Table 3. Schematic representation of assessments at study visits.
| Screening
Visit 1 |
Baseline
Visit 2 |
Visit 3 | Visit 4 | Visit 5 | Visit 6 | Visit 7 | |
|---|---|---|---|---|---|---|---|
| Day | -1 to-30 | 0 | 30 | 60 | 90 | 180 | 360 Or at
study end |
| Month | 1 | 2 | 3 | 6 | 12 | ||
| Time window | +/- 7
days |
+/- 7
days |
+/- 7
days |
+/- 7
days |
+/- 7
days |
||
| Informed consent | x | ||||||
| Review inclusion/exclusion criteria | x | x | |||||
| Medical & family history | x | ||||||
| Randomisation | x | ||||||
| Aldesleukin/ Placebo administration | x
a Twice-weekly, three days apart – with FU
call daily for first week |
||||||
| Patient AE diary | Twice-weekly, between treatments.
Completed by patient/family at home |
||||||
|
Provide instructions for DBS at home
and provision of Ensure Plus |
x | ||||||
| Twice-weekly home visits from nurse | Twice per week to deliver IMP and
provide Ensure, if needed. |
||||||
| Concomitant medications | x | x | x | x | x | x | x |
| Height, weight and BMI | x | x | x | x | x | x | x |
| Pubertal stage | x | ||||||
| Physical examination | x | x | x | x | x | x | x |
|
Vital signs (temperature, Heart rate,
blood pressure) |
x | x | x | x | x | x | x |
| Serum | x | x b | x | ||||
| Random C-peptide (venous) | x | ||||||
| HbA1c (local) | x e | x | x | x | |||
| HbA1c (central) | x | x | x b | x | |||
| PBMC | x | x | x | x b | x | ||
| Treg levels | x | x | x | x | x b | x | |
| TBNK FACS assay | x | x | x | x | x b | x | |
| DBS for C-peptide c | weekly at home | Monthly until study
end |
|||||
| Full blood count d | x | x | x | x | x | ||
| Liver function tests d | x | x | x | x | x | ||
| Renal function tests d | x | x | x | x | x | ||
| Electrolytes d | x | x | x | x | x | ||
| Thyroid function d | x | x | x | ||||
| Pregnancy test (post-menarcheal girls) | x | x | x | x | x | x | |
| Insulin dose data | x | x | x | x | x | x | |
| Adverse events review | x | x | x | x | x | x | |
AE: adverse event; BMI: body mass index; HbA1c: haemoglobin A1c; IMP: investigational medical product; PBMC: Peripheral blood mononuclear cells; TBNK FACS: T-cell, B-cell and NK cell Fluorescence activated cell sorting; Treg: T regulatory cells
a Treatment should start on day of randomisation or within 7 days thereafter. First dose of IMP or placebo should be given in a hospital setting and the patient observed for a period afterwards. All subsequent doses are administered at home by the research nurse, twice-weekly, three days apart. Wherever possible, doses should be taken on the same days each week e.g. Tuesday and Friday or Monday and Thursday.
bshould be taken before final injection.
cDBS will be also collected at home every week in-between monthly visits until 6 months post treatment start, and then monthly from 7–12 months. Samples should always be taken prior to administration of study drug. The first at-home DBS will be supervised by a research nurse.
dLocal results may be used if available, and taken within 2 weeks of the visit, or within 4 weeks of the screening/baseline visit.
eThis should be local HbA1c data, either from point of care (finger prick), or venous samples