Motor Neurone Disease - Systematic Multi-Arm Adaptive Randomised Trial

NCT ID: NCT04302870

Last Updated: 2025-06-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Clinical Phase

PHASE2/PHASE3

Total Enrollment

1150 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-02-27

Study Completion Date

2030-12-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

MND-SMART is investigating whether selected drugs can slow down the progression of motor neuron disease (MND) and improve survival.

The study is 'multi-arm' meaning more than one treatment will be tested at the same time. The trial started with 3 arms; drug 1 (memantine), drug 2 (trazodone) and placebo (dummy drug). A third drug, amantadine, was added in April 2023. A fourth drug, tacrolimus, was added in March 2025 in Edinburgh and across all sites in April 2025. The first two drugs, memantine and trazodone, were removed from the trial in September 2023 due to lack of benefit. The trial currently has 4 recruiting arms; amantadine, liquid placebo (matched to amantadine), tacrolimus, and tablet placebo (matched to tacrolimus). This allows the evaluation of each drug versus placebo. Participants will be randomly allocated between the treatment arms they are eligible for. Medicines being tested are already approved for use in other conditions.

MND-SMART has an 'adaptive' design. This means medicines being studied can change according to emerging results. Treatments shown to be ineffective can be dropped and new drugs can be added over the duration of the study. This will allow many treatments, over time, to be efficiently and definitively evaluated.

The medicines being tested have been selected following a rigorous process involving a systematic, unbiased, and comprehensive review of past clinical trials data, as well as information from pre-clinical research (studies in laboratories), for MND and other related neurodegenerative disorders. Drugs have been ranked for inclusion in MND-SMART by a group of independent MND experts according to set criteria. These include consideration of how the drugs work, their safety profiles, and the quality of previous studies.

New drugs will be selected for investigation in MND-SMART based on continuous review of constantly updated scientific evidence as well as findings from state-of-the-art human stem cell based drug discovery platforms. These can be added by substantial amendment to the protocol.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

For further information, please visit: https://mnd-smart.org/

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Motor Neuron Disease, Amyotrophic Lateral Sclerosis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Memantine

Group Type EXPERIMENTAL

Memantine Hydrochloride Oral Solution

Intervention Type DRUG

Memantine hydrocholoride taken once daily

Trazodone

Group Type EXPERIMENTAL

Trazodone Hydrochloride oral solution

Intervention Type DRUG

Trazodone Hydrochloride taken once daily

Placebo (liquid)

Group Type PLACEBO_COMPARATOR

Placebo oral solution

Intervention Type DRUG

Placebo taken once daily

Amantadine

Group Type EXPERIMENTAL

Amantadine Hydrochloride Oral Solution

Intervention Type DRUG

Amantadine Hydrochloride taken once daily

Tacrolimus

Group Type EXPERIMENTAL

Tacrolimus 1Mg Cap

Intervention Type DRUG

Tacrolimus 1Mg overencapsulated tablet taken once daily

Placebo (tablet)

Group Type PLACEBO_COMPARATOR

Placebo capsule

Intervention Type DRUG

Placebo taken once daily

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Memantine Hydrochloride Oral Solution

Memantine hydrocholoride taken once daily

Intervention Type DRUG

Trazodone Hydrochloride oral solution

Trazodone Hydrochloride taken once daily

Intervention Type DRUG

Placebo oral solution

Placebo taken once daily

Intervention Type DRUG

Amantadine Hydrochloride Oral Solution

Amantadine Hydrochloride taken once daily

Intervention Type DRUG

Tacrolimus 1Mg Cap

Tacrolimus 1Mg overencapsulated tablet taken once daily

Intervention Type DRUG

Placebo capsule

Placebo taken once daily

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Confirmed diagnosis of MND. This includes the following subtypes: ALS by El Escorial Criteria (possible, probable, and definite) or Gold Coast Criteria, Primary Lateral Sclerosis, and Progressive Muscular Atrophy
* Over 18
* Women of childbearing potential according to CTFG guidelines must have a negative pregnancy test within 7 days prior to, or at, the baseline visit
* Women of childbearing potential and fertile men must be using an appropriate method of contraception to avoid any unlikely teratogenic effects of the selected drugs from time of consent, to 4 weeks after treatment inclusive
* Willing and able to comply with the trial protocol and ability to understand and complete questionnaires
* Written informed consent (in the case of limb dysfunction verbal consent can be given in the presence of a witness who can sign)

Exclusion Criteria

* Patients diagnosed with Frontotemporal Dementia (FTD-MND) or any other significant psychiatric disorder that prevents informed consent being given.
* Alcoholism (current self-reported - at the investigator's discretion)
* Active suicide ideation assessed using the Columbia-Suicide Severity Rating Scale
* On concurrent investigational devices and medication (including biological therapy)
* Pregnancy or breast-feeding females
* If ALT, ALP, bilirubin or GGT \>3 times the upper limit of normal.
* If creatinine clearance (creatinine clearance or eGFR) \<35 ml/min.
* If TSH \<0.2mU/l (if possible to test free T4, then Serum free T4 \>25pmol/l)
* If corrected QT interval on 12 lead ECG \>500 ms
* Patient's diagnosed with ventricular arrhythmias, significant heart block (at the investigator's discretion)) or in the immediate recovery period after myocardial infarction (\< 6 weeks).
* Patients who the PI considers will not be able to comply with the study protocol.


