Trial · NCT05630001
Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.
NCT05630001, Phase 3, Completed, Novartis Pharmaceuticals; results first posted 2025-12-15
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan. Trials, record NCT05630001. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05630001
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.
- NCT number
- NCT05630001
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 52 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Interventions
- Iptacopan
- First posted
- 2022-11-29
- Last updated
- 2026-01-13
- Results first posted
- 2025-12-15
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Hb Levels as Mean of Visits Between Day 126 and Day 168 Compared to Baseline Tested for Non-inferiority | Baseline, Day 126 to Day 168 | Change in hemoglobin (Hb) levels as mean of visits between Day 126 and Day 168 compared to baseline. Baseline is defined as as the mean of three Hb assessments conducted at the central laboratory: two during screening and the third on Day 1. The estimation of change from baseline in Hb levels was handled by the hypothetical strategy where participants were assumed as if they did not receive RBC transfusions while on treatment (RBC transfusions were expected to be rare). Assuming that participants had stable Hb levels at study entry, the mean change from baseline in Hb level between Day 126 and Day 168 was expected to be unchanged should participants have continued on anti-C5 treatment. Non-inferiority of iptacopan was therefore tested by the null hypothesis (H0) against the alternate hypothesis (H1) comparing the mean change from baseline in Hb level in iptacopan between Day 126 and Day 168 (μ) to -1 g/dL: H0: μ \<= -1, H1: μ \> -1. |
| Secondary | Change in Hb Levels as Mean of Visits Between Day 126 and Day 168 Compared to Baseline Tested for Superiority | Baseline, Day 126 to Day 168 | Change in hemoglobin (Hb) levels as mean of visits between Day 126 and Day 168 compared to baseline. Baseline is defined as as the mean of three Hb assessments conducted at the central laboratory: two during screening and the third on Day 1. |
| Secondary | Proportion of Hematological Responders to Iptacopan Treatment | Day 126 to Day 168 | Response defined as Hb ≥12 g/dL assessed between visits Day 126 and Day 168 in the absence of RBC transfusions, on three out of four measurements taken at the visits occurring in last six weeks |
| Secondary | Proportion of Participants Who Remain Free From Transfusions | Day 1 to Day 168 | Number of participants with absence of administration of packed RBC transfusions between Day 1 and Day 168 |
| Secondary | Change From Baseline in Absolute Reticulocytes Count (ARC) Levels | Baseline, Day 126 to Day 168 | Change from baseline in ARC levels as mean of visits between Day 126 and Day 168 |
| Secondary | Percentage Change From Baseline in Lactate Dehydrogenase (LDH) Levels | Baseline, Day 126 to Day 168 | Percentage change from baseline in LDH levels as mean of visits between Day 126 and Day 168 |
| Secondary | Change From Baseline in Treatment Satisfaction Score Using TSQM-9 Questionnaire | Baseline, Day 84 and Day 168 | Difference in scores of the Treatment Satisfaction Questionnaire for Medication(TSQM-9) between baseline and Day 84 and Day 168 assessed after switching from SoC (anti-C5) to iptacopan. TSQM-9 is a patient reported outcomes measure that was designed to assess patients' satisfaction with medication across three domains of effectiveness, convenience and global satisfaction. The TSQM-9 contains 3 questions in each domain. Domain scores range from 0 - 100 with higher scores representing better outcomes for the domain. |
| Secondary | Change From Baseline in Fatigue Score Using FACIT-F Questionnaire | Baseline, Day 84 and Day 168 | Change from baseline in patient-reported scores for the functional assessment of chronic illness therapy - Fatigue (FACIT-F) collected at Day 84 and Day 168. The FACIT-F is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function. All FACIT scales are scored so that a high score is better. As each of the 13 items of the FACIT-F scale ranges from 0-4, the range of possible scores is 0-52, with 0 being the worst possible score and 52 the best. |
| Secondary | Percentage of Patients Who Had Breakthrough Hemolysis (BTH) Event | Up to 168 Days | Wilson method is used to calculate the confidence interval for the proportion of patients who had events. The breakthrough is defined clinical if either there is a decrease in hemoglobin levels equal to or more than 2 g/dL (compared to the latest assessment) or if patients present signs or symptoms of gross hemoglobinuria, painful crisis, dysphagia or any other significant clinical PNH-related signs \& symptoms, in presence of laboratory evidence of intravascular hemolysis. |
| Secondary | Percentage of Patients Who Had Major Adverse Vascular Events (MAVEs) | Up to 168 Days | A MAVE is defined as: acute peripheral vascular occlusion, amputation (non-traumatic; nondiabetic), cerebral arterial occlusion/cerebrovascular accident, cerebral venous occlusion, dermal thrombosis, gangrene (non-traumatic; nondiabetic), hepatic/portal vein thrombosis (Budd-Chiari syndrome), mesenteric/visceral arterial thrombosis or infarction, mesenteric/visceral vein thrombosis or infarction, myocardial infarction, pulmonary embolus, renal arterial thrombosis, renal vein thrombosis, thrombophlebitis / deep vein thrombosis, transient ischemic attack, unstable angina or other. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in Hb Levels as Mean of Visits Between Day 126 and Day 168 Compared to Baseline Tested for Non-inferiority | Baseline, Day 126 to Day 168 | Full Analysis Set (FAS) comprised all participants with confirmed eligibility to whom study treatment was assigned. The number of participants analyzed corresponds to the number of subjects who had values at baseline and Day 126 and Day 168. | g/dL | MEAN |
| SECONDARY | Change in Hb Levels as Mean of Visits Between Day 126 and Day 168 Compared to Baseline Tested for Superiority | Baseline, Day 126 to Day 168 | Full Analysis Set (FAS) comprised all participants with confirmed eligibility to whom study treatment was assigned. The number of participants analyzed corresponds to the number of subjects who had values at baseline and Day 126 and Day 168. | g/dL | MEAN |
| SECONDARY | Proportion of Hematological Responders to Iptacopan Treatment | Day 126 to Day 168 | Full Analysis Set (FAS) comprised all participants with confirmed eligibility to whom study treatment was assigned. The number of participants analyzed corresponds to the number of subjects who had values at Day 126 and Day 168. | Proportion of participants | NUMBER |
| SECONDARY | Proportion of Participants Who Remain Free From Transfusions | Day 1 to Day 168 | Full Analysis Set (FAS) comprised all participants with confirmed eligibility to whom study treatment was assigned. | Proportion of participants | NUMBER |
| SECONDARY | Change From Baseline in Absolute Reticulocytes Count (ARC) Levels | Baseline, Day 126 to Day 168 | Full Analysis Set (FAS) comprised all participants with confirmed eligibility to whom study treatment was assigned. The number of participants analyzed corresponds to the number of subjects who had values at Day 126 and Day 168. | 10^9 cells/L | MEAN |
| SECONDARY | Percentage Change From Baseline in Lactate Dehydrogenase (LDH) Levels | Baseline, Day 126 to Day 168 | Full Analysis Set (FAS) comprised all participants with confirmed eligibility to whom study treatment was assigned. The number of participants analyzed corresponds to the number of subjects who had values at Day 126 and Day 168. | Percent change from baseline in LDH | MEAN |
| SECONDARY | Change From Baseline in Treatment Satisfaction Score Using TSQM-9 Questionnaire | Baseline, Day 84 and Day 168 | Full Analysis Set (FAS) comprised all participants with confirmed eligibility to whom study treatment was assigned. The number of participants analyzed corresponds to the number of subjects who had values at baseline, Day 84 and Day 168. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in Fatigue Score Using FACIT-F Questionnaire | Baseline, Day 84 and Day 168 | Full Analysis Set (FAS) comprised all participants with confirmed eligibility to whom study treatment was assigned. The number of participants analyzed corresponds to the number of subjects who had values at Day 84 and Day 168. | score on a scale | MEAN |
