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Ethics statement
The studies involving humans were approved by the local Ethical Committee with Authorization Number AOU 0010923/19 on 12/04/2019 and AIFA Authorization Number AIFA/SC/P/33830 on 25/03/2019. EudraCT Number 2018-003458-26. The studies were conducted in accordance with the local legislation and institutional requirements....
PMC10613634
Author contributions
GN, MMe, CM
AC: Conceptualization, Data curation, Formal Analysis, Funding acquisition, Investigation, Methodology, Project administration, Resources, Software, Supervision, Validation, Visualization, Writing – original draft, Writing – review & editing. AC-L: Conceptualization, Data curation, Formal Analysis, Investigation, Metho...
PMC10613634
Conflict of interest
CM
GG and CM received a research grant and a speaker honorarium from Gilead, ViiV, MERCK, and Jansen. GG and CM are on the advisory boards of Gilead, ViiV, and MERCK. JM received a speaker honorarium from Gilead and ViiV.The remaining authors declare that the research was conducted in the absence of any commercial or fina...
PMC10613634
Publisher’s note
All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article, or claim that may be made by its manufacturer, is not guaranteed or ...
PMC10613634
Supplementary material
The Supplementary Material for this article can be found online at: Click here for additional data file.Box-plots of primary endpoint CD4, CD8 count and CD4/CD8 ratio and adjusted prediction with 95% CI from fitting the mixed linear model.Click here for additional data file.Box-plots of monocytes and IL-6 and adjusted ...
PMC10613634
References
PMC10613634
Subject terms
T1D, Diabetes
TYPE 1 DIABETES, DIABETES
Previous studies showed a low-grade enterovirus infection in the pancreatic islets of patients with newly diagnosed type 1 diabetes (T1D). In the Diabetes Virus Detection (DiViD) Intervention, a phase 2, placebo-controlled, randomized, parallel group, double-blind trial, 96 children and adolescents (aged 6–15 years) wi...
PMC10667091
Main
T1D, enteroviral infection, Diabetes
ENTEROVIRAL INFECTION, AUTOIMMUNE RESPONSE, ENTEROVIRUS, DISEASE, TYPE 1 DIABETES, DIABETES
Type 1 diabetes (T1D) is characterized by progressive loss of pancreatic β cell function that leads to lifelong dependence on insulin therapyThe disease is the result of a complex interplay between genetic predisposition, the immune system and environmental factorsIn the Diabetes Virus Detection study (DiViD), pancreat...
PMC10667091
Results
PMC10667091
Participants
The trial included a screening period from the day of diagnosis until baseline (up to 3 weeks), a 26-week treatment period and a 26-week off-therapy follow-up period, and an ongoing extended follow-up of 2 additional years (to be reported). Out of 96 randomized participants, 47 (19 females and 28 males) were randomized...
PMC10667091
Primary endpoint
The primary endpoint was endogenous insulin production at 12 months, as assessed by the 2-h serum C-peptide rea under the curve (AUC) during a mixed meal tolerance test (MMTT). Endogenous insulin production was measured at baseline, and then at 3, 6 and 12 months. The primary endpoint was analyzed using a linear mixed ...
PMC10667091
Discussion
T1D, hemolysis, autoimmunity, hypoglycemia
DISEASE PROGRESSION, VIRUS, SIDE EFFECT, PERSISTENT INFECTION, AUTOIMMUNE RESPONSE, INFLAMMATION, HEMOLYSIS, DYSFUNCTION, HYPOGLYCEMIA, SECONDARY, AUTOIMMUNITY, PATHOGENESIS
In this phase 2 trial involving children and adolescents with newly diagnosed T1D, combination therapy with the antiviral drugs pleconaril and ribavirin for 6 months resulted in a higher endogenous insulin production than placebo 12 months from baseline. The treatment was safe and tolerable.Viruses contribute to the pa...
PMC10667091
Methods
PMC10667091
Trial design
T1D, Diabetes
DIABETES
We conducted this phase 2, randomized, placebo-controlled, double-blind, parallel-group clinical trial at two sites: Oslo University Hospital, Norway and Steno Diabetes Center Copenhagen/Herlev University Hospital, Copenhagen, Denmark. Participants were children and adolescents aged 6–15 years with newly diagnosed T1D,...
PMC10667091
Participants and randomization
T1D, hemolytic anemia, cardiac disease, Diabetes
DISEASES, HEMOLYTIC ANEMIA, DISEASE, CARDIAC DISEASE, DIABETES, IMPAIRED RENAL FUNCTION
Participants were aged between 6 and 15 years, had received a diagnosis of stage 3 T1D according to the American Diabetes Association criteriaThe main inclusion criteria, which had to be fulfilled at screening before receiving the study agent, were: (1) diagnosed with T1D (International Statistical Classification of Di...
PMC10667091
Treatment
VIRUS, CHRONIC VIRAL INFECTIONS
Pleconaril and ribavirin were administered as oral solutions (to enable weight-based dosing) at home as separate mixtures for 26 weeks. Combination treatment was chosen to increase and broaden the antiviral effect and to reduce the risk of emergence of drug-resistant virus variants. A 6-month treatment was chosen based...
PMC10667091
Endpoints
The primary endpoint was endogenous insulin production, as assessed according to the 2-h serum C-peptide AUC during an MMTT at 12 months. The AUC was calculated at each visit using the trapezoidal rule on five measurements collected during the 2-h test (at 0, 15, 30, 60 and 90 min, respectively). Secondary endpoints in...
PMC10667091
Laboratory methods
Enterovirus
ENTEROVIRUS
All laboratory analyses were performed with the same methods throughout the trial for all participants. All sampling and biobanking followed the INNODIA Master Protocol and Standard of ProceduresHbA1c was analyzed using international standard methods (coefficient of variantion (CV) 2%). Glycated albumin (%) was determi...
PMC10667091
Statistical analysis
ADVERSE EVENTS, SECONDARY, RECRUITMENT, SENSITIVITY
The sample size calculation is based on the efficacy continuous variable ‘2-h AUC C-peptide’. Because this variable is strongly skewed to the right, a logarithmic transformation (log(Both efficacy and safety analyses included all participants who had received at least one dose of pleconaril and ribavirin or placebo.The...
PMC10667091
Reporting summary
Further information on research design is available in the
PMC10667091
Online content
Any methods, additional references, Nature Portfolio reporting summaries, source data, extended data, supplementary information, acknowledgements, peer review information; details of author contributions and competing interests; and statements of data and code availability are available at 10.1038/s41591-023-02576-1.
PMC10667091
Supplementary information
Supplementary treatment description, Tables 1 and 2 and legend to Extended Data Fig. 1.Reporting Summary
PMC10667091
Extended data
PMC10667091
Extended data
is available for this paper at 10.1038/s41591-023-02576-1.
PMC10667091
Supplementary information
The online version contains supplementary material available at 10.1038/s41591-023-02576-1.
PMC10667091
Acknowledgements
P.
LARSEN, SONNE, JUVENILE DIABETES
We thank the participants and their families for their tremendous efforts during the COVID-19 pandemic lockdowns. We thank our colleagues at the local pediatric departments in Norway for their help recruiting the participants for the trial. We thank the study nurses T. Roald and C. Steinhovden, Oslo University Hospital...
PMC10667091
Author contributions
CRF
VIRUS, CRF
K.D.-J. is the principal investigator of the DiViD studies, including this intervention trial. He conceived the project, drafted the protocol, organized the staff and collaborations with partners, partnered with Apodemus/Curovir AB for the trial, organized the applications to the medicine agencies and ethics committees...
PMC10667091
Peer review
PMC10667091
Data availability
Data collected for the study and presented herein will be made available to others when the end-of-trial reports, after 3 years of follow-up, have been published. Anonymized participant data can be obtained upon reasonable request from the corresponding author. Proposals will be reviewed on the basis of scientific meri...
PMC10667091
Code availability
All the code used for the descriptive tables and the analysis of the primary and safety endpoints is publicly available at
PMC10667091
Competing interests
enterovirus-induced
DISEASES
H.H. is a shareholder and member of the board of Vactech Ltd, which develops vaccines against picornaviruses. N.L. and J.W. are employees of Apodemus/Curovir AB, which own the rights to the commercialization of the results of this trial. Curovir AB is developing antiviral drugs for enterovirus-induced diseases. The oth...
PMC10667091
References
PMC10667091
Keywords
HYPOPLASTIC LEFT HEART SYNDROME
The Single Ventricle Reconstruction (SVR) Trial was a randomized prospective trial designed to determine survival advantage of the modified Blalock-Taussig-Thomas shunt (BTTS) vs the right ventricle to pulmonary artery conduit (RVPAS) for patients with hypoplastic left heart syndrome. The primary aim of the long-term f...
PMC10435402
Introduction
hypoplastic left heart syndrome, valve regurgitation, congenital heart disease
HYPOPLASTIC LEFT HEART SYNDROME
The Single Ventricle Reconstruction (SVR) Trial was the first of its kind—a prospective randomized trial in congenital heart surgery designed to evaluate the effect of systemic to pulmonary shunt type on short-term survival [Cardiac magnetic resonance imaging (CMR) is the gold standard for the assessment of right and l...
PMC10435402
Materials and Methods
inferior vena cava (IVC)/Fontan, PHN, expiratory breath, inspiratory breath
MINOR, PHN
This study is a descriptive design to evaluate the use of CMR in the SVR long-term follow-up cohort using standard CMR methods [As a part of the SVR III cohort study, participants were asked to undergo a non-contrast, non-sedated CMR exam to assess single ventricle function, blood flow in the major venous and arterial ...
PMC10435402
Results
SECONDARY
At the start of SVRIII, 313 patients were eligible for enrollment, and 237 patients enrolled with a median age of 10.76 years [IQR 10.28, 11.35] at the time of consent, Fig. This is a flow chart demonstrating the breakdown of eligible patients enrolled in SVR III and the total number of CMR exams with adequate imaging ...
PMC10435402
Discussion
congenital heart disease, paralysis
GENETIC SYNDROMES
In the long-term follow-up of the Single Ventricle Reconstruction Trial [Earlier data from the SVR Trial suggested that RV function as measured by echocardiography was impaired prior to Fontan surgery in patients who were palliated with a RVPAS versus those who received a mBTTS [While 75% of the patients in this study ...
PMC10435402
Funding
HEART
Open access funding provided by SCELC, Statewide California Electronic Library Consortium.  This study was funded by NIH 1 U10 HL068270, Pediatric Heart Network - NHLBI (Goldberg).
PMC10435402
Declarations
PMC10435402
Conflict of interest
MC Consultant for Longeveron, JD Scientific Advisory Board for Alcor Scientific.
PMC10435402
References
PMC10435402
Summary
Contributed equally.
PMC10275696
Background
The efficacy of on-demand HIV pre-exposure prophylaxis (PrEP) for men in sub-Saharan Africa has not been evaluated, and the on-demand PrEP dosing requirement for insertive sex remains unknown.
PMC10275696
Methods
HIV-negative males 13–24 years, requesting voluntary medical male circumcision (VMMC), were enrolled into an open-label randomised controlled trial (NCT03986970), and randomised 1:1:1:1:1:1:1:1:1 to control arm or one of eight arms receiving emtricitabine-tenofovir disoproxil fumarate (F/TDF) or emtricitabine-tenofovir...
PMC10275696
Findings
144 participants were analysed. PrEP with F/TDF or F/TAF prevented
PMC10275696
Interpretation
A double dose of either F/TDF or F/TAF given once either 5 or 21 h before
PMC10275696
Keywords
PMC10275696
Research in context
PMC10275696
Evidence before this study
We searched PubMed and
PMC10275696
Added value of this study
TAF-FTC
This clinical trial uses Both drugs showed significant PEP activity in foreskin explants however, protection was limited after 48 h, with TAF-FTC providing more durable protection compared to TFV-FTC.
