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Pegcetacoplan Long Term Safety and Efficacy Extension Study

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03531255
Recruitment Status : Active, not recruiting
First Posted : May 21, 2018
Last Update Posted : July 26, 2021
Information provided by (Responsible Party):
Apellis Pharmaceuticals, Inc.

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

Condition or disease Intervention/treatment Phase
PNH Drug: Pegcetacoplan Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 160 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : August 27, 2018
Estimated Primary Completion Date : August 20, 2022
Estimated Study Completion Date : August 20, 2022

Arm Intervention/treatment
Experimental: 1,080 mg pegcetacoplan administered subcutaneously
1,080mg pegcetacoplan administered subcutaneously twice weekly or every three days.
Drug: Pegcetacoplan
Complement (C3) Inhibitor

Primary Outcome Measures :
  1. Incidence and severity of treatment-emergent adverse events [ Time Frame: Baseline to 2 Years ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03531255

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Sponsors and Collaborators
Apellis Pharmaceuticals, Inc.
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Responsible Party: Apellis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03531255    
Other Study ID Numbers: APL2-307
First Posted: May 21, 2018    Key Record Dates
Last Update Posted: July 26, 2021
Last Verified: July 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No