Clinical trial • Phase I/II | Phase III • Endocrinology
ZIMISLECEL for Type 1 diabetes mellitus | Impaired hypoglycemic awareness | Severe hypoglycemia
Phase I/II | Phase III trial of ZIMISLECEL for Type 1 diabetes mellitus | Impaired hypoglycemic awareness | Severe hypoglycemia.
Overview
- Trial Therapeutic Area
- Endocrinology
- Trial Disease
- Type 1 diabetes mellitus | Impaired hypoglycemic awareness | Severe hypoglycemia
- Trial Stage
- Phase I/II | Phase III
- Drug Modality
- Cell therapy
Key dates
- Initial CTIS Submission Date
- 05-09-2024
- First CTIS Authorization Date
- 02-10-2024
Trial design
open-label, none/not specified-controlled Phase I/II | Phase III trial across 6 sites in France, Germany, Italy and others.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 36
- Trial Duration For Participant
- 365
Eligibility
Recruits 36 Vulnerable population selected (isVulnerablePopulationSelected = true). Subject information sheets and informed consent forms for adults are provided (L1_SIS and ICF Main Adult, Pre-Screening, Pregnancy ICF and country-specific ICFs). No information on assent or parental consent for minors is provided in the available CTIS data..
- Vulnerable Population
- Vulnerable population selected (isVulnerablePopulationSelected = true). Subject information sheets and informed consent forms for adults are provided (L1_SIS and ICF Main Adult, Pre-Screening, Pregnancy ICF and country-specific ICFs). No information on assent or parental consent for minors is provided in the available CTIS data.
Inclusion criteria
- {"criterion_text":"-Clinical history and laboratory evidence of T1D"}
- {"criterion_text":"-At least 2 episodes of severe hypoglycemia (confirmed by independent adjudication for subjects in Parts B and C) in the 12 months prior to signing of informed consent at Screening."}
- {"criterion_text":"-Reduced awareness of hypoglycemia at Screening"}
- {"criterion_text":"-Consistent use of continuous glucose monitor (CGM) for at least 3 months before Screening. In regions where CGM is not standard of care for T1D, subjects are exempted from the requirement for use of CGM before Screening."}
- {"criterion_text":"-Compatible blood group (A or AB)"}
Exclusion criteria
- {"criterion_text":"-Prior islet cell transplant, organ transplant, or cell therapy"}
- {"criterion_text":"-Advanced complications associated with diabetes including untreated proliferative retinopathy, skin ulcers, or amputations attributable to diabetes."}
- {"criterion_text":"-Subjects who have any 1 of the following criteria: o Insulin requirements: >0.8 U/(kg*day), >55 U/day, or <10 U/day; o HbA1c: <6.0% or >9.5%"}
- {"criterion_text":"-Clinically significant active infection or chronic infection such as hepatitis B, hepatitis C, human immunodeficiency virus (HIV), and/or tuberculosis (TB); or invasive aspergillus, histoplasmosis, or coccidioidomycosis infection within 1 year prior to signing of informed consent at Screening."}
- {"criterion_text":"-Negative screen for Epstein-Barr virus (EBV) by immunoglobulin G (IgG) determination"}
Endpoints
Primary endpoints
- {"endpoint_text":"-Part A - Safety and tolerability based on treatment-emergent adverse events (TEAEs; including incidence and severity of adverse events [AEs] and serious adverse events [SAEs]), clinical laboratory values, vital signs, standard 12-lead ECGs, and imaging findings","definition_or_measurement_approach":"Safety and tolerability assessed by incidence and severity of TEAEs (including AEs and SAEs), clinical laboratory values, vital signs, standard 12-lead ECGs, and imaging findings."}
- {"endpoint_text":"-Part B and C - The following endpoints apply to the analysis of all infused subjects who intended to receive 0.8 × 10E9 total SC-islet cells with 1 infusion: •Proportion of subjects who are insulin independent at Day 365 after VX-880 infusion","definition_or_measurement_approach":"Proportion of infused subjects who are insulin independent at Day 365 post-infusion (analysis population: all infused subjects who intended to receive 0.8 × 10^9 SC-islet cells in one infusion)."}
Secondary endpoints
- {"endpoint_text":"-Part B and C: Secondary Endpoints - Proportion of subjects free of SHEs from Day 90 through Day 365 (inclusive) and HbA1c <7.0% at Day 365 after VX 880 infusion","definition_or_measurement_approach":"Proportion free of severe hypoglycemic events (SHEs) between Day 90 and Day 365 inclusive; and proportion with HbA1c <7.0% at Day 365 post-infusion."}
- {"endpoint_text":"-Part B and C: Secondary Endpoints - Change from baseline in HbA1c at Day 365 after VX-880 infusion","definition_or_measurement_approach":"Change from baseline in HbA1c measured at Day 365 post-infusion compared to baseline."}
- {"endpoint_text":"-Part B and C: Secondary Endpoints- Proportion of subjects who achieve insulin independence and are insulin independent 12 months later, with absence of SHEs","definition_or_measurement_approach":"Proportion of subjects who achieve insulin independence and remain insulin independent 12 months later without SHEs."}
- {"endpoint_text":"-Part B and C: Secondary Endpoints- Proportion of subjects who maintain insulin independence for at least 1 year","definition_or_measurement_approach":"Proportion of subjects maintaining insulin independence for ≥1 year post-achievement."}
- {"endpoint_text":"-Part B and C: afety and tolerability based on TEAEs (including incidence and severity of AEs and SAEs), clinical laboratory values, vital signs, standard 12-lead ECGs, and imaging findings","definition_or_measurement_approach":"Safety and tolerability endpoints measured by TEAE incidence and severity, clinical lab values, vital signs, 12-lead ECGs, and imaging findings for Parts B and C."}
Recruitment
- Digital Remote Recruitment
- Yes
- Planned Sample Size
- 36
- Recruitment Window Months
- 90
- Consent Approach
- Informed consent obtained from adult subjects using subject information sheets and informed consent forms (L1_SIS and ICF Main Adult, Pre-Screening, Pregnancy ICF and country-specific ICFs). Documents provided in multiple languages (English, French, German, Italian, Dutch, Norwegian as indicated by country-specific ICFs). No information on assent or parental consent for minors is provided in the CTIS data.
