ALSPhase 2TCZALS-001NCT02469896
A Trial of Tocilizumab in ALS Subjects
A Phase 2 study testing Tocilizumab in people with ALS and Amyotrophic Lateral Sclerosis, run by Barrow Neurological Institute at 5 sites in United States.
Trial summary
This research study is being done to find out if tocilizumab, also known as Actemra™, can help with Amyotrophic Lateral Sclerosis (ALS). The investigators also want to find out if tocilizumab is safe to take without causing too many side effects. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- ALS, Amyotrophic Lateral Sclerosis, Lou Gehrig's Disease
- What is being tested?
- Tocilizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 22 participants
- Where
- 5 sitesUnited States
- Study dates
- Nov 2015to 11 Jul 2018
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants with ALS (El Escorial criteria: possible, laboratory-supported probable, probable or definite)
- Capable of providing informed consent and complying with trial procedures.
- High inflammatory profile of PBMC gene expression
- Upright SVC ≥40% of predicted value for gender, height and age at Screening and in the opinion of the investigator is able to comply with and complete the trial.
- Women must not be able to become pregnant for the duration of the study.
- Negative tuberculosis blood or skin test at Screening
Show all 14Show fewer
- Not taking riluzole, or on a stable dosage for at least 30 days prior to Screening.
- Subjects medically able to undergo lumbar puncture (LP)
- Subjects must agree not to take live attenuated vaccines 30 days before Screening, throughout the duration of the trial and for 60 days following the subject's last dose of study drug
- Geographic accessibility to the study site
- Additional MRI-PET Inclusion Criteria (MGH only):
- High or mixed affinity to bind TSPO protein (Ala/Ala or Ala/Thr) (see section 7.1)
- Upper Motor Neuron Burden (UMNB) Scale Score ≥25 (out of 45) at the Screening Visit.
- Able to safely undergo MRI-PET scans based on the opinion of the site investigator.
Who cannot join
- Prior use of Tocilizumab, cell-depleting therapies, alkylating agents, total lymphoid irradiation
- Stem cell therapies
- Dependence on mechanical ventilation as defined as being unable to lay supine without it, unable to sleep without it, or continuous daytime use
- Presence of tracheostomy at Screening
- Exposure to any anti-inflammatory agent currently under investigation for the treatment of patients with ALS (off label use or investigational) within 30 days prior to the Screening Visit (examples include NP001 and Lunasin). Medications that do not have an anti-inflammatory mechanism, such as mexiletine or retigabine are allowed if on stable dose for 30 days prior to Screening visit
- Treatment with a prohibited medication within 30 days of the Screening Visit
Show all 29Show fewer
- Treatment with intravenous gamma globulin, plasmapheresis or Prosorba column within 6 months of Screening
- Presence of diaphragm pacing system at Screening.
- Primary or secondary immunodeficiency (history of or currently active) unless related to primary disease under investigation
- History of or active diverticulitis, diverticulosis requiring antibiotic treatment, peptic ulcer disease, or gastrointestinal (GI) tract perforation, or chronic ulcerative lower GI disease such as Crohn's disease, ulcerative colitis or other symptomatic lower GI conditions that might predispose to perforations
- Known active current or history of recurrent bacterial, viral, fungal, mycobacterial or other opportunistic infections.
- History of severe allergic or anaphylactic reactions to human, humanized or murine monoclonal antibodies
- Presence of any of the following clinical conditions: bleeding diathesis, or any other clinical condition that would, in the opinion of the investigator, place the patient at increased risk during LP. Drug abuse or alcoholism within the past 12 months. Unstable cardiac, pulmonary, renal, hepatic, endocrine, hematologic, or active infectious disease, including current or prior malignancy. Rheumatic autoimmune disease, mixed connective tissue disease, scleroderma, polymyositis, or significant systemic involvement secondary to rheumatoid arthritis. Evidence of active malignant disease, malignancies diagnosed within the previous 5 years, or breast cancer diagnosed within the previous 5 years. Human immunodeficiency virus infection or other immunodeficient state.Uncontrolled hypertension defined as systolic blood pressure > 170 or diastolic blood pressure > 110. Unstable psychiatric illness defined as psychosis or untreated major depression within 90 days of the Screening Visit
- Any major episode of infection requiring hospitalization or treatment with IV antibiotics within 4 weeks of screening
- Screening alanine aminotransferase (ALT), aspartate aminotransferase (AST), or total bilirubin > than 1.5 times the upper limit of normal (ULN), serum creatinine > 1.6 mg/dL in female patients and > 1.9 mg/dL in male patients (patients with serum creatinine values exceeding limits may be eligible for the study if their estimated glomerular filtration rate (GFR) are >30), hemoglobin < 85 g/L, white blood cells < 3.0 x 109/L, absolute neutrophil count of <2000/mm3, absolute lymphocyte count < 0.5 x 109/L, platelet concentration of <100,000/mm3, positive Hepatitis B surface antigen (HBsAg)
- Pregnant women or women currently breastfeeding
- No history of chicken pox infection or no history of varicella zoster vaccination
- Any reason in the opinion of the investigator that the patient may not be able to comply with study procedures, complete the study or is unsuitable for immunosuppressive therapy.
- Additional MR-PET Exclusion Criteria (MGH only):
- Any contraindication to undergo MRI studies such as
- History of a cardiac pacemaker or pacemaker wires
- Metallic particles in the body
- Vascular clips in the head
- Prosthetic heart valves
- Claustrophobia
- Radiation exposure that exceeds the site's current guidelines
- Current use of tobacco products including cigarettes, e-cigarettes, cigars, snuff and chewing tobacco, or nicotine replacement products such as gum, or patch
- Taking any other anti-inflammatory or immune modulating medications except for over the counter NSAIDs
- Unwilling or unable to discontinue benzodiazepine usage (other than Lorazepam, Clonazepam, or Zolpidem) for one day prior to scanning
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Penn State College of Medicine Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
University of Kansas Medical Center
Kansas City, Kansas, 66160
Barrow Neurological Institute
Phoenix, Arizona, 85013
Wake Forest University School of Medicine
Winston-Salem, North Carolina, 27157
About this study
Official title: A Phase 2 Randomized, Placebo Controlled Trial of Tocilizumab in ALS Subjects
This research study is being done to find out if tocilizumab, also known as Actemra™, can help with Amyotrophic Lateral Sclerosis (ALS). The investigators also want to find out if tocilizumab is safe to take without causing too many side effects.
Currently ALS has no cure and 2 modestly effective treatment to slow the progression of the disease. Although not the initial cause of ALS, the immune system plays a role in the death of motor neurons. The immune cells that participate in this process are stimulated by a substance called interleukin-6 (IL-6) whose effect is blocked by tocilizumab and thus, may slow the death of motor neurons and slow the disease.
Read the detailed description
This is a multicenter, randomized, double-blind, placebo-controlled 16-week study evaluating the safety and tolerability of tocilizumab in subjects with ALS.
The primary objective of the study is to determine the safety and tolerability of intravenous administration of 8 mg/kg of tocilizumab every 4 weeks vs. matched intravenous placebo administered every 4 weeks over an 8 week period.
The secondary objectives of the study are to describe the expression of pro-inflammatory genes in Peripheral Blood Mononuclear Cells (PBMCs) of sporadic ALS patients, to assess the ability of tocilizumab to reduce the expression of pro-inflammatory genes in PBMCs and pro-inflammatory cytokines in the cerebrospinal fluid (CSF) of patients with sporadic ALS and to assess the CSF penetration of tocilizumab. Mean peripheral benzodiazepine receptor 28 (PBR28) uptake will be measured in the motor cortices as regions of interest (ROIs), and will be compared between pre- and post-dose, for Massachusetts General Hospital (MGH) subjects.
Approximately 5 Northeast ALS Consortium (NEALS) Centers in the US will participate in the study. Twenty-four subjects will be randomized in the study.
This study will be conducted in subjects who meet the El Escorial criteria of possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS. At screening, eligible subjects must be at least 18 years old, must have a slow vital capacity (SVC) ≥ 40% of predicted capacity for age, height and gender (and in the opinion of the investigator is able to comply with and complete the trial), and must provide written informed consent prior to screening. Subjects on a stable dose of riluzole and those not taking riluzole, and women of child-bearing age at screening are eligible for inclusion as long as they meet specific protocol requirements. Detailed criteria are described in the body of the protocol.
Subjects participating in the magnetic resonance imaging - positron emission tomography (MRI-PET) portion of the study (MGH only) must meet the following additional criteria.High or mixed affinity to bind translocator protein (TSPO) (Ala/Ala or Ala/Thr,) Upper Motor Neuron Burden (UMNB) Scale Score ≥25 (out of 45) at the Screening Visit.
and have the ability to safely undergo MRI-PET scans based on the opinion of the site investigator.
Subjects will be randomly assigned in a 2:1 ratio to intravenous tocilizumab 8 mg/kg or matching placebo every 4 weeks over an 8 week period.