* Patients in the manic phase of bipolar disorder.
* Patients with history of proven peptic ulcer confirmed on endoscopy
* Patients with active epilepsy
* Already taking the IMP in this comparison
* Known hypersensitivity, including hereditary fructose intolerance, or adverse reaction to the active substances and their excipients (as per SPCs for this comparison) or any past medical history contraindicating use of the IMP in this comparison


* Poorly controlled hypertension (Systolic BP\>180 mmHg or Diastolic BP\>100mmHg)
* Poorly controlled diabetes (HbA1c\>6.4% or 48mmol/mol)
* Hypertrophic cardiomyopathy or history of QT prolongation (including family history), congestive heart failure, bradyarrhythmias, and electrolyte abnormalities
* History of bleeding disorders or significant haematological or immune diseases including, congenital or acquired immune deficiency, anaemia (Hb\<130g/L for males and Hb\<120 g/L in females) and thrombocytopenia (platelet count \<150 × 109/L), use of other biological agents and immunosuppressant medications including oral/IV steroids
* Active or chronic infection (at PI discretion)
* History of Hepatitis B or C
* History of lymphoma and active malignancy
* Risk of dehydration due to reduced oral intake and lack of parenteral route
* Patient's contraindicated to tacrolimus according to SPC section 4.3
* Use of concomitant medications that interacts with tacrolimus according to the SPC, including but not limited to strong CYP3A4 inhibitors (i.e. azoles, protease inhibitors) or CYP3A4 inducers (rifampicin, phenytoin, carbamazepine), barbiturates, macrolides, digoxin, statins, PPI inhibitors, ergotamine, tricyclic antidepressants, herbal supplements (St. John's wort, extracts of Schisandra sphenanthera)
* Inability to swallow capsules
* Already taking the IMP in this comparison
* Known hypersensitivity, including lactose and gelatin intolerance, or adverse reaction to the active substances and their excipients (as per SPCs for this comparison) or any past medical history contraindicating use of the IMP in this comparison
* Receipt of a live attenuated vaccine within four weeks prior to receipt of tacrolimus. These include, but are not limited to live influenza vaccine (Fluenz Tetra), Shingles (varicella zoster virus) Zostavax, Varicella (Varilrix, Varilvax), Oral typhoid (Ty21a), and yellow fever vaccines.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

University College, London

OTHER

Sponsor Role collaborator

University of Warwick

OTHER

Sponsor Role collaborator

NHS Lothian

OTHER_GOV

Sponsor Role collaborator

University of Edinburgh

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Professor Chandran

Role: STUDY_DIRECTOR

University of Edinburgh

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Southern Health and Social Care Trust, Craigavon Area Hospital

Portadown, County Armagh, United Kingdom

Site Status RECRUITING

Aberdeen Royal Infirmary

Aberdeen, , United Kingdom

Site Status RECRUITING

University Hospitals of Birmingham NHS Foundation Trust

Birmingham, , United Kingdom

Site Status RECRUITING

University Hospitals Sussex NHS Foundation Trust

Brighton, , United Kingdom

Site Status RECRUITING

West Suffolk NHS Foundation Trust

Bury St Edmunds, , United Kingdom

Site Status RECRUITING

Cambridge University Hospitals NHS Foundation Trust

Cambridge, , United Kingdom

Site Status RECRUITING

Cardiff and Vale University Local Health Board

Cardiff, , United Kingdom

Site Status RECRUITING

Clinical Research Centre , Ninewells Hospital

Dundee, , United Kingdom

Site Status RECRUITING

Anne Rowling Regenerative Neurology Clinic

Edinburgh, , United Kingdom

Site Status RECRUITING

Royal Devon and Exeter Hospital

Exeter, , United Kingdom

Site Status RECRUITING

Queen Elizabeth University Hospital Clinical Research Facility

Glasgow, , United Kingdom

Site Status RECRUITING

NHS Highland Clinical Research Facility, Raigmore Hospital

Inverness, , United Kingdom

Site Status RECRUITING

East Suffolk and North Essex NHS Foundation Trust

Ipswich, , United Kingdom

Site Status RECRUITING

Royal London Hospital

London, , United Kingdom

Site Status RECRUITING

St George's University Hospitals NHS Foundation Trust

London, , United Kingdom

Site Status RECRUITING

King's College Hospital NHS Foundation Trust

London, , United Kingdom

Site Status RECRUITING

Newcastle upon Tyne Hospitals NHS Foundation Trust

Newcastle upon Tyne, , United Kingdom

Site Status RECRUITING

Norfolk and Norwich University Hospitals NHS Foundation Trust

Norwich, , United Kingdom

Site Status RECRUITING

University Hospitals of Dorset NHS Trust

Poole, , United Kingdom

Site Status RECRUITING

Clinical Research Facility Salford Royal NHS Foundation Trust

Salford, , United Kingdom

Site Status RECRUITING

Sheffield Teaching Hospitals NHS Foundation Trust

Sheffield, , United Kingdom

Site Status RECRUITING

Clinical Research Facility University Hospital Southampton

Southampton, , United Kingdom

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

United Kingdom

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Professor Chandran

Role: CONTACT

0131 465 9612

Amy Stenson

Role: CONTACT

0131 242 9122

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Raeburn Forbes

Role: primary

Callum Duncan

Role: primary

Venkatamaran Srinivasan

Role: primary

Francesca Crawley

Role: primary

Rhys Roberts

Role: primary

Ken Dawson

Role: primary

Ian Morrison

Role: primary

Judith Newton

Role: primary

0131 465 9517

Timothy Harrower

Role: primary

George Gorrie

Role: primary

Javier Carod Artal

Role: primary

Clare Galton

Role: primary

Aleks Radunovic

Role: primary

Pablo Garcia Reitboeck

Role: primary

Tim Williams

Role: primary

Godwin Mamutse

Role: primary

Hisham Hamdalla

Role: primary

Christopher McDermott

Role: primary

Ashwin Pinto

Role: primary

References

Explore related publications, articles, or registry entries linked to this study.

Pal S, Chataway J, Swingler R, Macleod MR, Carragher NO, Hardingham G, Selvaraj BT, Smith C, Wong C, Newton J, Lyle D, Stenson A, Dakin RS, Ihenacho A, Colville S, Mehta AR, Stallard N, Carpenter JR, Parker RA, Keerie C, Weir CJ, Virgo B, Morris S, Waters N, Gray B, MacDonald D, MacDonald E, Parmar MKB, Chandran S; MND SMART Investigators. Safety and efficacy of memantine and trazodone versus placebo for motor neuron disease (MND SMART): stage two interim analysis from the first cycle of a phase 3, multiarm, multistage, randomised, adaptive platform trial. Lancet Neurol. 2024 Nov;23(11):1097-1107. doi: 10.1016/S1474-4422(24)00326-0. Epub 2024 Sep 19.

Reference Type DERIVED
PMID: 39307154 (View on PubMed)

Wong C, Gregory JM, Liao J, Egan K, Vesterinen HM, Ahmad Khan A, Anwar M, Beagan C, Brown FS, Cafferkey J, Cardinali A, Chiam JY, Chiang C, Collins V, Dormido J, Elliott E, Foley P, Foo YC, Fulton-Humble L, Gane AB, Glasmacher SA, Heffernan A, Jayaprakash K, Jayasuriya N, Kaddouri A, Kiernan J, Langlands G, Leighton D, Liu J, Lyon J, Mehta AR, Meng A, Nguyen V, Park NH, Quigley S, Rashid Y, Salzinger A, Shiell B, Singh A, Soane T, Thompson A, Tomala O, Waldron FM, Selvaraj BT, Chataway J, Swingler R, Connick P, Pal S, Chandran S, Macleod M. Systematic, comprehensive, evidence-based approach to identify neuroprotective interventions for motor neuron disease: using systematic reviews to inform expert consensus. BMJ Open. 2023 Feb 1;13(2):e064169. doi: 10.1136/bmjopen-2022-064169.

Reference Type DERIVED
PMID: 36725099 (View on PubMed)

Parker RA, Weir CJ, Pham TM, White IR, Stallard N, Parmar MKB, Swingler RJ, Dakin RS, Pal S, Chandran S. Statistical analysis plan for the motor neuron disease systematic multi-arm adaptive randomised trial (MND-SMART). Trials. 2023 Jan 16;24(1):29. doi: 10.1186/s13063-022-07007-z.

Reference Type DERIVED
PMID: 36647114 (View on PubMed)

Wong C, Dakin RS, Williamson J, Newton J, Steven M, Colville S, Stavrou M, Gregory JM, Elliott E, Mehta AR, Chataway J, Swingler RJ, Parker RA, Weir CJ, Stallard N, Parmar MKB, Macleod MR, Pal S, Chandran S. Motor Neuron Disease Systematic Multi-Arm Adaptive Randomised Trial (MND-SMART): a multi-arm, multi-stage, adaptive, platform, phase III randomised, double-blind, placebo-controlled trial of repurposed drugs in motor neuron disease. BMJ Open. 2022 Jul 7;12(7):e064173. doi: 10.1136/bmjopen-2022-064173.

Reference Type DERIVED
PMID: 35798516 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

AC18082

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

ALS Phase II Study of NX210c
NCT06365216 ACTIVE_NOT_RECRUITING PHASE2
Dimethyl Fumarate in Adrenomyeloneuropathy
NCT06513533 RECRUITING PHASE2/PHASE3