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NCT03610334

A Combined SAD and MAD Study to Investigate the Safety, Tolerability and Pharmacokinetic Profile of IFB-088

Completed Phase 1 Results posted Last updated 26 January 2022
What this trial tests

Phase 1 trial testing IFB-088 (2.5-60.0mg) oral capsule in Healthy Volunteers in 72 participants. Completed in 16 December 2019.

Timeline
21 June 2018
Primary endpoint
21 June 2019
16 December 2019

Quick facts

Lead sponsorInFlectis BioScience
PhasePhase 1
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designparallel
Maskingquadruple
Primary purposeother
Enrollment72
Start date21 June 2018
Primary completion21 June 2019
Estimated completion16 December 2019
Sites2 locations across France

Drugs / interventions tested

Conditions studied

Sponsor

InFlectis BioScience — full company profile →

Who can join

Adults 18 to 40, male only, with Healthy Volunteers. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Participants With Treatment Emergent Adverse Events Per Group Primary · SAD & MAD phases: Screening visit to End of study visit (7 to 14 days after last dosing) + 30 days

This safety outcome lists the number of subjects experiencing adverse events (AEs), whether not related, possibly or unlikely related to the study treatment. As all the parameters are developped in the pharmacovigilance section, please do refer to that section for further details.

GroupValue95% CI
SAD IFB-088 2.5mg1
SAD Placebo 2.5mg0
SAD IFB-088 5.0mg1
SAD Placebo 5.0mg1
SAD IFB-088 10.0mg2
SAD Placebo 10.0mg0
SAD IFB-088 20.0mg2
SAD Placebo 20.0mg0
SAD IFB-088 40.0mg2
SAD Placebo 40.0mg1
SAD IFB-088 60.0mg3
SAD Placebo 60.0mg0
Number of Participants With Change in Concomitant Medications Secondary · SAD & MAD phases: Continuous (Screening visit to End of study visit (7 to 14 days after last dosing) + 30 days)

modification in Concomitant medication(s) occuring during the study (if applicable)

GroupValue95% CI
SAD IFB-088 2.5mg0
SAD Placebo 2.5mg0
SAD IFB-088 5.0mg0
SAD Placebo 5.0mg0
SAD IFB-088 10.0mg0
SAD Placebo 10.0mg0
SAD IFB-088 20.0mg1
SAD Placebo 20.0mg0
SAD IFB-088 40.0mg0
SAD Placebo 40.0mg0
SAD IFB-088 60.0mg0
SAD Placebo 60.0mg0
SAD IFB-088 2.5mg0
SAD Placebo 2.5mg0
SAD IFB-088 5.0mg0
SAD Placebo 5.0mg0
SAD IFB-088 10.0mg0
SAD Placebo 10.0mg0
SAD IFB-088 20.0mg0
SAD Placebo 20.0mg0
SAD IFB-088 40.0mg1
SAD Placebo 40.0mg0
SAD IFB-088 60.0mg0
SAD Placebo 60.0mg0
SAD IFB-088 2.5mg0
SAD Placebo 2.5mg0
SAD IFB-088 5.0mg0
SAD Placebo 5.0mg0
SAD IFB-088 10.0mg0
SAD Placebo 10.0mg0
SAD IFB-088 20.0mg0
SAD Placebo 20.0mg0
SAD IFB-088 40.0mg0
SAD Placebo 40.0mg0
SAD IFB-088 60.0mg1
SAD Placebo 60.0mg0
SAD IFB-088 2.5mg0
SAD Placebo 2.5mg0
SAD IFB-088 5.0mg0
SAD Placebo 5.0mg0
SAD IFB-088 10.0mg0
SAD Placebo 10.0mg0
SAD IFB-088 20.0mg0
SAD Placebo 20.0mg0
SAD IFB-088 40.0mg0
SAD Placebo 40.0mg0
SAD IFB-088 60.0mg1
SAD Placebo 60.0mg0
Pharmacokinetic: Maximum Observed Plasma Concentration (Cmax) Secondary · Starting 1 hour prior to dosing on Day 1 and until 72 hours after last dosing (Day 17)

