Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher D⦠(NCT04145037) | Clinical Trial Compass
TerminatedPhase 1/2
Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease
Stopped: This study was voluntarily terminated by the sponsor to halt development and was not based on any safety or medical reasons.
United States, Canada8 participantsStarted 2019-05-30
Plain-language summary
This was a multinational, open-label study to assess the safety and efficacy of AVR-RD-02 in approximately 8 to 16 subjects (male or female) who are β₯18 and β€50 years of age and post pubertal at Screening with a confirmed diagnosis of Type 1 Gaucher disease (based on clinical phenotype, genotyping, and deficient GCase enzyme activity in whole blood).
Who can participate
Age range18 Years β 50 Years
SexALL
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Inclusion criteria
β. Subject was β₯18 and β€50 years old and post pubertal
β. Subject had a confirmed diagnosis of Type 1 Gaucher disease based on deficient GCase enzyme at Screening.
β. Female subjects of reproductive potential were counseled regarding the risks, benefits, limitations, and alternatives associated with female fertility preservation. Oocyte harvesting and cryopreservation were offered
β. Male subjects were willing to refrain from donating sperm at any time after receiving conditioning therapy. For subjects planning on (or for whom there is a possibility of) fathering children in the future, sperm cryopreservation before administration of the conditioning regimen was recommended.
β. All subjects who had not undergone successful surgical sterilization (ie, vasectomy, hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) agreed to remain sexually abstinent or use two effective methods of contraception while sexually active from the day of conditioning administration until 52 weeks post-gene therapy infusion. Two methods of contraception were required even with documented medical assessment of surgical success of sterilization.
β. For male subjects and for male spouses/partners of female subjects, condoms were an acceptable method of barrier contraception
β. For female subjects and for female spouses/partners of male subjects, acceptable methods of barrier contraception included diaphragm, cervical cap, or contraceptive sponge.
What they're measuring
1
Number of Clinically Significant Adverse Events (AEs) and Serious Adverse Events (SAEs) of AVR-RD-02
Timeframe: Baseline to 52 weeks post-AVR-RD-02 treatment follow-up
2
Vector Copy Number (VCN) in Peripheral Blood as Assessed by Quantitative Polymerase Chain Reaction (qPCR) and/or Droplet Digital Polymerase Chain Reaction (ddPCR)
Timeframe: Baseline to 52 weeks post-AVR-RD-02 treatment follow-up
3
Vector Copy Number (VCN) in Bone Marrow as Assessed by Quantitative Polymerase Chain Reaction (qPCR) and/or Droplet Digital Polymerase Chain Reaction (ddPCR)
Timeframe: Baseline to 52 weeks post-AVR-RD-02 treatment follow-up
4
Change From Baseline in Spleen Volume Assessed by Abdominal MRI
Timeframe: Baseline to 52 weeks post-AVR-RD-02 treatment follow-up
5
Change From Baseline in Liver Volume Assessed by Abdominal MRI
Timeframe: Baseline to 52 weeks post-AVR-RD-02 treatment follow-up
6
Change From Baseline in Hemoglobin Concentration
Timeframe: Baseline to 52 weeks post-AVR-RD-02 treatment follow-up
7
Change From Baseline in Platelet Count
Timeframe: Baseline to 52 weeks post-AVR-RD-02 treatment follow-up
β. Male and female subjects agreed to refrain from donating sperm and eggs, respectively, after undergoing conditioning.
Exclusion criteria
β. Subject had Type 2 or 3 Gaucher disease, had severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease, or had a tremor, peripheral neuropathy or symptoms of Parkinson's disease.
β. Hemoglobin value \<9.0 g/dL, or
β. Platelet count \<70 x 10Λ9/L, or
β. Spleen volume \>10 x normal, or
β. Pulmonary hypertension 3. Subject had experienced a prior anaphylactic or anaphylactoid reaction (of any severity) to ERT.
8
Change From Baseline in Plasma Lyso-Gb1 Levels by Liquid Chromatography Tandem Mass Spectrometry (LC/MS/MS)
Timeframe: Baseline to 52 weeks post-AVR-RD-02 treatment follow-up