Pegcetacoplan Long Term Safety and Efficacy Extension Study

PNH Clinical Trial

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)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03531255
• Other study ID #: APL2-307
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: August 27, 2018
• Est. completion date: June 2025

Study information

• Verified date: June 2023
• Source: Apellis Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 160
• Est. completion date: June 2025
• Est. primary completion date: June 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• PNH

Intervention

Drug:
• Pegcetacoplan
Complement (C3) Inhibitor

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Apellis Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Australia,  Belgium,  Bulgaria,  Canada,  Colombia,  France,  Germany,  Hong Kong,  Japan,  Korea, Republic of,  Malaysia,  Mexico,  Peru,  Philippines,  Russian Federation,  Serbia,  Singapore,  Spain,  Thailand,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence and severity of treatment-emergent adverse events		Baseline to 2 Years