Phase 1, FiH study to assess Safety, Tolerability and PK of NMD1343 in HV
Research type
Research Study
Full title
A Phase 1 Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of oral NMD1343 in Healthy Male and Female Volunteers
IRAS ID
1006840
Contact name
Thomas Petersen
Contact email
Sponsor organisation
NMD Pharma A/S
Eudract number
2022-003322-50
ISRCTN Number
ISRCTN12117857
Research summary
Research Summary:
The medicine to be tested in this trial is called NMD1343, being developed for neuromuscular diseases such as myasthenia gravis (MG) and spinal muscular atrophy (SMA). This is a first-time-in-human clinical trial, which means that this is the first time that the clinical trial medicine will be given to humans. Up to now, it has only been tested in animals. The main purpose of the clinical trial is to see how safe the clinical trial medicine is and how well it is tolerated after dosing. The clinical trial will also investigate how the clinical trial medicine is taken up, metabolised (chemically broken down), distributed through the body and excreted. \n\nThis trial comprises two study parts; Part 1 – which follows a single ascending dose design and Part 2 – which follows a multiple ascending doses design. The trial is a single centre, randomised, double-blind trial, in 88 healthy male participants aged 18 to 55 years old and female (non-childbearing potential) participants aged 18 to 60 years\n\nFor Part 1 of the study, the clinical trial consists of a Screening Period (Days -28 to Day -2), an in-house Treatment Period (Days -1 to 3) and a 7-day post-dosing Follow-up Visit (Day 7 ± 2). \nPart 1 of the study is further divided in 3 different parts. \n•\tPart 1.A: a single dose capsule at ascending dose levels in healthy male participants\n•\tPart 1.B: a single dose capsule in healthy female (non-childbearing potential) participants to investigate the gender effect\n•\tPart 1.C: a single dose capsule following a high fat breakfast in healthy male participants to determine the effect of food. One cohort who participate in Part 1.A will be invited to participate in Part 1.C (food effect).\n\nFor Part 2 of the study, the clinical trial consists of a Screening Period (Days -28 to Day -2), an in-house Assessment Period (Days -1 up to 12) and a post-dosing Follow-up Visit 7 ± 2 days after the last dose.
Summary of results:
The researchers found that NMD1343 was safe, and participants tolerated NMD1343 well at all doses. Researchers found that as the dose increased, the amount of NMD1343 in the body increased in a predictable way. After taking the tablet by mouth NMD1343 was quickly found in the blood of the participants. The highest concentration was measured in the blood between 30 minutes and 1 hour and 30 minutes after taking the tablets. Half of the NMD1343 was out of the body again between 1 hour and 2 hours and 36 minutes after taking the tablets. NMD1343 and its metabolites were quickly excreted from the participants’ body in the urine. Most of the excretion happened between 6 and 12 hours after taking the tablets. Further information on summary results can be found in Clinical Study Report V1.0, dated 06Jan2025 and Clinical Study Results.
REC name
London - Harrow Research Ethics Committee
REC reference
23/LO/0007
Date of REC Opinion
23 Feb 2023
REC opinion
Further Information Favourable Opinion