Amyotrophic Lateral SclerosisPhase 1NCT03981536
A Study to Evaluate AP-101 in Familial and Sporadic Amyotrophic Lateral Sclerosis (ALS)
A Phase 1 study testing AP-101 in people with Amyotrophic Lateral Sclerosis, run by AL-S Pharma at 3 sites in Canada.
Trial summary
Single ascending doses of AP-101 will be administered by intravenous (IV) infusion Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- AP-101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 3 sitesCanada
- Study dates
- 10 Oct 2019to 2 Nov 2020
- Phase
- Phase 1
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- All participants must adhere to contraception restrictions
- Female patients of non-childbearing potential due to:
- Menopause: spontaneous amenorrhea for at least 12 months not induced by a medical conditions such as anorexia nervosa and not taking medications that induced the amenorrhea (e.g., oral contraceptives, hormones, gonadotropin releasing hormones, anti-estrogens, selective estrogen receptor modulators, or chemotherapy)
- Surgical sterilization
- Have possible, probable, probable laboratory supported or definite and definite familial laboratory-supported ALS in accordance with the El-Escorial criteria
- Have familial or sporadic ALS.
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- With onset of ALS symptoms, specifically onset of muscle weakness within past 48 months
- Have slow vital capacity (SVC) of (greater than or equal to) ≥60%
- If on riluzole, must be on a stable dose
- If on edaravone, must have completed 2 cycles and are expected to remain on the same dose throughout the study
- Able to provide informed consent. If the patient is not able to provide written consent due to aggravation of disease condition, written informed consent may be provided by a legally authorized representative
- Have venous access sufficient to allow for blood sampling
- Have clinical laboratory test results within normal reference range for the population or study site, or results with acceptable deviations that are judged to be not clinically significant
Who cannot join
- Are currently enrolled in, or discontinued from, within the last 30 days, a clinical trial involving an investigational drug or device or off-label use of a drug or device, or any other type of medical research judged not to be scientifically or medically compatible with this study
- Have previously completed or withdrawn from this study
- Have a history or presence of medical illness including, but not limited to, any cognitive, cardiovascular, hepatic, hematological, renal, endocrine, or psychiatric, or any clinically significant laboratory abnormality that indicates a medical problem that would preclude study participation
- Show evidence of human immunodeficiency virus (HIV) infection and/or positive human HIV antibodies
- Show evidence of hepatitis C and/or positive hepatitis C antibody
- Show evidence of hepatitis B and/or positive hepatitis B surface antigen
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- Are women who are lactating.
- Have undergone a tracheostomy unless it was removed at least 6 months prior
- Are on feeding tube, unless the insertion of a feeding tube is considered prophylactic
- Are on nasal intermittent positive pressure ventilation (NIPPV) >4 hours per day or at the discretion of the medical monitor
- Have undergone stem cell therapy
Trial locations and contacts
3 sites in 1 country.
Canada3 sites
London Health Sciences Centre, University Hospital
London, Ontario, N6A 5A5
Montreal Neurological Institute & Hospital
Montreal, Quebec, H3A 2B4
Sunnybrook Health Sciences Centre, Toronto
Toronto, Ontario, M4N 3M5
About this study
Official title: A Multicenter, Open Label, Single-Ascending Dose Study to Evaluate Safety, Tolerability, and Pharmacokinetics of AP-101 in Familial and Sporadic Amyotrophic Lateral Sclerosis (ALS)
Single ascending doses of AP-101 will be administered by intravenous (IV) infusion
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- AP-101
- Keywords
- Familial Amyotrophic Lateral Sclerosis, Sporadic Amyotrophic Lateral Sclerosis
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Amyotrophic lateral sclerosis 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AP-101Drug
Administered by intravenous infusion (IV)
| Study group | Role | What they receive |
|---|---|---|
| AP-101: Dose Level 1 | Experimental | Single dose of AP-101 Receives: Drug: AP-101 |
| AP-101: Dose Level 2 | Experimental | Single dose of AP-101 Receives: Drug: AP-101 |
| AP-101: Dose Level 3 | Experimental | Single dose of AP-101 Receives: Drug: AP-101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With One or More Non-Serious Adverse Events (AEs) or Any Serious AEs (SAEs)
A clinical trial AE is any untoward medical event associated with the use of a drug or drug delivery system in humans, whether or not it is considered related to that drug or drug delivery system
Measured: Baseline up to day 84
Number of participants with abnormalities in vital signs, clinical laboratory assessments, physical or neurological examinations, or electrocardiograms (ECGs)
Vital signs include blood pressure, pulse rate, and body temperature
Measured: Baseline up to day 84
Secondary outcomes (4)
Maximum Observed Drug Concentration (Cmax)
In serum
Measured: Baseline up to day 84
Time of Maximum Drug Concentration (Tmax)
In serum
Measured: Baseline up to day 84
Area Under the Concentration Time Curve (AUC)
In serum
Measured: Baseline up to day 84
Pharmacokinetic Concentrations in Cerebrospinal Fluid (CSF)
Taken at screening, and then only one sample per participant post-dose, in the higher level doses
Measured: Screening, and at either 1 hour, 4 hours, 24 hours, 48 hours, 72 hours, or 168 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2019
- Study start10 Oct 2019
- Primary completion2 Nov 2020 (estimated)
- Study completion2 Nov 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies 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 1
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AL-S Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, AL-S Pharma SA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03981536
- Sponsor's protocol ID
- AP101-01
- Status last verified
- Jul 2020
- First submitted
- 30 May 2019
- Last updated
- 27 Oct 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open Label, Single-Ascending Dose Study to Evaluate Safety, Tolerability, and Pharmacokinetics of AP-101 in Familial and Sporadic Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT03981536
- Acronym / sponsor ID
- AP101-01
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- Familial Amyotrophic Lateral Sclerosis, Sporadic Amyotrophic Lateral Sclerosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- AL-S PharmaIndustry (pharma, biotech, devices)
- Study start
- 10 Oct 2019 (actual)
- Primary completion
- 2 Nov 2020 (estimated)
- Study completion
- 2 Nov 2020 (estimated)
- First posted
- 11 Jun 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Single ascending doses of AP-101 will be administered by intravenous (IV) infusion
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| AP-101: Dose Level 1 | Experimental | Drug: AP-101Single dose of AP-101 |
| AP-101: Dose Level 2 | Experimental | Drug: AP-101Single dose of AP-101 |
| AP-101: Dose Level 3 | Experimental | Drug: AP-101Single dose of AP-101 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- All participants must adhere to contraception restrictions
- Female patients of non-childbearing potential due to:
- Have possible, probable, probable laboratory supported or definite and definite familial laboratory-supported ALS in accordance with the El-Escorial criteria
- Have familial or sporadic ALS.
- With onset of ALS symptoms, specifically onset of muscle weakness within past 48 months
Key exclusion criteria (11 listed)
- Are currently enrolled in, or discontinued from, within the last 30 days, a clinical trial involving an investigational drug or device or off-label use of a drug or device, or any other type of medical research judged not to be scientifically or medically compatible with this study
- Have previously completed or withdrawn from this study
- Have a history or presence of medical illness including, but not limited to, any cognitive, cardiovascular, hepatic, hematological, renal, endocrine, or psychiatric, or any clinically significant laboratory abnormality that indicates a medical problem that would preclude study participation
- Show evidence of human immunodeficiency virus (HIV) infection and/or positive human HIV antibodies
- Show evidence of hepatitis C and/or positive hepatitis C antibody
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 |
|---|---|---|
| AP-101Administered by intravenous infusion (IV) | Drug | AP-101: Dose Level 1, AP-101: Dose Level 2, AP-101: Dose Level 3 |
Primary outcome
Number of Participants With One or More Non-Serious Adverse Events (AEs) or Any Serious AEs (SAEs)
Time frame: Baseline up to day 84
A clinical trial AE is any untoward medical event associated with the use of a drug or drug delivery system in humans, whether or not it is considered related to that drug or drug delivery system
Number of participants with abnormalities in vital signs, clinical laboratory assessments, physical or neurological examinations, or electrocardiograms (ECGs)
Time frame: Baseline up to day 84
Vital signs include blood pressure, pulse rate, and body temperature
4 secondary outcomes
Maximum Observed Drug Concentration (Cmax)
Time frame: Baseline up to day 84
Time of Maximum Drug Concentration (Tmax)
Time frame: Baseline up to day 84
Area Under the Concentration Time Curve (AUC)
Time frame: Baseline up to day 84
Pharmacokinetic Concentrations in Cerebrospinal Fluid (CSF)
Time frame: Screening, and at either 1 hour, 4 hours, 24 hours, 48 hours, 72 hours, or 168 hours
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Oct 2020.
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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.
Study Director study director
AL-S Pharma SA
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing AP-101 in people with Amyotrophic Lateral Sclerosis, run by AL-S Pharma at 3 sites in Canada.
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 13 inclusion and 11 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: status not confirmed, as of the sponsor's last update on 27 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Canada. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
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?
Number of Participants With One or More Non-Serious Adverse Events (AEs) or Any Serious AEs (SAEs) (time frame: Baseline up to day 84). 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?
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 27 Oct 2020.