Plasma samples are collected: SAD phase - cohort 1 (one intake for daily administration): Day 1 at predose, 0.16, 0.33, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 5, 8, 12, 24, 32 hours SAD phase - cohort 2 to 6 (two intakes for daily administration): Day 1 at predose, 0.33, 0.66, 1, 2, 3, 4, 5, 12, 12.25, 12.5, 13, 14, 16, 18, 24, 32 hours MAD phase (two intakes for daily administration): Day 1 and Day 14 at predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 13, 14, 17, 19 and 21h ; Day 2 (24 hours); Day 7 (predose, 1h and 2 h post dose); Day 15 (24, 30, 36 and 42 hours after Day 14); Day 16 (48, 60 hours afte

Cmax Dose 1
GroupValue95% CI
SAD IFB-088 2.5mg0.54± 34.5
SAD IFB-088 5.0mg0.45± 62.3
SAD IFB-088 10.0mg1.19± 75.5
SAD IFB-088 20.0mg1.39± 16.3
SAD IFB-088 40.0mg2.93± 35.2
SAD IFB-088 60.0mg4.26± 46.3
MAD IFB-088 15.0mg1.70± 87.1
MAD IFB-088 30.0mg2.52± 54.0
MAD IFB-088 50.0mg4.38± 49.8
Cmax Dose 2
GroupValue95% CI
SAD IFB-088 5.0mg0.37± 59.7
SAD IFB-088 10.0mg1.13± 44.2
SAD IFB-088 20.0mg1.52± 34.3
SAD IFB-088 40.0mg2.39± 31.2
SAD IFB-088 60.0mg4.52± 50.6
MAD IFB-088 15.0mg1.61± 47.2
MAD IFB-088 30.0mg3.58± 41.1
MAD IFB-088 50.0mg6.92± 52.3
Pharmacokinetic: Time to Reach the Maximum Concentration in Plasma (Tmax) Secondary · Starting 1 hour prior to dosing on Day 1 and until 72 hours after last dosing (Day 17)

Plasma samples are collected: SAD phase - cohort 1 (one intake for daily administration): Day 1 at predose, 0.16, 0.33, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 5, 8, 12, 24, 32 hours SAD phase - cohort 2 to 6 (two intakes for daily administration): Day 1 at predose, 0.33, 0.66, 1, 2, 3, 4, 5, 12, 12.25, 12.5, 13, 14, 16, 18, 24, 32 hours MAD phase (two intakes for daily administration): Day 1 and Day 14 at predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 13, 14, 17, 19 and 21h ; Day 2 (24 hours); Day 7 (predose, 1h and 2 h post dose); Day 15 (24, 30, 36 and 42 hours after Day 14); Day 16 (48, 60 hours afte

Tmax Dose 1
GroupValue95% CI
SAD IFB-088 2.5mg1.51.0 – 1.5
SAD IFB-088 5.0mg1.50.7 – 2.0
SAD IFB-088 10.0mg2.01.0 – 4.0
SAD IFB-088 20.0mg2.01.0 – 3.0
SAD IFB-088 40.0mg1.00.7 – 2.0
SAD IFB-088 60.0mg2.01.0 – 3.0
MAD IFB-088 15 mg2.01.0 – 2.0
MAD IFB-088 30 mg2.01.0 – 3.0
MAD IFB-088 50 mg2.01.0 – 3.0
Tmax Dose 2
GroupValue95% CI
SAD IFB-088 5.0mg2.01.0 – 4.0
SAD IFB-088 10.0mg2.02.0 – 2.0
SAD IFB-088 20.0mg2.01.0 – 2.0
SAD IFB-088 40.0mg1.01.0 – 2.0
SAD IFB-088 60.0mg2.00.5 – 4.0
MAD IFB-088 15 mg2.02.0 – 3.0
MAD IFB-088 30 mg2.02.0 – 3.0
MAD IFB-088 50 mg2.01.0 – 3.0
Pharmacokinetic: Terminal Half-life (t1/2) Secondary · Starting 1 hour prior to dosing on Day 1 and until 72 hours after last dosing (Day 17)

