Hemophilia Inhibitor Clinical Trials (INHIBIT) Platform

• Status: Withdrawn
• Trial end date: 2027-07-01
• Target enrollment: 0
• Participant gender: Male

Summary

This study will evaluate if Eloctate is superior to Emicizumab in reducing inhibitors in children with severe hemophilia when given before the first bleed (preemptive) and continued weekly to prevent bleeds (prophylaxis); and whether Eloctate immune tolerance induction (ITI) plus emicizumab is superior to Eloctate ITI alone in eradicating inhibitor formation in children and adults with severe hemophilia A.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Margaret Ragni; University of Pittsburgh
• Treatments: Antibodies, Bispecific; Factor VIII; Immunoglobulin Fc Fragments

Criteria

Prevention Trial, Inclusion Criteria:

• Male children >/= 4 months of age.

• Severe hemophilia A (FVIII < 0.01 U/ml)

• No previous bleed or surgery requiring treatment (except circumcision)

• No previous factor VIII product (except for circumcision)

• Willingness to comply with weekly prophylaxis for 48 weeks

• Willingness of parent/caregiver to keep a personal diary of bleeding frequency and factor treatment.

• Willingness to make monthly visits and coagulation testing at weeks 4, 12, 24, 36, and 48 (end of study)

Prevention Trial, Exclusion Criteria:

• Acquired hemophilia.

• Any bleeding disorder other than hemophilia A.

• Treatment with clotting factor previously, other than circumcision.

• Presence of an inhibitor to factor VIII.

• Use of an experimental drug(s).

• Surgery anticipated in the next 48 weeks.

• Life expectancy less than 5 years.

• Inability to comply with study requirements.

Eradication Trial, Inclusion Criteria:

• Male adults or children with no age limitation.

• Severe hemophilia A (FVIII <0.01 U/ml).

• Presence of an inhibitor to FVIII (anti-FVIII > 5.0 B.U.)

• Willingness to comply with study drugs for up to 48 weeks.

• Willingness to keep a personal diary of bleed frequency and drug treatment.

• Willingness to make monthly visits and coagulation testing at weeks 4, 12, 24, 36, and 48 (end of study).

Eradication Trial, Exclusion Criteria:

• Acquired hemophilia.

• Any bleeding disorder other than hemophilia A.

• Current use of Emicizumab, or if used, > 8 weeks since last treatment.

• Use of an experimental drug(s).

• Surgery anticipated in the next 48 weeks.

• Life expectancy less than 5 years.

• Inability to copy with study requirements.