A Study Investigating Coagadex in the Treatment AFXD Associated With AL Amyloidosis

Phase 3RecruitingNCT06963216

Kedrion S.p.A.15 enrolled

Overview

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.

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

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Acquired Factor X Deficiency

Interventions

Coagadex(R)BIOLOGICAL

Coagulation Factor X (Human)

Eligibility

Sex: ALLMin age: 18 YearsMax age: 70 Years

Medical Language ↔ Plain English

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.

Locations (2)

Mayo Clinic Arizona

Phoenix, Arizona, United States

RECRUITING

Mayo Clinic Rochester

Rochester, Minnesota, United States

RECRUITING

Outcomes

Primary Outcomes

Proportion of bleeding events responsive to Coagdex® therapy at 72 hours after initiation of treatment in the On-Demand Treatment Cohort

Proportion of bleeding events with a 'positive' response.

Time frame: at 72 hours

Proportion of bleeding events responsive to Coagdex® therapy at 72 hours after initiation of treatment in the Surgery/Invasive Procedures Cohort

Proportion of bleeding events with a 'fair, good, or excellent' response.

Time frame: at 72 hours

Secondary Outcomes

Proportion of bleeding events responsive to Coagdex® therapy at Different Timepoints After 72 hours in the On- Demand Cohort

Investigators will assess Coagadex® efficacy in the on-demand treatment cohort by looking at: -Percentage of responsive bleedings, -The ability to discontinue or reduce the dose and/or dosing frequency of Coagadex®.

Time frame: Day 4 to day 30

To assess the safety of Coagadex® by number and percentage of infusion related AEs, SAEs, coded by MedDRA

To assess the safety of Coagadex® in the on-demand treatment of bleeding and management of peri-operative/peri-procedural hemostasis in infusion related AEs and SAEs. AEs and SAEs will be evaluated following the first Coagadex® administration: assessment of seriousness, causality assessment; and incidence, frequency, and severity of AEs and incidence and frequency of SAEs.

Time frame: Day 0 through study completion, an average of up to 6 months.

Proportion of bleeding events responsive to Coagdex® therapy at Different Timepoints After 72 hours in Surgery/Invasive Procedures Cohort

Investigators will assess Coagadex® efficacy in the surgery/invasive procedures cohort by looking at: * The overall percentage of bleeding episodes successfully controlled, * The ability to discontinue or reduce the dose and/or dosing frequency of Coagadex®.

Central Contacts

Nicola Rovai

CONTACT

+39 335 6524750 n.rovai@kedrion.com

Data from ClinicalTrials.gov

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