Methods
- MyTomorrows digital patient recruitment platform (landing pages, patient navigator scripts) — country-specific (Netherlands, Germany, Italy, Norway, France) materials listed
- Online advertising — Google advertisements and Facebook advertisements (MyTomorrows Google/Facebook Advertisements) targeting patients
- Press releases and study-specific press release texts (country-specific)
- HCP outreach materials — HCP flyer, PI to physician letters, HCP slide decks and HCP websites to engage healthcare professionals
- Patient-facing materials — patient invitation letters, recruitment flyers, recruitment brochures, posters (country- and language-specific)
Geography
- Total Number Of Sites
- 6
- Total Number Of Participants
- 8
France
- Earliest CTIS Part Ii Submission Date
- 24-09-2024
- Latest Decision Or Authorization Date
- 07-10-2024
- Processing Time Days
- 13
- Number Of Sites
- 2
- Number Of Participants
- 1
Sites
- Site Name
- Les Hopitaux Universitaires De Strasbourg
- Department Name
- Service Endocrinologie-Diabète-Nutrition
- Contact Person Name
- Laurence Kessler
- Contact Person Email
- Laurence.Kessler@chru-strasbourg.fr
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- Sce Chir Generale & Endocrinienne
- Contact Person Name
- Francois Pattou
- Contact Person Email
- francois.pattou@univ-lille.fr
Germany
- Earliest CTIS Part Ii Submission Date
- 24-09-2024
- Latest Decision Or Authorization Date
- 08-10-2024
- Processing Time Days
- 14
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Universitaetsklinikum Carl Gustav Carus Dresden an der Technischen Universitaet Dresden AöR
- Department Name
- Medizinische Klinik und Poliklinik III/Klinik und Poliklinik f. Viszeral-,Thorax- und Gefäßchirurgie
- Contact Person Name
- Barbara Ludwig
- Contact Person Email
- Barbara.Ludwig@uniklinikum-dresden.de
Italy
- Earliest CTIS Part Ii Submission Date
- 24-09-2024
- Latest Decision Or Authorization Date
- 14-10-2024
- Processing Time Days
- 20
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Ospedale San Raffaele S.r.l.
- Department Name
- Department of Regenerative Medicine and Transplantation
- Contact Person Name
- Lorenzo Piemonti
- Contact Person Email
- piemonti.lorenzo@hsr.it
Netherlands
- Earliest CTIS Part Ii Submission Date
- 24-09-2024
- Latest Decision Or Authorization Date
- 02-10-2024
- Processing Time Days
- 8
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Leiden University Medical Center
- Department Name
- Leiden University Medical Centre
- Contact Person Name
- Eelco de Koning
- Contact Person Email
- e.j.p.de_koning@lumc.nl
Norway
- Earliest CTIS Part Ii Submission Date
- 24-09-2024
- Latest Decision Or Authorization Date
- 03-10-2024
- Processing Time Days
- 9
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Oslo University Hospital HF
- Contact Person Name
- Trond G Jenssen
- Contact Person Email
- trond.jenssen@medisin.uio.no
Sponsor
Primary sponsor
- Full Name
- Vertex Pharmaceuticals Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Investigational products
- Investigational Product Name
- VX-880 solution for infusion
- Active Substance
- ZIMISLECEL
- Modality
- Cell therapy
- Routes Of Administration
- INTRAPORTAL USE
- Route
- INTRAPORTAL
- Authorisation Status
- Investigational (Advanced Therapy IMP - ATIMP)
- Starting Dose
- 0.8 × 10^9 SC-islet cells (single infusion) for Parts B and C (intended dose for infused subjects)
- Frequency
- Single infusion (per protocol descriptions for Parts B and C)
- Maximum Dose
- 8000000000
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