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

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

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.

Study Overview

• Status: Recruiting
• Conditions: Paroxysmal Nocturnal Hemoglobinuria
• Intervention / Treatment: Drug: Pegcetacoplan

Detailed 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.

• Study Type: Observational
• Enrollment (Estimated): 200

Contacts and Locations

• Study Contact: Name: Matthias Noelte; Phone Number: +41797977276; Email: matthias.noelte@sobi.com
• Study Contact Backup: Name: Michael O'Malley; Email: michael.omalley@sobi.com

Study Locations

• Spain
  • Lugo, Spain
    • Recruiting
    • Hospital Lucus Augusti
    • Principal Investigator: Esperanza Lavilla, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Adult patients (≥18 years) with PNH, who started routine treatment with pegcetacoplan up to 12 months prior to enrollmentor at enrollment

Description

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.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in observed hemoglobin level from initiation of treatment with pegcetacoplan to 6 months	Hemoglobin level in g/dL.	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change of Lactate Dehydrogenase values from initiation of pegcetacoplan treatment to 6 months	Lactate Dehydrogenase (LDH) in U/L	6 months
Change in Absolute Reticulocyte Count (ARC) from initiation of pegcetacoplan treatment to 6 months	Absolute Reticulocyte Count (ARC) in 10^9/L	6 months
Change in indirect/ total bilirubin from initiation of pegcetacoplan treatment to 6 months	Indirect/ total bilirubin in umol/L	6 months
Change in Haptoglobin from initiation of pegcetacoplan treatment to 6 months	Haptoglobin in mg/dL	6 months
Change in Ferritin from initiation of pegcetacoplan treatment to 6 months	Ferritin in ug/L	6 months
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	6 months
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	12 months
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	18 months
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	24 months
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of study	Lactate Dehydrogenase (LDH) in U/L	6 months
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of study	Lactate Dehydrogenase (LDH) in U/L	12 months
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of study	Lactate Dehydrogenase (LDH) in U/L	18 months
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of study	Lactate Dehydrogenase (LDH) in U/L	24 months
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
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
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
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
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of study	Indirect/ total bilirubin in umol/L	6 months
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of study	Indirect/ total bilirubin in umol/L	12 months
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of study	Indirect/ total bilirubin in umol/L	18 months
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of study	Indirect/ total bilirubin in umol/L	24 months
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study	Haptoglobin in mg/dL	6 months
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study	Haptoglobin in mg/dL	12 months
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study	Haptoglobin in mg/dL	18 months
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study	Haptoglobin in mg/dL	24 months
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of study	Ferritin in ug/L	6 months
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of study	Ferritin in ug/L	12 months
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of study	Ferritin in ug/L	18 months
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of study	Ferritin in ug/L	24 months
Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	6 months
Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	12 months
Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	18 months
Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study	Hemoglobin in g/dL	24 months
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
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
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
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
Acute hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of study	Yes/No	6 months
Acute hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of study	Yes/No	12 months
Acute hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of study	Yes/No	18 months
Acute hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of study	Yes/No	24 months
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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

Collaborators and Investigators

• Sponsor: Swedish Orphan Biovitrum
• Investigators: Study Director: Stefan Lethagen, MD PhD Prof., Swedish Orphan Biovitrum; Principal Investigator: Regis Peffault de Latour, MD PhD Prof., Saint-Louis Hospital, Paris, France

Study record dates

Study Major Dates

• Study Start (Actual): June 26, 2023
• Primary Completion (Estimated): January 3, 2026
• Study Completion (Estimated): July 3, 2027

Study Registration Dates

• First Submitted: February 20, 2023
• First Submitted That Met QC Criteria: March 17, 2023
• First Posted (Actual): March 20, 2023

Study Record Updates

• Last Update Posted (Actual): July 13, 2023
• Last Update Submitted That Met QC Criteria: July 11, 2023
• Last Verified: July 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urologic Diseases; Urological Manifestations; Bone Marrow Diseases; Hematologic Diseases; Urination Disorders; Anemia; Proteinuria; Anemia, Hemolytic; Myelodysplastic Syndromes; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Hemoglobinuria; Hemoglobinuria, Paroxysmal
• Other Study ID Numbers: Sobi.PEGCET-304

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No