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NCT04901936 · ClinicalTrials.gov registry record · Phase 2

A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

A Phase 2 study of Paroxysmal Nocturnal Hemoglobinuria (PNH) and Paroxysmal Hemoglobinuria, sponsored by Apellis Pharmaceuticals.

Recruiting
Registry status
Phase 2
Development phase
12
Enrollment target
12
Study locations

NCT04901936 is a Phase 2 study of Paroxysmal Nocturnal Hemoglobinuria (PNH) and Paroxysmal Hemoglobinuria that is actively recruiting participants, run by Apellis Pharmaceuticals. The registered enrollment target is 12 participants, below the 123-participant average among 5 other Paroxysmal Nocturnal Hemoglobinuria (PNH) trials with a reported enrollment target (90% lower). The trial reports 12 study locations across 2 states.

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The verdict

NCT04901936, a Phase 2 study of Paroxysmal Nocturnal Hemoglobinuria (PNH) and Paroxysmal Hemoglobinuria, is actively recruiting participants, sponsored by Apellis Pharmaceuticals.

RECRUITING
Registry status
Phase 2
Development phase
12 participants
Enrollment target
12
Study locations

Study Summary

The purpose of this study is to evaluate the safety, effectiveness, and biological activity (how the investigational medication is processed by the body) of pegcetacoplan in 12-17 year-olds (adolescents) who have paroxysmal nocturnal hemoglobinuria (PNH).

Conditions Studied

Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Hemoglobinuria

Interventions

  • DRUG Pegcetacoplan

Study Locations (12)

Other

  • Motol University Hospital - Prague
  • Robert-Debré Hospital Paris - Paris
  • Hospital Ampang - Ampang
  • Radboud University Hospital Nijmegen - Nijmegen
  • University Medical Center Utrecht - Utrecht
  • University Children's Hospital - Belgrade
  • University Hospital Vall d'Hebron - Barcelona
  • University Hospital 12 de Octubre - Madrid
  • Phramongkutklao Hospital and College of Medicine - Bangkok
  • Maharaj Nakorn Chiang Mai hospital - Chiang Mai
  • St. Mary's Hospital - London

Georgia

  • Children's Hospital of Atlanta - Atlanta

Trial Details

FieldValue
Enrollment Target 12 participants
Start Date 2021-02-04
Est. Completion 2028-12
Phase Phase 2

Sponsor

Apellis Pharmaceuticals

21 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT04901936

The ClinicalTrials.gov registry entry for NCT04901936 describes a study currently listed as recruiting, categorized as Phase 2. The registered enrollment target is 12 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 123-participant average among 5 other Paroxysmal Nocturnal Hemoglobinuria (PNH) trials with a reported enrollment target (90% lower). The listed sponsor is Apellis Pharmaceuticals, which has 21 total studies on file at ClinicalTrials.gov.

The record links to 2 conditions, with Paroxysmal Nocturnal Hemoglobinuria (PNH) appearing as the primary indexed condition, and to 1 intervention - of which Pegcetacoplan is the first listed.

NCT04901936 reports 12 study locations spanning 2 distinct geographic areas - top geographies include Other, Georgia.

Frequently Asked Questions

What is clinical trial NCT04901936 about?

NCT04901936 is a clinical study titled "A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)". The purpose of this study is to evaluate the safety, effectiveness, and biological activity (how the investigational medication is processed by the body) of pegcetacoplan in 12-17 year-olds (adolescents) who have paroxysmal nocturnal hemoglobinuria (PNH).

What is the current status of trial NCT04901936?

This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 12 participants. The study started on 2021-02-04. Estimated completion is 2028-12.

What conditions does trial NCT04901936 study?

This clinical trial studies the following conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Hemoglobinuria.

What interventions are being tested in trial NCT04901936?

The interventions under investigation include: Pegcetacoplan (DRUG).

Who is sponsoring clinical trial NCT04901936?

This trial is sponsored by Apellis Pharmaceuticals, which has 21 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT04901936 being conducted?

This trial has 12 study locations across Georgia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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