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Pegcetacoplan Early Access Program for PNH

Primary Purpose

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Status
No longer available
Phase
Locations
United States
Study Type
Expanded Access
Intervention
Pegcetacoplan
Sponsored by
Apellis Pharmaceuticals, Inc.
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Paroxysmal Nocturnal Hemoglobinuria (PNH)

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All Sexes

Inclusion Criteria:

  • Patients with a confirmed diagnosis of PNH, who are transfusion dependent and are not responding to C5 treatment

Exclusion Criteria:

  • N/A

Sites / Locations

  • Cancer Specialists of North Florida
  • Tenneessee Oncology-Nashville
  • Seattle Children's Hospital
  • MultiCare Deaconess Cancer and Blood Specialty Center - North Spokane

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
January 25, 2021
Last Updated
June 2, 2021
Sponsor
Apellis Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04729062
Brief Title
Pegcetacoplan Early Access Program for PNH
Official Title
Early Access Program (EAP) For The Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) With Pegcetacoplan
Study Type
Expanded Access

2. Study Status

Record Verification Date
June 2021
Overall Recruitment Status
No longer available
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Apellis Pharmaceuticals, Inc.

4. Oversight

5. Study Description

Brief Summary
This is an Early Access Program (EAP) which will provide access to pegcetacoplan for eligible participants with paroxysmal nocturnal hemoglobinuria (PNH).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Paroxysmal Nocturnal Hemoglobinuria (PNH)

7. Study Design

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Pegcetacoplan
Intervention Description
Complement (C3) Inhibitor

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Eligibility Criteria
Inclusion Criteria: Patients with a confirmed diagnosis of PNH, who are transfusion dependent and are not responding to C5 treatment Exclusion Criteria: N/A
Facility Information:
Facility Name
Cancer Specialists of North Florida
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32256
Country
United States
Facility Name
Tenneessee Oncology-Nashville
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37203
Country
United States
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
MultiCare Deaconess Cancer and Blood Specialty Center - North Spokane
City
Spokane
State/Province
Washington
ZIP/Postal Code
99204
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Pegcetacoplan Early Access Program for PNH

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