临床试验Nct页

Summary
EudraCT Number:2022-003318-35
Sponsor's Protocol Code Number:VX22-264-101
National Competent Authority:Netherlands - Competent Authority
Clinical Trial Type:EEA CTA
Trial Status:Ongoing
Date on which this record was first entered in the EudraCT database:2023-01-06
Trial results
Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL
A. Protocol Information
A.1Member State ConcernedNetherlands - Competent Authority
A.2EudraCT number2022-003318-35
A.3Full title of the trial
A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-264 in Subjects With Type 1 Diabetes Mellitus
A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
A Safety, Tolerability, and Efficacy Study of VX-264 in Subjects With Type 1 Diabetes
A.4.1Sponsor's protocol code numberVX22-264-101
A.7Trial is part of a Paediatric Investigation Plan No
A.8EMA Decision number of Paediatric Investigation Plan
B. Sponsor Information
B.Sponsor: 1
B.1.1Name of SponsorVertex Pharmaceuticals Incorporated
B.1.3.4CountryUnited States
B.3.1 and B.3.2Status of the sponsorCommercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1Name of organisation providing supportVertex Pharmaceuticals Incorporated
B.4.2CountryIreland
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1Name of organisationVertex Pharmaceuticals Incorporated
B.5.2Functional name of contact pointClinical Trials and Medical Info
B.5.3 Address:
B.5.3.1Street Address50 Northern Avenue
B.5.3.2Town/ cityBoston
B.5.3.3Post codeMA 02210
B.5.3.4CountryUnited States
B.5.4Telephone number+1877 634 8789
B.5.6E-mailmedicalinfo@vrtx.com
D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3IMP RoleTest
D.2 Status of the IMP to be used in the clinical trial
D.2.1IMP to be used in the trial has a marketing authorisation No
D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
D.2.5.1Orphan drug designation number
D.3 Description of the IMP
D.3.2Product code VX-264
D.3.4Pharmaceutical form Implant
D.3.4.1Specific paediatric formulation No
D.3.7Routes of administration for this IMPImplantation
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8INN - Proposed INNVX-264
D.3.9.3Other descriptive nameVX-264
D.3.9.4EV Substance CodeSUB295649
D.3.10 Strength
D.3.10.1Concentration unit Other
D.3.10.2Concentration typenot less then
D.3.10.3Concentration number75000000
D.3.11 The IMP contains an:
D.3.11.1Active substance of chemical origin No
D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
The IMP is a:
D.3.11.3Advanced Therapy IMP (ATIMP) Yes
D.3.11.3.1Somatic cell therapy medicinal product Yes
D.3.11.3.2Gene therapy medical product No
D.3.11.3.3Tissue Engineered Product No
D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Yes
D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
D.3.11.5Radiopharmaceutical medicinal product No
D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
D.3.11.7Plasma derived medicinal product No
D.3.11.8Extractive medicinal product No
D.3.11.9Recombinant medicinal product No
D.3.11.10Medicinal product containing genetically modified organisms No
D.3.11.11Herbal medicinal product No
D.3.11.12Homeopathic medicinal product No
D.3.11.13Another type of medicinal product No
D.8 Information on Placebo
E. General Information on the Trial
E.1 Medical condition or disease under investigation
E.1.1Medical condition(s) being investigated
Type 1 Diabetes Mellitus
E.1.1.1Medical condition in easily understood language
Type 1 Diabetes
E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
MedDRA Classification
E.1.2 Medical condition or disease under investigation
E.1.2Version 20.0
E.1.2Level PT
E.1.2Classification code 10012601
E.1.2Term Diabetes mellitus
E.1.2System Organ Class 10027433 - Metabolism and nutrition disorders
E.1.3Condition being studied is a rare disease No
E.2 Objective of the trial
E.2.1Main objective of the trial
1. Evaluate the safety and tolerability of VX-264 in subjects with T1D (Part A and Part B).
2. Evaluate VX-264 function in subjects with T1D (Part B and Part C).
E.2.2Secondary objectives of the trial
1. Evaluate the effect of VX-264 on exogenous insulin dose (Part C).
2. Evaluate the effect of VX-264 on insulin independence (Part C).
3. Evaluate the effect of VX-264 on metabolic control (Part C).
4. Evaluate the safety and tolerability of VX-264 (Part C).
E.2.3Trial contains a sub-study No
E.3Principal inclusion criteria
1. Subjects (male and female) between the ages of 18 and 65 years (inclusive) on the date of first informed consent.
2. HbA1c ≥6.0% and ≤9.5%.
3. Clinical history and laboratory evidence of T1D based on the American Diabetes Association/European Association for the Study of Diabetes algorithm 13 for investigation of suspected T1D including:
o insulin dependence for ≥5 years at time of Screening, and
o peak stimulated C-peptide level during MMTT <50 pmol/L (<0.15 ng/mL)
4. Consistent use of CGM for at least 4 weeks before Screening and willingness to use only the study-provided CGM for the duration of the study.
5. Body habitus supportive of implantation of 6 VX-264 units.
Please refer to Section 8.1 of the Protocol for additional inclusion criteria.
E.4Principal exclusion criteria
1. Prior islet cell transplant, organ transplant, or cell therapy.
2. Advanced complications associated with diabetes including untreated proliferative retinopathy, diabetic nephropathy, skin ulcers, or amputations attributable to diabetes.
3. Insulin requirement:
a) Parts A and B >40 U/day, or <10 U/day
b) Part C: >55 U/day, or <10 U/day
c) >0.8 U/kg/day.
4. Subjects with ≥2 or more episodes of severe hypoglycemia in the 12 months prior to signing of informed consent at Screening.
