E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Non-inferiority and safety/tolerance of Hyqvia compared to IVIg in 20 MMN patients with at least one conduction block on EMG and stable on IVIg. |
|
E.1.1.1 | Medical condition in easily understood language |
Immunoglobulins given under the skin as a way of treaatment compared to admission directly in the blood. Safety and effect are monitored. |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Test safety/tolerance of Hyqvia compared to IVIg |
|
E.2.2 | Secondary objectives of the trial |
Test non-inferiority of Hyqvia compared to IVIg |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
(1) Age at onset of MMN, 18 – 65 years.
(2) The presence of asymmetrical limb weakness at onset or motor involvement having a motor nerve distribution in at least two peripheral nerve distributions, predominant upper limb involvement, disabling weakness MRC grade 4 or less in at least one muscle.
(3) Decreased or absent tendon reflexes in affected limbs.
(4) Electrophysiological evidence of one site with definite motor conduction block or one site with probable conduction block according to previously defined criteria.
(5) Response to IGIV according to criteria that were described in previous studies.
(6) stable on IGIV maintenance treatment in the year preceding the study.
(7) Patients have given written informed consent, prior to the study, with the understanding that consent may be withdrawn at any time without prejudice.
|
|
E.4 | Principal exclusion criteria |
A potential subject who meets any of the following criteria will be excluded from participation in this study:
(1) Bulbar signs or symptoms.
(2) Upper motor neuron signs (spasticity, hyperreflexia, extensor plantar response).
(3) Sensory symptoms and signs with sensory deficits on examination (except for vibration sense) and abnormal results of sensory nerve conduction studies
(4) Other neuropathies (e.g. diabetic, lead, porphyric or vasculitic neuropathy, chronic inflammatory demyelinating polyneuropathy, Lyme neuroborreliosis, post radiation neuropathy, hereditary neuropathy with liability to pressure palsies, Charcot-Marie-Tooth neuropathies, meningeal carcinomatosis).
(5) Treatment with other immunosuppressive drugs (cyclophosphamide, azathioprine, cyclosporin) in the 6 months preceding the study.
(6) Female patient who is pregnant or breast-feeding or of childbearing potential.
Confirmation that the patient is not pregnant will be established by a negative b-HCG test within a 7-day period before inclusion in the study. Lack of childbearing potential is met by a) being post-menopausal, b) being surgically sterile, c) practising contraception with an oral contraceptive, intra-uterine device, diaphragm or condom with spermacide or d) being sexually inactive.
(7) Age < 18 years.
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
Test safety/tolerance of Hyqvia compared to IVIg |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Final timepoint :After around 9 months usage of Hyqvia per patient.
In total eight visits of the outpatient clinic will be organized to monitor safety. In case of intolerability as described in the protocol the patient can be re-switsched to IVIg. |
|
E.5.2 | Secondary end point(s) |
Test non-inferiority of Hyqvia compared to IVIg. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Final timepoint :After around 9 months usage of Hyqvia per patient.
In total eight visits of the outpatient clinic will be organized to monitor non-inferiority. In case of clinical deterioration as described in the protocol the patient can be re-switsched to IVIg. |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | Yes |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | Yes |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
Same medicine is compared in different ways of administration (IV vs SC) and hyaluronidase is added |
|
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
LVLS
If during the study data analysis shows clear inferioroty of Hyqvia compared to IVIg the study will be ended. Also if unacceptable (S)AEs are popping up compared to IVIg |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |