A study to assess the safety, tolerability, and efficacy of VX-264 in patients with type 1 diabetes mellitus

Canada, Germany, Italy, Netherlands, Switzerland, United Kingdom, United States (ICTRP)

ID di studio ICTRP
EUCTR2022-003318-35 (ICTRP)

Titolo ufficiale (approvato dal comitato etico)
A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-264 in Subjects With Type 1 Diabetes Mellitus (BASEC)

Titolo accademico
A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-264 in Subjects With Type 1 Diabetes Mellitus (ICTRP)

Titolo pubblico
A Safety, Tolerability, and Efficacy Study of VX-264 in Subjects With Type 1 Diabetes (ICTRP)

Malattie studiate
Type 1 Diabetes Mellitus
MedDRA version: 20.0Level: PTClassification code 10012601Term: Diabetes mellitusSystem Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] (ICTRP)

Intervento studiato

Product Code: VX-264
Pharmaceutical Form: Implant
INN or Proposed INN: VX-264
Other descriptive name: VX-264
Concentration unit: Other
Concentration type: not less then
Concentration number: 75000000-

(ICTRP)

Tipo di studio
Interventional clinical trial of medicinal product (ICTRP)

Disegno dello studio
Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1 (ICTRP)

Criteri di inclusione/esclusione
Gender:
Female: yes
Male: yes

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.
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 16
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1
(ICTRP)

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.


Endpoint primari e secondari
Main Objective: 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).;Secondary Objective: 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).;Primary 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.;Timepoint(s) of evaluation of this end point: Please refer to the protocol (ICTRP)

Secondary 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;Timepoint(s) of evaluation of this end point: Please refer to the protocol (ICTRP)

Data di registrazione
06.01.2023 (ICTRP)

Inclusione del primo partecipante
17.03.2023 (ICTRP)

Sponsor secondari
non disponibile

Contatti aggiuntivi
Clinical Trials and Medical Info, medicalinfo@vrtx.com, +1877 634 8789, Vertex Pharmaceuticals Incorporated (ICTRP)

ID secondari
VX22-264-101, 2022-003318-35-DE (ICTRP)

Risultati-Dati individuali dei partecipanti
non disponibile

Ulteriori informazioni sullo studio
https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2022-003318-35 (ICTRP)