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

Kedrion Biopharma

Status and phase: Begins enrollment this month; Phase 3

Conditions: Acquired Factor X Deficiency

Treatments: Biological: Coagadex(R)

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT06963216

KB074

IRAS UK # 1011593 (Other Identifier)

Details and patient eligibility

About

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.

Full 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

Enrollment: 15 estimated patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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15 participants in 2 patient groups

On-Demand Treatment Cohort

Experimental group

Description:

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.)

Treatment:

Biological: Coagadex(R)

Surgery/Invasive Procedures Cohort

Experimental group

Description:

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.)

Treatment:

Biological: Coagadex(R)