PMC10275696
Implications of all the available evidence
Understanding dosing requirements for insertive sex is necessary to provide guidance for optimal use. The high concentration of protection found in this study of foreskins from men in Uganda and South Africa provides for the data for insertive sex in men from SSA. Our data suggest that on-demand PrEP could be simplifie...
PMC10275696
Methods
PMC10275696
Study design and participants
HIV negative males aged 13–24 years were recruited from VMMC clinics at Chris Hani Baragwanath Academic Hospital, Soweto, South Africa or Entebbe General Hospital, Entebbe, Uganda. Eligibility criteria included being clinically eligible for VMMC, weighing >35 kg, and being able to give written informed consent. Full el...
PMC10275696
Randomization and masking
Participants were randomised in a 1:1:1:1:1:1:1:1:1 ratio to control arm or one of the eight treated arms. Random allocation sequence was generated by an independent statistician using Stata, stratified by country and using block size 9. Participants who were not circumcised following randomisation were excluded accord...
PMC10275696
Ethical approvals
RECRUITMENT
Written informed consent was obtained from all participants aged ≥18 years and emancipated minors (in Uganda); for those <18 years and not emancipated minors, assent with parental consent was obtained. The trial was conducted in accordance with the principles of the Declaration of Helsinki and Good Clinical Practice an...
PMC10275696
Outcomes
SECONDARY
The primary outcome was HIV-1 p24 concentration in participants' foreskin tissue up to day 15 following The secondary outcomes included p24 concentrations in PBMCs; drug concentration in foreskin tissue, foreskin CD4+/CD4-cells, blood (plasma and PBMCs); additional effect of
PMC10275696
Sampling
BLOOD, COLD
Study samples were collected during VMMC visit 5 h or 21 h ± 40 min after the last PrEP dosing. Blood and foreskin tissue, including inner and outer, were collected. VMMC was performed using the dorsal slit method according to local guidelines. Tissue was placed immediately in cold culture media DMEM. Samples were imme...
PMC10275696
Isolation of peripheral blood mononuclear cells
PD
LYSIS
PBMC were isolated by density-gradient centrifugation using Lymphoprep™ (Stem Cell Technologies, Vancouver, Canada) followed by erythrocyte lysis (ACK Lysing buffer, Gibco, Waltham, MA, USA). For pharmacodynamic (PD) analysis, cells were resuspended in RPMI 1640 supplemented with 10% fetal bovine serum (FBS), 2 mM
PMC10275696
Ex vivo challenge of foreskin tissue and PBMCs
infection
INFECTION
Foreskin was cut into 2 mmExplants from the control arm were used as baseline of infection. Additional explants from the control arm were dosed
PMC10275696
Isolation of CD4+ and CD4-cells from foreskin
Foreskin tissue was cut into 4 mm
PMC10275696
Bioanalytical methods
Concentrations (denoted as [drug or metabolite]) of TFV, FTC, and the pro-drug TAF, were measured in plasma and foreskin tissue. Concentrations of the active phosphorylated intracellular metabolites – tenofovir-diphosphate (TFV-DP) and emtricitabine-triphosphate (FTC-TP) were determined in PBMCs, foreskin tissue, and i...
PMC10275696
Statistical analysis
The planned sample size of 144 participants was based on feasibility of conducting this number of experiments based on previous
PMC10275696
Role of the funding source
Funders had no role in the data collection, data analysis or data interpretation. In addition, EDCTP2 and Vetenskapsrådet had no role in study design or writing of the report. All authors had full access to all study data and had final responsibility for the decision to submit for publication.
PMC10275696
Discussion
TAF
HIV INFECTIONS
CHAPS is a trial of on-demand PrEP in young men in Uganda and South Africa, evaluating F/TAF for PrEP and PEP timing and dosing to prevent HIV acquisition after The aim of the study was to provide guidance for dosing requirements and time to protection following starting PrEP. Our primary outcome demonstrated that an o...
PMC10275696
Contributors
CH and JS contributed equally to conceptualisation, formal analysis, investigation, methodology, resources, supervision, validation, writing – original draft; LE: conceptualisation, formal analysis, investigation, methodology, resources, supervision, validation, writing – original draft; LL: project administration, res...
