Amyotrophic Lateral SclerosisPhase 1/2NeurALSNCT07660614
A Study of LTX-002 in Adult Participants With Amyotrophic Lateral Sclerosis
A Phase 1/2 study testing LTX-002 in people with Amyotrophic Lateral Sclerosis, run by Leal Therapeutics, Inc at 5 sites in 4 countries.
Trial summary
This study will examine what happens when patients with amyotrophic lateral sclerosis (ALS) are given an investigational medication (study drug) known as LTX-002. Specifically, the researchers will be looking at safety, tolerability (if someone has any side effects from the drug), pharmacokinetics (what the body does… Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- LTX-002
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 56 participants
- Where
- 5 sitesin 4 countries
- Study dates
- 29 Apr 2026to Jun 2031
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of ALS per Gold Coast criteria
- ALS symptom onset less than 36 months prior to Screening
- Slow vital capacity ≥ 50% of predicted value
- Body mass index ≥18 and ≤40 kg/m2
Who cannot join
- Current evidence or history of a clinically significant medical condition that, in the Investigator's judgement, would impact the participant's safety, interpretation of study results, or place the participant at high risk of poor treatment compliance or of not completing the study
- History of brain or spinal abnormalities on magnetic imaging (MRI) or computed tomography (CT) that might interfere with the lumbar puncture (LP), cerebrospinal fluid (CSF) circulation or safety assessments
- Prior treatment with antisense oligonucleotide (ASO), small interfering RNA, stem cell therapy, or gene therapy for any indication
- Tracheostomy
- HIV, Hepatitis B or C infection (acute or chronic)
- Presence of implanted shunt (CSF) or vascular device
- Risk for uncontrolled bleeding
- Pregnant or breastfeeding
Trial locations and contacts
5 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
5 sites in 4 countries
Italy2 sites, 2 recruiting
- Not yet recruiting
Istituto Neurologico "Carlo Besta"
Milan
Nilo Riva, MD, +49 02 2394 3062, nilo.riva@istituto-besta.it
- Recruiting
AOU Città della Salute e della Scienza di Torino
Torino
Andrea Calvo, MD, +39 011 670 9171, andrea.calvo@unito.it
Germany1 site, 1 recruiting
- Recruiting
University Hospital Schleswig-Holstein, Campus Lübeck
Lübeck
Julian Grosskreutz, MD, +49 451 500 43450, julian.grosskreutz@neuro.uni-luebeck.de
Netherlands1 site, 1 recruiting
- Recruiting
Universitair Medisch Centrum Utrecht
Utrecht
Leonard Van den Berg, MD, +31 088-755 7939, l.h.vandenberg@umcutrecht.nl
Sweden1 site, 1 recruiting
- Recruiting
Karolinska Universitetssjukhuset
Stockholm
Staffan Rosenborg, MD, +46-8-123 810 48, staffan.rosenborg@regionstockholm.se
About this study
Official title: A First-in-Human, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of Intrathecally Administered LTX-002 in Adult Participants With Amyotrophic Lateral Sclerosis
This study will examine what happens when patients with amyotrophic lateral sclerosis (ALS) are given an investigational medication (study drug) known as LTX-002. Specifically, the researchers will be looking at safety, tolerability (if someone has any side effects from the drug), pharmacokinetics (what the body does to the study drug) and pharmacodynamics (what the study drug does to the body). The study will also investigate the effect of the drug on indicators of the severity of ALS, such as markers in blood and in the cerebrospinal fluid (the fluid that surrounds the brain and spinal cord, CSF) and on measures of the participant's ability to move, speak, and breathe.
Read the detailed description
Eligible participants will be asked to visit the study site 9 times and stay overnight once. In addition, there will be telephone or video calls with the study staff on 8 separate occasions. Procedures will include:
- A review of medical history and medications
- Physical exams, including a neurologic (nervous system) assessment
- Electrocardiograms (a measure of the heart's electrical activity)
- Blood, urine and cerebrospinal fluid (CSF) collection
- Measures of lung function
- Measures of speech
- Measures of muscle strength
- Scales to assess ALS symptoms and overall health, and feelings about suicide
- Administration of the study drug (or placebo) by injection into the CSF through a lumbar (lower spinal area) injection
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- LTX-002, Placebo (aCSF)
- Keywords
- ALS
- 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.
LTX-002Drug
LTX-002 is an antisense oligonucleotide (ASO) targeting SPTLC1 messenger RNA (mRNA). Several dose levels will be tested.
Placebo (aCSF)Other
Sterile aCSF solution formulation intended for intrathecal administration.
| Study group | Role | What they receive |
|---|---|---|
| LTX-002 | Experimental | LTX-002 is an antisense oligonucleotide (ASO) targeting SPTLC1 messenger RNA (mRNA). Several dose levels will be tested. Receives: Drug: LTX-002 |
| Placebo (aCSF) | Placebo comparator | Sterile aCSF solution formulation intended for intrathecal administration. Receives: Other: Placebo (aCSF) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability (Adverse Events)
Incidence and severity of adverse events (AEs), treatment-emergent adverse events (TEAEs) and serious adverse events
Measured: Screening to Day 169
Safety and Tolerability (Clinical Laboratory Tests)
Clinical laboratory tests including serum chemistry and hematology; urinalysis
Measured: Screening to Day 169
Safety and Tolerability (Vital Signs)
Vital signs will be collected, including blood pressure (mm Hg), heart rate (beats per minute), respiratory rate (breaths per minute), body temperature (°C or °F), total body weight (kilograms), and height (centimeters). Body Mass Index (BMI) will be calculated using the values of total body weight and height, with the formula BMI = weight in kg/(height in cm)^2.
Measured: Screening to Day 169
Safety and Tolerability (Physical and Neurological exams)
Physical and neurological exams will be performed periodically to ensure participant safety. Height (centimeters or inches), weight (kilograms or pounds) and body mass index (BMI; kilograms/meters^2) will be measured.
Measured: Screening to Day 169
Safety and Tolerability (ECGs - heart rate)
Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including heart rate
Measured: Screening to Day 169
Safety and Tolerability (ECGs - QRS)
Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including the QRS complex (normally 70-100 ms)
Measured: Time Frame: Screening to Day 169
Safety and Tolerability (ECGs - QT)
Description: Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including the QT interval measure
Measured: Screening to Day 169
Safety and Tolerability (ECGs - QTc)
Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including QTc (heart rate-corrected QT interval)
Measured: Screening to Day 169
Safety and Tolerability (ECGs - PR intervals)
Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including PR intervals (normally 120-200 ms)
Measured: Screening to Day 169
Secondary outcomes (10)
Pharmacokinetics of LTX-002 (CSF and plasma levels)
The amount of LTX-002 in the cerebrospinal fluid (CSF) and plasma will be measured.
Measured: Day 1 to Day 169
Pharmacokinetics of LTX-002 (Cmax)
The maximum observed plasma concentration (Cmax) of LTX-002 will be calculated.
Measured: Day 1 to Day 169
Pharmacokinetics of LTX-002 (Tmax)
The time to maximum observed plasma concentration (Tmax) of LTX-002 will be calculated.
Measured: Day 1 through Day 169
Pharmacokinetics of LTX-002 (AUC0-∞)
The area under the plasma-time concentration curve from time 0 extrapolated to infinity (AUC0-∞) of LTX-002 will be calculated.
Measured: Day 1 to Day 169
Pharmacokinetics of LTX-002 (AUC0-t)
The area under the plasma concentration-time curve from time 0 to the last measurable concentration (AUC0-t) of LTX-002 will be calculated.
Measured: Day 1 to Day 169
Pharmacokinetics of LTX-002 (Tlag)
The delay between the time of dosing and the time of appearance of the first measurable concentration (Tlag) of LTX-002 in plasma will be calculated.
Measured: Day 1 to Day 169
Pharmacokinetics of LTX-002 (Vd/F)
The apparent volume of distribution (Vd/F) of LTX-002 in plasma, if applicable, will be calculated.
