Table 2.
Clinical trials with growth factors.
| NCT number | NTF | Delivery method | Phase and status of the trial | Cohort size | Outcomes | References | Year |
|---|---|---|---|---|---|---|---|
| PROTEIN INFUSION | |||||||
| Not provided | CNTF | SC | Phase I, terminated | 57 | No adverse neurologic effects, safe, and tolerated | (70) | 1995 |
| Not provided | SC | Phase I, terminated | 570 | No beneficial effect, adverse events dose related, increased number of death at the highest dose, no beneficial effect on ALS progression | (71) | 1996 | |
| Not provided | SC | Phase II/III | 730 | Disease progression not modified, minor adverse side effects | (72) | 1996 | |
| Not provided | IT | Phase I | 4 | Pain syndromes dose-related, no systemic side effect, no improvement, or worsen of motor function | (73) | 1997 | |
| Not provided | BDNF | SC | Phase I/II, terminated | 283 | Tolerated, Trend of improved survival, less deterioration of predicted FVC | (74) | 1995 |
| Not provided | BDNF | SC | Phase III | 1 135 | Disease progression not modified, Patients with early respiratory impairment and with altered bowel function showed benefit | (75) | 1999 |
| Not provided | BDNF | IT | Phase I/II, terminated | 25 | Well tolerated, feasible | (76) | 2000 |
| Not provided | BDNF | IT | Phase III, terminated | 17 | No adverse events, no effect | (77) | 2003 |
| Not provided | BDNF | IT | Phase II/III, terminated | 13 | No effect | (78) | 2005 |
| Not provided | IGF-1 | SC | Not specify | 266 | Slowed the progression of functional impairment, slow the decline in health-related quality of life | (79) | 1997 |
| Not provided | SC | Not specify | 183 | Safe and well-tolerated, no effect | (80) | 1998 | |
| Not provided | IT | Not specify | 9 | No serious adverse effect, modest beneficial effect | (81) | 2005 | |
| NCT00035815 | SC | Phase III, completed | 330 | No benefit | (82) | 2008 | |
| Not provided | G-CSF | SC | Phase I, terminated | 13 | Safe, less decline of ALSFRS score | (83) | 2009 |
| Not provided | SC | Phase I, terminated | 39 | Safe, no significative effect on ALSFRS score | (84) | 2010 | |
| NCT00397423 | Not specify | Phase II, completed | 40 | Not available | |||
| NCT01999803 | VEGF | ICV | Phase I, terminated | 15 | Not available | ||
| NCT02269436 | ICV | Phase I, terminated | 11 | Not available | |||
| NCT01384162 | ICV | Phase I/II, terminated | 15 | Not available | |||
| STEM CELLS | |||||||
| NCT number | Type of stem cells | Delivery method | Phase and status of the trial | Cohort size | Results | References | Year |
| NCT01348451 | NSC | ISP | Phase I | 12 | No major adverse events | (85, 86) | 2012 |
| NCT01730716 | NSC | ISP | Phase II, unknown status | 18 | Not available | ||
| NCT02943850 | NPC | ISP | Phase I/IIa, active, not recruiting | 18 | Not available | ||
| NCT01640067 | NSC | ISP | Phase I, completed | 6 | Safe approach, no increase of disease progression | (87) | 2015 |
| NCT00781872 | MSC | IT, IV | Phase I/II, terminated | 19 | Safe and feasible, ALS-FRS score stable the first 6 months | (88) | 2010 |
| NCT03085706 | PBMC | ISP | Phase NA, completed | 14 | Not available | ||
| NCT01933321 | HSC | IT | Phase II/III, completed | 14 | Not available | ||
| NCT01609283 | MSC | IT | Phase I, active, not recruiting | 27 | Not available | ||
| NCT01142856 | MSC | IT | Phase I, completed | 1 | Not available | ||
| NCT00855400 | MSC | ISP | Phase I/II completed | 11 | No severe adverse event, no acceleration in the rate of decline, possible neurotrophic activity | (89) | 2012 |
| NCT02286011 | MC | IM | Phase I, active, not recruiting | 20 | Not available | ||
| NCT00855400 | MC | ISP | Phase I, completed | 11 | Safe approach, no worsening of the disease | (90) | 2016 |
| NCT03268603 | MSC | IT | Phase II, recruiting | 60 | Not available | ||
| NCT01254539 | MSC | ISP, IT | Phase I/II, completed | 63 | Infusion of MSC produces spinal changes unrelated with clinical events and disease worsening | (91) | 2013 |
| NCT01363401 | MSC | IT | Phase I/II, completed | 64 | Possible benefit lasting at least 6 months with safety | (92) | 2018 |
| NCT02917681 | MSC | IT | Phase I/II, recruiting | 28 | Not available | ||
| NCT02987413 | MSC | IT | Phase I, completed | 3 | Not available | ||
| NCT02290886 | MSC | IV | Phase I/II, active, not recruiting | 52 | Not available | ||
| NCT01051882 | MSC | IM or IT | Phase I/II, completed | 12 | Safe and tolerated, no serious adverse event, possible benefits on ALS-FRS score, and percentage of FVC | (93) | 2016 |
| NCT01777646 | MSC | IM + IT | Phase IIa, completed | 14 | |||
| NCT03280056 | MSC | IT | Phase III, Recruiting | 200 | Not available | ||
| NCT02017912 | MSC | IM, IT | Phase II, completed | 48 | Not available | ||
| NCT01759797 | MSC | IV | Phase I/II, completed | 6 | No adverse events, ALS-FRS score reduced, FVC percentage reduced | (94) | 2019 |
| NCT01771640 | MSC | IT | Phase I, completed | 8 | |||
FVC, force vital capacity; HSC, hematopoietic stem cells; I, intramuscular; ISP, intraspinal; IT, intrathecal; IV, intravenous; MC, mononuclear cell; MSC, mesenchymal stem cells; NPC, neuronal progenitor cells; NSC, neural stem cells; NTF, neurotrophic factor, PBMC, peripheral blood mononuclear cell; SC, subcutaneous.