Clinical trial NCT03531255: Pegcetacoplan Long Term Safety and Efficacy Extension Study
Official title: An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: PNH
Lead sponsor: Apellis Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2018-05-21
Enrollment: 160
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is an Open-label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed a pegcetacoplan study.


Eligibility criteria: Inclusion criteria:

1. Subjects at least 18 years of age with PNH who have participated in a pegcetacoplan clinical trial. Subjects who received treatment with pegcetacoplan must have experienced clinical benefit and adequate tolerability in the opinion of the investigator.

   Note: Subjects with PNH who completed a pegcetacoplan clinical trial without receiving pegcetacoplan (or without receiving pegcetacoplan for long enough to demonstrate clinical benefit) may be enrolled in this study if, in the opinion of the Investigator, the subject is expected to demonstrate clinical benefit upon the initiation or continuation of pegcetacoplan therapy.
2. Vaccination against Neisseria meningitidis types A, C, W, Y and B, Streptococcus pneumoniae and Haemophilus influenzae Type B (Hib) either within 2 years prior to Day 1 dosing of this study, or within 14 days after starting treatment with pegcetacoplan. Vaccination is mandatory unless documented evidence exists that subjects are nonresponders to vaccination as evidenced by titers or display titer levels within acceptable local limits. Immunization status checks will be performed to determine whether subjects require primary or booster vaccinations.
3. Willing and able to give written informed consent.
4. Willing and able to self-administer pegcetacoplan (administration by caregiver will be allowed)
5. Women of childbearing potential (WOCBP) defined as any females who have experienced menarche and who are NOT permanently sterile or postmenopausal must have a negative pregnancy test and must agree to continue to use an approved method of contraception for the duration of the study and 90 days after their last dose of study drug. Note: Postmenopausal is defined as 12 consecutive months with no menses without an alternative medical cause.
6. Males must agree to continue to use an approved method of contraception and must agree to refrain from donating sperm for the duration of the study and 90 days after their last dose of study drug.

Exclusion Criteria:

1. Subjects who have withdrawn from a pegcetacoplan clinical study.
2. Any condition that could increase the subject's risk by participating in the study.
3. Any comorbidity or condition (such as malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data.
4. History or presence of hypersensitivity or idiosyncratic reaction to compounds related to the investigational product or SC administration.
5. Known infection with hepatitis B, C, or HIV.
6. Hereditary complement deficiency.
7. History of bone marrow transplant.
8. Concurrent severe aplastic anemia (SAA), defined as currently receiving immunosuppressive therapy for SAA including but not limited to cyclosporin A, tacrolimus, mycophenolate mofetil or anti-thymocyte globulin.
9. History of meningococcal disease.
10. Concomitant treatment with any complement inhibitor (eg, eculizumab, ravulizumab).
11. Pregnancy, breastfeeding, or positive pregnancy test.

