Paroxysmal Nocturnal HemoglobinuriaAPPEXNCT05842486
Hematological Response in Patients With Paroxysmal Nocturnal Hemoglobinuria Treated With Anti-C5 Antibody: an External Control Arm Study for Iptacopan Use in Anti-C5 naïve Patients
An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
The aim of this non-interventional secondary use of data study is to evaluate hematological response in patients with paroxysmal nocturnal hemoglobinuria and anemia in the 6-month period after initiation of anti-C5 antibody treatment using real-world data obtained from multiple datasets. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- Anti-C5 antibody treatment
- How many people
- 92 participants
- Where
- 1 siteFrance
- Study dates
- 18 Jan 2023to 1 Mar 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: The real-world cohort consists of patients with diagnosis of paroxysmal nocturnal hemoglobinuria and anemia initiating anti-C5 therapy. Patient data originate from the Hôpital Saint-Louis, France and St. James's University Hospital, Leeds, United Kingdom.
Who may be able to join
- Incident users of anti-C5 antibody treatment (i.e., first use of anti-C5 antibody with no evidence of use prior to that)
- Confirmed clinical diagnosis of PNH (including PNH clone size >10% in RBC percent/white blood cell (WBC) percent)
- Adult patients ≥18 years of age
- Baseline Hb <10 g/dL.
Who cannot join
- Patients who met any of the following criteria were not eligible for inclusion.
- Bone marrow transplantation within one year prior to initiating anti-C5 treatment
- Hereditary complement deficiency
- Reticulocyte count <60x10^9/L.
- Pregnancy
Trial locations and contacts
1 site in 1 country.
About this study
The aim of this non-interventional secondary use of data study is to evaluate hematological response in patients with paroxysmal nocturnal hemoglobinuria and anemia in the 6-month period after initiation of anti-C5 antibody treatment using real-world data obtained from multiple datasets. The results will be used to contextualize results from the APPOINT-PNH (NCT04820530) trial with iptacopan.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- Anti-C5 antibody treatment
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Anti-C5 antibody treatmentOther
Anti-C5 antibody treatment (i.e., eculizumab and ravulizumab)
| Study group | Role | What they receive |
|---|---|---|
| Real-world cohort | The real-world cohort consists of patients with diagnosis of paroxysmal nocturnal hemoglobinuria and anemia initiating anti-C5 therapy. Receives: Other: Anti-C5 antibody treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Increase from baseline in hemoglobin
Increase from baseline in hemoglobin of ≥ 2 g/dL in the absence of transfusions occurring after Day 14 post index date
Measured: between Day 100 and Day 200
Secondary outcomes (2)
Hemoglobin Response
Achieving hemoglobin of ≥ 12 g/dL in the absence of blood transfusions occurring after Day 14 post index date
Measured: between Day 100 and Day 200
Transfusion avoidance
Transfusion avoidance (dichotomous endpoint) defined proportion of subjects without transfusions during the initial 6-month treatment period starting from 2 Weeks after index date (i.e., date of first anti-C5 antibody treatment for PNH).
Measured: Occurrence between Day 15 and Day 200
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 May 2023
- Study start18 Jan 2023
- Primary completion1 Mar 2023
- Study completion1 Mar 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 92 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- Leeds Cancer Centre at St. James's University Hospital
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05842486
- Sponsor's protocol ID
- CLNP023N12002R
- Acronym
- APPEX
- Status last verified
- Mar 2023
- First submitted
- 24 Apr 2023
- Last updated
- 6 May 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Holt M, Kelly RJ, Fermont JM, Ansari S, Dahlke M, Brindel I, Maafa L, de Fontbrune FS, de Latour RP. Effectiveness of Iptacopan Versus C5 Inhibitors in Complement Inhibitor-Naive Patients With Paroxysmal Nocturnal Haemoglobinuria. EJHaem. 2025 May 20;6(3):e270055. doi: 10.1002/jha2.70055. eCollection 2025 Jun.
PubMed 40395624
Study at a glance
From the registry record, last updated by the sponsor on 6 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hematological Response in Patients With Paroxysmal Nocturnal Hemoglobinuria Treated With Anti-C5 Antibody: an External Control Arm Study for Iptacopan Use in Anti-C5 naïve Patients
- ClinicalTrials.gov ID
- NCT05842486
- Acronym / sponsor ID
- APPEX / CLNP023N12002R
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 92 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- Leeds Cancer Centre at St. James's University Hospital
- Study start
- 18 Jan 2023 (actual)
- Primary completion
- 1 Mar 2023 (actual)
- Study completion
- 1 Mar 2023 (actual)
- First posted
- 6 May 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this non-interventional secondary use of data study is to evaluate hematological response in patients with paroxysmal nocturnal hemoglobinuria and anemia in the 6-month period after initiation of anti-C5 antibody treatment using real-world data obtained from multiple datasets. The results will be used to contextualize results from the APPOINT-PNH (NCT04820530) trial with iptacopan.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Real-world cohort | – | Other: Anti-C5 antibody treatmentThe real-world cohort consists of patients with diagnosis of paroxysmal nocturnal hemoglobinuria and anemia initiating anti-C5 therapy. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The real-world cohort consists of patients with diagnosis of paroxysmal nocturnal hemoglobinuria and anemia initiating anti-C5 therapy. Patient data originate from the Hôpital Saint-Louis, France and St. James's University Hospital, Leeds, United Kingdom.
Key inclusion criteria (4 listed)
- Incident users of anti-C5 antibody treatment (i.e., first use of anti-C5 antibody with no evidence of use prior to that)
- Confirmed clinical diagnosis of PNH (including PNH clone size >10% in RBC percent/white blood cell (WBC) percent)
- Adult patients ≥18 years of age
- Baseline Hb <10 g/dL.
Key exclusion criteria (5 listed)
- Patients who met any of the following criteria were not eligible for inclusion.
- Bone marrow transplantation within one year prior to initiating anti-C5 treatment
- Hereditary complement deficiency
- Reticulocyte count <60x10^9/L.
- Pregnancy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Anti-C5 antibody treatmentAnti-C5 antibody treatment (i.e., eculizumab and ravulizumab) | Other | Real-world cohort |
Primary outcome
Increase from baseline in hemoglobin
Time frame: between Day 100 and Day 200
Increase from baseline in hemoglobin of ≥ 2 g/dL in the absence of transfusions occurring after Day 14 post index date
2 secondary outcomes
Hemoglobin Response
Time frame: between Day 100 and Day 200
Transfusion avoidance
Time frame: Occurrence between Day 15 and Day 200
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 May 2023.
- Derived from this study: PubMed 40395624Holt M, Kelly RJ, Fermont JM, Ansari S, Dahlke M, Brindel I, Maafa L, de Fontbrune FS, de Latour RP. Effectiveness of Iptacopan Versus C5 Inhibitors in Complement Inhibitor-Naive Patients With Paroxysmal Nocturnal Haemoglobinuria. EJHaem. 2025 May 20;6(3):e270055. doi: 10.1002/jha2.70055. eCollection 2025 Jun.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Increase from baseline in hemoglobin (time frame: between Day 100 and Day 200). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05842486, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 May 2023.