Plasma samples are collected: SAD phase - cohort 1 (one intake for daily administration): Day 1 at predose, 0.16, 0.33, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 5, 8, 12, 24, 32 hours SAD phase - cohort 2 to 6 (two intakes for daily administration): Day 1 at predose, 0.33, 0.66, 1, 2, 3, 4, 5, 12, 12.25, 12.5, 13, 14, 16, 18, 24, 32 hours MAD phase (two intakes for daily administration): Day 1 and Day 14 at predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 13, 14, 17, 19 and 21h ; Day 2 (24 hours); Day 7 (predose, 1h and 2 h post dose); Day 15 (24, 30, 36 and 42 hours after Day 14); Day 16 (48, 60 hours afte

GroupValue95% CI
SAD IFB-088 2.5mg4.64± 33.3
SAD IFB-088 5.0mg4.61± 56.5
SAD IFB-088 10.0mg5.92± 33.8
SAD IFB-088 20.0mg6.21± 26.9
SAD IFB-088 40.0mg5.76± 10.8
SAD IFB-088 60.0mg5.17± 11.8
MAD IFB-088 15 mg6.97± 33.1
MAD IFB-088 30 mg5.70± 22.9
MAD IFB-088 50 mg6.34± 28.7
Pharmacokinetic: Area Under Plasma Concentration-time Curve From Hour 0 to Last Sample With Measurable Plasma Concentrations (AUClast) Secondary · Starting 1 hour prior to dosing on Day 1 and until 72 hours after last dosing (Day 17)

Plasma samples are collected: SAD phase - cohort 1 (one intake for daily administration): Day 1 at predose, 0.16, 0.33, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 5, 8, 12, 24, 32 hours SAD phase - cohort 2 to 6 (two intakes for daily administration): Day 1 at predose, 0.33, 0.66, 1, 2, 3, 4, 5, 12, 12.25, 12.5, 13, 14, 16, 18, 24, 32 hours MAD phase (two intakes for daily administration): Day 1 and Day 14 at predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 13, 14, 17, 19 and 21h ; Day 2 (24 hours); Day 7 (predose, 1h and 2 h post dose); Day 15 (24, 30, 36 and 42 hours after Day 14); Day 16 (48, 60 hours afte

GroupValue95% CI
SAD IFB-088 2.5mg2.25± 50.6
SAD IFB-088 5.0mg3.80± 95.2
SAD IFB-088 10.0mg12.5± 47.9
SAD IFB-088 20.0mg17.4± 41.7
SAD IFB-088 40.0mg27.4± 32.5
SAD IFB-088 60.0mg51.1± 40.0
MAD IFB-088 15 mg18.7± 49.5
MAD IFB-088 30 mg40.8± 35.7
MAD IFB-088 50 mg77.2± 48.8
Pharmacokinetic: Apparent Volume of Distribution (Vd/F) Secondary · Starting 1 hour prior to dosing on Day 1 and until 72 hours after last dosing (Day 17)

Plasma samples are collected: SAD phase - cohort 1 (one intake for daily administration): Day 1 at predose, 0.16, 0.33, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 5, 8, 12, 24, 32 hours SAD phase - cohort 2 to 6 (two intakes for daily administration): Day 1 at predose, 0.33, 0.66, 1, 2, 3, 4, 5, 12, 12.25, 12.5, 13, 14, 16, 18, 24, 32 hours MAD phase (two intakes for daily administration): Day 1 and Day 14 at predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 13, 14, 17, 19 and 21h ; Day 2 (24 hours); Day 7 (predose, 1h and 2 h post dose); Day 15 (24, 30, 36 and 42 hours after Day 14); Day 16 (48, 60 hours afte

GroupValue95% CI
SAD IFB-088 10.0mg5093± 16.9
SAD IFB-088 20.0mg10055± 33.2
SAD IFB-088 40.0mg10408± 16.4
SAD IFB-088 60.0mg10550± 65.5
MAD IFB-088 15 mg8916± 41.1
MAD IFB-088 30 mg6860± 43.0
MAD IFB-088 50 mg7042± 54.5
Pharmacokinetic: Apparent Total Body Clearance (CL/F) Secondary · Starting 1 hour prior to dosing on Day 1 and until 72 hours after last dosing (Day 17)

Plasma samples are collected: SAD phase - cohort 1 (one intake for daily administration): Day 1 at predose, 0.16, 0.33, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 5, 8, 12, 24, 32h SAD phase - cohort 2 to 6 (two intakes for daily administration): Day 1 at predose, 0.33, 0.66, 1, 2, 3, 4, 5, 12, 12.25, 12.5, 13, 14, 16, 18, 24, 32h MAD phase (two intakes for daily administration): Day 1 and Day 14 at predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 13, 14, 17, 19 and 21h ; Day 2 (24 hours); Day 7 (predose, 1h and 2 h post dose); Day 15 (24, 30, 36 and 42 hours after Day 14); Day 16 (48, 60 hours after Day 14).

GroupValue95% CI
SAD IFB-088 10.0mg736± 43.0
SAD IFB-088 20.0mg1146± 45.1
SAD IFB-088 40.0mg1279± 13.9
SAD IFB-088 60.0mg1388± 61.6
MAD IFB-088 15 mg926± 34.7
MAD IFB-088 30 mg826± 38.3
MAD IFB-088 50 mg843± 28.0
Pharmacokinetic: Renal Clearance (CLr) Secondary · Starting 1 hour prior to dosing on Day 1 and until 48 hours after last dosing (Day 16)

Urine samples are collected: SAD phase - cohort 1 (one intake for daily administration): Day 1 at predose, 0-4 hours, 4-8 hours, 8-16 hours,16-32 hours SAD phase - cohort 2 to 6 (two intakes for daily administration): Day 1 at predose, 0-4, 4-8, 8-12, 12-24, 24-32 hours MAD phase (two intakes for daily administration): Day 1 and Day 14: predose, 0-4, 4-12, 12-24 hours; Day 6: predose, 0-4 and 4-12 hours; Day 15: 24-36, 36-48 hours after Day 14

GroupValue95% CI
SAD IFB-088 5.0mg167± 55.8
SAD IFB-088 10.0mg75.3± 44.6
SAD IFB-088 20.0mg117± 22.3
SAD IFB-088 40.0mg106± 32.4
SAD IFB-088 60.0mg60.5± 37.6
MAD IFB-088 15 mg76.6± 34.0
MAD IFB-088 30 mg84.4± 45.0
MAD IFB-088 50 mg66.2± 36.4
Pharmacokinetic: Percent of Drug Recovered in Urine (Ae %Dose) Secondary · Starting 1 hour prior to dosing on Day 1 and until 48 hours after last dosing (Day 16)

Urine samples are collected: SAD phase - cohort 1 (one intake for daily administration): Day 1 at predose, 0-4 hours, 4-8 hours, 8-16 hours,16-32 hours SAD phase - cohort 2 to 6 (two intakes for daily administration): Day 1 at predose, 0-4, 4-8, 8-12, 12-24, 24-32 hours MAD phase (two intakes for daily administration): Day 1 and Day 14: predose, 0-4, 4-12, 12-24 hours; Day 6: predose, 0-4 and 4-12 hours; Day 15: 24-36, 36-48 hours after Day 14

GroupValue95% CI
SAD IFB-088 5.0mg0.52± 67.7
SAD IFB-088 10.0mg0.55± 49.6
SAD IFB-088 20.0mg0.61± 46.2
SAD IFB-088 40.0mg0.44± 44.9
SAD IFB-088 60.0mg0.29± 39.7
MAD IFB-088 15 mg0.61± 52.5
MAD IFB-088 30 mg0.74± 56.8
MAD IFB-088 50 mg0.63± 39.7
Number of Participants With Clinically Significant Change in Physical Evaluation During the Study Secondary · SAD & MAD phases: Screening; Day 2 (SAD) or Day 17 (MAD); and at End of study visit (7 to 14 days after last dosing)

the following parameters are assessed during the physical evaluation visit: Cardiovascular system (see specific outcome measure), Digestive system (included spleen organ), alcohol consumption (Ethylotest), General condition, Liver and biliary tracts, Lymphatic system, Muco-cutaneous system, Neck/Thyroide, Nervous system, ENTsystem, Respiratory system, Visual system

