Please note that the ANZCTR website will be unavailable from 1pm until 2pm (AEST) on Wednesday the 26th of June for website maintenance. Please be sure to log out of the system in order to avoid any loss of data. Thank you and apologies for any inconvenience caused.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial registered on ANZCTR

Registration number
Ethics application status
Date submitted
Date registered
Date last updated
Type of registration
Prospectively registered

Titles & IDs
Public title
Study to Assess the Efficacy and Safety of Tecfidera in Patients with Amyotrophic Lateral Sclerosis - TEALS Study
Scientific title
Phase 2 Randomised Placebo Controlled Double Blind Study to Assess the Efficacy and Safety of Tecfidera in Patients with Amyotrophic Lateral Sclerosis (TEALS Study)
Secondary ID [1] 294497 0
Nil Known
Universal Trial Number (UTN)
Trial acronym
TEALS study
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Amyotrophic Lateral Sclerosis 307269 0
Condition category
Condition code
Neurological 306388 306388 0 0
Neurodegenerative diseases
Musculoskeletal 306412 306412 0 0
Other muscular and skeletal disorders

Study type
Description of intervention(s) / exposure
The TEALS study is assessing the effects of Dimethyl Fumarate (trade name tecfidera ) in Amyotrophic Lateral Sclerosis. The dosage is 240 mg twice daily for a period of 36 weeks. Mode of administration is an oral tablet. Adherence to intervention will be drug tablet return.
Intervention code [1] 300794 0
Treatment: Drugs
Comparator / control treatment
The placebo is a micro crystalline cellulose capsule.
Control group

Primary outcome [1] 305395 0
The primary outcome for this study is Amyotrophic Lateral Sclerosis progression determined using the ALS functional Rating Scale-revised.
Timepoint [1] 305395 0
Timepoints for the primary outcome will be assessed at screening, week 0, week 12, week 24, week 36 and 40 weeks.
Secondary outcome [1] 345064 0
1. Survival status at 40 weeks. Assessed form hospital records

Timepoint [1] 345064 0
Time-points for the secondary outcome will be assessed at 40 weeks.
Secondary outcome [2] 345168 0
2. Change in lower motor dysfunction as measured by:
-Higher Neurophysiological Index (NPI) and Split Hand Index (SI).
-Improved muscle strength, as measured by power grip dynamometry and Medical Research Council (MRC) Score.

This is a composite outcome measure.
Timepoint [2] 345168 0
Timepoints for the secondary outcome will be assessed at 40 weeks.
Secondary outcome [3] 345169 0
3. Change in respiratory function as measured by:
-Forced vital capacity (FVC).
-Sniff nasal inspiratory pressure (SNIP).
Composite outcome measure.
Timepoint [3] 345169 0
Timepoints for the secondary outcome will be assessed at 40 weeks.
Secondary outcome [4] 345170 0
4. Change in urinary neurotrophin receptor P75 levels.
Timepoint [4] 345170 0
Timepoints for the secondary outcome will be assessed at screening, week 12, week 24, week 36 and 40 weeks.
Secondary outcome [5] 345171 0
5. Change in clinical scores will be measured using the ALS Specific Quality of Life-Revised (ALSSQOL-R) questionnaire
Timepoint [5] 345171 0
Timepoints for the secondary outcome will be assessed at 40 weeks.

Key inclusion criteria
1. Male and female patients aged 18 to 85 years at the time of the Screening Visit.
2. Able to provide informed consent and comply with study procedures.
3. Sporadic ALS diagnosed as definite, probable, or possible according to the Awaji criteria as determined by a neurologist subspecializing in ALS.
4. Disease duration at recruitment less than 24 months from diagnosis.
5. Patient must have the results of magnetic resonance imaging scan of brain and spinal cord undergone within 2 years (24 months) prior to the Screening Visit.
6. Forced vital capacity >60% of predicted value as adjusted for gender, height, and age at the Screening Visit.
7. Must be on a stable dose of riluzole for at least 30 days prior to the Screening Visit.
8. Patient who has established care with a neurologist at 1 of the 5 specialised ALS clinics involved in the study and will maintain this clinical care throughout the study.
9. If a patient is referred from a third party (neurologist or a State based ALS organisation) they should be willing to transfer care to the neurologist participating in the study.
10. Patients may participate in clinical registries, but will be excluded if they are participating in a clinical study involving an alternative investigational treatment.
11. Women of childbearing potential must have a negative urine pregnancy test at screening and Baseline, and be surgically sterile or postmenopausal, or using highly effective methods of contraception throughout the study and for 30 days after the last dose of IP.
Minimum age
18 Years
Maximum age
85 Years
Both males and females
Can healthy volunteers participate?
Key exclusion criteria
1. Dependence on mechanical ventilation at the time of screening.
2. Gastrostomy at the time of Screening/Baseline Visit. If the patient has a gastrostomy tube inserted post randomisation they will be allowed to continue in the study.
3. Participation in any other IP study or using an IP (within 12 weeks prior to screening).
4. Known hypersensitivity to Tecfidera or any excipients in this product.
5. Presence of a monogenic cause of ALS (e.g. known mutation in superoxide dismutase-1 (SOD1), expansion in c9orf72 etc.).
6. Taking immunosuppressive medications.
7. Positive test for human immunodeficiency virus (HIV), hepatitis B (+HbsAg), or hepatitis C.
8. Presence of any of the following clinical conditions at the time of screening:
-Unstable medical disease (such as unstable angina, heart failure, chronic obstructive pulmonary disease, liver disease or renal disease), or active infectious diseases (such as hepatitis B or C or tuberculosis), or current malignancy.
-Unstable psychiatric illness defined as psychosis or untreated major depression within 90 days of the Screening Visit. This exclusion criteria is based on a prior psychiatric diagnosis that is unstable as determined by the patient’s treating Psychiatrist.
-Dementia as previously diagnosed by a medical practitioner.

9. Safety Laboratory Criteria at the Screening Visit:
-Alanine aminotransferase >3 × the upper limit of normal (ULN).
-Total bilirubin, lactate, triglycerides, amylase, or lipase >2 × the ULN.
-Patient has impaired renal function defined as creatinine clearance of <40 mL/min via Cockroft Gault method.
-Absolute neutrophil count of <2 × 109/L.
-Absolute lymphocyte count of <0.5 × 109/L.
-Platelet concentration of <100 × 109/L.
-Haemoglobin <100 g/L.

10. Female patients who are pregnant or lactating, or intend to become pregnant during the study period.

Study design
Purpose of the study
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Central randomisation by computer
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Simple randomisation using a randomisation table created by computer software (i.e. computerised sequence generation). This will be overseen by IQUVIA (CRO) and Cenduit.
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Other design features
Phase 2
Type of endpoint/s
Statistical methods / analysis
The calculation of the sample size was based on the following criteria: (i) mean difference of 7 points in the ALSFRS-R between active and control groups at 9 months; (ii) common standard deviation (SD) of 8%; (iii) a value of 0.05; (iv) power 0.9; (v) active:control group (m-value) 2:1; and (vi) 20% dropout rate assumed for both groups.

Based on previous clinical studies and literature; it is assumed that the mean difference is 7 and common SD is 8% in the ALSFRS-R between active and control groups. A clinically important response rate on Tecfidera is defined as 0.9. A 2:1 (Dimethyl Fumarate:Placebo) allocation, 90% power and 5% statistical significance rate estimate 48:24 evaluable patients will be required for this study. With a 20% estimated non-evaluable rate, it is planned to assign 90 patients (60:30) to randomised treatment.

Recruitment status
Not yet recruiting
Date of first participant enrolment
Date of last participant enrolment
Date of last data collection
Sample size
Accrual to date
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 10553 0
Westmead Hospital - Westmead
Recruitment hospital [2] 10554 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [3] 10555 0
Royal Brisbane & Womens Hospital - Herston
Recruitment hospital [4] 10556 0
Calvary Health Care Bethlehem Ltd - Caulfield
Recruitment hospital [5] 10557 0
Flinders Medical Centre - Bedford Park
Recruitment hospital [6] 10558 0
Fiona Stanley Hospital - Murdoch
Recruitment postcode(s) [1] 22273 0
2050 - Camperdown
Recruitment postcode(s) [2] 22272 0
2145 - Westmead
Recruitment postcode(s) [3] 22275 0
3162 - Caulfield
Recruitment postcode(s) [4] 22274 0
4029 - Herston
Recruitment postcode(s) [5] 22276 0
5042 - Bedford Park
Recruitment postcode(s) [6] 22277 0
6150 - Murdoch

Funding & Sponsors
Funding source category [1] 299121 0
Name [1] 299121 0
Country [1] 299121 0
Primary sponsor type
University of Sydney
Camperdown NSW 2006
Secondary sponsor category [1] 298381 0
Name [1] 298381 0
Address [1] 298381 0
Country [1] 298381 0

Ethics approval
Ethics application status
Ethics committee name [1] 300055 0
Westmead Area Local Health District
Ethics committee address [1] 300055 0
Research Office, Level 2, REN Building
Westmead Hospital, Hawkesbury & Darcy Roads, Westmead NSW 2145
Ethics committee country [1] 300055 0
Date submitted for ethics approval [1] 300055 0
Approval date [1] 300055 0
Ethics approval number [1] 300055 0

Brief summary
The primary purpose of this study is to assess whether dimethyl fumarate will slow down disease progression in sporadic ALS. The study hypothesis is based on findings that the regulatory T cells, which form an important component of the immune system, slow down disease progression in ALS. increasing the levels and function of regulatory T cells could slow down disease progression in ALS. Dimethyl fumarate effectively increases the function of regulatory T cells and it is hoped that this increase in T cell function will significantly slow disease progression in ALS when compared to conventional treatment.
Trial website
Trial related presentations / publications
Public notes

Principal investigator
Name 82410 0
Prof Steve Vucic
Address 82410 0
Department of Neurology
Westmead Hospital
Cnr Hawkesbury and Darcy Roads
Westmead NSW 2145
Country 82410 0
Phone 82410 0
Fax 82410 0
Email 82410 0
Contact person for public queries
Name 82411 0
Prof Steve Vucic
Address 82411 0
Department of Neurology
Westmead Hospital
Cnr Hawkesbury and Darcy Roads
Westmead NSW 2145
Country 82411 0
Phone 82411 0
Fax 82411 0
Email 82411 0
Contact person for scientific queries
Name 82412 0
Prof Steve Vucic
Address 82412 0
Department of Neurology
Westmead Hospital
Cnr Hawkesbury and Darcy Roads
Westmead NSW 2145
Country 82412 0
Phone 82412 0
Fax 82412 0
Email 82412 0

No information has been provided regarding IPD availability

What supporting documents are/will be available?

No Supporting Document Provided

Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
SourceTitleYear of PublicationDOI
EmbaseDimethyl fumarate and its esters: A drug with broad clinical utility?.2020
EmbaseBiomarkers in amyotrophic lateral sclerosis: A review of new developments.2020
EmbaseIn a search for efficient treatment for amyotrophic lateral sclerosis: Old drugs for new approaches.2021
EmbaseHarnessing regulatory T cell neuroprotective activities for treatment of neurodegenerative disorders.2020
EmbaseDimethyl fumarate: A review of preclinical efficacy in models of neurodegenerative diseases.2022
EmbaseClinical studies in amyotrophic lateral sclerosis.2022
EmbaseNew developments and opportunities in drugs being trialed for amyotrophic lateral sclerosis from 2020 to 2022.2022
EmbaseImproving clinical trial outcomes in amyotrophic lateral sclerosis.2021
EmbasePhase 2 randomized placebo controlled double blind study to assess the efficacy and safety of tecfidera in patients with amyotrophic lateral sclerosis (TEALS Study): Study protocol clinical trial (SPIRIT Compliant).2020
Dimensions AIThe involvement of regulatory T cells in amyotrophic lateral sclerosis and their therapeutic potential2020
N.B. These documents automatically identified may not have been verified by the study sponsor.