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Emicizumab PUPs and Nuwiq ITI Study

NCT04030052 · Status: WITHDRAWN · Phase: PHASE3 · Type: INTERVENTIONAL

Last updated 2024-01-05

No results posted yet for this study

Summary

This study prospectively investigates the safety, FVIII immunogenicity, and hemostatic efficacy of prophylactic HEMLIBRA® given with a concomitant low dose recombinant factor VIII (rFVIII) known as NUWIQ®, in HA infants and children \<3 years old who have had little to no previous exposure to FVIII. In addition, the study investigates the safety and efficacy of a novel FVIII ITI regimen in children \<21 with existing low and high titer inhibitors (LTI and HTI).

Conditions

Interventions

DRUG

Nuwiq (low dose protocol)

After receiving HEMLIBRA® for 1-6 months, rFVIII (NUWIQ®) will be given at low dose (25 ±5 units/kg/dose) every 7-14 days as part of a low dose factor exposure program and for on demand use for acute bleeding episodes/procedures. NUWIQ® will be administered intravenously (IV) via peripheral infusion. If the infant has a central line such as a PICC line or mediport this can be used.

DRUG

HEMLIBRA

Four weekly subcutaneous (SQ) injections of HEMLIBRA® loading doses of 3 mg/kg will be given. A total of 12 mg/kg within the first month is allowed for the loading doses. Maintenance dosing will follow, and will either be 1.5 mg/kg/dose weekly, 3 mg/kg/dose biweekly (every 2 weeks), or 6 mg/kg/dose every 4 weeks depending on the recommended dosing.

DRUG

Nuwiq (Atlanta protocol)

After completing HEMLIBRA® loading doses, participants will receive intravenous (IV) infusions of NUWIQ® 3 times per week, 100 units/kg the Atlanta protocol. Infusions will be given at least 36 hours from the previous NUWIQ® injection. Participants will continue on the HEMLIBRA® SQ - NUWIQ® IV treatment regimen for up to 12 months of NUWIQ® treatment.

Sponsors & Collaborators

  • Genentech, Inc.

    collaborator INDUSTRY

  • Emory University

    lead OTHER

Principal Investigators

  • Robert Sidonio, MD · Emory University

Study Design

Allocation
NON_RANDOMIZED
Purpose
PREVENTION
Masking
NONE
Model
PARALLEL

Eligibility

Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-02-17
Primary Completion
2023-01-19
Completion
2023-01-19
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04030052 on ClinicalTrials.gov