Amicar for Craniosynostosis Surgery

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Children's National Research Institute

Must not be taking: Anticoagulants

Disqualifiers: Coagulation disorder, Thrombotic event, Renal issues, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Intraoperative period

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether Amicar (ε-aminocaproic acid) can reduce blood loss in children undergoing surgery for craniosynostosis, a condition where a baby's skull bones fuse too early. Researchers believe Amicar might decrease blood loss during surgery and lessen the need for blood transfusions. The trial compares children receiving Amicar with those receiving a placebo, an inactive substance. Children diagnosed with craniosynostosis and scheduled for craniofacial surgery may be suitable candidates for this trial. As a Phase 2 trial, this research measures how well Amicar works in an initial, smaller group of children.

Will I have to stop taking my current medications?

If you are currently taking anticoagulant medications like Coumadin, heparin, or aspirin, you will not be eligible to participate in this trial. The protocol does not specify about other medications, so it's best to discuss your current medications with the trial team.

Is there any evidence suggesting that Amicar is likely to be safe for humans?

Research has shown that Amicar (ε-aminocaproic acid) is likely safe for children. Although specific data on its use in craniosynostosis surgery is limited, existing studies have shown promising results. One study found that Amicar reduced blood loss and the need for blood transfusions during surgery, which is encouraging.

Amicar is generally well-tolerated, with no major safety concerns reported in the reviewed studies. The research suggests it can be a helpful and safe option for managing blood loss in surgeries such as craniofacial reconstruction. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for craniosynostosis surgery, which often involve blood transfusions and antifibrinolytic agents like tranexamic acid, Amicar (ε-aminocaproic acid) offers a unique approach. It works by inhibiting enzymes that break down blood clots, potentially reducing bleeding during surgery. Researchers are excited because Amicar's mechanism could lead to fewer complications with blood loss and transfusions, making surgery safer for patients.

What evidence suggests that Amicar is effective in reducing blood loss during craniofacial surgery?

Research shows that Amicar (a medication) can help reduce blood loss during surgery. Studies have found that it lowers the need for blood transfusions in children undergoing surgery to correct facial and skull bone issues. This is important because losing less blood and needing fewer transfusions make surgery safer and recovery faster. Although few studies focus specifically on surgeries for craniosynostosis (a condition affecting skull shape), existing research is encouraging. In this trial, participants will receive either Amicar or a placebo (normal saline) to evaluate its effectiveness. Amicar is also more affordable than other treatments like tranexamic acid. ^{1 2 3 4 6}

Who Is on the Research Team?

Srijaya K Reddy, MD, MBA

Principal Investigator

Vanderbilt University Medical Center

Are You a Good Fit for This Trial?

This trial is for children aged 2-36 months diagnosed with craniosynostosis requiring complex craniofacial reconstruction surgery. They must have parental consent to participate and no history of coagulation disorders, abnormal renal function, or prior thrombotic events. Children on anticoagulant therapy or with known hypersensitivity to Amicar are excluded.

Inclusion Criteria

My child has been diagnosed with craniosynostosis and needs surgery.

Written informed consent from parent/guardian

I am a child aged between 2 and 36 months.

Exclusion Criteria

I have blood in my urine.

I have a history of kidney abnormalities.

Subjects with a prior history of a thrombotic event

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Amicar or placebo during craniofacial reconstructive surgery to assess its efficacy in reducing blood loss

Intraoperative period

Immediate Postoperative Monitoring

Participants are monitored for blood loss and perioperative complications within the first 72 hours after surgery

72 hours

Follow-up

Participants are monitored for safety and effectiveness, including blood donor exposure, within the first 30 days after surgery

30 days

What Are the Treatments Tested in This Trial?

Interventions

• Amicar (ε-aminocaproic acid)

Trial Overview The study tests if Amicar reduces blood loss during and after craniofacial surgery in children compared to a saline solution. The hypothesis suggests that patients receiving Amicar will need fewer blood transfusions perioperatively.

How Is the Trial Designed?

Group I: Amicar (ε-aminocaproic acid) Experimental Treatment 1 Intervention

Treatment group 1: Amicar 100 mg/kg (0.4 mL/kg) IV loading dose, followed by an intraoperative continuous infusion at 40 mg/kg/hr (0.16 mL/kg/hr) to be continued until skin closure.

Group II: normal saline Placebo Group 1 Intervention

Treatment group 2: Equal volume of normal saline (placebo control) at the same rate as treatment group 1.

Who Is Running the Clinical Trial?

Children's National Research Institute

Lead Sponsor

Trials

227

Recruited

258,000+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT02229968

Efficacy of Amicar for Children Having Craniofacial Surgery The purpose of this study is to determine whether Amicar (ε-aminocaproic acid) is effective in reducing blood loss in children undergoing craniofacial ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9209391/

Epsilon Aminocaproic Acid's Safety and Efficacy in Pediatric ... This study compiles the literature assessing the safety and efficacy of EACA in reducing blood loss and transfusion volumes in children

3. clinicaltrials.gov clinicaltrials.gov/study/NCT02229968?term=AMINOCAPROIC%20ACID&viewType=Table&rank=2

Efficacy of Amicar for Children Having Craniofacial Surgery The purpose of this study is to determine whether Amicar (ε- aminocaproic acid ) is effective in reducing blood loss in children undergoing craniofacial ...

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/35747005/

Epsilon Aminocaproic Acid's Safety and Efficacy in ... There are not many studies of EACA in craniosynostosis repair, but the existing literature shows promising results for EACA's efficacy and ...

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0952818016309114

Blood loss and transfusion requirements with epsilon ... Amicar is significantly less expensive than more frequently studied tranexamic acid. •. Amicar use has been described in cranial vault reconstruction surgery. •.

6. researchgate.net researchgate.net/publication/360773221_Epsilon_Aminocaproic_Acid's_Safety_and_Efficacy_in_Pediatric_Surgeries_Including_Craniosynostosis_Repair_A_Review_of_the_Literature

(PDF) Epsilon Aminocaproic Acid's Safety and Efficacy in ... Compared with the control cohort, craniosynostosis OS utilizing TXA demonstrated significantly lower intraoperative RBC transfusion volumes ( ...