Acquired Factor X DeficiencyPhase 3NCT06963216
A Study Investigating Coagadex in the Treatment AFXD Associated With AL Amyloidosis
A Phase 3 study testing Coagadex(R) in people with Acquired Factor X Deficiency, run by Kedrion S.p.A..
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Acquired Factor X Deficiency
- What is being tested?
- Coagadex(R)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Jun 2026to 10 Jun 2026
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
About this study
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
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.
Read the 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
- Conditions
- Acquired Factor X Deficiency
- Being tested
- Coagadex(R)
- Keywords
- Factor X Deficiency, Blood Coagulation Disorders, Hematologic Diseases, Hemorrhagic Disorders, Light Chain amyloidosis, AL amyloidosis
- Medical subject headings
- Factor X Deficiency, Blood Coagulation Disorders, Hematologic Diseases, Hemorrhagic Disorders, Immunoglobulin Light-chain Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Coagadex(R)Biological
Coagulation Factor X (Human)
| Study group | Role | What they receive |
|---|---|---|
| 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.) Receives: 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.) Receives: Biological: Coagadex(R) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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
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®.
Measured: 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.
Measured: 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®.
Measured: Day 4 to Day 14
Coagadex® exposure
Assess the number of study drug exposure days (overall and per participant) and the Coagadex® exposure per event.
Measured: Day 1 to Day 30
Assess Post-treatment FX Levels
Assess in the on-demand treatment cohort and surgery/invasive procedures cohort by FX:C IU/mL
Measured: Day 1 to Day 30
Assess bleeds at other sites.
Description: Assessed in the surgery/invasive procedures cohort by looking at: Number of sites per participant including severity, duration, location, and cause.
Measured: Last Coagadex® until study completion, an average of up to 6 months.
Assess the pharmacokinetics(PK) of Coagadex® in peri-operative/peri-procedural hemostasis in Surgery/Invasive Procedures Cohort.
To assess the pharmacokinetics(PK) of Coagadex® in peri-operative/peri-procedural hemostasis by PK parameter Maximum Plasma Concentration [Cmax]
Measured: Day 0 pre-dose to Day 5
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 May 2025
- Study startJun 2026 (estimated)
- Primary completion10 Jun 2026
- Study completion10 Jun 2026
Why it stopped: Study was discontinued for non-safety reasons and the rapidly evolving treatment landscape for AL amyloidosis.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acquired Factor X DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kedrion S.p.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mirella Calcinai, MD, Study director, Medical Director, Clinical Development
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06963216
- Sponsor's protocol ID
- KB074
- Other IDs
- IRAS UK # 1011593
- Status last verified
- Nov 2026
- First submitted
- 14 Apr 2025
- Last updated
- 5 Nov 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Publishing of data and IPD that underlie results in the publication will be determined at study completion to comply with ICMJE minimum requirements.
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06963216
- Acronym / sponsor ID
- KB074
- Conditions
- Acquired Factor X Deficiency
- Keywords
- Factor X Deficiency, Blood Coagulation Disorders, Hematologic Diseases, Hemorrhagic Disorders, Light Chain amyloidosis, AL amyloidosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study was discontinued for non-safety reasons and the rapidly evolving treatment landscape for AL amyloidosis.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Kedrion S.p.A.Industry (pharma, biotech, devices)
- Study start
- Jun 2026 (estimated)
- Primary completion
- 10 Jun 2026 (actual)
- Study completion
- 10 Jun 2026 (actual)
- First posted
- 8 May 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| On-Demand Treatment Cohort | Experimental | Biological: Coagadex(R)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.) |
| Surgery/Invasive Procedures Cohort | Experimental | Biological: Coagadex(R)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.) |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Coagadex(R)Coagulation Factor X (Human) | Biological | On-Demand Treatment Cohort, Surgery/Invasive Procedures Cohort |
Primary outcome
Proportion of bleeding events responsive to Coagdex® therapy at 72 hours after initiation of treatment in the On-Demand Treatment Cohort
Time frame: at 72 hours
Proportion of bleeding events with a 'positive' response.
Proportion of bleeding events responsive to Coagdex® therapy at 72 hours after initiation of treatment in the Surgery/Invasive Procedures Cohort
Time frame: at 72 hours
Proportion of bleeding events with a 'fair, good, or excellent' response.
7 secondary outcomes
Proportion of bleeding events responsive to Coagdex® therapy at Different Timepoints After 72 hours in the On- Demand Cohort
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
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
Time frame: Day 4 to Day 14
Coagadex® exposure
Time frame: Day 1 to Day 30
Assess Post-treatment FX Levels
Time frame: Day 1 to Day 30
Assess bleeds at other sites.
Time frame: Last Coagadex® until study completion, an average of up to 6 months.
Assess the pharmacokinetics(PK) of Coagadex® in peri-operative/peri-procedural hemostasis in Surgery/Invasive Procedures Cohort.
Time frame: Day 0 pre-dose to Day 5
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2026.
- Individual participant dataSharing planned: No. Publishing of data and IPD that underlie results in the publication will be determined at study completion to comply with ICMJE minimum requirements.
Related trials for Acquired Factor X Deficiency
RecruitingObservational study
Longitudinal Cohort of Thrombosis and Hemostasis Diseases
Immune Thrombocytopenia, Thrombotic Thrombocytopenic Purpura
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mirella Calcinai, MD study director
Medical Director, Clinical Development
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Coagadex(R) in people with Acquired Factor X Deficiency, run by Kedrion S.p.A..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 5 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of bleeding events responsive to Coagdex® therapy at 72 hours after initiation of treatment in the On-Demand Treatment Cohort (time frame: at 72 hours). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Nov 2026.