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Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant

Study acronym: TigetT10_MPSIH
StatusActive, Not Recruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗

Record History

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  1. Primary completion date: Jan 2028 to Jan 2035v10 Show 6 more changesShow less
    • Primary endpoints revised (7)
    • Study completion date: Mar 2028 to Mar 2035
    • Interventions: Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the… to Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LVV, encoding for the…
    • Secondary endpoints: details revised at 7 of 7 entries
    • Publications: 13 to 3
    • Study description expanded, about 4 words
  2. Primary endpoints revised (7)v9 Show 4 more changesShow less
    • Primary completion date: Mar 2028 to Jan 2028
    • Eligibility criteria revised
    • Secondary endpoints revised (7)
    • Study description revised
  3. Lead sponsor: IRCCS San Raffaele to Orchard Therapeuticsv8
  4. Primary and study completion dates: Jan 2025 to Mar 2028v7
  5. Primary and study completion dates: Jan 2023 to Jan 2025
    • Secondary endpoints: details revised at 7 of 7 entries
    • Study arms: 0 to 1
    • Study description shortened, about 1 word
    v6
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  1. Primary endpoints: 10 to 7
    • Secondary endpoints: details revised at 6 of 7 entries
    • Eligibility criteria shortened, about 1 word
    v5
  2. Primary and study completion dates: Dec 2021 to Jan 2023v4 Show 4 more changesShow less
    • Trial status: Recruiting to Active, Not Recruiting
    • Secondary endpoints: details revised at 7 of 7 entries
    • Study description shortened, about 18 words
    • Site details revised
  3. Primary and study completion dates: Sep 2020 to Dec 2021v3 Show 5 more changesShow less
    • Enrollment target: 6 to 8
    • Eligibility criteria expanded, about 18 words
    • Secondary endpoints: 6 to 7
    • Primary endpoints revised (10)
    • Study description expanded, about 27 words
  4. Trial status: Not Yet Recruiting to Recruiting
    • Start date changed from estimate of Apr 2018 to May 11, 2018
    • Site details revised
    v2
  1. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
This is a phase I/II study evaluating safety and efficacy of autologous hematopoietic stem and progenitor cells genetically modified with IDUA lentiviral vector encoding for the human α-L-iduronidase gene for the treatment of patients affected by Mucopolysaccharidosis Type I, Hurler variant
Trial Details
NCT Number NCT03488394
Lead Sponsor Orchard Therapeutics
Collaborators: Fondazione Telethon
Conditions
(from sponsor)		 Mucopolysaccharidosis IH
Related terms Mucopolysaccharidosis I
Enrollment 8 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov