EUCTR2019-001283-30-DKActive, not recruitingPhase 1
Phase 1/2, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease
Spark Therapeutics0 sites20 target enrollmentStarted: October 5, 2021Last updated:
Conditions
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Sponsor
- Enrollment
- 20
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional clinical trial of medicinal product
Eligibility Criteria
- Sex
- All
Inclusion Criteria
- •1. Be able to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local privacy regulations;
- •2. Are male or female =18 years of age with a confirmed diagnosis (e.g., GAA genetic testing) of LOPD, or based on a documented deficiency of GAA enzyme activity;
- •3. Have received a marketed ERT for at least the previous 24 months and maintained a stable dose, frequency and compliance for the past 6 months with no dose variation and no longer improving on ERT;
- •4. Have clinically moderate, LOPD characteristics;
- •a. Able to walk =75 meters on the 6MWT (assistive devices permitted) but less than 500 meters assessed at two timepoints prior to initiating immunosuppression (Day -6) with <10% variance between the assessments. If the variance is =10%, a third timepoint will be collected;
- •b. Percentage of the predicted FVC =30% and =80% in the upright position;
- •5. Agree to use reliable contraception for a minimum of 6 months after administration of SPK-3006 or 12 weeks after the final dose of sirolimus, whichever occurs later. Female candidates of child-bearing potential must have a negative pregnancy test prior to initiating immunosuppression (Day -6) and on Day 0 prior to administration of SPK-3006. See Section 13 (Appendix 1) for guidance on reliable
- •contraception and the definition of women of child-bearing potential (WOCBP);
- •6. Agree to refrain from blood, plasma, platelets, egg or sperm and organ donation after receiving SPK-3006.
- •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 20
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
Exclusion Criteria
- •1. Active hepatitis B and/or C
- •All candidates must be screened for both active hepatitis B and C, regardless of prior known history.
- •a. Screening for hepatitis B
- •All candidates must have a single sample at Screening for each of the following tests: hepatitis B surface antigen (HBsAg), total hepatitis B core antibody (anti-HBc), and a nucleic acid test for hepatitis B virus DNA (HBV-DNA viral assay)
- •i. A candidate is not eligible if either HBsAg is positive or HBV-DNA is positive/detectable.
- •ii. A candidate is eligible if the anti-HBc is positive and both HBsAg and HBV-DNA are negative, as this would be consistent with a prior infection of hepatitis B. Anti-HBc must be obtained in all candidates to discriminate between acute infection and possible reactivation of hepatitis B during the trial in candidates with no prior history of hepatitis B.
- •iii. A candidate who is currently undergoing antiviral therapy for chronic hepatitis B is not eligible.
- •b. Screening for hepatitis C
- •i. A candidate who is currently undergoing antiviral therapy for chronic hepatitis C is not eligible.
- •ii. All other candidates, including those who have never been treated or who have completed antiviral therapy for chronic hepatitis C must have a nucleic acid test for hepatitis C viral RNA (HCV-RNA single load assay) at Screening
- •A candidate is not eligible if the HCV-RNA load assay is positive/detectable.
- •Candidates treated with anti-viral therapy for chronic hepatitis C, must have completed anti-viral therapy at least 6 months prior to Screening and have a negative HCV-RNA at Screening.
- •Candidates with a documented or self-reported history of hepatitis C must have a single negative HCV-RNA at Screening
- •2. Significant underlying liver disease. A candidate is not eligible if any of the following pre-existing diagnoses, which are indicative of significant underlying liver disease, are present in the medical record
- •Liver cirrhosis
- •Portal hypertension
- •Hepatic encephalopathy
- •Gamma-glutamyl transferase (GGT) >1.2X ULN adjusted for age and gender; or
- •Bilirubin >1.2X ULN adjusted for age and gender. Candidates with asymptomatic elevated bilirubin (e.g., Gilbert syndrome) can be considered after discussion with the Medical Monitor.
- •All candidates who do not have the pre-existing diagnoses listed above must have the following assessments performed at Screening
- •Measurement of serum albumin. A candidate is not eligible if the serum albumin level is below the testing laboratory's lower limit of normal
- •Liver fibrosis = stage 3. The following results are indicative of fibrosis
- •>= stage 3 and exclude the candidate from participation
- •- FibroScan, with a score > 8.3 kPa units
- •- FibroTest/ FibroSURE with a result > 0.48
- •- AST-Platelet Ratio Index (APRI) >1;
- •Of note, if results from more than one modality for evaluation of liver fibrosis are available (e.g., results from both FibroScan and FibroSURE are available) the result from FibroScan should be consulted for determination of liver fibrosis as it will take precedence over other modalities.
- •3. Human immunodeficiency virus (HIV) infection
- •4. Prior hypersensitivity to rhGAA
- •5. Pre-existing AAV-Spark100 neutralizing antibody titers >1:1
- •6. High titer antibody responses to rhGAA (anti-GAA >1:30,000)
- •7. Participation in a clinical study with an investigational drug in the past
- •6 months (vaccination studies are accepted; observation studies are accepted after discussion with the Medical Monitor);
- •8. Requires any invasi
Investigators
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