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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05776472




Registration number
NCT05776472
Ethics application status
Date submitted
20/02/2023
Date registered
20/03/2023

Titles & IDs
Public title
A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Scientific title
A Single Arm, Long-term, Multicentre Observational Study to Evaluate Effectiveness of Pegcetacoplan Under Real World Conditions in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Secondary ID [1] 0 0
Sobi.PEGCET-304
Universal Trial Number (UTN)
Trial acronym
COMPLETE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Paroxysmal Nocturnal Hemoglobinuria 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Treatment: Drugs - Pegcetacoplan

Treatment: Drugs: Pegcetacoplan
Pegcetacoplan will be prescribed according to the label in patients with PNH.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in observed hemoglobin level from initiation of treatment with pegcetacoplan to 6 months
Timepoint [1] 0 0
6 months
Secondary outcome [1] 0 0
Change of Lactate Dehydrogenase values from initiation of pegcetacoplan treatment to 6 months
Timepoint [1] 0 0
6 months
Secondary outcome [2] 0 0
Change in Absolute Reticulocyte Count (ARC) from initiation of pegcetacoplan treatment to 6 months
Timepoint [2] 0 0
6 months
Secondary outcome [3] 0 0
Change in indirect/ total bilirubin from initiation of pegcetacoplan treatment to 6 months
Timepoint [3] 0 0
6 months
Secondary outcome [4] 0 0
Change in Haptoglobin from initiation of pegcetacoplan treatment to 6 months
Timepoint [4] 0 0
6 months
Secondary outcome [5] 0 0
Change in Ferritin from initiation of pegcetacoplan treatment to 6 months
Timepoint [5] 0 0
6 months
Secondary outcome [6] 0 0
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [6] 0 0
6 months
Secondary outcome [7] 0 0
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [7] 0 0
12 months
Secondary outcome [8] 0 0
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [8] 0 0
18 months
Secondary outcome [9] 0 0
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [9] 0 0
24 months
Secondary outcome [10] 0 0
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [10] 0 0
6 months
Secondary outcome [11] 0 0
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [11] 0 0
12 months
Secondary outcome [12] 0 0
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [12] 0 0
18 months
Secondary outcome [13] 0 0
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [13] 0 0
24 months
Secondary outcome [14] 0 0
Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [14] 0 0
6 months
Secondary outcome [15] 0 0
Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [15] 0 0
12 months
Secondary outcome [16] 0 0
Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [16] 0 0
18 months
Secondary outcome [17] 0 0
Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [17] 0 0
24 months
Secondary outcome [18] 0 0
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [18] 0 0
6 months
Secondary outcome [19] 0 0
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [19] 0 0
12 months
Secondary outcome [20] 0 0
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [20] 0 0
18 months
Secondary outcome [21] 0 0
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [21] 0 0
24 months
Secondary outcome [22] 0 0
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [22] 0 0
6 months
Secondary outcome [23] 0 0
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [23] 0 0
12 months
Secondary outcome [24] 0 0
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [24] 0 0
18 months
Secondary outcome [25] 0 0
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [25] 0 0
24 months
Secondary outcome [26] 0 0
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [26] 0 0
6 months
Secondary outcome [27] 0 0
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [27] 0 0
12 months
Secondary outcome [28] 0 0
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [28] 0 0
18 months
Secondary outcome [29] 0 0
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [29] 0 0
24 months
Secondary outcome [30] 0 0
Hemoglobin = 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [30] 0 0
6 months
Secondary outcome [31] 0 0
Hemoglobin = 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [31] 0 0
12 months
Secondary outcome [32] 0 0
Hemoglobin = 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [32] 0 0
18 months
Secondary outcome [33] 0 0
Hemoglobin = 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [33] 0 0
24 months
Secondary outcome [34] 0 0
Increase in hemoglobin levels of = 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [34] 0 0
6 months
Secondary outcome [35] 0 0
Increase in hemoglobin levels of = 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [35] 0 0
12 months
Secondary outcome [36] 0 0
Increase in hemoglobin levels of = 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [36] 0 0
18 months
Secondary outcome [37] 0 0
Increase in hemoglobin levels of = 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [37] 0 0
24 months
Secondary outcome [38] 0 0
Acute hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [38] 0 0
6 months
Secondary outcome [39] 0 0
Acute hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [39] 0 0
12 months
Secondary outcome [40] 0 0
Acute hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [40] 0 0
18 months
Secondary outcome [41] 0 0
Acute hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of study
Timepoint [41] 0 0
24 months
Secondary outcome [42] 0 0
Annualized number of red blood cell (RBC) transfusions during pegcetacoplan treatment until end of study compared to the 12 month period before pegcetacoplan treatment
Timepoint [42] 0 0
12 months
Secondary outcome [43] 0 0
Annualized number of red blood cell (RBC) units during pegcetacoplan treatment until end of study compared to the 12 month period before pegcetacoplan treatment
Timepoint [43] 0 0
12 months
Secondary outcome [44] 0 0
Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of study
Timepoint [44] 0 0
6 months
Secondary outcome [45] 0 0
Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of study
Timepoint [45] 0 0
12 months
Secondary outcome [46] 0 0
Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of study
Timepoint [46] 0 0
18 months
Secondary outcome [47] 0 0
Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of study
Timepoint [47] 0 0
24 months
Secondary outcome [48] 0 0
Quality of life at initiation of treatment with pegcetacoplan and every 6 months until end of study
Timepoint [48] 0 0
6 months
Secondary outcome [49] 0 0
Quality of life at initiation of treatment with pegcetacoplan and every 6 months until end of study
Timepoint [49] 0 0
12 months
Secondary outcome [50] 0 0
Quality of life at initiation of treatment with pegcetacoplan and every 6 months until end of study
Timepoint [50] 0 0
18 months
Secondary outcome [51] 0 0
Quality of life at initiation of treatment with pegcetacoplan and every 6 months until end of study
Timepoint [51] 0 0
24 months
Secondary outcome [52] 0 0
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.
Timepoint [52] 0 0
12 months
Secondary outcome [53] 0 0
Patient treatment satisfaction every 6 months until end of study
Timepoint [53] 0 0
6 months
Secondary outcome [54] 0 0
Patient treatment satisfaction every 6 months until end of study
Timepoint [54] 0 0
12 months
Secondary outcome [55] 0 0
Patient treatment satisfaction every 6 months until end of study
Timepoint [55] 0 0
18 months
Secondary outcome [56] 0 0
Patient treatment satisfaction every 6 months until end of study
Timepoint [56] 0 0
24 months
Secondary outcome [57] 0 0
Physician treatment satisfaction every 6 months until end of study
Timepoint [57] 0 0
6 months
Secondary outcome [58] 0 0
Physician treatment satisfaction every 6 months until end of study
Timepoint [58] 0 0
12 months
Secondary outcome [59] 0 0
Physician treatment satisfaction every 6 months until end of study
Timepoint [59] 0 0
18 months
Secondary outcome [60] 0 0
Physician treatment satisfaction every 6 months until end of study
Timepoint [60] 0 0
24 months

