Clinical trial NCT06963216: A Study Investigating Coagadex in the Treatment AFXD Associated With AL Amyloidosis
Official title: 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
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE3
Conditions: Acquired Factor X Deficiency
Keywords: Factor X Deficiency
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhagic Disorders
Light Chain amyloidosis
AL amyloidosis
Lead sponsor: Kedrion S.p.A.
Sponsor class: INDUSTRY
Collaborators: name: Worldwide Clinical Trials
First posted: 2025-05-08
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the efficacy and safety of Coagadex in treating active bleeds and to manage peri-operative bleeding in participants with acquired factor X deficiency associated with AL amyloidosis.

Detailed description: This study is designed to evaluate the efficacy and safety of Coagadex® in treating active bleeds and to manage peri-operative bleeding in participants with aFXD associated with AL amyloidosis. Coagadex® is a plasma-derived human coagulation factor concentrate expected to treat bleeding episodes in patients with aFXD associated with AL amyloidosis by delivering concentrated Factor X to address the clotting deficiency caused by the disease.

aFXD is a rare condition (prevalence of \<1 / 1 000 000 \[Orphanet 2024\]) and may occur due to infections, neoplastic and chronic diseases (Lee G et al., 2012). aFXD is considered severe when the FX coagulant activity (FX:C) level is \< 25% of normal (normal range 70 - 150 IU/dL), and moderate when FX:C level is 25% to 50% of normal (Choufani et al., 2001).

AL amyloidosis is the most common cause of acquired FX Deficiency (aFXD). In AL amyloidosis, amyloid fibrils bind to and reduce the levels of circulating FX and shorten its half-life, causing aFXD, leading to an increase in risk of bleeding. Bleeding may occur both in patients with moderate and severe deficiency

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.

Primary outcomes:
measure: Proportion of bleeding events responsive to Coagdex® therapy at 72 hours after initiation of treatment in the On-Demand Treatment Cohort
timeFrame: at 72 hours
measure: Proportion of bleeding events responsive to Coagdex® therapy at 72 hours after initiation of treatment in the Surgery/Invasive Procedures Cohort
timeFrame: at 72 hours
Secondary outcomes:
measure: Proportion of bleeding events responsive to Coagdex® therapy at Different Timepoints After 72 hours in the On- Demand Cohort
timeFrame: Day 4 to day 30
measure: To assess the safety of Coagadex® by number and percentage of infusion related AEs, SAEs, coded by MedDRA
timeFrame: Day 0 through study completion, an average of up to 6 months.
measure: Proportion of bleeding events responsive to Coagdex® therapy at Different Timepoints After 72 hours in Surgery/Invasive Procedures Cohort
timeFrame: Day 4 to Day 14
measure: Coagadex® exposure
timeFrame: Day 1 to Day 30
measure: Assess Post-treatment FX Levels
timeFrame: Day 1 to Day 30
measure: Assess bleeds at other sites.
timeFrame: Last Coagadex® until study completion, an average of up to 6 months.
measure: Assess the pharmacokinetics(PK) of Coagadex® in peri-operative/peri-procedural hemostasis in Surgery/Invasive Procedures Cohort.
timeFrame: Day 0 pre-dose to Day 5
Other outcomes:
Locations:
Study officials:
affiliation: Medical Director, Clinical Development
name: Mirella Calcinai, MD
role: STUDY_DIRECTOR
References: