This trial is not currently recruiting.
This study is a First in Human, open label, Single Ascending Dose (SAD) and Multiple Ascending Dose (MAD) study to determine the safety and Pharmacokinetics (PK) characteristics of mEphA4-Fc in HVs and patients with ALS. Measurements of clinical and lung function endpoints associated with ALS will also be assessed.
There will be two parts. Part 1 (SAD) of the study will be conducted in five cohorts of 4 subjects each. Part 2 (MAD) of the study will be conducted in two cohorts of 4 ALS patients each. mEphA4-Fc will be administered as an IV infusion. Each infusion of mEphA4-Fc administration should be completed within 75-90 minutes.
Each cohort will be comprised of 4 HVs each to receive mEphA4-Fc. mEphA4-Fc will be given in sequential, escalating doses. Incremental doses starting from a dose selected according to the NOAEL strategy for FIH studies will be administered and the planned increments will not exceed 3 fold. The minimum and maximum doses for each cohort are limited by body weight. The actual dose level for a cohort will be ultimately decided by the Safety Review Committee based on a review of data from the proceeding cohort/s.
The first multiple dose cohort may be initiated prior to the completion of the fifth SAD cohort and after completion of the fourth SAD cohort. The multiple ascending dose will be comprised of two cohorts of 4 ALS patients each to receive mEphA4-Fc. The frequency of administration, dose increments and number of cohorts will be reviewed based on review of all data by the Safety Review Committee.
Each cohort will be comprised of 4 subjects each to receive 4 doses of mEphA4-Fc, over 28 days. The proposed time duration between each dose will be 7 days, but will be reviewed by the Safety Review Committee.
Primary outcome [1]
Safety and tolerability of mEphA4-Fc assessed from Incidence and severity of adverse events (AEs).
Primary outcome [2]
To characterise the pharmacokinetics (PK) of mEphA4-Fc.
Secondary outcome [1]
To evaluate the potential effects of treatment on ALS function rating score revised (ALSFRS-R).
Secondary outcome [2]
To evaluate the potential effects of treatment on forced vital capacity (FVC).
Dr Richard Friend- Clive Berghofer Cancer Research Centre (CBCRC)
+61 403 415 925
r.friend@nucleusnetwork.com.au
