Table 4.
Adverse events reported during the study.
| 12-month double-blind, placebo-controlled phase | MD1003 (n = 103) | Placebo (n = 51) |
|---|---|---|
| Any AE, n (%) | 84 (81.6) | 43 (84.3) |
| AEs occurring in >2% of MD1003-treated patients | ||
| Urinary tract infection | 10 (9.7) | 6 (11.8) |
| Bronchitis | 5 (4.9) | 6 (11.8) |
| Nasopharyngitis | 5 (4.9) | 3 (5.9) |
| Multiple sclerosis relapse | 5 (4.9) | 4 (7.8) |
| Hyperthyroidisma | 6 (5.8) | – |
| Gastroenteritis | 4 (3.9) | 2 (3.9) |
| Constipation | 4 (3.9) | 2 (3.9) |
| Back pain | 4 (3.9) | 3 (5.9) |
| Headache | 4 (3.9) | 3 (5.9) |
| Muscle spasticity | 4 (3.9) | 6 (11.8) |
| Nausea | 3 (2.9) | 2 (3.9) |
| Oedema peripheral | 3 (2.9) | 1 (2.0) |
| Cystitis | 3 (2.9) | 1 (2.0) |
| Fall | 3 (2.9) | 2 (3.9) |
| Dizziness | 3 (2.9) | 2 (3.9) |
| Oropharyngeal pain | 3 (2.9) | – |
| Any TRAE, n (%) | 20 (19.4) | 12 (23.5) |
| TRAEs occurring in >2% of MD1003-treated patients | ||
| Headache | 3 (2.9) | – |
| Any severe AE,b n (%) | 5 (4.9) | 4 (7.8) |
| Any SAE,c n (%) | 20 (19.4) | 12 (23.5) |
| SAEs occurring in more than one patient | ||
| Multiple sclerosis relapse | 5 (4.9) | 4 (7.8) |
| 12-month extension phase | MD1003 > MD1003 (n = 91) | Placebo > MD1003 (n = 42) |
| Any AE, n (%) | 49 (53.8) | 25 (59.5) |
| AEs occurring in >2% of MD1003 > MD1003 patients | ||
| Urinary tract infection | 8 (8.8) | 6 (14.3) |
| Multiple sclerosis relapse | 7 (7.7) | 2 (4.8) |
| Bronchitis | 3 (3.3) | 2 (4.8) |
| Hyperthyroidisma | 3 (3.3) | 1 (2.4) |
| Fall | 3 (3.3) | 1 (2.4) |
| Abdominal pain | 3 (3.3) | – |
| Eczema | 3 (3.3) | – |
| Hypercholesterolaemia | 2 (2.2) | 1 (2.4) |
| Nasopharyngitis | 2 (2.2) | – |
| Back pain | 2 (2.2) | – |
| Headache | 2 (2.2) | – |
| Oedema peripheral | 2 (2.2) | – |
| Musculoskeletal pain | 2 (2.2) | – |
| Any TRAE, n (%) | 7 (7.7) | 5 (11.9) |
| TRAEs occurring in >2% of MD1003 > MD1003 patients | ||
| None | – | – |
| Any severe AE,b n (%) | 6 (6.6) | 2 (4.8) |
| Any SAE,c n (%) | 14 (15.4) | 6 (14.3) |
| SAEs occurring in more than one patient | ||
| Multiple sclerosis relapse | 6 (6.6) | 2 (4.8) |
AE: adverse event; MS: multiple sclerosis; SAE: serious adverse event; TRAE: treatment-related adverse event.
Six patients had apparent hyperthyroidism in the placebo-controlled phase and five patients during the extension phase. One patient in the placebo-controlled phase was confirmed to have Basedow’s disease; the remaining AEs reported as hyperthyroidism were believed to be due to abnormal thyroid function tests due to interference with the biotin-based assay systems used. An additional case of suspected hyperthyroidism in the MD1003 > MD1003 group was identified but not reported as an AE.
Severe events during the placebo-controlled phase were isolated cases of urinary tract infection, humerus fracture, suicide, mucocutaneous rash and rosacea in the MD1003 arm and isolated cases of fatigue, intracranial haematoma, MS relapse and bipolar disorder in the placebo arm. Severe events during the extension phase were isolated cases of vertigo, hyperthyroidism, hypoglycaemia, rectal cancer, MS relapse, lung disorder and menopause in the MD1003 > MD1003 group (seven events in six patients) and isolated cases of thyroiditis and myopathy in the placebo > MD1003 group.
An adverse event resulting in death, hospitalisation, disability, congenital anomaly or was otherwise life threatening.
AE: adverse event; SAE: serious adverse event; TRAE: treatment-related adverse event.