The Hemophilia Inhibitor Eradication Trial

Study Results

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-11-01

Study Completion Date

2022-06-27

Brief Summary

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This is a multi-center randomized phase III clinical trial, the Inhibitor Eradication Trial, in which Eloctate ITI plus Emicizumab will be compared with Eloctate ITI alone to eradicate inhibitors in severe hemophilia A.

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Detailed Description

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This is a multi-center randomized phase III clinical trial, the Inhibitor Eradication Trial, in which Eloctate ITI plus Emicizumab will be compared with Eloctate ITI alone to eradicate inhibitors in patients with severe hemophilia A This adaptive design is necessary as randomized trials in rare diseases are often not possible. The INHIBIT Clinical Trials Platform includes two linked trials, the Inhibitor Prevention Trial (Prevention Trial) and the Inhibitor Eradication Trial (Eradication Trial) that will be conducted at up to 41 U.S. hemophilia treatment centers (HTCs) affiliated with universities. The Inhibitor Eradication Trial is a 48-week randomized phase III trial, in which 90 previously treated patients (PTPs) with severe hemophilia A and high-responding inhibitors (anti-VIII \> 0.6 B.U.), will be enrolled. Subjects will include individuals with severe hemophilia A who develop inhibitors during the linked Inhibitor Prevention Trial and adults or children at the same HTCs refractory to or never undergoing immune tolerance induction (ITI). Once enrolled, subjects who meet all the inclusion and none of the exclusion criteria, will be randomized to weekly Eloctate ITI plus weekly Emicizumab vs. weekly Eloctate ITI alone to eradicate inhibitor formation, defined as anti-FVIII\<0.6 B.U. Blood draws will be minimized to 6 timepoints, pre, 4, 12, 24, 36, and 48 weeks, and validated for small volumes, 3.8 cc (¾ tsp) each. The Inhibitor Eradication Trial is considered greater than minimal risk as study drug is given before the first bleed and special inhibitor studies are obtained. (NB: The Inhibitor Eradication Trial (PRO19070080) is linked to the Inhibitor Prevention Trial (PRO19040140), as part of the INHIBIT Clinical Trials Platform, and both trials will be conducted efficiently in the same hemophilia treatment centers (HTCs), with the same MDs, coordinators, visit frequency, blood sampling, and assays.

Conditions

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Hemophilia A With Inhibitor

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

This is a phase III open-label, randomized controlled trial comparing immune tolerance induction with one drug, with or without a second drug in the eradication of hemophilia inhibitors.

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Eloctate ITI plus Emicizumab

Arm A: Eloctate 100 IU/kg every other day by intravenous infusion plus Emicizumab 1.5 mg/kg subcutaneously (following 3 mg/kg/wk x 4 induction) in children and adults with severe hemophilia A and anti-FVIII inhibitor, continued up to 48 weeks.

Group Type EXPERIMENTAL

Eloctate ITI

Intervention Type DRUG

This is a factor VIII-Fc infusion protein.

Emicizumab

Intervention Type DRUG

This is a bispecific monoclonal antibody FVIII mimic.

Eloctate ITI

Arm B: Eloctate 100 IU/kg every other day by intravenous infusion in children and adults with severe hemophilia A and anti-FVIII inhibitor, continued up to 48 weeks.

Group Type ACTIVE_COMPARATOR

Eloctate ITI

Intervention Type DRUG

This is a factor VIII-Fc infusion protein.

Interventions

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Eloctate ITI

This is a factor VIII-Fc infusion protein.

Intervention Type DRUG

Emicizumab

This is a bispecific monoclonal antibody FVIII mimic.

Intervention Type DRUG

Other Intervention Names

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rFVIIIFc Hemlibra

Eligibility Criteria

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Inclusion Criteria

1. Male adults or children \> 4 months of age.
2. Severe hemophilia A (FVIII \< 0.01 U/ml).
3. Current or past high-responding inhibitor, anti-FVIII \>= 5.0 B.U., ITI-refractory or ITI-naive.

Exclusion Criteria

1. Acquired hemophilia or any bleeding disorder other than hemophilia A.
2. Current use of Emicizumab, or if used, \> 8 weeks since last treatment.
3. Use of an experimental drug(s).
4. Surgery anticipated in the next 48 weeks.
5. Life expectancy less than 5 years.
6. Patient/parent/caretaker unable or unwilling to keep a personal diary of bleeding frequency and study drug treatment, make monthly visits and blood draws at weeks 4, 8, 12, 24, 36, and 48.
7. Other illness, condition, or reason in the opinion of the investigator that would make the patient unsuitable for the trial.

Eligible Sex

MALE

Accepts Healthy Volunteers

No