This research study protocol allows the subject to receive up to 3 infusions of Tocilizumab. Even if the treatment is shown to be of benefit, additional infusions of Tocilizumab beyond that allowed in the protocol cannot be given to the subject while she/he is participating in this study.
Subjects will remain on randomized, placebo-controlled, double-blind treatment until the Week 8 visit. Each randomized subject will also have a Week 12 Follow-up visit and Week 16 End-of-Study visit to assess for adverse events (AEs), changes in concomitant medications, to administer the ALS Functional Rating Scale (ALSFRS-R) and selected study procedures.
- Being tested
- Tocilizumab, Placebo
- Keywords
- ALS, tocilizumab, biomarker
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TocilizumabDrug
Also called Actemra
IV Infusion
PlaceboOther
Also called Saline
IV Infusion
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | 8 subjects will receive matching IV placebo every 4 weeks for 3 months. Receives: Other: Placebo |
| Active drug | Active comparator | 14 subjects will receive 8mg/kg of IV tocilizumab every 4 weeks for 3 months. Receives: Drug: Tocilizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients Tolerant to Study Drug
Tolerability will be assessed by on the proportion of participants remaining on study drug through all 3 doses and remaining on study and free from possibly drug-related and dose-limiting SAEs to the end of follow-up. Safety will be assessed by the occurrence of severe adverse events (SAEs), overall rates of adverse events (AEs), clinically significant abnormal laboratory tests, and changes in vital signs.
Measured: 16 weeks
Rates of All-cause Mortality
Safety will be assessed by the occurrence of all-cause mortality.
Measured: 16 weeks
Secondary outcomes (8)
Rate of Decline in Slow Vital Capacity (SVC)
Efficacy will be assessed by the change in the rate of change of SVC as measured by change in percent predicted per month. The SVC is a measure of lung capacity that is reported as the percent of the predicted value expected based on gender and height. In ALS patients, this measure declines over time as a result of progressive respiratory muscle weakness.
Measured: 16 weeks
Rate of Decline ALS Functional Rating Scale Revised (ALSFRS-R)
Efficacy will be assessed by the mean change in ALSFRS-R total score.The ALSFRS-R scale measures the functional capabilities of an ALS patient in multiple domains such as swallowing, speech, fine motor, and breathing functions. It ranges from a maximum score of 48 for normal functioning to 0 for death or dependance on mechanical ventilation and declines by approximately 1 point per month on average for an ALS patient.
Measured: 16 weeks
Rate of Decline Handheld Dynamometry (HHD)
Efficacy will be assessed by the change in the rate of change of HHD upper and lower extremity mega-scores. HHD utilizes an electronic pressure sensor to measure strength of individual muscles in kilograms. To calculate megascores, the mean and standard deviation of each muscle or muscle group, without regard to laterality, will be calculated from the baseline assessment of all participants. Strength estimates of each bilateral muscle or muscle group will be converted to Z scores by subtracting the relevant mean and dividing by the relevant standard deviation. Z scores for all upper extremity measurements (shoulder flexion, elbow flexion, elbow extension, wrist extension, and first dorsal interosseous contraction) and all lower extremity measurements (hip flexion, knee flexion, knee extension, and ankle dorsiflexion) will be averaged to yield upper and lower extremity megascores. Larger values indicate greater strength.
Measured: 16 weeks
Change in Peripheral Blood Mononuclear Cell (PBMC) Gene Expression
Target engagement will be assessed by comparing the PBMC fold change in cytokine gene expression from baseline to week 4-16 average of ALS patients receiving drug versus placebo.
Measured: 16 weeks
Changes in Cytokine Levels in the Plasma
Target engagement will be assessed by mean change in plasma cytokine concentration between weeks 4 and 16 in ALS subjects receiving placebo or active drug.
Measured: 16 weeks
Change in Mean Concentration Cytokines in the Cerebrospinal Fluid (CSF)
Target engagement will be assessed by the mean change in CSF cytokine concentration between baseline and week 8 in ALS subjects receiving placebo or active drug.
Measured: 8 weeks
Change in CSF Soluble Interleukin-6 (sIL-6) Receptor Concentrations
Target engagement will be assessed by comparing the mean change in CSF sIL-6 receptor concentrations (ng/mL) between baseline and week 8 of the placebo and active drug groups.
Measured: 8 weeks
Peripheral Benzodiazepine Receptor 28 (PBR28) Positron Emission Tomography (PET)
Measure the effects of tocilizumab on reducing glial activation measured by PBR28 PET in a subset of trial participants.
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jun 2015 (estimated)
- Study startNov 2015
- Primary completion11 Jul 2018
- Study completion11 Jul 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for ALSStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Barrow Neurological Institute
- Sponsor type
- University, hospital or other
- Collaborators
- ALS Association, Barrow Neurological Foundation, Massachusetts General Hospital, Genentech, Inc.
- Responsible party
- Principal investigator, Shafeeq Ladha, MD, Barrow Neurological Institute
- Investigators
- Shafeeq Ladha, MD, Principal investigator, Barrow Neurological Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02469896
- Sponsor's protocol ID
- 2015TCZALS-001
- Acronym
- TCZALS-001
- Status last verified
- Dec 2019
- First submitted
- 3 Jun 2015
- Last updated
- 18 Dec 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Milligan C, Atassi N, Babu S, Barohn RJ, Caress JB, Cudkowicz ME, Evora A, Hawkins GA, Wosiski-Kuhn M, Macklin EA, Shefner JM, Simmons Z, Bowser RP, Ladha SS. Tocilizumab is safe and tolerable and reduces C-reactive protein concentrations in the plasma and cerebrospinal fluid of ALS patients. Muscle Nerve. 2021 Sep;64(3):309-320. doi: 10.1002/mus.27339. Epub 2021 Jun 24.
PubMed 34075589
Study at a glance
From the registry record, last updated by the sponsor on 18 Dec 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Randomized, Placebo Controlled Trial of Tocilizumab in ALS Subjects
- ClinicalTrials.gov ID
- NCT02469896
- Acronym / sponsor ID
- TCZALS-001 / 2015TCZALS-001
- Keywords
- ALS, tocilizumab, biomarker
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Barrow Neurological InstituteUniversity, hospital or other
- Collaborators
- ALS Association, Barrow Neurological Foundation, Massachusetts General Hospital, Genentech, Inc.
- Study start
- Nov 2015
- Primary completion
- 11 Jul 2018 (actual)
- Study completion
- 11 Jul 2018 (actual)
- First posted
- 12 Jun 2015 (estimated)
- Results
- Posted 18 Dec 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This research study is being done to find out if tocilizumab, also known as Actemra™, can help with Amyotrophic Lateral Sclerosis (ALS). The investigators also want to find out if tocilizumab is safe to take without causing too many side effects.
Currently ALS has no cure and 2 modestly effective treatment to slow the progression of the disease. Although not the initial cause of ALS, the immune system plays a role in the death of motor neurons. The immune cells that participate in this process are stimulated by a substance called interleukin-6 (IL-6) whose effect is blocked by tocilizumab and thus, may slow the death of motor neurons and slow the disease.
Detailed description
This is a multicenter, randomized, double-blind, placebo-controlled 16-week study evaluating the safety and tolerability of tocilizumab in subjects with ALS.
The primary objective of the study is to determine the safety and tolerability of intravenous administration of 8 mg/kg of tocilizumab every 4 weeks vs. matched intravenous placebo administered every 4 weeks over an 8 week period.
The secondary objectives of the study are to describe the expression of pro-inflammatory genes in Peripheral Blood Mononuclear Cells (PBMCs) of sporadic ALS patients, to assess the ability of tocilizumab to reduce the expression of pro-inflammatory genes in PBMCs and pro-inflammatory cytokines in the cerebrospinal fluid (CSF) of patients with sporadic ALS and to assess the CSF penetration of tocilizumab. Mean peripheral benzodiazepine receptor 28 (PBR28) uptake will be measured in the motor cortices as regions of interest (ROIs), and will be compared between pre- and post-dose, for Massachusetts General Hospital (MGH) subjects.
Approximately 5 Northeast ALS Consortium (NEALS) Centers in the US will participate in the study. Twenty-four subjects will be randomized in the study.
This study will be conducted in subjects who meet the El Escorial criteria of possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS. At screening, eligible subjects must be at least 18 years old, must have a slow vital capacity (SVC) ≥ 40% of predicted capacity for age, height and gender (and in the opinion of the investigator is able to comply with and complete the trial), and must provide written informed consent prior to screening. Subjects on a stable dose of riluzole and those not taking riluzole, and women of child-bearing age at screening are eligible for inclusion as long as they meet specific protocol requirements. Detailed criteria are described in the body of the protocol.
Subjects participating in the magnetic resonance imaging - positron emission tomography (MRI-PET) portion of the study (MGH only) must meet the following additional criteria.High or mixed affinity to bind translocator protein (TSPO) (Ala/Ala or Ala/Thr,) Upper Motor Neuron Burden (UMNB) Scale Score ≥25 (out of 45) at the Screening Visit.
and have the ability to safely undergo MRI-PET scans based on the opinion of the site investigator.
Subjects will be randomly assigned in a 2:1 ratio to intravenous tocilizumab 8 mg/kg or matching placebo every 4 weeks over an 8 week period.
This research study protocol allows the subject to receive up to 3 infusions of Tocilizumab. Even if the treatment is shown to be of benefit, additional infusions of Tocilizumab beyond that allowed in the protocol cannot be given to the subject while she/he is participating in this study.
Subjects will remain on randomized, placebo-controlled, double-blind treatment until the Week 8 visit. Each randomized subject will also have a Week 12 Follow-up visit and Week 16 End-of-Study visit to assess for adverse events (AEs), changes in concomitant medications, to administer the ALS Functional Rating Scale (ALSFRS-R) and selected study procedures.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Other: Placebo8 subjects will receive matching IV placebo every 4 weeks for 3 months. |
| Active drug | Active comparator | Drug: Tocilizumab14 subjects will receive 8mg/kg of IV tocilizumab every 4 weeks for 3 months. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Participants with ALS (El Escorial criteria: possible, laboratory-supported probable, probable or definite)
- Capable of providing informed consent and complying with trial procedures.
- High inflammatory profile of PBMC gene expression
- Upright SVC ≥40% of predicted value for gender, height and age at Screening and in the opinion of the investigator is able to comply with and complete the trial.
- Women must not be able to become pregnant for the duration of the study.
Key exclusion criteria (29 listed)
- Prior use of Tocilizumab, cell-depleting therapies, alkylating agents, total lymphoid irradiation
- Stem cell therapies
- Dependence on mechanical ventilation as defined as being unable to lay supine without it, unable to sleep without it, or continuous daytime use
- Presence of tracheostomy at Screening
- Exposure to any anti-inflammatory agent currently under investigation for the treatment of patients with ALS (off label use or investigational) within 30 days prior to the Screening Visit (examples include NP001 and Lunasin). Medications that do not have an anti-inflammatory mechanism, such as mexiletine or retigabine are allowed if on stable dose for 30 days prior to Screening visit
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TocilizumabAlso: ActemraIV Infusion | Drug | Active drug |
| PlaceboAlso: SalineIV Infusion | Other | Placebo |
Primary outcome
Number of Patients Tolerant to Study Drug
Time frame: 16 weeks
Tolerability will be assessed by on the proportion of participants remaining on study drug through all 3 doses and remaining on study and free from possibly drug-related and dose-limiting SAEs to the end of follow-up. Safety will be assessed by the occurrence of severe adverse events (SAEs), overall rates of adverse events (AEs), clinically significant abnormal laboratory tests, and changes in vital signs.
Rates of All-cause Mortality
Time frame: 16 weeks
Safety will be assessed by the occurrence of all-cause mortality.
8 secondary outcomes
Rate of Decline in Slow Vital Capacity (SVC)
Time frame: 16 weeks
Rate of Decline ALS Functional Rating Scale Revised (ALSFRS-R)
Time frame: 16 weeks
Rate of Decline Handheld Dynamometry (HHD)
Time frame: 16 weeks
Change in Peripheral Blood Mononuclear Cell (PBMC) Gene Expression
Time frame: 16 weeks
Changes in Cytokine Levels in the Plasma
Time frame: 16 weeks
Change in Mean Concentration Cytokines in the Cerebrospinal Fluid (CSF)
Time frame: 8 weeks
Change in CSF Soluble Interleukin-6 (sIL-6) Receptor Concentrations
Time frame: 8 weeks
Peripheral Benzodiazepine Receptor 28 (PBR28) Positron Emission Tomography (PET)
Time frame: 8 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Dec 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 34075589Milligan C, Atassi N, Babu S, Barohn RJ, Caress JB, Cudkowicz ME, Evora A, Hawkins GA, Wosiski-Kuhn M, Macklin EA, Shefner JM, Simmons Z, Bowser RP, Ladha SS. Tocilizumab is safe and tolerable and reduces C-reactive protein concentrations in the plasma and cerebrospinal fluid of ALS patients. Muscle Nerve. 2021 Sep;64(3):309-320. doi: 10.1002/mus.27339. Epub 2021 Jun 24.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Shafeeq Ladha, MD principal investigator
Barrow Neurological Institute
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Tocilizumab in people with ALS and Amyotrophic Lateral Sclerosis, run by Barrow Neurological Institute at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 29 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Dec 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 18 Dec 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Patients Tolerant to Study Drug (time frame: 16 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02469896, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Dec 2019.