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Preventing Inhibitor Recurrence Indefinitely

NCT04621916 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 52

Last updated 2023-10-05

No results posted yet for this study

Summary

This study will enroll children who have hemophilia A with inhibitors who successfully completed immune tolerance induction per the ISTH criteria (negative inhibitor titer, recovery \>66% of expected, and half-life of \>6 hours with their current FVIII concentrate). Previous to emicizumab, there was only one option for these patients which was to continue FVIII therapy in a prophylaxis mode to prevent bleeding. There was a sense that the ongoing FVIII served to maintain tolerance however no evidence for this notion exists and in fact what limited data is available suggests that continuing FVIII may not be necessary simply to maintain tolerance.

To figure out this question, this will be a randomized, controlled 2 arm study which will randomize patients post-successful ITI to emicizumab plus weekly FVIII (for maintenance of tolerance) versus emicizumab alone. Patients will be followed for up to 2 years. We aim to enroll 52 subjects. The FVIII weekly arm can use any factor VIII concentrate and emicizumab is standard of care for inhibitor and non-inhibitor patients.

Conditions

Interventions

DRUG

FVIII

This study will evaluate the inhibitor recurrence with or without ongoing FVIII exposure in patients with hemophilia A on emicizumab prophylaxis after a successful immune tolerance induction.

DRUG

Emicizumab

This study will evaluate the inhibitor recurrence with or without ongoing FVIII exposure in patients with hemophilia A on emicizumab prophylaxis after a successful immune tolerance induction.

Sponsors & Collaborators

Principal Investigators

  • Guy Young, MD · Professor of Pediatrics, Director of Hemostasis and Thrombosis Center

Study Design

Allocation
RANDOMIZED
Purpose
PREVENTION
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
0 Years
Max Age
12 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-09-21
Primary Completion
2024-09-20
Completion
2025-04-20
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Diseases
Companies

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 NCT04621916 on ClinicalTrials.gov