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.

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. - Extension study of AAV5-hFIX in severe/moderately severe haemophilia B

Status

Active, not recruiting

Phases

Phase 2

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON54158

Enrollment

Registered

2020-08-27

Start date

2021-03-19

Completion date

Unknown

Last updated

2024-09-16

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

Conditions

Christmas disease

Interventions

None listed

Eligibility

Age

18 Years to 99 Years

Inclusion criteria

Inclusion criteria: 1. Subjects 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.

Exclusion criteria

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

Design outcomes

Primary

Measure	Time frame
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 - ALT/aspartate aminotransferase (AST) levels - Liver pathology (assessed by ultrasound) - Alpha-fetoprotein (AFP)	—

Secondary

Measure	Time frame
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, any procedures and Quality of Life. Secondary efficacy endpoints include the following: - Endogenous FIX activity - Utilization of FIX-replacement therapy - Annualized bleeding rate; including the following: o All bleeds (treated and untreated) o Spontaneous bleeds o Traumatic bleeds o Joint bleeds - Procedures (including major and minor surgeries) - Short form (SF-36) and EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) QoL scores - Hemophilia Joint Health Score (HJHS)	—

Measure

Time frame

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, any procedures and Quality of Life. Secondary efficacy endpoints include the following: - Endogenous FIX activity - Utilization of FIX-replacement therapy - Annualized bleeding rate; including the following: o All bleeds (treated and untreated) o Spontaneous bleeds o Traumatic bleeds o Joint bleeds - Procedures (including major and minor surgeries) - Short form (SF-36) and EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) QoL scores - Hemophilia Joint Health Score (HJHS)

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Countries

Netherlands

Outcome results