5. Previous abdominal or abdominal wall surgery, or history of peritonitis, that can impact VX-264 placement or put the patient at higher risk of surgical complications
Please refer to Section 8.2 of the Protocol for additional exclusion criteria.
E.5 End points
E.5.1Primary end point(s)
Part A: Safety and tolerability assessments.
Part B:
- Safety and tolerability assessments,
- Change from baseline in peak C-peptide during MMTT at Day 90.
Part C: Change from baseline in peak C-peptide during MMTT at Day 90.
E.5.1.1Timepoint(s) of evaluation of this end point
Please refer to the protocol
E.5.2Secondary end point(s)
Part C:
• Change from baseline in peak C-peptide during MMTT over time
• Change from baseline in C-peptide AUC during MMTT over time
• Proportion of subjects with peak C-peptide ≥100, ≥200, ≥400, and ≥1000 pmol/L (≥0.30, ≥0.60, ≥1.21, and ≥3.02 ng/mL) during MMTT over time
• Change from baseline in average total daily insulin dose over time
• Proportion of subjects with a reduction of at least 50% insulin dose from baseline and HbA1c <7.0% at Day 365
• Proportion of subjects who are insulin independent at one point in time between Day 180 and Day 365
• Change from baseline on metabolic control: HbA1c values; continuous glucose monitoring (CGM)-derived time-in-range (TIR) over time; glucose variability (CGM, CV%); and glucose management indicator (GMI)
• Safety and tolerability assessments
E.5.2.1Timepoint(s) of evaluation of this end point
Please refer to the protocol
E.6 and E.7 Scope of the trial
E.6Scope of the trial
E.6.1Diagnosis No
E.6.2Prophylaxis No
E.6.3Therapy Yes
E.6.4Safety Yes
E.6.5Efficacy Yes
E.6.6Pharmacokinetic No
E.6.7Pharmacodynamic No
E.6.8Bioequivalence No
E.6.9Dose response No
E.6.10Pharmacogenetic No
E.6.11Pharmacogenomic No
E.6.12Pharmacoeconomic No
E.6.13Others No
E.7Trial type and phase
E.7.1Human pharmacology (Phase I) Yes
E.7.1.1First administration to humans Yes
E.7.1.2Bioequivalence study No
E.7.1.3Other No
E.7.1.3.1Other trial type description
E.7.2Therapeutic exploratory (Phase II) Yes
E.7.3Therapeutic confirmatory (Phase III) No
E.7.4Therapeutic use (Phase IV) No
E.8 Design of the trial
E.8.1Controlled No
E.8.1.1Randomised No
E.8.1.2Open Yes
E.8.1.3Single blind No
E.8.1.4Double blind No
E.8.1.5Parallel group No
E.8.1.6Cross over No
E.8.1.7Other No
E.8.2 Comparator of controlled trial
E.8.2.1Other medicinal product(s) No
E.8.2.2Placebo No
E.8.2.3Other No
E.8.2.4Number of treatment arms in the trial1
E.8.3 The trial involves single site in the Member State concerned Yes
E.8.4 The trial involves multiple sites in the Member State concerned No
E.8.5The trial involves multiple Member States Yes
E.8.5.1Number of sites anticipated in the EEA3
E.8.6 Trial involving sites outside the EEA
E.8.6.1Trial being conducted both within and outside the EEA Yes
E.8.6.2Trial being conducted completely outside of the EEA No
E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
Canada
United States
Netherlands
Switzerland
Germany
Italy
United Kingdom
E.8.7Trial has a data monitoring committee Yes
E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
LPLV
E.8.9 Initial estimate of the duration of the trial
E.8.9.1In the Member State concerned years3
E.8.9.1In the Member State concerned months3
E.8.9.1In the Member State concerned days0
E.8.9.2In all countries concerned by the trial years3
E.8.9.2In all countries concerned by the trial months3
E.8.9.2In all countries concerned by the trial days0
F. Population of Trial Subjects
F.1 Age Range
F.1.1Trial has subjects under 18 No
F.1.1.1In Utero No
F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
F.1.1.3Newborns (0-27 days) No
F.1.1.4Infants and toddlers (28 days-23 months) No
F.1.1.5Children (2-11years) No
F.1.1.6Adolescents (12-17 years) No
F.1.2Adults (18-64 years) Yes
F.1.2.1Number of subjects for this age range: 16
F.1.3Elderly (>=65 years) Yes
F.1.3.1Number of subjects for this age range: 1
F.2 Gender
F.2.1Female Yes
F.2.2Male Yes
F.3 Group of trial subjects
F.3.1Healthy volunteers No
F.3.2Patients Yes
F.3.3Specific vulnerable populations Yes
F.3.3.1Women of childbearing potential not using contraception No
F.3.3.2Women of child-bearing potential using contraception Yes
F.3.3.3Pregnant women No
F.3.3.4Nursing women No
F.3.3.5Emergency situation No
F.3.3.6Subjects incapable of giving consent personally No
F.3.3.7Others No
F.4 Planned number of subjects to be included
F.4.1In the member state2
F.4.2 For a multinational trial
F.4.2.1In the EEA 3
F.4.2.2In the whole clinical trial 17
F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
All subjects who receive VX-264 will be asked to enroll in a long-term follow-up study upon completing 2 years of follow-up in the VX22-264-101 study.
G. Investigator Networks to be involved in the Trial
N. Review by the Competent Authority or Ethics Committee in the country concerned
N.Competent Authority Decision Authorised
N.Date of Competent Authority Decision2022-12-12
N.Ethics Committee Opinion of the trial applicationFavourable
N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
N.Date of Ethics Committee Opinion2023-03-17
P. End of Trial
P.End of Trial StatusOngoing

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