Coagadex to treat acquired Factor X deficiency in AL amyloidosis

A Phase III Open-Label Multi-Center Study of the Pharmacokinetics, Safety, and Efficacy of Coagadex in the Treatment of Adults With Acquired Factor X Deficiency Associated With Light Chain Amyloidosis

This study will test whether Coagadex can stop active bleeds and prevent bleeding during surgery in adults with acquired Factor X deficiency from AL amyloidosis.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 15 (estimated)
• Ages: 18 Years to 70 Years
• Sex: All
• Sponsor: Kedrion S.p.A. (Industry-sponsored)
• Locations: 3 sites (Phoenix, Arizona and 2 other locations)
• Trial ID: NCT06963216 on ClinicalTrials.gov

What this trial studies

This phase 3 interventional study gives a plasma-derived Factor X concentrate (Coagadex) to adults with acquired Factor X deficiency (aFXD) associated with AL amyloidosis to treat active bleeding and manage peri-operative bleeding. Participants receive Coagadex when they have an active bleed or around the time of procedures to restore Factor X levels and help clot formation. The trial is conducted at multiple specialist centers in the United States and the United Kingdom and follows participants for safety and bleeding outcomes. Because aFXD in AL amyloidosis is very rare, the study focuses on carefully documenting responses and any adverse events in this specific population.

Who should consider this trial

Good fit: Adults aged 18–70 with acquired Factor X deficiency associated with AL amyloidosis and Factor X activity ≤50 IU/dL who agree to use effective contraception if of reproductive potential are the intended participants.

Not a fit: People who are pregnant or breastfeeding, have Factor X inhibitors, are on anticoagulant therapy, have uncontrolled hypertension or recent significant thrombosis, or recently received other Factor X products are unlikely to be eligible or to benefit from participation.

Why it matters

Potential benefit: If successful, Coagadex could reduce bleeding episodes and make surgeries and procedures safer for people with aFXD due to AL amyloidosis.

How similar studies have performed: Concentrated Factor X products have been used successfully in congenital Factor X deficiency and in case reports of acquired deficiency, but randomized or large-scale data in AL amyloidosis are limited, so this phase 3 effort is building higher-quality evidence.

Eligibility criteria

Inclusion Criteria:

1. Male or female participants ages 18-70 years old.
2. Diagnosis of aFXD associated with AL amyloidosis with FX:C ≤ 50 IU/dL.
3. Woman of Childbearing Potential (defined in Appendix 2) and men of reproductive potential who agree to use an effective contraception measure during the study.

Exclusion Criteria:

1. Female participants that are pregnant or lactating.
2. Presence of FX inhibitors.
3. Uncontrolled arterial hypertension.
4. On anticoagulant therapy (not antiplatelets), unstable heart disease, clinically significant thrombotic event in the past 180 days or any other condition that the Investigator believes is likely to interfere with evaluation of the study drug or with satisfactory conduct of the trial.
5. Any factor X containing product taken within 2 weeks of first Coagadex® administration.
6. Participation in another interventional clinical study with an investigational drug, biologic, or device within 30 days prior to screening.
7. Hemodynamically unstable due to any cause or even after blood transfusion, fluid resuscitation and pharmacologic or volume replacement pressor therapy.
8. Prior history of bleeding disorder other than aFXD.

Where this trial is running

Phoenix, Arizona and 2 other locations

• Mayo Clinic Arizona — Phoenix, Arizona, United States (Recruiting)
• Mayo Clinic Rochester — Rochester, Minnesota, United States (Recruiting)
• Cancer Clinical Trials Unit — London, London, United Kingdom (Recruiting)

Study contacts

• Study coordinator: Nicola Rovai

Conditions Acquired Factor X Deficiency; Factor X Deficiency; Blood Coagulation Disorders; Hematologic Diseases; Hemorrhagic Disorders; Light Chain amyloidosis; AL amyloidosis