PMC10275696
Data sharing statement
Deidentified participant data and a corresponding data dictionary will be available together with the study protocol, with publication and upon request to the corresponding author. This will be made available on LSHTM Data Compass repository. The Trial Management Group will approve data sharing requests.
PMC10275696
Declaration of interests
CH has received research grants from EDCTP, Vetenskapsrådet and Gilead Sciences. LE has received research grants from EDCTP, and Gilead Sciences. LL has received research grants from EDCTP, Gilead Sciences, Roche Diagnostic, DO has received research grants from EDCTP, AS has received research grants from EDCTP, AP has ...
PMC10275696
References
PMC10275696
Supplementary data
PMC10275696
Supplementary material
PMC10275696
Protocol The CHAPS Trial Version 1.2
PMC10275696
CHAPS Study Team list
PMC10275696
Acknowledgements
We would like to acknowledge all study participants and their parents for their involvement in this study. This study was funded by EDCTP2 programme from the Supplementary data related to this article can be found at
PMC10275696
Background
Ankle sprains
Ankle sprains are frequent injuries in general practice. However, no effective treatment is available yet.
PMC10755994
Aim
lateral ankle sprains
To examine the effectiveness of an unsupervised e-health-supported neuromuscular training programme in combination with usual care compared with usual care alone in patients with acute lateral ankle sprains in general practice.
PMC10755994
Design and setting
lateral ankle sprain
Randomised controlled trial with 1-year follow-up among patients (14–65 years) who visited the GP with an acute lateral ankle sprain within 3 weeks of injury.
PMC10755994
Method
pain
The intervention group received, in addition to usual care, an unsupervised e-health-supported neuromuscular training programme and the control group received usual care alone. The primary outcome was self-reported re-sprains during 52 weeks of follow-up. Secondary outcomes were ankle function, pain in rest and during ...
PMC10755994
Results
SECONDARY
In total, 165 participants (mean age 38.3 years and 69 [41.8%] male) were included. No statistically significant difference in the occurrence of a re-sprain were found between the intervention 20.7% (17/82) and control group 24.1% (20/83) (hazard ratio 1.14, 95% confidence interval = 0.59 to 2.21). Also, no statistical...
PMC10755994
Conclusion
re-sprains
The rate of re-sprains was relatively high and an unsupervised e-health-supported neuromuscular training programme does not yield meaningful effects and does not encourage adherence in preventing re-sprains in patients in general practice. More research is necessary to indicate the best treatment modality and way of de...
PMC10755994
Introduction
injuries of the musculoskeletal, lateral ankle sprains, re-sprains, ankle sprains
RECURRENCE
Acute lateral ankle sprains (LASs) are one of the most common injuries of the musculoskeletal system. The incidence rate in the general population is 2.15 per 1000 person-years in the US, with the highest incidence seen in patients aged between 15 and 24.Given the relatively high risk of re-sprains, effective treatment...
PMC10755994
Method
PMC10755994
Trial design
fits
The trAPP-study was undertaken according to a previously published protocol.How this fits in
PMC10755994
Participants
fracture
EVENTS
Patients with an acute LAS (14–65 years) who visited a GP within the 3 weeks after the injury were eligible for inclusion. Exclusion criteria were a LAS during the previous year, a fracture, or no understanding of the Dutch language. Interested patients were referred to the research team by the GP. Additionally, partic...
PMC10755994
Randomisation procedure
Participants were randomised by a computer-generated randomisation list (block sizes two, four, and six) with a 1:1 allocation ratio, to receive either the app-based programme, in addition to GP-led usual care (intervention group) or GP-led usual care alone (control group). An independent data manager created the rando...
PMC10755994
Interventions
The control group only received GP-led usual care. The content of usual care was based on the Dutch Guideline for GPs and best practice.The intervention group received, in addition to GP-led usual care, an 8-week standardised neuromuscular training programme. The free application ‘Versterk je enkel’ (‘Strengthen your a...
