A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal Nocturnal Hemoglobinuria Clinical Trial

— COMPLETE  

Official title:

A Single Arm, Multicentre Observational Study to Evaluate Effectiveness of Pegcetacoplan Under Real World Conditions in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05776472
• Other study ID #: Sobi.PEGCET-304
• Status: Recruiting
• Start date: June 26, 2023
• Est. completion date: July 3, 2027

Study information

• Verified date: July 2023
• Source: Swedish Orphan Biovitrum
• Contact: Matthias Noelte
• Phone: +41797977276
• Email: matthias.noelte[@]sobi.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This is a 24-month multicenter, observational study designed to describe the real world effectiveness of pegcetacoplan in patients with PNH. Patients meeting the eligibility criteria will be enrolled in the study at the enrollment visit and followed prospectively for 24 (+/- 3) months. The scope of the study is to collect both retrospective and prospective data. The main part of the study will be prospective,collecting data on effectiveness, safety, patient- and clinician-reported outcomes and health care resource use.

Description:

As pegcetacoplan is a new product on the market, with a new mechanism of action, there is an urgent need to provide data to treaters, payers and the PNH community on the real-world usage and effectiveness of pegcetacoplan. This study aims to fill part of that knowledge gap and to add to the knowledge base regarding the use of pegcetacoplan in routine medical practice. Another important rationale for this study is to provide information on pre and post pegcetacoplan treatment outcomes.

The study plans to include approximately 200 patients at 70 sites in Europe, Middle East, Canada and Australia. Patients meeting the eligibility criteria will be enrolled in the study at the enrollment visit and followed prospectively for 24 (+/- 3) months. Patients will come to their routine visits and the available data from each visit will be collected.

The scope of the study is to collect both retrospective and prospective data. The main part of the study will be prospective, collecting data on effectiveness, safety, patient- and clinician-reported outcomes and health care resource use. The study will also collect retrospective data before pegcetacoplan treatment start, which will consist of information on PNH treatment, blood transfusions and healthcare resource use. Data will be collected for up to 12 months prior to pegcetacoplan treatment start. As patients may have been treated with pegcetacoplan for up to 12 months prior to enrollment, retrospective data may be collected for up to 24 months. This means that the total data collection period including both the retrospective and the prospective part is up to 48 (+/- 3) months.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 200
• Est. completion date: July 3, 2027
• Est. primary completion date: January 3, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Patients =18 years of age with a documented PNH diagnosis.

• Patient started routine treatment with pegcetacoplan for PNH up to 12 months before enrollment or prescribed pegcetacoplan at enrollment. Decision to initiate treatment shall be made by the treating physician and independently from the decision to include the patient in the study.

• Patient is willing and able to provide written informed consent to participate in the study in a manner approved by the Institutional Review Board/Independent Ethics Committee and local regulations.

Exclusion Criteria:

• Enrollment in a concurrent clinical interventional study, or intake of an Investigational Medicinal Product (IMP), within three months prior to the start of the current pegcetacoplan treatment.

• Initiated current treatment with pegcetacoplan in an interventional study.

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Nocturnal Hemoglobinuria

Intervention

Drug:
• Pegcetacoplan
Pegcetacoplan will be prescribed according to the label in patients with PNH.

Locations

Country	Name	City	State
Spain	Hospital Lucus Augusti	Lugo

Sponsors (1)

Lead Sponsor	Collaborator
Swedish Orphan Biovitrum

Country where clinical trial is conducted

Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in observed hemoglobin level from initiation of treatment with pegcetacoplan to 6 months	Hemoglobin level in g/dL.	6 months
Secondary	Change of Lactate Dehydrogenase values from initiation of pegcetacoplan treatment to 6 months	Lactate Dehydrogenase (LDH) in U/L	6 months
Secondary	Change in Absolute Reticulocyte Count (ARC) from initiation of pegcetacoplan treatment to 6 months	Absolute Reticulocyte Count (ARC) in 10^9/L	6 months
Secondary	Change in indirect/ total bilirubin from initiation of pegcetacoplan treatment to 6 months	Indirect/ total bilirubin in umol/L	6 months
Secondary	Change in Haptoglobin from initiation of pegcetacoplan treatment to 6 months	Haptoglobin in mg/dL	6 months
Secondary	Change in Ferritin from initiation of pegcetacoplan treatment to 6 months	Ferritin in ug/L	6 months
Secondary	Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	6 months
Secondary	Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	12 months
Secondary	Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	18 months
Secondary	Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	24 months
Secondary	Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of study	Lactate Dehydrogenase (LDH) in U/L	6 months
Secondary	Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of study	Lactate Dehydrogenase (LDH) in U/L	12 months
Secondary	Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of study	Lactate Dehydrogenase (LDH) in U/L	18 months
Secondary	Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of study	Lactate Dehydrogenase (LDH) in U/L	24 months
Secondary	Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of study	Absolute Reticulocyte Count (ARC) in 10^9/L	6 months
Secondary	Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of study	Absolute Reticulocyte Count (ARC) in 10^9/L	12 months
Secondary	Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of study	Absolute Reticulocyte Count (ARC) in 10^9/L	18 months
Secondary	Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of study	Absolute Reticulocyte Count (ARC) in 10^9/L	24 months
Secondary	Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of study	Indirect/ total bilirubin in umol/L	6 months
Secondary	Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of study	Indirect/ total bilirubin in umol/L	12 months
Secondary	Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of study	Indirect/ total bilirubin in umol/L	18 months
Secondary	Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of study	Indirect/ total bilirubin in umol/L	24 months
Secondary	Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study	Haptoglobin in mg/dL	6 months
Secondary	Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study	Haptoglobin in mg/dL	12 months
Secondary	Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study	Haptoglobin in mg/dL	18 months
Secondary	Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study	Haptoglobin in mg/dL	24 months
Secondary	Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of study	Ferritin in ug/L	6 months
Secondary	Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of study	Ferritin in ug/L	12 months
Secondary	Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of study	Ferritin in ug/L	18 months
Secondary	Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of study	Ferritin in ug/L	24 months
Secondary	Hemoglobin = 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	6 months
Secondary	Hemoglobin = 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	12 months
Secondary	Hemoglobin = 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	18 months
Secondary	Hemoglobin = 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	24 months
Secondary	Increase in hemoglobin levels of = 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	6 months
Secondary	Increase in hemoglobin levels of = 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	12 months
Secondary	Increase in hemoglobin levels of = 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	18 months
Secondary	Increase in hemoglobin levels of = 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	24 months
Secondary	Acute hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of study	Yes/No	6 months
Secondary	Acute hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of study	Yes/No	12 months
Secondary	Acute hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of study	Yes/No	18 months
Secondary	Acute hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of study	Yes/No	24 months
Secondary	Annualized number of red blood cell (RBC) transfusions during pegcetacoplan treatment until end of study compared to the 12 month period before pegcetacoplan treatment	Total number of RBC transfusions	12 months
Secondary	Annualized number of red blood cell (RBC) units during pegcetacoplan treatment until end of study compared to the 12 month period before pegcetacoplan treatment	Total number of RBC units	12 months
Secondary	Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of study	Patient reported outcome scale scores for the Functional assessment of chronic illness therapy (FACIT) - Fatigue. It is a 13-item scale and each item is scored on a 5-point Likert Scale ranging from "0-Not at all" to "4-Very much". The FACIT-fatigue score is obtained by summing all item scores. The score range from 0 to 52, higher score indicating less fatigue and lower score indicating more fatigue.	6 months
