Status and phase
Conditions
Treatments
About
Fifty patients with amyotrophic lateral sclerosis that is progressing rapidly will be randomized to receive either the monoclonal antibody IC14 or placebo to be given intravenously over two hours twice weekly for 12 weeks. Blood and urine tests will be done to measure biomarkers in order to evaluate clinical response and to monitor for safety. Other evaluations include patient questionnaires about function, quality of life and mental function; pulmonary function test; and sniff nasal pressure.
Full description
This will be a placebo-controlled, double-blind, parallel-group comparison.
Fifty patients with rapidly progressive ALS will be randomised to receive one of the following regimens:
The primary endpoint is:
• Treatment-related change in disease biomarker profiles [e.g., neurofilaments (Nf), urinary p75 neurotrophin receptor (p75NTR), cytokines, and soluble CD14].
The secondary endpoints are:
Sex
Ages
Volunteers
Inclusion criteria
Signed informed consent prior to initiation of any study-specific procedures.
Familial or sporadic MND defined as clinically possible, probable, or definite by Awaji-Shima Consensus Recommendations.
Rapidly progressive MND as defined by a decline of 3 or more points in the ALSFRS-R score during the prior 3 months.
First symptoms of MND within 3 years of informed consent.
Age between 18 and 75 years at time of informed consent.
Seated Forced Vital Capacity (FVC) ≥ 65% of predicted value.
Not taking riluzole or edaravone or on a stable dose of riluzole or edaravone for at least 3 months prior to screening visit.
Adequate bone marrow reserve, renal and liver function:
Females of childbearing potential should be using and committed to continue using one of the following acceptable birth control methods:
To be considered of non-childbearing potential, females should be surgically sterilized (bilateral tubal ligation, hysterectomy, or bilateral oophorectomy at least 2 months prior to study) or be post-menopausal and at least 3 years since last menses.
Males with female partners of childbearing potential must use contraception through study completion.
Able to give informed consent and able to comply with all study visits and all study procedures.
Exclusion criteria
Dependence on mechanical ventilation, defined as being unable to lay supine without it, unable to sleep without it, or continuous daytime use; presence of tracheostomy at screening; or presence of diaphragm pacing system at screening.
Treatment with a drug or device within the last 30 days that has not received regulatory approval.
Treatment within 12 months with immunomodulator or immunosuppressant agent (including but not limited to cyclophosphamide, cyclosporine, interferon-α, interferon-β-1a, rituximab, alemtuzumab, azathioprine, etanercept, infliximab, adalimumab, certolizumab, golimumab, anakinra, rilonacept, secukinumab, tocilizumab, mycophenolate mofetil, methotrexate, haematopoietic stem cell transplantation, anti-sense drugs, gene therapy, cell-depleting agents, total lymphoid irradiation). Treatment with intravenous immunoglobulin within 2 months or dimethyl fumarate within 3 months. Non-steroidal anti-inflammatory drugs are acceptable.
Known active current or history of recurrent bacterial, viral, fungal, mycobacterial or other opportunistic infections; or major episode of infection requiring hospitalization or treatment with IV antibiotics within 4 weeks.
Live-attenuated vaccines within 30 days before dosing. Subjects must agree to forego live-attenuated vaccines throughout the study, including 12 weeks after the last dose of study drug.
History of severe allergic or anaphylactic reactions to human, humanized or murine monoclonal antibodies.
Presence of any of the following clinical conditions:
Pregnancy or breastfeeding.
Deprivation of freedom by administrative or court order.
Primary purpose
Allocation
Interventional model
Masking
0 participants in 2 patient groups, including a placebo group
Loading...
Data sourced from clinicaltrials.gov
Clinical trials
Research sites
Resources
Legal