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 design
Purpose
Duration
Selection
Timing
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
26/06/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
15/08/2029
Actual
Sample size
Target
200
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD
Recruitment hospital [1] 0 0
Liverpool Hospital - Liverpool
Recruitment hospital [2] 0 0
Calvary Mater Newcastle - Waratah
Recruitment hospital [3] 0 0
Sunshine Coast University Hospital - Birtinya
Recruitment postcode(s) [1] 0 0
2170 - Liverpool
Recruitment postcode(s) [2] 0 0
2298 - Waratah
Recruitment postcode(s) [3] 0 0
4575 - Birtinya
Recruitment outside Australia
Country [1] 0 0
Belgium
State/province [1] 0 0
Brugge
Country [2] 0 0
Belgium
State/province [2] 0 0
Bruxelles
Country [3] 0 0
Belgium
State/province [3] 0 0
Liège
Country [4] 0 0
Belgium
State/province [4] 0 0
Turnhout
Country [5] 0 0
Canada
State/province [5] 0 0
Ontario
Country [6] 0 0
Canada
State/province [6] 0 0
Quebec
Country [7] 0 0
Czechia
State/province [7] 0 0
Brno
Country [8] 0 0
Czechia
State/province [8] 0 0
Praha 2
Country [9] 0 0
Finland
State/province [9] 0 0
Helsinki
Country [10] 0 0
France
State/province [10] 0 0
Alpes Maritimes
Country [11] 0 0
France
State/province [11] 0 0
Gironde
Country [12] 0 0
France
State/province [12] 0 0
Hauts De Seine
Country [13] 0 0
France
State/province [13] 0 0
Isere
Country [14] 0 0
France
State/province [14] 0 0
Loire Atlantique
Country [15] 0 0
France
State/province [15] 0 0
Meurthe Et Moselle
Country [16] 0 0
France
State/province [16] 0 0
Nord
Country [17] 0 0
France
State/province [17] 0 0
Paris
Country [18] 0 0
France
State/province [18] 0 0
Rhone
Country [19] 0 0
Germany
State/province [19] 0 0
Baden Wuerttemberg
Country [20] 0 0
Germany
State/province [20] 0 0
Bayern
Country [21] 0 0
Germany
State/province [21] 0 0
Hessen
Country [22] 0 0
Germany
State/province [22] 0 0
Nordrhein Westfalen
Country [23] 0 0
Germany
State/province [23] 0 0
Sachsen
Country [24] 0 0
Germany
State/province [24] 0 0
Thueringen
Country [25] 0 0
Spain
State/province [25] 0 0
Lugo

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Swedish Orphan Biovitrum
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Stefan Lethagen, MD PhD Prof.
Address 0 0
Swedish Orphan Biovitrum
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Michael O'Malley
Address 0 0
Country 0 0
Phone 0 0
+41797977276
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05776472