Secondary	Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of study	Patient reported outcome scale scores for the Functional assessment of chronic illness therapy (FACIT) - Fatigue. It is a 13-item scale and each item is scored on a 5-point Likert Scale ranging from "0-Not at all" to "4-Very much". The FACIT-fatigue score is obtained by summing all item scores. The score range from 0 to 52, higher score indicating less fatigue and lower score indicating more fatigue.	12 months
Secondary	Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of study	Patient reported outcome scale scores for the Functional assessment of chronic illness therapy (FACIT) - Fatigue. It is a 13-item scale and each item is scored on a 5-point Likert Scale ranging from "0-Not at all" to "4-Very much". The FACIT-fatigue score is obtained by summing all item scores. The score range from 0 to 52, higher score indicating less fatigue and lower score indicating more fatigue.	18 months
Secondary	Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of study	Patient reported outcome scale scores for the Functional assessment of chronic illness therapy (FACIT) - Fatigue. It is a 13-item scale and each item is scored on a 5-point Likert Scale ranging from "0-Not at all" to "4-Very much". The FACIT-fatigue score is obtained by summing all item scores. The score range from 0 to 52, higher score indicating less fatigue and lower score indicating more fatigue.	24 months
Secondary	Quality of life at initiation of treatment with pegcetacoplan and every 6 months until end of study	Patient reported outcome scale scores for Quality of life questionnaire for patients with Aplastic Anemia and/or Paroxysmal Nocturnal Hemoglobinuria (QLQ AA/PNH). The QLQ AA/PNH questionnaire consists of 54 items relating to how the patient has been feeling lately with 4 response options: 'Not at all', 'A little', 'Moderately' and 'Very'. Scoring guidelines that have been validated for use in clinical studies will be used to calculate patients' scores.	6 months
Secondary	Quality of life at initiation of treatment with pegcetacoplan and every 6 months until end of study	Patient reported outcome scale scores for Quality of life questionnaire for patients with Aplastic Anemia and/or Paroxysmal Nocturnal Hemoglobinuria (QLQ AA/PNH). The QLQ AA/PNH questionnaire consists of 54 items relating to how the patient has been feeling lately with 4 response options: 'Not at all', 'A little', 'Moderately' and 'Very'. Scoring guidelines that have been validated for use in clinical studies will be used to calculate patients' scores.	12 months
Secondary	Quality of life at initiation of treatment with pegcetacoplan and every 6 months until end of study	Patient reported outcome scale scores for Quality of life questionnaire for patients with Aplastic Anemia and/or Paroxysmal Nocturnal Hemoglobinuria (QLQ AA/PNH). The QLQ AA/PNH questionnaire consists of 54 items relating to how the patient has been feeling lately with 4 response options: 'Not at all', 'A little', 'Moderately' and 'Very'. Scoring guidelines that have been validated for use in clinical studies will be used to calculate patients' scores.	18 months
Secondary	Quality of life at initiation of treatment with pegcetacoplan and every 6 months until end of study	Patient reported outcome scale scores for Quality of life questionnaire for patients with Aplastic Anemia and/or Paroxysmal Nocturnal Hemoglobinuria (QLQ AA/PNH). The QLQ AA/PNH questionnaire consists of 54 items relating to how the patient has been feeling lately with 4 response options: 'Not at all', 'A little', 'Moderately' and 'Very'. Scoring guidelines that have been validated for use in clinical studies will be used to calculate patients' scores.	24 months
Secondary	Health care resource use: Annualized number of hospitalizations and emergency room visits during pegcetacoplan treatment until end of study compared to the 12-month period before pegcetacoplan treatment.	Number of hospitalizations and emergency room visits	12 months
Secondary	Patient treatment satisfaction every 6 months until end of study	Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	6 months
Secondary	Patient treatment satisfaction every 6 months until end of study	Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	12 months
Secondary	Patient treatment satisfaction every 6 months until end of study	Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	18 months
Secondary	Patient treatment satisfaction every 6 months until end of study	Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	24 months
Secondary	Physician treatment satisfaction every 6 months until end of study	Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	6 months
Secondary	Physician treatment satisfaction every 6 months until end of study	Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	12 months
Secondary	Physician treatment satisfaction every 6 months until end of study	Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	18 months
Secondary	Physician treatment satisfaction every 6 months until end of study	Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	24 months