Table 1.
Previous studies and trials examining IGF-1 as a potential treatment in several childhood-onset neurodevelopmental disorders.
| References | Disorder | Treatment | Dose | Sample SIze | Study type | Treatment duration | Findings | Conclusion | Company (if applicable) |
|---|---|---|---|---|---|---|---|---|---|
| Khwaja et al., 2014 | RTT | rhIGF-1 | 40–120 ug/kg bd (MAD) followed by 12 weeks at max. dose | 12 | Clinical trial | 6 months | Improvement in apnoea index neurobehavioural parameters, measures of mood and anxiety Reversal of alpha band desynchronization on EEG | Safety and preliminary efficacy supported | – |
| Pini et al., 2012 | RTT | rhIGF-1 | 0.05 mg/kg bd first and last week; 0.1 mg/kg bd in between | 6 | Clinical study | 6 months | No adverse events | Safety of IGF-1 supported | – |
| Pini et al., 2014 | RTT | rhIGF-1 | 0.1 mg/kg bd | Single case study | 6 months | No adverse events | Demonstrated safety of repeated doses in a single patient | – | |
| Pini et al., 2016 | RTT | rhIGF-1 | 0.05 mg/kg bd first and last week; 0.1 mg/kg bd in between | 10 (incl. Pini et al., 2012) | Clinical study | 6 months | Significant improvement in Rett Severity Score (RSS) and International Scoring System (RSS) Significant improvement in social/cognitive testing endurance | Prelimary efficacy of IGF-1 supported | – |
| NCT01703533 | RTT | NNZ-2566 | 35 mg/kg or 70 mg/kg bd (note: oral administration) | 56 | Phase II Trial | 28 days | No adverse events Significant improvement in Motor-Behavior Assessment Change Index, Clinical Global Impression of Improvement and Caregiver Top 3 Concerns | Safety and preliminary efficacy supported | Neuren Pharmaceuticals Ltd. see disclosure (Neuren Pharmaceuticals Ltd.)a |
| NCT01894958 | FXS | NNZ-2566 | 35 mg/kg or 70 mg/kg bd (note: oral administration) | 45 | Phase II Trial | 56 days | No serious adverse events Significant improvement in group and individual level analysis of specified core measures | Safety and preliminary efficacy supported | Neuren Pharmaceuticals Ltd.b |
| Kolevzon et al., 2014 | PMDS | rhIGF-1 | 0.04 mg/kg bd to a maximum of 0.12 mg/kg bd | 9 | Phase II Trial | 3 months | No serious adverse events Significant improvement on both the Aberrant Behavior Checklist and Repetitive Behavior Scale | Safety and preliminary efficacy supported | Neuren Pharmaceuticals Ltd. |
RTT, Rett Syndrome; FXS, Fragile X Syndrome; PMDS, Phelan McDermid Syndrome, ASD, Autism Spectrum Disorder.
Neuren Pharmaceuticals Ltd. ASX Announcement 7th Dec 2015. Melbourne, Australia. Neuren's trofinetide successful in proof of concept Phase 2 clnical trial in Fragile X Syndrome. http://www.neurenpharma.com/IRM/PDF/1557/TrofinetidesuccessfulinPhase2trialinFragileX Last accessed 28th July 2016.
Neuren Pharmaceuticals Ltd. Neuren (Neu) ASX Announcement 12th Nov 2014. Melbourne, Australia. Neuren's NZ-2566 successful in demonstrating clinical benefit in Rett syndrome Phase 2 trial. http://www.neurenpharma.com/IRM/PDF/1447/NeurensuccessfulinRettsyndromePhase2trial Last accessed 28th July 2016.