Coagadex for Factor X Deficiency
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a treatment called Coagadex, a clotting factor replacement therapy, for individuals with a specific bleeding disorder linked to AL amyloidosis. The trial aims to determine if Coagadex can effectively treat active bleeds and manage bleeding during surgeries. Participants are divided into two groups: one for those requiring treatment during bleeding events and another for those needing surgery. Individuals diagnosed with this bleeding disorder and who have experienced bleeding issues might be suitable candidates. As a Phase 3 trial, this is the final step before FDA approval, providing patients an opportunity to contribute to the potential availability of a new treatment.
Will I have to stop taking my current medications?
The trial requires that you stop taking any anticoagulant therapy (blood thinners) and any factor X containing products at least 2 weeks before starting Coagadex. If you're on other medications, the protocol doesn't specify, so it's best to discuss with the trial team.
Is there any evidence suggesting that Coagadex is likely to be safe for humans?
Research has shown that Coagadex is generally well-tolerated by people with hereditary Factor X deficiency. In earlier studies, some participants experienced mild side effects, such as headaches and injection site reactions, including redness or swelling. Serious allergic reactions are rare but have been reported. Coagadex is not recommended for individuals who have previously had severe allergic reactions to it.
The FDA has already approved Coagadex for treating hereditary Factor X deficiency, which helps reassure its safety. Since this trial is in a later phase, earlier research has already found the treatment to be relatively safe. It is important to consult a doctor to determine if this treatment is appropriate.12345Why do researchers think this study treatment might be promising for factor X deficiency?
Coagadex is unique because it specifically targets Factor X deficiency, a rare bleeding disorder, by replacing the missing or deficient clotting factor. Unlike other treatments that may use general clotting agents, Coagadex is derived from human plasma and is designed to precisely address the deficiency. This targeted approach can result in more effective management of bleeding episodes, potentially reducing the frequency and severity of bleeds. Researchers are excited about Coagadex because it offers a tailored treatment option that could improve the quality of life for individuals with Factor X deficiency.
What evidence suggests that Coagadex might be an effective treatment for factor X deficiency?
Research shows that Coagadex effectively treats factor X deficiency, a condition causing bleeding problems. Previous studies found that Coagadex stopped bleeding episodes 98% of the time, with minimal side effects. In this trial, participants in the On-Demand Treatment Cohort will use Coagadex to treat bleeding as it occurs and have experienced good results. Meanwhile, those in the Surgery/Invasive Procedures Cohort will receive Coagadex to reduce bleeding risk during surgeries and other medical procedures. Overall, Coagadex has demonstrated strong results in managing bleeding for people with factor X deficiency.36789
Who Is on the Research Team?
Mirella Calcinai, MD
Principal Investigator
Medical Director, Clinical Development
Are You a Good Fit for This Trial?
This trial is for individuals with acquired factor X deficiency (FXD) linked to AL amyloidosis, having blood Factor X levels ≤ 50 IU/dL. Women who can bear children and men able to father must agree to use effective contraception during the study.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Run-In Period
Participants without an active bleeding event will enter the Run-In Period, which lasts up to 180 days
On-Demand Treatment
Participants receive Coagadex® for active bleeding events, with dosing based on incremental recovery
Surgery/Invasive Procedures
Participants undergo pharmacokinetic sampling and receive Coagadex® based on PK profile for surgical interventions
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Coagadex
Trial Overview
The study tests Coagadex(R), focusing on its effectiveness and safety in treating active bleeding episodes and managing bleeding during surgery in patients with FXD associated with AL amyloidosis.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Participants enrolled at least 3 weeks prior to a scheduled surgical intervention will undergo pharmacokinetic sampling. The dose of Coagadex® will be determined based on the patient's PK profile. (During this 3-week period, if the participant develops an acute bleed, they will cross over into the On-Demand Treatment Cohort for treatment.)
Participants without an active bleeding event will enter the Run-In Period, which lasts up to 180 days. Upon a bleeding event participants will receive a first dose of 40 IU/kg of Coagadex®. Additional dose(s) will be based on the incremental recovery (IR). (If an allowed Emergent Surgery becomes necessary in the Run-In Period, the participant will cross over into the Surgery/Invasive Procedures Cohort.)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Kedrion S.p.A.
Lead Sponsor
Worldwide Clinical Trials
Collaborator
Citations
Plasma‐derived factor X concentrate compassionate use for ...
This analysis supports the use of pdFX as a safe, effective treatment for hereditary FXD. Routine prophylaxis with pdFX may reduce bleed frequency.
Registry Study of COAGADEX® Patients With Moderate or ...
This is a non-interventional, multicenter, post-marketing registry study in three patients with moderate or severe hereditary FX deficiency, to assess Coagadex ...
Clinical Results (On-demand Treatment)
In clinical studies, COAGADEX was proven effective for on-demand treatment. See benefits & risks.
Efficacy, safety and pharmacokinetics of a new high‐purity ...
Treatment success rate was 98%; only 6 adverse events in 2 subjects were possibly pdFX related. •. On‐demand pdFX 25 IU kg−1 was effective and safe in women/ ...
Factor X Concentrate Treatment Schedule and Dosing ... - PMC
Here, we demonstrate the effective use of FX concentrate to control bleeding symptoms and effectively facilitate invasive procedures.
Package Insert - COAGADEX
Sixteen subjects aged 12 to 58 years with moderate to severe hereditary Factor X deficiency. (basal FX:C < 5 IU/dL) received doses of COAGADEX for ...
Factor X Deficiency Diagnosis and Lab Results
COAGADEX is contraindicated in patients who have had life-threatening hypersensitivity reactions to COAGADEX. Important Safety Information for COAGADEX.
COAGADEX Prescribing Information
COAGADEX is contraindicated in patients who have had life-threatening hypersensitivity reactions to COAGADEX. Important Safety Information for COAGADEX.
Plasma‐derived factor X concentrate compassionate use ...
Plasma‐derived factor X concentrate compassionate use for hereditary factor X deficiency: Long‐term safety and efficacy in a retrospective data‐collection study.
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