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Multi-center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)

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ClinicalTrials.gov Identifier: NCT03531255
Recruitment Status : Enrolling by invitation
First Posted : May 21, 2018
Last Update Posted : September 7, 2018
Sponsor:
Information provided by (Responsible Party):
Apellis Pharmaceuticals, Inc.

Brief Summary:
This is an Open Label, Non-Randomized, Multi-Center Extension Study for ongoing and planned Phase IB, Phase II, and Phase III studies with APL-2 in subjects with PNH.

Condition or disease Intervention/treatment Phase
PNH Drug: APL-2 Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 109 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase III, Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)
Actual Study Start Date : August 27, 2018
Estimated Primary Completion Date : December 20, 2020
Estimated Study Completion Date : December 20, 2020


Arm Intervention/treatment
Experimental: APL-2 1,080mg twice weekly
1,080mg APL-2 will be administered subcutaneously twice weekly.
Drug: APL-2
Complement (C3) Inhibitor

Experimental: APL-2 1,080mg every three days
1,080mg APL-2 will be administered subcutaneously every three days.
Drug: APL-2
Complement (C3) Inhibitor

Experimental: APL-2 1,080mg twice weekly or every three days
1,080mg APL-2 will be administered subcutaneously twice weekly or 1,080mg APL-2 will be administered subcutaneously every three days.
Drug: APL-2
Complement (C3) Inhibitor




Primary Outcome Measures :
  1. Incidence and severity of hematologic treatment-emergent adverse events [ Time Frame: 48 Weeks ]

Secondary Outcome Measures :
  1. Mean change from baseline to 48 weeks in hemoglobin levels (g/dL) [ Time Frame: 48 Weeks ]


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
Criteria

Inclusion Criteria:

  1. Subjects with PNH who have participated in an APL-2 clinical trial, and have experienced clinical benefit in the opinion of the Investigator.
  2. Willing and able to give written informed consent.
  3. Women of child-bearing potential (WOCBP) must have a negative pregnancy test and must agree to use protocol defined methods of contraception for the duration of the study and 60 days after their last dose of study drug.
  4. Males must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study and 60 days after their last dose of study drug.

Exclusion Criteria:

  1. Subjects who have withdrawn from an APL-2 clinical study
  2. Any condition that could increase the subject's risk by participating in the study

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


Locations
United States, California
University of Southern California
Los Angeles, California, United States, 90033
United States, Florida
Lakes Research
Miami Lakes, Florida, United States, 33014
United States, Ohio
Cleveland Clinic
Cleveland, Ohio, United States, 44195
Sponsors and Collaborators
Apellis Pharmaceuticals, Inc.

Responsible Party: Apellis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03531255     History of Changes
Other Study ID Numbers: APL2-307
First Posted: May 21, 2018    Key Record Dates
Last Update Posted: September 7, 2018
Last Verified: September 2018

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

Additional relevant MeSH terms:
Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases