A single-center, open single-arm study where the safety, tolerability and efficacy of subcutaneously administered ILB will be evaluated in patients with Amyotrophic Lateral Sclerosis.

• Register: EUCTR
• Last refreshed on: 10 December 2019
• Main ID: EUCTR2017-005065-47-SE
• Date of registration: 19/03/2018
• Prospective Registration: Yes
• Primary sponsor: TikoMed AB
• Public title: A single-center, open single-arm study where the safety, tolerability and efficacy of subcutaneously administered ILB will be evaluated in patients with Amyotrophic Lateral Sclerosis.
• Scientific 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
• Date of first enrolment: 15/05/2018
• Target sample size: 15
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-005065-47
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Sweden

Contacts

• Name: Lars Bruce
• Affiliation: TikoMed AB

• Name: Lars Bruce
• Affiliation: TikoMed AB

Key inclusion & exclusion criteria

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

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 9
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 6

Exclusion criteria:
1. Unable to understand information about the trial or are expected not to collaborate with the study team.
2. Concurrent serious disease, other than ALS.
3. Pregnancy.
4. Patients of child-bearing potential not willing to use adequate double contraception with <1 % failure rate after the screening visit until the last visit.
5. Addiction to drugs or alcohol.
6. Confirmed HIV, Hepatitis B or C.
7. Known bleeding disorders or abnormal bleeding events.
8. Treatment with anticoagulant drugs warfarin and novel oral anticoagulants (NOAC) within the last 14 days prior to screening.
9. Treatment with Riluzole or Lamotrigine within the last 28 days prior to study drug administration.
10. Hypersensitivity to dextran sulfate
11. Poor venous access.
12. Patients with clinically significant abnormal PK-INR, fibrinogen, von Willebrand factor and activated partial thromboplastin time (APTT) at screening


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Body processes [G] - Immune system processes [G12]

Amyotrophic Lateral Sclerosis (ALS)

Intervention(s)

Product Name: ILB
Product Code: ILB
Pharmaceutical Form: Solution for injection
INN or Proposed INN: ILB
CAS Number: 9011-18-1
Other descriptive name: DEXTRAN SULFATE SODIUM
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 20-

Primary Outcome(s)

Main Objective: To study safety and tolerability of subcutaneously administered ILB in patients diagnosed with ALS.

Secondary Objective: To evaluate efficacy of subcutaneously administered ILB in patients diagnosed with ALS.

Timepoint(s) of evaluation of this end point: At 9 visits over 4 months.

Primary end point(s): To evaluate safety by:
* Adverse events.
* Physical exam.
* Vital signs: blood pressure, heart rate, and body temperature.
* ECG assessment.
* Haematology and clinical chemistry lab tests.

Secondary Outcome(s)

Secondary end point(s): To evaluate efficacy by:
* ALSFRS-R functional rating scale.
* Norris functional rating scale.
* Plasma and CSF-analysis for biomarker Neurofilament light chain (NFL).
* Plasma, blood and serum analysis for biomarkers for neurological disorders.
* Respiratory function (FVC).
* Evaluation of quality of Life and autonomous symptoms.
* Levels of hepatocyte growth factor, pharmacokinetics and activated partial thromboplastin time.

Timepoint(s) of evaluation of this end point: At specific timepoints during up to 9 visits over several months.

Secondary ID(s)

TM-105

Source(s) of Monetary Support

TikoMed AB

Ethics review

Status: Approved
Approval date:
Contact: