Clinical trial NCT04085601: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH
Official title: A Phase 3, Randomized, Multicenter, Open-Label, Controlled Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Lead sponsor: Apellis Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2019-09-11
Enrollment: 53
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Evaluation of the Efficacy and Safety of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria .


Eligibility criteria: Inclusion Criteria:

* Be at least 18 years old (inclusive).
* Have LDH ≥1.5 x ULN at the screening visit.
* Have PNH diagnosis, confirmed by high sensitivity flow cytometry (granulocyte or monocyte clone \>10%).
* Have Hb less than the lower limit of normal (LLN) at the screening visit.
* Have ferritin greater than/equal to the LLN, or total iron binding capacity (TIBC) less than/equal to ULN at the screening visit, based on central laboratory reference ranges. If a subject is receiving iron supplements at screening, the Investigator must ensure that the subject's dose has been stable for 4 weeks prior to screening, and it must be maintained throughout the study. Subjects not receiving iron at screening must not start iron supplementation during the course of the study.
* Body mass index (BMI) ≤ 35 kg/m2 at the screening visit.
* Have a platelet count of \>50,000/mm3 at the screening visit.
* Have an absolute neutrophil count \>500/mm3 at the screening visit.

Exclusion Criteria:

* Treatment with any complement inhibitor (eg, eculizumab) within 3 months prior to screening.
* Hereditary complement deficiency.
* History of bone marrow transplantation.
* Concomitant use of any of the following medications is prohibited if not on a stable regimen for the time period indicated below prior to screening:

  * Erythropoietin or immunosuppressants for at least 8 weeks
  * Systemic corticosteroids for at least 4 weeks
  * Vitamin K antagonists (eg, warfarin) with a stable international normalized ratio (INR) for at least 4 weeks
  * Iron supplements, vitamin B12, or folic acid for at least 4 weeks
  * Low-molecular-weight heparin for at least 4 weeks

Primary outcomes:
measure: Number of Subjects Who Achieved Hemoglobin (Hb) Stabilization
timeFrame: From Baseline (Day 1) up to Week 26
measure: Change From Baseline in Lactate Dehydrogenase (LDH) Concentration At Week 26
timeFrame: Baseline (Day 1) and Week 26
Secondary outcomes:
measure: Number of Subjects With an Hb Response in the Absence of Transfusions
timeFrame: Baseline and Week 26
measure: Change From Baseline in Absolute Reticulocyte Count (ARC) at Week 26
timeFrame: Baseline and Week 26
measure: Change From Baseline in Hb Concentration at Week 26
timeFrame: Baseline and Week 26
measure: Percentage of Subjects Who Received Transfusion or Decrease of Hb >2 g/dL From Baseline
timeFrame: At Week 26
measure: Percentage of Subjects With Transfusion Avoidance
timeFrame: At Week 26
measure: Number of PRBC Units Transfused From Baseline Through Week 26
timeFrame: Up to Week 26
measure: Change From Baseline in Functional Assessment of Chronic Illness Therapy- (FACIT-Fatigue) Scale Score at Week 26
timeFrame: Baseline and Week 26
measure: Percentage of Subjects With Hb Normalization Levels at Week 26
timeFrame: Baseline and Week 26
measure: Percentage of Subjects With LDH Normalization at Week 26
timeFrame: At Week 26
measure: Change From Baseline in European Organization for Research and Treatment of Cancer (EORTC) 30-item Core Quality of Life Questionnaire (QLQ-C30) Scores at Week 26
timeFrame: Baseline and Week 26
measure: Change From Baseline in Linear Analog Assessment (LASA) Scales Score at Week 26
timeFrame: Baseline and Week 26
measure: Percentage of Subjects With ARC Normalization
timeFrame: At Week 26
measure: Number of Subjects With Failure of Hb Stabilization
timeFrame: Up to Week 26
measure: Time to First PRBC Transfusion
timeFrame: Up to Week 26
Other outcomes:
Locations:
city: Cali
country: Colombia
facility: Julian Coronel Medical Center
city: Medellín
country: Colombia
facility: Research Center of the Colombian Clinical Life Cancer Foundation
city: Hong Kong
country: Hong Kong
facility: Queen Mary Hospital
city: Kwai Chung
country: Hong Kong
facility: Princess Margaret Hospital
city: Sha Tin
country: Hong Kong
facility: Prince and Wales Hospital
city: Ampang
country: Malaysia
facility: Hospital Ampang
state: Selangor
city: Kuala Lumpur
country: Malaysia
facility: University Malaya Medical Centre
city: Monterrey
country: Mexico
facility: Hospital Universitario Dr.Jose Eleuterio Gonzalez
city: Jesús María
country: Peru
facility: Hospital Cayetano Heredia
state: Lima
city: San Martin de Porres
country: Peru
facility: Hospital Cayetano Heredia
state: Lima
city: Lima
country: Peru
facility: Hospital Nacional Dos de Mayo
city: Benguet
country: Philippines
facility: Baguio General Hospital
city: Cebu City
country: Philippines
facility: Perpetual Succour Hospital
city: Lipa City
country: Philippines
facility: Mary Mediatrix Medical Center
city: Makati City
country: Philippines
facility: Makati Medical Centre
city: Pasig City
country: Philippines
facility: The Medical City
city: Quezon City
country: Philippines
facility: St. Lukes Medical Centre
city: Lubin
country: Poland
facility: Independent Public Clinical Hospital
city: Warsaw
country: Poland
facility: Institute of Hematology and Transfusiology
city: Wrocław
country: Poland
facility: EMC Medical Institute
city: Belgrade
country: Serbia
facility: Clinical Centre of Serbia
city: Singapore
country: Singapore
facility: Singapore General Hospital (SGH)
city: Bangkok
country: Thailand
facility: Ramathibodi Hospital
city: Bangkok
country: Thailand
facility: Siriraj Hospital
city: Bangkok
country: Thailand
facility: Hospital for Tropical disease
city: Bangkok
country: Thailand
facility: Phramongkutklao Hospital
city: Chiang Mai
country: Thailand
facility: Maharaj Nakorn Chiang Mai Hospital
city: Khon Kaen
country: Thailand
facility: Srinagaring Hospital
city: Pathum Thani
country: Thailand
facility: Thammasat University Hospital
city: Songkhla
country: Thailand
facility: Songklanagaring Hospital
Study officials:
References:
citation: Del Pozo Martin Y. 2021 ASH annual meeting. Lancet Haematol. 2022 Feb;9(2):e92-e93. doi: 10.1016/S2352-3026(21)00384-7. Epub 2021 Dec 16. No abstract available.