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Amyotrophic Lateral SclerosisPhase 2NCT03613571

A Study to Evaluate the Safety, Tolerability and Efficacy of ILB in Patients With Amyotrophic Lateral Sclerosis

A Phase 2 study testing ILB in people with Amyotrophic Lateral Sclerosis, run by TikoMed AB at 1 site in Sweden.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This is a Phase 2a single-centre, open single-arm study in patients with Amyotrophic Lateral Sclerosis (ALS) of intermediate progression rate. Eligible subjects will be administered weekly doses of ILB. A total of 5 subcutaneous (s.c.) doses will be administered at the study clinic. Read more about this study

Who is it for?
Ages 18 to 80 years
Conditions the study is for
Amyotrophic Lateral Sclerosis
What is being tested?
ILB
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
13 participants
Where
1 siteSweden
Study dates
15 Aug 2018to 20 Aug 2019
Phase
Phase 2
Are there results?
None posted in the registry

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 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Willing and able to give written informed consent for participation in the study.
  • Clinical diagnosis of Amyotrophic Lateral Sclerosis (ALS).
  • Male or female patients between 18 to 80 years (inclusive).
  • Forced Vital Capacity (FVC) 65% of predicted value for gender, height and age at screening.
  • Evaluated with ALSFRS-R and Norris clinical rating scales for at least the past 4 weeks before study drug administration.

Who cannot join

  • Unable to understand information about the study or are expected not to collaborate with the study team.
  • Concurrent serious disease, other than ALS, at the discretion of the nvestigator.
  • Pregnancy.
  • Patients of childbearing potential not willing to use adequate double contraception with less than 1 percentage failure rate after the screening visit until the last visit.
  • Addiction to drugs or alcohol.
  • Confirmed HIV, hepatitis B or hepatitis C.
Show all 12Show fewer
  • Known bleeding disorders or abnormal bleeding events.
  • Treatment with anticoagulant drugs warfarin and novel oral anticoagulants (NOAC) within the last 14 days prior to screening.
  • Treatment with Riluzole or Lamotrigine within the last 28 days prior to study drug administration.
  • Hypersensitivity to dextran sulfate.
  • Poor venous access.
  • Patients with clinically significant abnormal PK-INR, fibrinogen, von Willebrand factor and activated partial thromboplastin time (APTT) at screening.

Trial locations and contacts

1 site in 1 country.

Sweden1 site

  • Sahlgrenska University Hospital

    Gothenburg

Other trials in Sweden

About this study

Official title: A Single-centre, Open Single-arm Study Where the Safety, Tolerability and Efficacy of Subcutaneously Administered ILB Will be Evaluated in Patients With Amyotrophic Lateral Sclerosis

This is a Phase 2a single-centre, open single-arm study in patients with Amyotrophic Lateral Sclerosis (ALS) of intermediate progression rate. Eligible subjects will be administered weekly doses of ILB. A total of 5 subcutaneous (s.c.) doses will be administered at the study clinic.

The study consists of 10 visits; One 2-part screening visit, 5 ILB administration visits, and 3 follow-up visits. Each individual patient's study participation will be 4 months, including the screening and follow-up visits. Fifteen patients are planned to be included.

Read more

The primary objective of the study is to evaluate the safety and tolerability of ILB in patients diagnosed with ALS.

ILB is a solution for subcutaneous (s.c.) injection in saline solution. The dose administered will depend on the subject's body weight at the second study visit, prior to the first ILB administration.

No formal sample size calculation has been performed for this study. The proposed sample size is considered sufficient in this early phase 2 development to provide adequate information on the patients. Categorical data will be presented as counts and percentages. Continuous data will be summarised using descriptive statistics.

Being tested
ILB
Medical subject headings
Amyotrophic Lateral Sclerosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ILBDrug

    The investigational medicinal product ILB will be given as single short-term s.c. injections in the abdomen, the thigh or the buttock, in that order of priority. Subjects will be observed for at least 3 hours after injection.The IMP is a sterile, colourless to pale yellow solution for subcutaneous injection.

Study groups in NCT03613571
Study groupRoleWhat they receive
ILBExperimentalILB treatment
Receives: Drug: ILB

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Frequency, seriousness and intensity of Treatment-emergent Adverse Events (TEAEs)

    A TEAE is any adverse event (AE) not present prior to the initiation of IMP administration or any event already present that worsens in either intensity or frequency following exposure to the IMP. AEs (including baseline events) identified using any of the following methods will be recorded:

    • AEs spontaneously reported by the subject
    • AEs observed by the Investigator or medical personnel
    • AEs elicited based on non-leading questions from the Investigator or medical personnel

    Measured: up to 3 months

  2. Change in physical status

    A complete physical examination according to clinical praxis will be performed, including assessment of the head, eyes, ears, nose, throat (EENT), cardiac, peripheral vascular, pulmonary, musculoskeletal, neurologic, abdominal, lymphatic and dermatological functions.

    According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS)

    Change from baseline at 3 months.

    Measured: up to 3 months

  3. Change in vital signs - blood pressure

    Percent change in blood pressure (mmHg) from baseline at 3 months

    Measured: up to 3 months

  4. Change in electrocardiogram (ECG) recordings

    Change in single 12-lead ECG (PQ/PR (ms), QRS (ms), QT (ms) and QTcF (ms)) from baseline at 3 months

    Measured: up to 3 months

  5. Change in vital signs - heart rate

    Percent change in heart rate (bpm, beats per minute) from baseline at 3 months

    Measured: up to 3 months

  6. Change in vital signs - body temperatue

    Percent change in body temperature (degrees Celsius) from baseline at 3 months

    Measured: up to 3 months

  7. Change in safety laboratory measurements - sodium, potassium, chloride, calcium, glucose (non-fasting)

    According to clinical praxis, laboratory tests for sodium, potassium, chloride, calcium, glucose (non-fasting) will be analysed. Unit of measure is mmol/L

    According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS)

    Change from baseline at 3 months.

    Measured: up to 3 months

  8. Change in safety laboratory measurements - albumin

    According to clinical praxis, laboratory test for albumin will be analysed. Unit of measure is g/L

    According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS)

    Change from baseline at 3 months.

    Measured: up to 3 months

  9. Change in safety laboratory measurements - AST, ALT, CK, alkaline phosphatase

    According to clinical praxis, laboratory tests for aspartate amino-transferase (AST), alanine amino-transferase (ALT), creatine kinase (CK) and alkaline phosphatase will be analysed. Unit of measure is micro-kat/L

    According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS)

    Change from baseline at 3 months.

    Measured: up to 3 months

  10. Change in safety laboratory measurements - creatinine and total bilirubin

    According to clinical praxis, laboratory tests for creatinine and total bilirubin will be analysed. Unit of measure is micro-mol/L

    According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS)

    Change from baseline at 3 months.

    Measured: up to 3 months

  11. Change in safety laboratory measurements - myoglobin

    According to clinical praxis, laboratory test for myoglobin will be analysed. Unit of measure is micro-g/L

    According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS)

    Change from baseline at 3 months.

    Measured: up to 3 months

  12. Change in safety laboratory measurements - CRP

    According to clinical praxis, laboratory test for C-reactive protein (CRP) will be analysed. Unit of measure is milli-g/L

    According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS)

    Change from baseline at 3 months.

    Measured: up to 3 months

  13. Change in hematology laboratory measurements - Hemoglobin and fibrinogen

    According to clinical praxis, laboratory tests for hemoglobin (Hb) and fibrinogen will be analysed. Unit of measure is g/L

    According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS)

    Change from baseline at 3 months.

    Measured: up to 3 months

  14. Change in hematology laboratory measurements - Red blood cell count

    According to clinical praxis, laboratory test for blood cell count (RBC) will be analysed. Unit of measure is 10x12/L

    According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS)

    Change from baseline at 3 months.

    Measured: up to 3 months

  15. Change in hematology laboratory measurements - WBC, platelets, basophils, eosinophils, lymphocytes, monocytes, neutrophils

    According to clinical praxis, laboratory tests for white blood cell count (WBC), platelets, basophils, eosinophils, lymphocytes, monocytes and neutrophils will be analysed. Unit of measure is 10x9/L

    According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS)

    Change from baseline at 3 months.

    Measured: up to 3 months

  16. Change in hematology laboratory measurements - APTT

    According to clinical praxis, laboratory test for activated partial thromboplastin time (aPTT) will be analysed. Unit of measure is s

    According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS)

    Change from baseline at 3 months.

    Measured: up to 3 months

  17. Change in hematology laboratory measurements - PK-INR

    According to clinical praxis, laboratory test for prothrombin kinase international normalized ratio (PK-INR) will be analysed. Unitless measure

    According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS)

    Change from baseline at 3 months.

