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HEMLIBRA for Hemophilia A

Mindy_L_Simpson@rush.edu, Indianapolis, IN
Hemophilia AHEMLIBRA - Drug
Eligibility
< 65
All Sexes

Study Summary

This trial looks at the safety and efficacy of a new drug, HEMLIBRA®, given to infants and children with hemophilia A.

Treatment Effectiveness

Phase-Based Effectiveness

2 of 3
Phase 3
This is further along than 85% of similar trials

Similar Trials

1
Factor IX
1
AAV2/8-HLP-FVIII-V3
1
L-citrulline

Study Objectives

6 Primary · 11 Secondary · Reporting Duration: Weekly for 4 weeks, monthly for 5 months, and every 3 months until study end (up to 36 months)

12 months follow up
Number of target joint bleeding events over time (≥3 bleeds in the same joint over the last 24 weeks)
Baseline, 36 months
Change in Adapted Inhib-QoL scale score
Change in CATCH scale score
Month 36
Annualized bleeding rate (ABR)
Cumulative incidence of inhibitors to FVIII
Microbiota composition of stool in infants with vs. without inhibitors
Number of Immune Tolerance Induction (ITI) failure cases
Number of Immune Tolerance Induction (ITI) partial failure cases
Number of Immune Tolerance Induction (ITI) partial response cases
Number of Immune Tolerance Induction (ITI) partial success cases
Number of Immune Tolerance Induction (ITI) success cases
Number of adverse events
Hemorrhage
Number of infusions of rFVIII or rFVIIa for treatment of an acute bleeding episode
Month 36
Change in blood levels of emicizumab (HEMLIBRA®) in young children (1 month to 24 months of age)
Month 36
Change in blood levels of anti-Emicizumab antibodies
Change in blood levels of anti-FVIII antibodies

Trial Safety

Phase-Based Safety

3 of 3
This is further along than 85% of similar trials

Similar Trials

1
Factor IX
1
AAV2/8-HLP-FVIII-V3
2
L-citrulline

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
No Placebo Group
All patients enrolled in this trial will receive the new treatment.
Approved for 5 Other Conditions
This treatment demonstrated efficacy for 5 other conditions.
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Trial Design

2 Treatment Groups

Untreated/minimally treated moderate HA no inhibitors
1 of 2
Treated any moderate HA with existing inhibitors
1 of 2

Experimental Treatment

60 Total Participants · 2 Treatment Groups

Primary Treatment: HEMLIBRA · No Placebo Group · Phase 3

Untreated/minimally treated moderate HA no inhibitorsExperimental Group · 2 Interventions: HEMLIBRA, Nuwiq (low dose protocol) · Intervention Types: Drug, Drug
Treated any moderate HA with existing inhibitorsExperimental Group · 2 Interventions: HEMLIBRA, Nuwiq (Atlanta protocol) · Intervention Types: Drug, Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: weekly for 4 weeks, monthly for 5 months, and every 3 months until study end (up to 36 months)

Who is running the clinical trial?

Genentech, Inc.Industry Sponsor
1,513 Previous Clinical Trials
568,216 Total Patients Enrolled
14 Trials studying Hemophilia A
830 Patients Enrolled for Hemophilia A
Emory UniversityLead Sponsor
1,559 Previous Clinical Trials
2,765,711 Total Patients Enrolled
7 Trials studying Hemophilia A
186 Patients Enrolled for Hemophilia A
Robert Sidonio, MDPrincipal InvestigatorEmory University
2 Previous Clinical Trials
150 Total Patients Enrolled
1 Trials studying Hemophilia A
20 Patients Enrolled for Hemophilia A

Eligibility Criteria

Age < 65 · All Participants · 10 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have received two or fewer treatments of certain blood products such as pdFVIII, rFVIII, FFP, Cryoprecipitate, or PRBCs.
Your kidneys are working well enough (based on certain tests) to participate in the study.
You must have a test to confirm that there is no inhibitor in your blood within four weeks of joining the study.
You have not developed antibodies against FVIII since you were born. You may also participate in the ATHN 8 study.
Requirements to be eligible for Part B of the study.
Your liver is working well. Your bilirubin levels and AST/ALT levels are within a certain range.
You have moderately severe hemophilia A, which means you have very low levels of clotting factor VIII (less than or equal to 2%).
You are under 3 years old when you give your permission to participate.
The person taking care of you (parent or legal guardian) has given written permission for you to participate in the study.
You have enough healthy blood cells (hemoglobin level above 8 g/dL and platelet count above 100,000 µL).
References

Frequently Asked Questions

Could you walk me through the other scientific research that has been conducted on HEMLIBRA?

"There are 20 ongoing clinical trials for HEMLIBRA, with 8 of them in Phase 3. The primary location for these studies is Nimes, Maryland; however, there are a total of 452 locations running HEMLIBRA trials." - Anonymous Online Contributor

Unverified Answer

At how many separate venues is this research project being undertaken?

"Currently, this study is being conducted out of Rush University Medical Center in Chicago, Illinois; Verisiti, WI in Milwaukee, Wisconsin; and University of North Carolina - Hemophilia and Thrombosis Center in Chapel Hill, North Carolina. There are also 8 other locations where this trial is taking place." - Anonymous Online Contributor

Unverified Answer

Are there any more room in this clinical trial for new patients?

"The study is currently looking for participants, as stated on clinicaltrials.gov. This particular clinical trial was first posted on February 17th, 2022 and was edited more recently on the 21st of the same month." - Anonymous Online Contributor

Unverified Answer

HEMLIBRA has been approved for what condition(s)?

"HEMLIBRA is indicated for the treatment of parenteral drug administration. It may also be used off-label to treat conditions like bleeding and hemophilia A." - Anonymous Online Contributor

Unverified Answer

How many human subjects are participating in this research?

"In order to proceed with the trial, a total of 60 eligible patients are needed. The clinical research will be conducted by Genentech, Inc. at various sites; two examples include Rush University Medical Center (Chicago, Illinois) and Verisiti (Milwaukee, Wisconsin)." - Anonymous Online Contributor

Unverified Answer

Could you please tell me the risks associated with HEMLIBRA?

"HEMLIBRA's safety has been well studied in prior Phase 3 clinical trials, so it received a score of 3." - Anonymous Online Contributor

Unverified Answer

What is the significance of this research?

"The primary outcome that will be measured over the course of this 36 month follow-up interval is the cumulative incidence of FVIII inhibitors. However, other important data points that will be collected include change in blood levels of emicizumab (HEMLIBRA®), anti-emicizumab antibodies, and adapted Inhib-QoL scale score." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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