An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of… (NCT05815004) | Clinical Trial Compass
WithdrawnPhase 2/3
An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3
Stopped: Further development of programs halted
0Started 2023-10
Plain-language summary
The purpose of this study is to evaluate the efficacy and safety of autologous hemotopoietic stem cell (HSC) gene therapy, AVR-RD-02, compared to enzyme replacement therapy, for the treatment of Gaucher disease Type 3 in male and female participants aged 2 to 25 years.
The study will consist of 2 parts - Core (Part 1) followed by the ERT-crossover (Part 2)
Who can participate
Age range2 Years – 25 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Participant and/or parent, caregiver, or legal representative must be willing and able to provide written informed consent/assent for the study in accordance with applicable regulations and guidelines and to comply with all study visits and procedures, including the use of any data collection device(s) that may be used to directly record participant data.
✓. Participant is ≥2 to ≤ 25 years old, at the time of providing informed consent or assent.
✓. Participant has a confirmed diagnosis of Gaucher disease Type 3 based on all of the following:
✓. Biallelic GBA1 gene mutation
✓. Deficient GCase enzyme activity in blood
✓. Clinical phenotype with the presence of gaze palsy, predominantly horizontal and with slow or absent saccades
✓. Participant has the presence of one or both of the following within 3 months of screening:
✓. Ataxia (score ≥1) based on the modified scale for the assessment and rating of ataxia total score (mSARA)
Exclusion criteria
✕. Participant has a diagnosis of Gaucher disease Type 1 or Type 2.
✕. Participant has any one of the following:
✕. Hemoglobin value of \<9.0 g/dL
✕. Platelet count of \<70 × 109/L
What they're measuring
1
Change from baseline in a multidomain endpoint as assessed by Global Statistical Test (GST)
Timeframe: Baseline to 52 weeks post AVR-RD-02 infusion (AVR-RD-02 arm) or Baseline to 52 weeks (ERT arm)