Clinical trial dataset supporting: Mavodelpar in Patients with Primary Mitochondrial Myopathy: A Phase 1 Trial

This dataset includes the clinical trial dataset and clinical trial documentation supporting: Mavodelpar in Patients with Primary Mitochondrial Myopathy: A Phase 1 Trial (Stefanetti et al.)DetailsAuthor: Stefanetti et al.Contact: [e-mail] registration: ClinicalTrials.gov ID: NCT03862846Raw Data Files:The following files underlying the clinical trial data (.excel files) are included:1_NMDAS. Raw data for: Table S1: Baseline Disease Characteristics2_CK and Aldolase. Raw data for: Fig 2: Evaluation of plasma biochemistry3_Pharmacokinetics. Raw data for: Pharmacokinetic (PK) analyses4_TEAEs. Raw data for: Table 1: Summary of Treatment-Emergent Adverse Events– Part A; Table 2: Most Frequent (≥2 participants (5 %)) TEAEs by System Organ Class and Preferred Term – Part A; Table S2: Treatment-Emergent Adverse Events (TEAEs) classified as Gastrointestinal (GI) Disorders (System Organ Class) for the Full Analysis Set – Part A; Table S3: Adverse Events Leading to Interruption in Dosing or Discontinuation of Mavodelpar (REN001) During Parts A or B).5_Peak exercise test_CWR_12MWT_STS. Raw data for: Fig 3: Exploratory efficacy outcomes with improvement; Table 3: Summary of Efficacy Exploratory Endpoints.6_NMDAS_SF-36_MFIS_BPI. Raw data for: Fig 3: Exploratory efficacy outcomes with improvement; Table 3: Summary of Efficacy Exploratory Endpoints.7_Vital signs_ECG. Raw data for: Table S4: Vital signs and ECGs.8_Laboratory Parameters. Raw data for: Table S5: Laboratory Parameters9_Serum Biomarkers_Acylcarnitine. Raw data for: Table S7: Pharmacodynamic (PD) Endpoints10_MFIS. Raw data for: Table S13: Modified Fatigue Impact11_BPI. Raw data for: Table S14: Brief Pain Inventory (BPI) – (Short Form)RNA Sequencing DataRNA sequencing data are deposited in the NCBI Sequence Read Archive under accession number: PRJNA1044583 - Study DocumentationThe following clinical trial study documents (PDF files) are included:1_REN001-101_Clinical Study Protocol V1.0_07Aug2018 – Version 1.0 (7th August 2018).2_REN001-101_Clinical Study Protocol_V2.0_Amendment 1.0_15May2019 – Amendment 1, Version 2.0 (15th May 2019).3_REN001-101_Clinical Study Protocol_V3.0_Amendment 2.0_07Aug2019 – Amendment 2, Version 3.0 (7th August 2019).4_REN001_101_Clinical Protocol V4.0_Amendment 3.0_11Nov19 – Amendment 3, Version 4.0 (11th November 2019).5_REN001-101_SAP_V1.0_07Mar2019 – Version 1.0 (7th March 2019).6_REN001-101_SAP_V2.0_03Dec2020 – Version: 2.0 (3rd December 2020).7_Summary of REN001-101 Protocol Amendments.8_Participant Information Sheet9_Informed Consent FormData RetentionData will remain publicly available for the lifetime of the repository.