PIONEER
PravastatIn tO preveNt prEtErm biRth (PIONEER): a
parallel group randomised placebo-controlled trial
Chief Investigator
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Institution
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Dates
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Funding Stream
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Grant Ref
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Amount
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Dr Katherine Birchenall
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University Hospitals Bristol and Weston NHS Foundation
Trust
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01/08/2023 - 31/07/2028 (60 months)
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NIHR Efficacy and Mechanism Evaluation
(EME)
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NIHR152798
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£2,673,531
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Summary
Research question:
1)Is Pravastatin better than placebo at
extending gestational length of pregnancy?
2)If so, does Pravastatin have this effect
through changes to the cervicovaginal inflammatory profile, vaginal
and gut microbiota or serum lipids?
Background:
Preterm birth (PTB) is a global concern. In
2019, nearly one million children under 5 years died due to
complications of prematurity. Risk for poor outcome is inversely
proportional to gestational age (GA) at birth. Rates of PTB have
not significantly altered over recent
decades.
Aims and
objectives:
1)Evaluate the efficacy of Pravastatin versus
placebo in reducing PTB in those at risk:
a)Assess difference between groups in mean GA
(days) at birth;
b)Estimate difference between groups with
respect to a range of secondary outcomes.
2)Investigate mechanisms via which Pravastatin
prevents PTB by assessing the effect of Pravastatin vs placebo on
maternal:
a)Cervicovaginal inflammatory
profile;
b)Vaginal and gut
microbiota;
c)Serum lipid
profile.
Design:
Multi-site parallel group placebo-controlled
RCT with blinding and internal pilot, with integrated monitoring
and feedback to maximise recruitment and adherence to study
medication, with an embedded mechanistic investigation, and a
QuinteT Recruitment Intervention to optimise recruitment and
safeguard informed consent. Population: Pregnant people referred to
PTB prevention clinics across the UK. Intervention: 20mg
Pravastatin every day, from the 2nd trimester until 37+0 weeks or
birth. Comparator: Placebo, taken as intervention. Primary outcome:
GA at birth. Secondary outcomes: Defined maternal and neonatal core
set for evaluation of PTB prevention interventions; childhood
assessment at 2 years corrected age via PARCA-R
questionnaire.
Mechanistic
sub-study: For 250 participants,
mechanistic studies will assess maternal: 1)Cervicovaginal
fluid inflammatory profile; 2)Vaginal microbiota profile; 3)Serum
lipid profile; 4)Stool microbiota profile.
Sample size: A difference of one week in GA is the minimum
clinicians require to consider interventions as effective in PTB
prevention. The estimated standard deviation (4.2 weeks) was taken
from the OPPTIMUM trial of Progesterone prophylaxis for PTB. A
sample size of 750 participants (375 per group), allows an effect
size of 0.24 to be detected with 90% power at the 5% 2-tailed
significance level, assuming complete data for primary outcome and
treatment compliance. Analyses: Conducted according to
intention-to-treat, following CONSORT reporting guidelines.
Randomised participants who do not complete treatment will be
included in the primary analysis. All models will compare treatment
groups and adjust for site and previous PTB. Sensitivity analyses,
restricted to participants who take at least 80% of the study
medication will be conducted. Subgroup analysis to investigate
whether prior large loop excision of the transformation zone
(LLETZ) procedure affects Pravastatin efficacy will be performed.
Timelines for delivery: Total duration 60 months (m), comprising:
set up (8m); internal pilot (12m); main trial recruitment (24m);
follow-up (8m), analysis, reporting and archiving (8m). An internal
pilot phase will include the first 12m of recruitment, and
progression criteria will need to have been met to ensure
continuation to the main study. Impact: If this study shows that
low-cost Pravastatin increases GA at birth when compared with
placebo, this study could change management of PTB and reduce PTB
rates globally.
Further Information
https://www.fundingawards.nihr.ac.uk/award/NIHR152798