Measured: Day 1 to Day 169
Pharmacokinetics of LTX-002 (λz)
The apparent terminal elimination rate constant (λz) of LTX-002 in plasma, if applicable, will be calculated.
Measured: Day 1 to Day 169
Pharmacokinetics of LTX-002 (T1/2)
The half-life (T1/2) of LTX-002 in plasma, if applicable, will be calculated.
Measured: Day 1 to Day 169
Pharmacokinetics of LTX-002 (CL/F)
The apparent clearance (CL/F) of LTX-002 in plasma, if applicable, will be calculated.
Measured: Day 1 to Day 169
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2026
- Study start29 Apr 2026
- Primary completionJun 2031 (estimated)
- Study completionJun 2031 (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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Participants (8 per cohort, randomized in a 6:2 ratio to receive LTX-002 or placebo) will receive a total of three IT injections of LTX-002 (or placebo) on Days 1, 29, and 85
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 56 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Leal Therapeutics, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Leal Therapeutics, Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07660614
- Sponsor's protocol ID
- LTX-002-101
- Other IDs
- 2025-522039-33-00, 2025
- Acronym
- NeurALS
- Status last verified
- Aug 2026
- First submitted
- 18 May 2026
- Last updated
- 18 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A First-in-Human, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of Intrathecally Administered LTX-002 in Adult Participants With Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT07660614
- Acronym / sponsor ID
- NeurALS / LTX-002-101
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 56 participants (estimated)
- Lead sponsor
- Leal Therapeutics, IncIndustry (pharma, biotech, devices)
- Study start
- 29 Apr 2026 (actual)
- Primary completion
- Jun 2031 (estimated)
- Study completion
- Jun 2031 (estimated)
- First posted
- 22 Jun 2026 (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.
This study will examine what happens when patients with amyotrophic lateral sclerosis (ALS) are given an investigational medication (study drug) known as LTX-002. Specifically, the researchers will be looking at safety, tolerability (if someone has any side effects from the drug), pharmacokinetics (what the body does to the study drug) and pharmacodynamics (what the study drug does to the body). The study will also investigate the effect of the drug on indicators of the severity of ALS, such as markers in blood and in the cerebrospinal fluid (the fluid that surrounds the brain and spinal cord, CSF) and on measures of the participant's ability to move, speak, and breathe.
Detailed description
Eligible participants will be asked to visit the study site 9 times and stay overnight once. In addition, there will be telephone or video calls with the study staff on 8 separate occasions. Procedures will include:
- A review of medical history and medications
- Physical exams, including a neurologic (nervous system) assessment
- Electrocardiograms (a measure of the heart's electrical activity)
- Blood, urine and cerebrospinal fluid (CSF) collection
- Measures of lung function
- Measures of speech
- Measures of muscle strength
- Scales to assess ALS symptoms and overall health, and feelings about suicide
- Administration of the study drug (or placebo) by injection into the CSF through a lumbar (lower spinal area) injection
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Participants (8 per cohort, randomized in a 6:2 ratio to receive LTX-002 or placebo) will receive a total of three IT injections of LTX-002 (or placebo) on Days 1, 29, and 85
| Arm | Type | Receives |
|---|---|---|
| LTX-002 | Experimental | Drug: LTX-002LTX-002 is an antisense oligonucleotide (ASO) targeting SPTLC1 messenger RNA (mRNA). Several dose levels will be tested. |
| Placebo (aCSF) | Placebo comparator | Other: Placebo (aCSF)Sterile aCSF solution formulation intended for intrathecal administration. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Diagnosis of ALS per Gold Coast criteria
- ALS symptom onset less than 36 months prior to Screening
- Slow vital capacity ≥ 50% of predicted value
- Body mass index ≥18 and ≤40 kg/m2
Key exclusion criteria (8 listed)
- Current evidence or history of a clinically significant medical condition that, in the Investigator's judgement, would impact the participant's safety, interpretation of study results, or place the participant at high risk of poor treatment compliance or of not completing the study