    Measured: up to 3 months

Secondary outcomes (9)

  1. Change in functional rating with Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R)

    The ALSFRS-R provides a physician-generated estimate of the patient's degree of functional impairment, which can be evaluated serially to objectively assess any response to treatment or progression of disease. The ALSFRS-R includes questions that ask the physician to rate his/her impression of the patients level of functional impairment in performing one of twelve common tasks. Each task is rated on a five-point scale from 0 = can't do, to 4 = normal ability. Individual item scores are summed to produce a reported score of between 0=worst and 48=best

    Absolute change from baseline at 3 months

    Measured: up to 3 months

  2. Change in functional rating with Norris rating scale

    The Norris rating scale provides a physician-generated estimate of the patient's degree of functional impairment, which can be evaluated serially to objectively assess any response to treatment or progression of disease. The Norris rating scale includes questions that ask the physician to rate his/her impression of the patients level of functional impairment in performing one of 34 common tasks and bodily functions. Each task or function is rated on a four-point scale from 0 = can't do, to 3 = normal ability. Individual item scores are summed to produce a reported score of between 0=worst and 100=best

    Absolute change from baseline at 3 months

    Measured: up to 3 months

  3. Change in pulmonary function (FVC) from baseline

    Measured: up to 3 months

  4. Change in Quality of Life (QoL) assessed by visual analogue scale (VAS)

    Questionnaire with three questions for patient and next-of-kin self reporting of:

    • general health status
    • physical health status
    • mental health status

    Each item is rated on a millimeter scale from 0 = very bad, to 100 = very good

    Absolute change from baseline at 3 months

    Measured: up to 1.5 months

  5. Change in functional rating of autonomous and sensory symptoms

    The autonomous and sensory rating scale provides a physician-generated estimate of the patient's degree of functional impairment, which can be evaluated serially to objectively assess any response to treatment or progression of disease. The rating scale includes questions that ask the physician to rate his/her impression of the patients level of impairment in 16 autonomous and sensory functions. Each function is rated on a four-point scale from 0 = not impaired, to 3 = very impaired.

    Absolute change from baseline at 3 months

    Measured: up to 3 months

  6. Change in maximum plasma concentration (Cmax) of ILB

    Measured: up to 1 month

  7. Change in exposure (Area Under the Curve, AUC) of ILB

    Measured: up to 1 month

  8. Changes in APTT (effect APTT) from day of dosing (day 1)

    Measured: up to 1 month

  9. Change in plasma concentration of hepatocyte growth factor (HGF)

    Measured: up to 1 month

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Aug 2018
  2. Study start15 Aug 2018
  3. Primary completion20 Aug 2019
  4. Study completion20 Aug 2019

Why it stopped: Inclusion was stopped with 13 of 15 patients recruited according to protocol. Patients responded with no safety signals. Study stop was due to slow recruitment.

Resources

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
13 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03613571
Sponsor's protocol ID
1.5, 2019-06-25
Other IDs
2017-005065-47, 2017
Status last verified
Jun 2023
First submitted
15 Jun 2018
Last updated
12 Jun 2023
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No

Publications

  • Logan A, Nagy Z, Barnes NM, Belli A, Di Pietro V, Tavazzi B, Lazzarino G, Lazzarino G, Bruce L, Persson LI. A phase II open label clinical study of the safety, tolerability and efficacy of ILB(R) for Amyotrophic Lateral Sclerosis. PLoS One. 2022 May 25;17(5):e0267183. doi: 10.1371/journal.pone.0267183. eCollection 2022.

    PubMed 35613082

  • Lazzarino G, Mangione R, Belli A, Di Pietro V, Nagy Z, Barnes NM, Bruce L, Ropero BM, Persson LI, Manca B, Saab MW, Amorini AM, Tavazzi B, Lazzarino G, Logan A. ILB(R) Attenuates Clinical Symptoms and Serum Biomarkers of Oxidative/Nitrosative Stress and Mitochondrial Dysfunction in Patients with Amyotrophic Lateral Sclerosis. J Pers Med. 2021 Aug 14;11(8):794. doi: 10.3390/jpm11080794.

    PubMed 34442438

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ILB in people with Amyotrophic Lateral Sclerosis, run by TikoMed AB at 1 site in Sweden.

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 5 inclusion and 12 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: stopped early, as of the sponsor's last update on 12 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Sweden. 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. 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?

The study was stopped early (reason given: Inclusion was stopped with 13 of 15 patients recruited according to protocol. Patients responded with no safety signals. Study stop was due to slow recruitment.). Results are not available in the source record.

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?

Frequency, seriousness and intensity of Treatment-emergent Adverse Events (TEAEs) (time frame: up to 3 months). The study lists 17 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 NCT03613571, which also lists 2 linked publications.

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 12 Jun 2023.

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