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Coagulation Factor

Novel Replacement Therapies for Hemophilia A

Q: What diseases can be managed with Extended half-life factor VIII-based replacement therapy?

Perioperative blood loss can be managed with Extended half-life factor VIII-based replacement therapy, which is also beneficial for treating hemophilia A related hemorrhages and bleeding episodes that occur even in the presence of inhibitors.

Q: Is this trial a pioneering effort in its field?

At present, 34 countries across 102 cities are running 14 ongoing experiments related to Extended half-life factor VIII-based replacement therapy. The initial trial for this therapy was conducted in 2015 by Baxalta (now Shire), and it involved 120 participants who completed Phase 3 of the drug approval stage. Since then, 82 more trials have been concluded.

Q: How many participants are being recruited for this research initiative?

Affirmative. The statistics found on clinicaltrials.gov illustrate that this trial, which was originally posted April 15th 2021, is currently enrolling patients. 28 subjects must be recruited from a single medical centre.

Q: Has the FDA provided their stamp of approval for extended half-life factor VIII-based replacement therapy?

Through Phase 4 trials, the safety of Extended half-life factor VIII-based replacement therapy has been approved and it thus scored a 3.

Q: Is it possible for individuals to still join this clinical exploration?

The information on clinicaltrials.gov illustrates that this trial is still seeking participants, after being first posted in April 2021 and most recently updated in April 2022.

Q: Have there been prior investigations of the efficacy of Extended half-life factor VIII based substitution therapy?

Presently, 14 Extended half-life factor VIII-based replacement therapy trials are ongoing with 8 in the third phase. Dallas, <a href="https://www.withpower.com/clinical-trials/texas">Texas</a> houses the majority of these experiments; however, a total of 335 sites across America offer this type of treatment.

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

Timeline

Screening 3 weeks

Treatment Varies

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

Awards & highlights

Study 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.Check my eligibility

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.See study design

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

Select...

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

Select...

I am under 18 years old.

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Changes in early joint-health markers

Changes in hemostatic potential

Secondary outcome measures

Hemorrhage

Changes in health-related quality of life and physical activity

Joints

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 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Jessica GarciaLead Sponsor

University of Texas Southwestern Medical CenterLead Sponsor

1,047 Previous Clinical Trials

1,053,771 Total Patients Enrolled

1 Trials studying Hemophilia A

11 Patients Enrolled for Hemophilia A

Jessica Garcia, MD4.8119 ReviewsPrincipal Investigator - University of Texas Southwestern Medical Center

CHRISTUS Santa Rosa Hospital - Medical Center

Medical School - University of Texas, Southwestern, Doctor of Medicine

5Patient Review

He was very personable and took the time to listen to my needs. He provided referrals to other specialists that I needed.

Media Library

Adynovate (Coagulation Factor) Clinical Trial Eligibility Overview. Trial Name: NCT04690322 — Phase 4

Hemophilia A Research Study Groups: Extended half-life factor VIII-based replacement therapy, Non-Factor VIII-based replacement therapy

Hemophilia A Clinical Trial 2023: Adynovate Highlights & Side Effects. Trial Name: NCT04690322 — Phase 4

Adynovate (Coagulation Factor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04690322 — Phase 4

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What diseases can be managed with Extended half-life factor VIII-based replacement therapy?

"Perioperative blood loss can be managed with Extended half-life factor VIII-based replacement therapy, which is also beneficial for treating hemophilia A related hemorrhages and bleeding episodes that occur even in the presence of inhibitors."

Answered by AI

Is this trial a pioneering effort in its field?

"At present, 34 countries across 102 cities are running 14 ongoing experiments related to Extended half-life factor VIII-based replacement therapy. The initial trial for this therapy was conducted in 2015 by Baxalta (now Shire), and it involved 120 participants who completed Phase 3 of the drug approval stage. Since then, 82 more trials have been concluded."

Answered by AI

How many participants are being recruited for this research initiative?

"Affirmative. The statistics found on clinicaltrials.gov illustrate that this trial, which was originally posted April 15th 2021, is currently enrolling patients. 28 subjects must be recruited from a single medical centre."

Answered by AI

Has the FDA provided their stamp of approval for extended half-life factor VIII-based replacement therapy?

"Through Phase 4 trials, the safety of Extended half-life factor VIII-based replacement therapy has been approved and it thus scored a 3."

Answered by AI

Is it possible for individuals to still join this clinical exploration?

"The information on clinicaltrials.gov illustrates that this trial is still seeking participants, after being first posted in April 2021 and most recently updated in April 2022."

Answered by AI

Have there been prior investigations of the efficacy of Extended half-life factor VIII based substitution therapy?

"Presently, 14 Extended half-life factor VIII-based replacement therapy trials are ongoing with 8 in the third phase. Dallas, Texas houses the majority of these experiments; however, a total of 335 sites across America offer this type of treatment."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies

Jessica Garcia 2021. "POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04690322.

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