Asset Details
Do you wish to reserve the book?
StratosPHere 2: statistical analysis plan for a response-adaptive randomised placebo-controlled phase II trial to evaluate hydroxychloroquine and phenylbutyrate in pulmonary arterial hypertension caused by mutations in BMPR2
Hey, we have placed the reservation for you!
By the way, why not check out events that you can attend while you pick your title.
Oops! Something went wrong.
Looks like we were not able to place the reservation. Kindly try again later.
Are you sure you want to remove the book from the shelf?
StratosPHere 2: statistical analysis plan for a response-adaptive randomised placebo-controlled phase II trial to evaluate hydroxychloroquine and phenylbutyrate in pulmonary arterial hypertension caused by mutations in BMPR2
Do you wish to request the book?
StratosPHere 2: statistical analysis plan for a response-adaptive randomised placebo-controlled phase II trial to evaluate hydroxychloroquine and phenylbutyrate in pulmonary arterial hypertension caused by mutations in BMPR2
Please be aware that the book you have requested cannot be checked out. If you would like to checkout this book, you can reserve another copy
We have requested the book for you!
Your request is successful and it will be processed during the Library working hours. Please check the status of your request in My Requests.
Oops! Something went wrong.
Looks like we were not able to place your request. Kindly try again later.
Journal Article
StratosPHere 2: statistical analysis plan for a response-adaptive randomised placebo-controlled phase II trial to evaluate hydroxychloroquine and phenylbutyrate in pulmonary arterial hypertension caused by mutations in BMPR2
2025
Overview
Background
The StratosPHere 2 trial will evaluate the efficacy of hydroxychloroquine and phenylbutyrate in pulmonary arterial hypertension caused by mutations in BMPR2 by focussing on the novel biomarker and other endpoints including safety.
Study design
StratosPHere 2 is a three armed, placebo-controlled, phase 2 trial with two strata based on the mutation groups. It is response adaptive where the allocation of treatments follows a Bayesian response-adaptive randomisation algorithm. An expected number of 20 patients will be randomised in each stratum to one of the three arms containing hydroxychloroquine, phenylbutyrate and placebo. The primary outcome is a novel endpoint considering the change in the bone morphogenetic receptor type 2 (BMPR2).
Method
The final primary analysis on the efficacy of each active treatment against control is assessed using a one-sided nonparametric Wilcoxon test computed on the continuous biomarker data collected up to 8 weeks from the start of treatment.
Discussion
This manuscript presents the key elements of the StratosPHere 2 implementation and statistical analysis plan. This is submitted to the journal before the first interim analysis to preserve the scientific integrity under a response-adaptive design framework.
The StratosPHere 2 trial closely follows published guidelines for the content of Statistical Analysis Plans in clinical trials.
Trial registration
The ISRCTN Registry ISRCTN10304915 (22/09/2023)
Publisher
BioMed Central,BioMed Central Ltd,Springer Nature B.V,BMC
Subject
/ Analysis
/ Antihypertensive Agents - adverse effects
/ Antihypertensive Agents - therapeutic use
/ Arterial Pressure - drug effects
/ Arterial Pressure - genetics
/ Bone Morphogenetic Protein Receptors, Type II - genetics
/ Clinical Trials, Phase II as Topic
/ Humans
/ Hydroxychloroquine - adverse effects
/ Hydroxychloroquine - therapeutic use
/ Medicine
/ Mutation
/ Patients
/ Phenylbutyrates - adverse effects
/ Phenylbutyrates - therapeutic use
/ Pulmonary Arterial Hypertension - drug therapy
/ Pulmonary Arterial Hypertension - genetics
/ Pulmonary Arterial Hypertension - physiopathology
/ Randomized Controlled Trials as Topic
/ Statistics for Life Sciences
/ Update