PMC10755994
Outcomes
Ankle Disability, pain
Participants completed, after baseline, online questionnaires at 4, 8, 12, 16, 21, 26, 31, 35, 39, 43, 47, and 52 weeks’ follow-up. The baseline questionnaire included questions on demographics, educational level (‘low’, ‘middle’, or ‘high’), comorbidities, paid job (‘yes, <16 hours’, ‘yes, >16 hours’, or ‘no’), sports...
PMC10755994
Use of co-intervention
pain
AIDS
The use of co-interventions was monitored during follow-up by monthly questionnaires and included information on visits to a healthcare professional (for example, GP, sports physician, specialist) and on self-initiated aids and treatment (for example, pain medication, brace, or taping).
PMC10755994
Adherence
The intervention group completed an extra weekly questionnaire on the number of exercises performed. Adherence was determined by the total number of exercises performed per week during the programme and it was defined by completing ≥75% of the total number of exercises in the programme.
PMC10755994
Sample size
A difference of 19% in the incidence of recurrent LASs between the two groups after 1-year follow-up was considered as clinically relevant.
PMC10755994
Statistical analysis
ankle sprain, pain
REGRESSION, EVENT, RECURRENCE, SECONDARY
Differences between the two groups were analysed following the intention-to-treat principle. Cox regression analysis, with adjustment for sex, was performed for comparing recurrence risk between groups and presented as a hazard ratio (HR) with a 95% confidence interval (CI). The first self-reported recurrent LAS was us...
PMC10755994
Not in line with published protocol
In contrast to what is stated in the previously published study protocol, the cost-effectiveness analysis has not yet been performed, and therefore it is not reported in this manuscript.
PMC10755994
Results
From November 2014 until January 2018, 386 patients were interested in the study (Flowchart of the inclusion of patients in the trAPP-study. Mixed-multilevel model analyses allowed inclusion of all participants who provided outcome data in at least one follow-up measurement. Baseline characteristics of the trAPP-study ...
PMC10755994
Primary outcome
re-sprains
Overall, 22.4% (37/165) of the participants reported one or more re-sprains during follow-up: 24.1% (20/83) in the control group and 20.7% (17/82) in the intervention group. A total of 51 re-sprains were reported by these 37 participants (26 in the intervention and 25 in the control group). There was no statistically s...
PMC10755994
Secondary outcomes
SECONDARY
No differences were observed in any of the secondary outcome measures between the two groups at 12, 26, and 52 weeks (Secondary study outcomes during 1-year follow-up
PMC10755994
Adherence
The number of responses to the specific intervention questionnaires ranged from 74.4% (61/82; week 2) to 51.2% (42/82; weeks 4 and 5). Twenty participants (20/82; 24.4%) were adherent with the intervention programme (that is, performed ≥75% of total prescribed exercises) and only five participants (5/82; 6.1%) were com...
PMC10755994
Discussion
PMC10755994
Summary
In this trial, the effectiveness of an unsupervised e-health-supported training programme, in addition to GP-led usual care, was examined among 165 participants with a LAS in general practice. In contrast with Hupperets
PMC10755994
Strengths and limitations
RECURRENCE
The current study is a high-quality pragmatic RCT evaluating an e-health intervention in addition to usual care about the recurrence rate of LASs in general practice. Nevertheless, some limitations need to be addressed. First, the number of included participants was lower than anticipated and the loss to follow-up was ...
PMC10755994
Comparison with existing literature
ankle sprain, ankle injuries, pain
To the authors’ knowledge, this is the first RCT to evaluate an unsupervised e-health-supported training programme for patients with an acute LAS in general practice. Rehabilitation programmes for ankle injuries, such as proprioceptive training programmes, have been studied previously in both general and sport populati...
PMC10755994
Implications for research and practice
sprain
An unsupervised e-health supported neuromuscular training programme, in addition to GP-led usual care, in its current form is not effective and does not encourage adherence in the prevention of a recurrent sprain during 1 year of follow-up in patients with an acute LAS in general practice. More research is needed to de...
PMC10755994