| SECONDARY | Percentage of Patients Who Had Breakthrough Hemolysis (BTH) Event | Up to 168 Days | Full Analysis Set (FAS) comprised all participants with confirmed eligibility to whom study treatment was assigned. | Percentage of patients with BTH events | NUMBER |
| SECONDARY | Percentage of Patients Who Had Major Adverse Vascular Events (MAVEs) | Up to 168 Days | Full Analysis Set (FAS) comprised all participants with confirmed eligibility to whom study treatment was assigned. | Percentage of patients with MAVEs | NUMBER |
Source ClinicalTrials.gov
Results by group16
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in Hb Levels as Mean of Visits Between Day 126 and Day 168 Compared to Baseline Tested for Non-inferiority | LNP023 200mg b.i.d. | 2.01 | 95% Confidence Interval 1.74 to 2.29 | g/dL | |
| Change in Hb Levels as Mean of Visits Between Day 126 and Day 168 Compared to Baseline Tested for Superiority | LNP023 200mg b.i.d. | 2.01 | 95% Confidence Interval 1.74 to 2.29 | g/dL | |
| Proportion of Hematological Responders to Iptacopan Treatment | LNP023 200mg b.i.d. | 92.7 | 95% Confidence Interval 84.6 to 98.1 | Proportion of participants | |
| Proportion of Participants Who Remain Free From Transfusions | LNP023 200mg b.i.d. | 100.0 | 95% Confidence Interval 93.1 to 100.0 | Proportion of participants | |
| Change From Baseline in Absolute Reticulocytes Count (ARC) Levels | LNP023 200mg b.i.d. | -89.19 | 95% Confidence Interval -95.47 to -82.92 | 10^9 cells/L | |
| Percentage Change From Baseline in Lactate Dehydrogenase (LDH) Levels | LNP023 200mg b.i.d. | -1.30 | 95% Confidence Interval -6.56 to 4.26 | Percent change from baseline in LDH | |
| Change From Baseline in Treatment Satisfaction Score Using TSQM-9 Questionnaire | LNP023 200mg b.i.d. | TSQMS1-Effectiveness Day 84 | 15.08 | 95% Confidence Interval 9.60 to 20.57 | score on a scale |
| Change From Baseline in Treatment Satisfaction Score Using TSQM-9 Questionnaire | LNP023 200mg b.i.d. | TSQMS1-Effectiveness Day 168 | 12.54 | 95% Confidence Interval 5.58 to 19.49 | score on a scale |
| Change From Baseline in Treatment Satisfaction Score Using TSQM-9 Questionnaire | LNP023 200mg b.i.d. | TSQMS1-Convenience Day 84 | 20.34 | 95% Confidence Interval 13.68 to 26.99 | score on a scale |
| Change From Baseline in Treatment Satisfaction Score Using TSQM-9 Questionnaire | LNP023 200mg b.i.d. | TSQMS1-Convenience 168 | 23.86 | 95% Confidence Interval 17.62 to 30.10 | score on a scale |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | LNP023 200mg b.i.d. | 52 | |
| Overall Study | COMPLETED | LNP023 200mg b.i.d. | 51 | |
| Overall Study | NOT COMPLETED | LNP023 200mg b.i.d. | 1 |
Source ClinicalTrials.gov
Adverse events10
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Diarrhoea | Gastrointestinal disorders | LNP023 200mg b.i.d. | 6 | 52 | |
| other | Nausea | Gastrointestinal disorders | LNP023 200mg b.i.d. | 6 | 52 | |
| other | Fatigue | General disorders and administration site conditions | LNP023 200mg b.i.d. | 4 | 52 | |
| other | Nasopharyngitis | Infections and infestations | LNP023 200mg b.i.d. | 6 | 52 | |
| other | Upper respiratory tract infection | Infections and infestations | LNP023 200mg b.i.d. | 3 | 52 | |
| other | Headache | Nervous system disorders | LNP023 200mg b.i.d. | 9 | 52 | |
| other | Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | LNP023 200mg b.i.d. | 3 | 52 | |
| serious | Pyrexia | General disorders and administration site conditions | LNP023 200mg b.i.d. | 1 | 52 | |
| serious | Pneumonia bacterial | Infections and infestations | LNP023 200mg b.i.d. | 1 | 52 | |
| serious | Subdural haematoma | Injury, poisoning and procedural complications | LNP023 200mg b.i.d. | 1 | 52 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | LNP023 200mg b.i.d. | 0 | 52 |
| Total, other adverse events | LNP023 200mg b.i.d. | 25 | 52 |
| Total, serious adverse events | LNP023 200mg b.i.d. | 2 | 52 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT05630001
GET https://conductscience.org/api/trials/v1/NCT05630001/registered