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Coagulation Factor

Novel Replacement Therapies for Hemophilia A

Phase 4

Recruiting

Led By Jessica Garcia, MD

Research Sponsored by Jessica Garcia

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subjects with moderate hemophilia A (baseline factor VIII activity 1-5%) or severe hemophilia A (baseline factor VIII activity <1%) on prophylactic standard half-life FVIII infusions OR subjects with moderate or severe hemophilia A who have not started prophylactic treatment

Less than 18 years of age

Must not have

Subjects with a history of ≥ 2 target joints

Currently using medications known to impact bone and mineral metabolism (e.g., bisphosphonates, corticosteroids, estrogen, testosterone, calcitonin, thyroid hormone therapy);

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, 1 month, 6 months, and 12 months from start of study

Awards & highlights

Drug Has Already Been Approved

No Placebo-Only Group

Pivotal Trial

Summary

This trial will compare two standard treatments for Hemophilia A to see which is better.

Who is the study for?

This trial is for young individuals under 18 with moderate to severe Hemophilia A, either on standard treatments or not yet treated. It's not for those with FVIII inhibitors, a history of joint surgery (synovectomy), more than one target joint, or conditions affecting bone health.

What is being tested?

The study compares two types of Hemophilia A treatments: extended half-life factor VIII replacement therapy and non-FVIII based therapy. Patients are randomly chosen to receive one of these standard care options.

What are the potential side effects?

Possible side effects from the therapies include allergic reactions, development of inhibitors (antibodies against the treatment making it less effective), vein inflammation due to infusion, and potential long-term effects on joints and bones.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have moderate or severe hemophilia A and may or may not be on standard treatment.

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I am under 18 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had issues with two or more specific joints.

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I am taking medication that affects bone health.

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I have had surgery to remove a synovial membrane.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, 1 month, 6 months, and 12 months from start of study

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, 1 month, 6 months, and 12 months from start of study

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, 1 month, 6 months, and 12 months from start of study for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Changes in early joint-health markers

Changes in hemostatic potential

Secondary study objectives

Hemorrhage

Changes in health-related quality of life and physical activity

Joints

Awards & Highlights

Drug Has Already Been Approved

The FDA has already approved this drug, and is just seeking more data.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Active Control

Group I: Extended half-life factor VIII-based replacement therapyActive Control2 Interventions

Subjects who are either already on prophylactic standard half-life FVIII products or have not started prophylactic treatment will be randomized to start prophylactic extended half-life FVIII products or non-factor product (emicizumab). Both therapies are considered the current standard of care. The study has 4 planned visits at baseline, 1 month, 6 months, and 12 months. They will coincide with the standard of care visits.

Group II: Non-Factor VIII-based replacement therapyActive Control1 Intervention

0 / 5Eligibility criteria met