Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
LTX-002 · 1 trial · 1 indication
Incidence and severity of adverse events (AEs), treatment-emergent adverse events (TEAEs) and serious adverse events
Clinical laboratory tests including serum chemistry and hematology; urinalysis
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.
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.
Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including heart rate
Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including the QRS complex (normally 70-100 ms)
Description: Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including the QT interval measure
Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including QTc (heart rate-corrected QT interval)
Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including PR intervals (normally 120-200 ms)
| Arm | Type | Description |
|---|---|---|
| LTX-002 | EXPERIMENTAL | LTX-002 is an antisense oligonucleotide (ASO) targeting SPTLC1 messenger RNA (mRNA). Several dose levels will be tested. |
| Placebo (aCSF) | PLACEBO_COMPARATOR | Sterile aCSF solution formulation intended for intrathecal administration. |
| Name | Type | Description |
|---|---|---|
| LTX-002 | DRUG | 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. |
Inclusion Criteria: * 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 Exclusion Criteria: * Current evidence or history of a clinically significant medical condit...