Primary outcomes:
measure: Incidence and severity of treatment-emergent adverse events
timeFrame: Baseline to 2 Years
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: University of Southern California
state: California
city: Denver
country: United States
facility: Denver Health Medical Center
state: Colorado
city: Jacksonville
country: United States
facility: Cancer Specialists of North Florida
state: Florida
city: Miami
country: United States
facility: Lakes Research
state: Florida
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Indianapolis
country: United States
facility: Investigative Clinical Research of Indiana
state: Indiana
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Memphis
country: United States
facility: Baptist Cancer Center
state: Tennessee
city: Melbourne
country: Australia
facility: Royal Melbourne Hospital
state: Victoria
city: Brussels
country: Belgium
facility: Cliniques Universitaires Saint-luc
city: Roeselare
country: Belgium
facility: AZ Delta
city: Sofia
country: Bulgaria
facility: Acibadem City Clinic Tokuda Hospital
city: Sofia
country: Bulgaria
facility: National Specialized Hospital for Active Treatment of Haematologic Diseases
city: Calgary
country: Canada
facility: University of Calgary
state: Alberta
city: Edmonton
country: Canada
facility: Alberta Health Services
state: Alberta
city: Toronto
country: Canada
facility: Toronto General Hospital
state: Ontario
city: Cali
country: Colombia
facility: IPS Centro Medico Julian Coronel SAS
state: Valle Del Cauca
city: Chalon-sur-Saône
country: France
facility: Hospital Center Chalon Sur Saone William Morey
city: Lille
country: France
facility: CHRU de Lille
city: Paris
country: France
facility: Hopital Saint-Louis
city: Pierre-Bénite
country: France
facility: Lyon Sud Hospital Center
city: Pringy
country: France
facility: Centre Hospitalier Annecy Genevois - Site Annecy
city: Saint-Quentin
country: France
facility: Centre Hospitalier de Saint-Quentin
city: Ulm
country: Germany
facility: Universitatsklinikum Ulm
state: BW
city: Aachen
country: Germany
facility: Uniklinik RWTH Aachen
state: NRW
city: Essen
country: Germany
facility: Essen University Hospital Department of Hematology
state: NRW
city: Hamburg
country: Germany
facility: University Medical Center Hamburg-Eppendorf
city: Hong Kong
country: Hong Kong
facility: Prince of Wales Hospital
city: Hong Kong
country: Hong Kong
facility: Queen Mary Hospital
city: Showa-ku
country: Japan
facility: Japanese Red Cross Nagoya Daini Hospital
state: Aichi
city: Matsumoto
country: Japan
facility: Shinshu University Hospital
state: Nagano
city: Bunkyō-Ku
country: Japan
facility: Juntendo University Hospital
state: Tokyo
city: Tanabe
country: Japan
facility: Kinan Hospital
state: Wakayama
city: Okayama
country: Japan
facility: Okayama University Hospital
city: Tokyo
country: Japan
facility: NTT Medical Center Tokyo
city: Junggu
country: Korea, Republic of
facility: Chungnam National University Hospital
state: Daejeon
city: Lembah Pantai
country: Malaysia
facility: University Malaya Medical Centre
state: Kuala Lumpur
city: Ampang
country: Malaysia
facility: Hospital Ampang
state: Selangor
city: Monterrey
country: Mexico
facility: Hospital Universitario "José Eleuterio González", UANL
city: Jesús María
country: Peru
facility: Hospital Edgardo Rebagliatti
state: Lima
city: Lima Cercado
country: Peru
facility: Hospital Nacional Dos De Mayo - Centro de Investigación
state: Lima
city: San Isidro
country: Peru
facility: Centro de Investigacion en Hematologia Clinica del Centro Medico Corpac
state: Lima
city: Cebu
country: Philippines
facility: Perpetual Succour Hospital
state: Metro Manila
city: Lipa City
country: Philippines
facility: Mary Mediatrix Medical Centre
state: Metro Manila
city: Makati City
country: Philippines
facility: Makati Medical Centre
state: Metro Manila
city: Pasig City
country: Philippines
facility: The Medical City
state: Metro Manila
city: Quezon City
country: Philippines
facility: St. Lukes Medical Centre
state: Metro Manila
city: Saint Petersburg
country: Russian Federation
facility: Pavlov First Saint Petersburg State Medical University
city: Tyumen
country: Russian Federation
facility: Therapeutic hospital of regional clinical hospital #1
city: Belgrade
country: Serbia
facility: Clinical Center of Serbia
city: Singapore
country: Singapore
facility: Singapore General Hospital
city: Las Palmas De Gran Canaria
country: Spain
facility: Hospital Univ. de Gran Canaria Dr.Negrin
state: Islas Canarias
city: Valencia
country: Spain
facility: Hospital Universitario Politecnico La Fe
city: Bangkok
country: Thailand
facility: Phramongkutklao Hospital
city: Bangkok
country: Thailand
facility: Ramathibodi Hospital
city: Bangkok
country: Thailand
facility: Siriraj Hospital
city: Chiang Mai
country: Thailand
facility: Maharaj Nakorn Chiang Mai Hospital
city: Khon Kaen
country: Thailand
facility: Srinagarind Hospital
city: Pathum Thani
country: Thailand
facility: Thammasat University Hospital
city: Songkhla
country: Thailand
facility: Songklanagarind Hospital
city: Leeds
country: United Kingdom
facility: Leeds Teaching Hospitals NHS Trust
Study officials:
References: