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Open-Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment -Naive Subjects With Classic Fabry Disease

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ClinicalTrials.gov Identifier: NCT03454893
Recruitment Status : Recruiting
First Posted : March 6, 2018
Last Update Posted : July 10, 2019
Sponsor:
Information provided by (Responsible Party):
AvroBio

Tracking Information
First Submitted Date  ICMJE December 20, 2017
First Posted Date  ICMJE March 6, 2018
Last Update Posted Date July 10, 2019
Actual Study Start Date  ICMJE February 21, 2018
Estimated Primary Completion Date June 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 5, 2018)
Incidence of Treatment-Emergent Adverse Events of AVR-RD-01 drug product [ Time Frame: baseline to 48 weeks post gene therapy ]
Incidence and severity of adverse events and serious adverse events
Original Primary Outcome Measures  ICMJE
 (submitted: February 27, 2018)
Safety and Tolerability of AVR-RD-01 drug product [ Time Frame: baseline to 48 weeks post gene therapy ]
Incidence and severity of adverse events and serious adverse events
Change History Complete list of historical versions of study NCT03454893 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Open-Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment -Naive Subjects With Classic Fabry Disease
Official Title  ICMJE An Open-Label, Multinational Study Of The Efficacy And Safety Of Ex Vivo, Lentiviral Vector-Mediated Gene Therapy AVR-RD-01 For Treatment-Naive Subjects With Classic Fabry Disease
Brief Summary This is a multinational, open-label study to assess the efficacy and safety of AVR-RD-01 in approximately 8 to 12 male subjects 16 years of age or older and postpubertal with a confirmed diagnosis of classic Fabry disease based on deficient AGA enzyme activity who have not previously received treatment with enzyme replacement therapy (ERT) and/or chaperone therapy at the time of Screening.
Detailed Description

The duration of each subject's participation in this study will be approximately 58 weeks (or 1 year, 6 weeks), comprised of a five study periods (Screening, Baseline, Pre-transplant, Transplant, and Post-transplant Follow-up). During the Screening Period (approximately 4 weeks), written informed consent (and assent, if applicable) will be obtained and the subject will complete other Screening procedures to confirm study eligibility. Once study eligibility is confirmed, subjects will enter the Baseline Period (up to 3 days) during which time assessments will be performed to establish a pre-transplant baseline. Once baseline assessments are complete, the subject will enter the Pre-transplant Period (approximately 6 weeks) during which time mobilization, apheresis, AVR-RD-01 drug product preparation and testing for release, and conditioning regimen administration to achieve partial myeloablation will take place. Following completion of the Pre-transplant Period, the subject will enter the Transplant Period (1 day) during which time AVR-RD-01 infusion will take place. After AVR-RD-01 infusion, the subject will enter the Post-transplant Follow-up Period (approximately 48 weeks), during which time periodic safety and efficacy assessments will be performed to assess measures of engraftment, clinical response, and safety post-transplant. Post-transplant follow-up will occur at the following time points: Week 1 (Days 1 through 7), Week 2 (Days 10 and 14), Week 4 (Day 28), Week 8 (Day 56), Week 12 (Day 84), Week 24 (Day 168), Week 36 (Day 216), and Week 48 (Day 336).

After study completion, consenting subjects will continue periodic safety and efficacy assessments for approximately 14 years (for a total of 15 years post-transplant follow-up) in a long-term follow-up study to AVRO-RD-01-201.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Fabry Disease
Intervention  ICMJE Drug: AVR-RD-01
AVR-RD-01 Drug Product (autologous CD34+ cell-enriched fraction that contains cells transduced with Lentiviral Vector/alpha-galactosidase A (AGA) encoding for the human AGA complementary deoxyribonucleic acid (cDNA) sequence
Study Arms  ICMJE Experimental: Single Assignment AVR-RD-01
AVR-RD-01 Drug Product (autologous CD34+ cell-enriched fraction that contains cells transduced with Lentiviral Vector/alpha-galactosidase A (AGA) encoding for the human AGA complementary deoxyribonucleic acid (cDNA) sequence
Intervention: Drug: AVR-RD-01
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: February 27, 2018)
12
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2020
Estimated Primary Completion Date June 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Subject is male, 16 years of age or older, and postpubertal,,
  2. Subject has a confirmed diagnosis of classic Fabry disease based on deficient AGA enzyme activity (defined as < 1% of normal).
  3. Subject has a history of GI symptoms including abdominal pain and diarrhea.

Exclusion Criteria:

  1. Subject has previously received ERT and/or chaperone therapy at any time for treatment of Fabry disease.
  2. Subject has eGFR < 60 mL/min/1.73 m² (ie, chronic kidney disease [CKD] stage ≥ 3) at Screening.
  3. Subject has a prior history of myocardial infarction (MI).
  4. Subject has a history of coronary artery disease (CAD) with angina requiring percutaneous transluminal coronary angioplasty (with or without stent placement) and/or coronary artery bypass graft (CABG).
  5. Subject has a history of moderate to severe valvular heart disease requiring valve replacement.
  6. Subject has a history of heart failure, moderate to severe diastolic dysfunction, and/or left ventricular ejection fraction (LVEF) ≤ 45% on echocardiogram (ECHO) performed at rest at Screening.
  7. Subject has a history of clinically significant cardiac arrhythmia (eg, heart block [second or third degree], atrial fibrillation requiring therapy (history of intermittent atrial fibrillation not requiring treatment is allowed), ventricular fibrillation, ventricular tachycardia, supraventricular tachycardia, or cardiac arrest).
  8. Subject has a prior history of stroke and/or transient ischemic attack (TIA).
  9. Subject has aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) ≥ 3 times the upper limit of normal (ULN) at Screening.
  10. Subject has a prior history of (or current) malignancy; the one exception is a prior history of resected basal cell carcinoma.
  11. Subject has previously received treatment with AVR-RD-01 or any other gene therapy.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 16 Years to 40 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: lisamarie fahy 6179148417 lisamarie.fahy@avrobio.com
Listed Location Countries  ICMJE Australia
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03454893
Other Study ID Numbers  ICMJE AVRO-RD-01-201
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party AvroBio
Study Sponsor  ICMJE AvroBio
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Shari Fallet, MD AvroBio
PRS Account AvroBio
Verification Date July 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP