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

NCT06963216 | Recruiting Phase 3 FDA Drug

• 2 other identifiers: KB074IRAS UK # 1011593
• Study Type: interventional
• Target: 15
• Locations: 2 countries
• Sites: 3

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.

Conditions

Acquired Factor X Deficiency

Keywords

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

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  at 72 hours

Secondary Outcomes (7)

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

  Day 4 to day 30

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

  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

  Day 4 to Day 14

• Coagadex® exposure

  Day 1 to Day 30

• Assess Post-treatment FX Levels

  Day 1 to Day 30

Study Arms (2)

On-Demand Treatment Cohort

EXPERIMENTAL

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.)

Biological: Coagadex(R)

Surgery/Invasive Procedures Cohort

EXPERIMENTAL

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.)

Biological: Coagadex(R)

Interventions

Coagadex(R) BIOLOGICAL

Coagulation Factor X (Human)

On-Demand Treatment Cohort; Surgery/Invasive Procedures Cohort

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• Female participants that are pregnant or lactating.
• Presence of FX inhibitors.
• Uncontrolled arterial hypertension.
• 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.
• Any factor X containing product taken within 2 weeks of first Coagadex® administration.
• Participation in another interventional clinical study with an investigational drug, biologic, or device within 30 days prior to screening.
• Hemodynamically unstable due to any cause or even after blood transfusion, fluid resuscitation and pharmacologic or volume replacement pressor therapy.
• Prior history of bleeding disorder other than aFXD.

Sponsors & Collaborators

• Kedrion S.p.A.: lead
• Worldwide Clinical Trials: collaborator

Study Sites (3)

Mayo Clinic Arizona

Phoenix, Arizona, 85054, United States

RECRUITING

Mayo Clinic Rochester

Rochester, Minnesota, 55905, United States

RECRUITING

Cancer Clinical Trials Unit

London, London, NW1 2PG, United Kingdom

RECRUITING

MeSH Terms

Conditions

Factor X Deficiency; Blood Coagulation Disorders; Hematologic Diseases; Hemorrhagic Disorders; Immunoglobulin Light-chain Amyloidosis

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Amyloidosis; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Paraproteinemias

Study Officials

• Mirella Calcinai, MD

  Medical Director, Clinical Development

  STUDY DIRECTOR

Central Study Contacts

Nicola Rovai

CONTACT

+39 335 6524750; n.rovai@kedrion.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Masking Details: None (open label)
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 14, 2025
• First Posted: May 8, 2025
• Study Start: February 1, 2026
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): September 1, 2027
• Last Updated: February 27, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (2); GB (1)