GroupValue95% CI
SAD IFB-088 2.5mg0
SAD Placebo 2.5mg0
SAD IFB-088 5.0mg0
SAD Placebo 5.0mg0
SAD IFB-088 10.0mg0
SAD Placebo 10.0mg0
SAD IFB-088 20.0mg0
SAD Placebo 20.0mg0
SAD IFB-088 40.0mg0
SAD Placebo 40.0mg0
SAD IFB-088 60.0mg0
SAD Placebo 60.0mg0
SAD IFB-088 2.5mg0
SAD Placebo 2.5mg0
SAD IFB-088 5.0mg0
SAD Placebo 5.0mg0
SAD IFB-088 10.0mg0
SAD Placebo 10.0mg0
SAD IFB-088 20.0mg0
SAD Placebo 20.0mg0
SAD IFB-088 40.0mg1
SAD Placebo 40.0mg0
SAD IFB-088 60.0mg0
SAD Placebo 60.0mg0
SAD IFB-088 2.5mg0
SAD Placebo 2.5mg0
SAD IFB-088 5.0mg0
SAD Placebo 5.0mg0
SAD IFB-088 10.0mg0
SAD Placebo 10.0mg0
SAD IFB-088 20.0mg0
SAD Placebo 20.0mg0
SAD IFB-088 40.0mg0
SAD Placebo 40.0mg0
SAD IFB-088 60.0mg0
SAD Placebo 60.0mg0
SAD IFB-088 2.5mg0
SAD Placebo 2.5mg0
SAD IFB-088 5.0mg0
SAD Placebo 5.0mg0
SAD IFB-088 10.0mg0
SAD Placebo 10.0mg0
SAD IFB-088 20.0mg0
SAD Placebo 20.0mg0
SAD IFB-088 40.0mg0
SAD Placebo 40.0mg0
SAD IFB-088 60.0mg0
SAD Placebo 60.0mg0
Number of Participants With Clinically Significant Change in Physiological Parameters During the Study Secondary · SAD & MAD phases: Screening; Day 2 (SAD) or Day 17 (MAD); and at End of study visit (7 to 14 days after last dosing)

Weight measured in kg and height measured in cm, were taken and Body Mass Index was calculated using those 2 values

GroupValue95% CI
SAD IFB-088 2.5mg0
SAD Placebo 2.5mg0
SAD IFB-088 5.0mg0
SAD Placebo 5.0mg0
SAD IFB-088 10.0mg0
SAD Placebo 10.0mg0
SAD IFB-088 20.0mg0
SAD Placebo 20.0mg0
SAD IFB-088 40.0mg0
SAD Placebo 40.0mg0
SAD IFB-088 60.0mg0
SAD Placebo 60.0mg0
SAD IFB-088 2.5mg0
SAD Placebo 2.5mg0
SAD IFB-088 5.0mg0
SAD Placebo 5.0mg0
SAD IFB-088 10.0mg0
SAD Placebo 10.0mg0
SAD IFB-088 20.0mg0
SAD Placebo 20.0mg0
SAD IFB-088 40.0mg0
SAD Placebo 40.0mg0
SAD IFB-088 60.0mg0
SAD Placebo 60.0mg0
SAD IFB-088 2.5mg6
SAD Placebo 2.5mg2
SAD IFB-088 5.0mg6
SAD Placebo 5.0mg2
SAD IFB-088 10.0mg6
SAD Placebo 10.0mg2
SAD IFB-088 20.0mg6
SAD Placebo 20.0mg2
SAD IFB-088 40.0mg6
SAD Placebo 40.0mg2
SAD IFB-088 60.0mg6
SAD Placebo 60.0mg2

Adverse events — posted to ClinicalTrials.gov

Time frame: 18 months. Reporting threshold: 1%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

SAD IFB-088 2.5mg
Serious: 0/6 (0%)
Deaths: 0/6
SAD IFB-088 5.0mg
Serious: 0/6 (0%)
Deaths: 0/6
SAD IFB-088 10.0mg
Serious: 0/6 (0%)
Deaths: 0/6
SAD IFB-088 20.0mg
Serious: 0/6 (0%)
Deaths: 0/6
SAD IFB-088 40.0mg
Serious: 0/6 (0%)
Deaths: 0/6
SAD IFB-088 60.0mg
Serious: 0/6 (0%)
Deaths: 0/6
SAD Placebo
Serious: 0/12 (0%)
Deaths: 0/12
MAD IFB-088 15.0mg
Serious: 0/6 (0%)
Deaths: 0/6
MAD IFB-088 30.0mg
Serious: 0/6 (0%)
Deaths: 0/6
MAD IFB-088 50.0mg
Serious: 0/6 (0%)
Deaths: 0/6
MAD Placebo
Serious: 0/6 (0%)
Deaths: 0/6
Other adverse events (19 terms — click to expand)

