A Phase I/IIb extension study assessing the long-term safety and efficacy of a gene therapy (AAV5-hFIX) previously administered to adult patients with severe or moderately severe haemophilia B during the CT-AMT-060-01 Phase I/II study.

A Phase I/IIb extension study assessing the long-term safety and efficacy of an adeno-associated viral vector containing a codon-optimized human factor IX gene (AAV5-hFIX) previously administered to adult patients with severe or moderately severe haemophilia B during the CT-AMT-060-01 Phase I/II study. - Phase I/IIb extension study of AAV5-hFIX in severe or moderately severe haemophilia B

Status

Active, not recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2020-000739-28-DK

Enrollment

Registered

2020-10-05

Start date

2020-11-13

Completion date

Unknown

Last updated

2021-05-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Haemophilia B MedDRA version: 20.0 Level: LLT Classification code 10018939 Term: Haemophilia B (Factor IX) System Organ Class: 100000004850

Interventions

Product Name: AAV5-hFIX Product Code: AMT-060 Pharmaceutical Form: Solution for infusion Current Sponsor code: AMT-060 Other descriptive name: AAV5-hFIX Concentration type: not less then Concentration

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: 1. Patients with congenital hemophilia B who completed Study CT-AMT-060-01 2. Able to provide informed consent following receipt of verbal and written information about the trial. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 9 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 1

Exclusion criteria

Exclusion criteria: Enrolled patients will have already been assessed based on the exclusion criteria for Study CT-AMT-060-01.

Design outcomes

Secondary

Measure	Time frame
Secondary end point(s): The secondary endpoints will focus on the long-term efficacy (6-10 years) after dosing of AAV5-hFIX on FIX activity, overall FIX utilization, bleeding events requiring FIX replacement therapy, any procedures, and QoL. Secondary efficacy endpoints include the following: - Endogenous FIX activity - Utilization of FIX-replacement therapy - Annualized bleeding rate (FIX-requiring); including the following: o All bleeds o Spontaneous bleeds o Traumatic bleeds o Joint bleeds - Procedures (including major and minor surgeries) - Short form (SF-36) and EQ-5D-5L QoL scores - Hemophilia Joint Health Score (HJHS) ;Timepoint(s) of evaluation of this end point: Endogenous FIX activity, Utilization of FIX-replacement therapy, FIX inhibitors and bleeding rates and procedures will be measured every 6 months QoL and HJHS will be measured every 12 months	—

Measure

Time frame

Secondary end point(s): The secondary endpoints will focus on the long-term efficacy (6-10 years) after dosing of AAV5-hFIX on FIX activity, overall FIX utilization, bleeding events requiring FIX replacement therapy, any procedures, and QoL. Secondary efficacy endpoints include the following: - Endogenous FIX activity - Utilization of FIX-replacement therapy - Annualized bleeding rate (FIX-requiring); including the following: o All bleeds o Spontaneous bleeds o Traumatic bleeds o Joint bleeds - Procedures (including major and minor surgeries) - Short form (SF-36) and EQ-5D-5L QoL scores - Hemophilia Joint Health Score (HJHS) ;Timepoint(s) of evaluation of this end point: Endogenous FIX activity, Utilization of FIX-replacement therapy, FIX inhibitors and bleeding rates and procedures will be measured every 6 months QoL and HJHS will be measured every 12 months

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Primary

Measure	Time frame
Main Objective: To assess the long-term safety (6-10 years after dosing, inclusive) of a systemic administration of AAV5-hFIX, an adeno-associated viral (AAV) vector containing a codon-optimized human coagulation Factor IX (hFIX) gene, to adult patients with severe or moderately severe haemophilia B..;Secondary Objective: To assess the long-term efficacy of a systemic administration of AAV5-hFIX, an adeno-associated viral (AAV) vector containing a codon-optimized human coagulation Factor IX (hFIX) gene, to adult patients with severe or moderately severe haemophilia B.;Primary end point(s): The primary endpoint is to demonstrate the long-term safety (6-10 years) after dosing of AAV5-hFIX. Primary safety endpoints include the following: - AEs possibly or probably related to previous AAV5-hFIX administration - Neutralizing FIX antibodies (FIX inhibitors) - ALT/AST levels - Liver pathology (assessed by ultrasound) - Alpha-fetoprotein (AFP) ;Timepoint(s) of evaluation of this end point: The individual subjects will be followed for an additional five years, 6-10 years after dosing of AAV5-hFIX. The occurrence of adverse events (AEs) related to previous AAV5-hFIX administration in patients will be continuously monitored. ALT/AST will be measuredevery 6 months Abdominal ultrasounds will be conducted at study entry (Visit 36) and study exit (Visit 45). Alpha-fetoprotein (AFP) and Anti-AAV5 antibodies will be measured every 12 months	—

Measure

Time frame

Main Objective: To assess the long-term safety (6-10 years after dosing, inclusive) of a systemic administration of AAV5-hFIX, an adeno-associated viral (AAV) vector containing a codon-optimized human coagulation Factor IX (hFIX) gene, to adult patients with severe or moderately severe haemophilia B..;Secondary Objective: To assess the long-term efficacy of a systemic administration of AAV5-hFIX, an adeno-associated viral (AAV) vector containing a codon-optimized human coagulation Factor IX (hFIX) gene, to adult patients with severe or moderately severe haemophilia B.;Primary end point(s): The primary endpoint is to demonstrate the long-term safety (6-10 years) after dosing of AAV5-hFIX. Primary safety endpoints include the following: - AEs possibly or probably related to previous AAV5-hFIX administration - Neutralizing FIX antibodies (FIX inhibitors) - ALT/AST levels - Liver pathology (assessed by ultrasound) - Alpha-fetoprotein (AFP) ;Timepoint(s) of evaluation of this end point: The individual subjects will be followed for an additional five years, 6-10 years after dosing of AAV5-hFIX. The occurrence of adverse events (AEs) related to previous AAV5-hFIX administration in patients will be continuously monitored. ALT/AST will be measuredevery 6 months Abdominal ultrasounds will be conducted at study entry (Visit 36) and study exit (Visit 45). Alpha-fetoprotein (AFP) and Anti-AAV5 antibodies will be measured every 12 months

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Countries

Denmark, Germany

Contacts

Public ContactRegulatory Affairs

uniQure biopharma B.V.

E.Destree@uniqure.com+31020240 6154 |

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026