Italian population of adult patients with PNH either naïve to iptacopan or transitioning from the Managed Access Program (MAP) starting from January 2023

You may qualify if:

  • signed informed consent: patient must provide written informed before any study assessment is collected; in case of deceased patients, informed consent is not required as per Art. 110, comma 1 of Legislative Decree n° 196, dated 30 June 2003 as amended by D.lgs. 101/2018 and the "Autorizzazione generale al trattamento dei dati personali effettuato per scopi di ricerca scientifica" n° 9/2016
  • Male and female,
  • ≥ 18 years of age,
  • documented diagnosis of PNH,
  • followed in Italian clinical sites,

You may not qualify if:

  • Concomitant participation in an interventional clinical study related to PNH or its treatment.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

Novartis Investigative Site

Alessandria, AL, 15121, Italy

RECRUITING

Novartis Investigative Site

Avellino, AV, 83100, Italy

RECRUITING

Novartis Investigative Site

Brescia, BS, 25123, Italy

RECRUITING

Novartis Investigative Site

Florence, FI, 50134, Italy

RECRUITING

Novartis Investigative Site

Palermo, PA, 90146, Italy

RECRUITING

Novartis Investigative Site

Roma, RM, 00161, Italy

RECRUITING

Novartis Investigative Site

Bassano del Grappa, VI, 36061, Italy

RECRUITING

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesMyelodysplastic SyndromesBone Marrow Diseases

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

+41613241111novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
OTHER
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 30, 2025

First Posted

November 14, 2025

Study Start

January 13, 2026

Primary Completion (Estimated)

August 31, 2028

Study Completion (Estimated)

August 31, 2028

Last Updated

April 16, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will not share

Locations

IT(7)