ReactionSystemSAD IFB-088 2.5mgSAD IFB-088 5.0mgSAD IFB-088 10.0mgSAD IFB-088 20.0mgSAD IFB-088 40.0mgSAD IFB-088 60.0mgSAD PlaceboMAD IFB-088 15.0mgMAD IFB-088 30.0mgMAD IFB-088 50.0mgMAD Placebo
headacheNervous system disorders———————————
hypertriglyceridemiaMetabolism and nutrition disorders———————————
nauseaGastrointestinal disorders———————————
pyrexiaGeneral disorders———————————
catheter site related reactionGeneral disorders———————————
influenza like illnessGeneral disorders———————————
nasopharyngitisInfections and infestations———————————
toothacheGastrointestinal disorders———————————
joint injuryInjury, poisoning and procedural complications———————————
diarrhoeaGastrointestinal disorders———————————
rhinitis allergicRespiratory, thoracic and mediastinal disorders———————————
ear painEar and labyrinth disorders———————————
orthostatic hypotensionVascular disorders———————————
nephrolithiasisRenal and urinary disorders———————————
haematuriaRenal and urinary disorders———————————
pollakyuriaRenal and urinary disorders———————————
paraesthesiaNervous system disorders———————————
abdominal painGastrointestinal disorders———————————
presyncopeNervous system disorders———————————

Data from ClinicalTrials.gov NCT03610334 adverse events section.

Sponsor's own description

This is the first study of single and multiple doses of IFB-088 in human subjects. The current study is designed to assess in the first part, the safety, tolerability, plasma and urine pharmacokinetics (PK) of single oral doses of IFB-088 in healthy subjects (Single Ascending Doses - SAD) and in a second part safety, tolerability, plasma and urine pharmacokinetics (PK) of multiple oral doses of IFB-088 in healthy subjects (Multiple Ascending Doses - MAD)

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Turn and Face the Strange: A New View on Phosphatases.
    Köhn M. · · 2020 · cited 80× · PMID 32341996 · DOI 10.1021/acscentsci.9b00909
  2. AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A.
    Gautier B, Hajjar H, Soares S, Berthelot J, et al · · 2021 · cited 59× · PMID 33883545 · DOI 10.1038/s41467-021-22593-3
  3. Emerging Therapies for Charcot-Marie-Tooth Inherited Neuropathies.
    Stavrou M, Sargiannidou I, Georgiou E, Kagiava A, et al · · 2021 · cited 52× · PMID 34205075 · DOI 10.3390/ijms22116048
  4. The unfolded protein response in amyotrophic later sclerosis: results of a phase 2 trial.
    Dalla Bella E, Bersano E, Antonini G, Borghero G, et al · · 2021 · cited 49× · PMID 33905493 · DOI 10.1093/brain/awab167
  5. Targeting Phosphatases and Kinases: How to Checkmate Cancer.
    Turdo A, D'Accardo C, Glaviano A, Porcelli G, et al · · 2021 · cited 40× · PMID 34778245 · DOI 10.3389/fcell.2021.690306
  6. Cellular responses to halofuginone reveal a vulnerability of the GCN2 branch of the integrated stress response.
    Pitera AP, Szaruga M, Peak-Chew SY, Wingett SW, et al · · 2022 · cited 21× · PMID 35466425 · DOI 10.15252/embj.2021109985
  7. Mechanisms and treatment strategies of demyelinating and dysmyelinating Charcot-Marie-Tooth disease.
    Hertzog N, Jacob C. · · 2023 · cited 17× · PMID 36926710 · DOI 10.4103/1673-5374.367834
  8. Therapeutic Development in Charcot Marie Tooth Type 1 Disease.
    Miniou P, Fontes M. · · 2021 · cited 11× · PMID 34201736 · DOI 10.3390/ijms22136755

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