- History of brain or spinal abnormalities on magnetic imaging (MRI) or computed tomography (CT) that might interfere with the lumbar puncture (LP), cerebrospinal fluid (CSF) circulation or safety assessments
- Prior treatment with antisense oligonucleotide (ASO), small interfering RNA, stem cell therapy, or gene therapy for any indication
- Tracheostomy
- HIV, Hepatitis B or C infection (acute or chronic)
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 |
|---|---|---|
| LTX-002LTX-002 is an antisense oligonucleotide (ASO) targeting SPTLC1 messenger RNA (mRNA). Several dose levels will be tested. | Drug | LTX-002 |
| Placebo (aCSF)Sterile aCSF solution formulation intended for intrathecal administration. | Other | Placebo (aCSF) |
Primary outcome
Safety and Tolerability (Adverse Events)
Time frame: Screening to Day 169
Incidence and severity of adverse events (AEs), treatment-emergent adverse events (TEAEs) and serious adverse events
Safety and Tolerability (Clinical Laboratory Tests)
Time frame: Screening to Day 169
Clinical laboratory tests including serum chemistry and hematology; urinalysis
Safety and Tolerability (Vital Signs)
Time frame: Screening to Day 169
Vital signs will be collected, including blood pressure (mm Hg), heart rate (beats per minute), respiratory rate (breaths per minute), body temperature (°C or °F), total body weight (kilograms), and height (centimeters). Body Mass Index (BMI) will be calculated using the values of total body weight and height, with the formula BMI = weight in kg/(height in cm)^2.
Safety and Tolerability (Physical and Neurological exams)
Time frame: Screening to Day 169
Physical and neurological exams will be performed periodically to ensure participant safety. Height (centimeters or inches), weight (kilograms or pounds) and body mass index (BMI; kilograms/meters^2) will be measured.
Safety and Tolerability (ECGs - heart rate)
Time frame: Screening to Day 169
Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including heart rate
Safety and Tolerability (ECGs - QRS)
Time frame: Time Frame: Screening to Day 169
Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including the QRS complex (normally 70-100 ms)
Safety and Tolerability (ECGs - QT)
Time frame: Screening to Day 169
Description: Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including the QT interval measure
Safety and Tolerability (ECGs - QTc)
Time frame: Screening to Day 169
Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including QTc (heart rate-corrected QT interval)
Safety and Tolerability (ECGs - PR intervals)
Time frame: Screening to Day 169
Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including PR intervals (normally 120-200 ms)
10 secondary outcomes
Pharmacokinetics of LTX-002 (CSF and plasma levels)
Time frame: Day 1 to Day 169
Pharmacokinetics of LTX-002 (Cmax)
Time frame: Day 1 to Day 169
Pharmacokinetics of LTX-002 (Tmax)
Time frame: Day 1 through Day 169
Pharmacokinetics of LTX-002 (AUC0-∞)
Time frame: Day 1 to Day 169
Pharmacokinetics of LTX-002 (AUC0-t)
Time frame: Day 1 to Day 169
Pharmacokinetics of LTX-002 (Tlag)
Time frame: Day 1 to Day 169
Pharmacokinetics of LTX-002 (Vd/F)
Time frame: Day 1 to Day 169
Pharmacokinetics of LTX-002 (λz)
Time frame: Day 1 to Day 169
Pharmacokinetics of LTX-002 (T1/2)
Time frame: Day 1 to Day 169
Pharmacokinetics of LTX-002 (CL/F)
Time frame: Day 1 to Day 169
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Aug 2026.
- Individual participant dataSharing planned: No
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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.
Medical Monitor study director
Leal Therapeutics, Inc
5 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing LTX-002 in people with Amyotrophic Lateral Sclerosis, run by Leal Therapeutics, Inc at 5 sites in 4 countries.
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 4 inclusion and 8 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: looking for participants now, as of the sponsor's last update on 18 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Italy, Germany, Netherlands, 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Trials Information (267-503-4800, clinical.trials@lealtx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and Tolerability (Adverse Events) (time frame: Screening to Day 169). The study lists 9 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 18 Aug 2026.