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Coagulation Factor Replacement Therapy
aPCC-Emicizumab Safety for Hemophilia A (SAFE Trial)
Phase 3
Recruiting
Led By Robert Sidonio, MD
Research Sponsored by Emory University
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Age ≥6 years of age at time of informed consent
Adequate hematologic function (Hgb >8 g/dL and platelet count >100,000 µL)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 2 years
Awards & highlights
SAFE Trial Summary
This trial will look at the safety and effectiveness of a blood clotting medication for people with hemophilia A who are also taking a different medication to prevent bleeding.
Who is the study for?
The SAFE Study is for children and adults over 6 years old with severe hemophilia A and inhibitors, who have not responded to standard treatments. Participants must have good blood, liver, and kidney function but cannot join if they have other bleeding disorders, high risk of clotting or bleeding conditions, HIV with low CD4 counts, a history of thromboembolic disease or significant reactions to monoclonal antibody therapies.Check my eligibility
What is being tested?
This study tests the safety and effectiveness of personalized doses of aPCC in combination with emicizumab during acute bleeding events or before procedures in those on emicizumab prophylaxis. It aims to understand how well this treatment works for people with hemophilia A who also have inhibitors.See study design
What are the potential side effects?
Potential side effects may include allergic reactions related to monoclonal antibodies or components in the injection. There's also a concern about increased risks of clotting (thrombosis) due to the nature of these medications.
SAFE Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am 6 years old or older.
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My blood counts are within the required range.
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My liver tests are within the required range.
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My kidney function is within the required range.
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My hemophilia A is moderately severe with very low FVIII levels.
SAFE Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 2 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 2 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Number of episodes of thrombotic events including thrombotic microangiopathy (TMA)
Number of serious adverse events
Number of serious bleeding episodes
Secondary outcome measures
Number of infusions of aPCC required to achieve hemostatic efficacy for treatment of an acute bleeding episode, or prevention of bleeding with emergent and non-emergent procedures
SAFE Trial Design
1Treatment groups
Experimental Treatment
Group I: Experimental treatmentExperimental Treatment3 Interventions
Personalized dose of aPCC-emicizumab will be administered to participants. The max dose allowed for aPCC will be 25 U/kg/dose every 8 hours, for no more than 72 hours without further discussion with the PI. If there is less than a "good' response in bleed event response efficacy as stated above at 48 hours or less than "moderate" for surgical event control, the local PI can consider the use of thrombin generation guided rFVIIa with max dose no more than 90 µg/kg/dose every 8 hours for 72 hours, with wean to occur for no more than 7 total days without further discussion with the PI.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
FEIBA
2016
Completed Phase 3
~60
rFVIIa
2005
Completed Phase 4
~230
Find a Location
Who is running the clinical trial?
Emory UniversityLead Sponsor
1,638 Previous Clinical Trials
2,560,512 Total Patients Enrolled
7 Trials studying Hemophilia A
166 Patients Enrolled for Hemophilia A
Takeda Pharmaceuticals North America, Inc.Industry Sponsor
57 Previous Clinical Trials
8,943 Total Patients Enrolled
Robert Sidonio, MDPrincipal InvestigatorEmory University
2 Previous Clinical Trials
108 Total Patients Enrolled
1 Trials studying Hemophilia A
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have had two tests showing high inhibitor levels in the past 2 years.I am not using and do not plan to use immune system medications during the study.I am 6 years old or older.I am HIV positive with a CD4 count below 200, or I am under 35 years old.My blood counts are within the required range.My liver tests are within the required range.My kidney function is within the required range.My hemophilia A is moderately severe with very low FVIII levels.I have a condition that could increase my risk of bleeding or blood clots.I have a bleeding disorder that is not hemophilia A.I am considering undergoing surgery within the next 2 years.You have had a serious allergic reaction in the past to other medications like emicizumab.I have been treated for or currently have signs of blood clots, excluding those from IV lines that resolved.
Research Study Groups:
This trial has the following groups:- Group 1: Experimental treatment
Awards:
This trial has 2 awards, including:- 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.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What is the most severe outcome that could result from aPCC-emicizumab?
"aPCC-emicizumab received a safety score of 3 because it is a phase 3 trial, which suggests that not only is there some evidence of its efficacy, but also that it has undergone multiple rounds of testing for safety."
Answered by AI
What is aPCC-emicizumab used to address most frequently?
"aPCC-emicizumab is not only used as an intervention for hemophilia b, but also for other conditions such as factor vii deficiency, hemophilia a, and factor ix inhibition."
Answered by AI
Can you give some examples of research that has been done on aPCC-emicizumab in the past?
"Currently, there are 19 ongoing clinical trials assessing the efficacy of aPCC-emicizumab. Out of these, 8 are in Phase 3. Notably, aPCC-emicizumab clinical trials are being conducted in Dallas, Texas and other parts of the world."
Answered by AI
Does this type of clinical trial have any precedence?
"86 cities across 24 countries are participating in 19 different clinical trials for aPCC-emicizumab. The very first study was completed in 2018 and it reached Phase 3 approval. Since then, there have been 65 more trials."
Answered by AI
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