A Phase 1 interventional study of NI112 and Placebo in Amyotrophic Lateral Sclerosis (ALS), sponsored by Sachi Bioworks Inc.. Not yet recruiting at 3 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-30.
Sponsored by Sachi Bioworks Inc. · Phase 1, Interventional, and Treatment
This is a first-in-human study of NI112, an investigational medicine given as an injection under the skin, in adults with amyotrophic lateral sclerosis (ALS). The purpose of the study is to find out how safe NI112 is, how well it is tolerated, and how the body handles it after a single dose. Up to 40 adults with ALS will take part at up to three study centers in the United States. Participants will be assigned by chance to receive either a single dose of NI112 or a single dose of placebo (sterile salt water). The study tests up to seven dose levels, one after another, beginning with the lowest dose. A Safety Review Board reviews the safety information from each group of participants before the next, higher dose is given to a new group. Each participant stays in the hospital for 24 hours after the injection for safety monitoring and blood sampling, and returns for follow-up visits on Day 8 and Day 28. The study also measures the amount of NI112 in blood and in spinal fluid.
This is a Phase 1, first-in-human, multi-center, randomized, double-blind, placebo-controlled, sequential single ascending dose-escalation study evaluating the safety, tolerability and pharmacokinetics of NI112 administered by subcutaneous injection to adults with amyotrophic lateral sclerosis. Up to seven sequential dose cohorts are planned at 2.5, 5, 10, 20, 40, 80 and 120 mg. Cohorts at 20 mg and below enroll 4 subjects randomized 3 active to 1 placebo; cohorts above 20 mg enroll 8 subjects randomized 6 active to 2 placebo. The 5 mg cohort may be omitted only under pre-specified pharmacokinetic criteria. The Safety Review Board may expand any of the first four cohorts to a maximum of 8 subjects, and total enrollment will not exceed 40 subjects. Within each new dose level the first two subjects, one active and one placebo, are dosed at least 48 hours apart and the Safety Review Board reviews all available safety data for that sentinel pair before the remaining subjects in the cohort are dosed. Safety Review Board authorization is required before each dose escalation, and dose increments do not exceed two-fold between consecutive cohorts except where the 5 mg cohort is omitted. Each participant is observed as an inpatient for 24 hours after dosing and completes safety follow-up through Day 28.
1,002 studies on the registry are indexed under Amyotrophic Lateral Sclerosis; 284 are open to participants now.
This study's planned enrollment of 40 is close to the median of 37 across 679 interventional studies indexed under Amyotrophic Lateral Sclerosis.
Browse Amyotrophic Lateral Sclerosis studies →This is the only study on the registry with Sachi Bioworks Inc. as lead sponsor.
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Able to understand the purpose and risks of the study and to provide signed, dated informed consent and authorization for use of protected health information. A legally authorized representative may consent if the subject is legally unable to do so.
Exclusion Criteria:
Positive test for, or history of, human immunodeficiency virus.
Drug: NI112 Injection 2.5 mg
Drug: NI112
Drug: NI112 Injection 5 mg
Drug: NI112
Drug: NI112 Injection 10 mg
Drug: NI112
Drug: NI112 Injection 20 mg
Drug: NI112
Drug: NI112 Injection 40 mg
Drug: NI112
Drug: NI112 Injection 80 mg
Drug: NI112
Drug: NI112 Injection 120 mg
Drug: NI112
Drug: Placebo
Drug: Placebo
Antisense Nanoligomer administered at 40 mg/mL, as a single subcutaneous injection on Day 1. Doses above 40 mg are divided across two injection sites with no more than 1.5 mL per site.
Sterile normal saline (0.9% sodium chloride), administered as a single subcutaneous injection on Day 1.
Number of Participants With incidence and severity of treatment-emergent adverse events
Number and percentage of participants with treatment-emergent adverse events, graded by severity according to NCI CTCAE version 5.0.
Time frame: Day 1 through Day 28
Number of Participants With incidence and severity of treatment-emergent serious adverse events
Number and percentage of participants with treatment-emergent serious adverse events, graded by severity according to NCI CTCAE version 5.0.
Time frame: Day 1 through Day 28
Number of Participants With Treatment-Emergent Clinically Significant Blood Pressure Abnormalities
Systolic and diastolic blood pressure, in mmHg, measured at screening, predose on Day 1, within one hour before the 2-hour and 4-hour postdose pharmacokinetic blood draws on Day 1, at 24 hours postdose on Day 2, on Day 8 and on Day 28. A value or finding is counted as clinically significant where it is newly present after dosing and the Investigator records it as clinically significant, applying the protocol definition: a finding that meets serious adverse event criteria, necessitates specific corrective therapy, or is deemed clinically significant by the Investigator. The single reported value is the NUMBER OF PARTICIPANTS with at least one such finding, by treatment arm.
Time frame: Day 1 through Day 28
Number of Participants With Treatment-Emergent Clinically Significant Pulse Rate Abnormalities
Pulse rate, in beats per minute, measured at screening, predose on Day 1, within one hour before the 2-hour and 4-hour postdose pharmacokinetic blood draws on Day 1, at 24 hours postdose on Day 2, on Day 8 and on Day 28. A value or finding is counted as clinically significant where it is newly present after dosing and the Investigator records it as clinically significant, applying the protocol definition: a finding that meets serious adverse event criteria, necessitates specific corrective therapy, or is deemed clinically significant by the Investigator. The single reported value is the NUMBER OF PARTICIPANTS with at least one such finding, by treatment arm.
Time frame: Day 1 through Day 28
Number of Participants With Treatment-Emergent Clinically Significant Respiratory Rate Abnormalities
Respiratory rate, in breaths per minute, measured at screening, predose on Day 1, within one hour before the 2-hour and 4-hour postdose pharmacokinetic blood draws on Day 1, at 24 hours postdose on Day 2, on Day 8 and on Day 28. A value or finding is counted as clinically significant where it is newly present after dosing and the Investigator records it as clinically significant, applying the protocol definition: a finding that meets serious adverse event criteria, necessitates specific corrective therapy, or is deemed clinically significant by the Investigator. The single reported value is the NUMBER OF PARTICIPANTS with at least one such finding, by treatment arm.
Time frame: Day 1 through Day 28
Number of Participants With Treatment-Emergent Clinically Significant Body Temperature Abnormalities
Body temperature, in °C, measured at screening, predose on Day 1, within one hour before the 2-hour and 4-hour postdose pharmacokinetic blood draws on Day 1, at 24 hours postdose on Day 2, on Day 8 and on Day 28. A value or finding is counted as clinically significant where it is newly present after dosing and the Investigator records it as clinically significant, applying the protocol definition: a finding that meets serious adverse event criteria, necessitates specific corrective therapy, or is deemed clinically significant by the Investigator. The single reported value is the NUMBER OF PARTICIPANTS with at least one such finding, by treatment arm.
Time frame: Day 1 through Day 28
Number of Participants With Treatment-Emergent Clinically Significant Neurological Examination Findings
A neurological examination is performed at screening, predose on Day 1, at 3 hours and 6 hours postdose on Day 1, at 24 hours postdose on Day 2, and on Day 8. A value or finding is counted as clinically significant where it is newly present after dosing and the Investigator records it as clinically significant, applying the protocol definition: a finding that meets serious adverse event criteria, necessitates specific corrective therapy, or is deemed clinically significant by the Investigator. The single reported value is the NUMBER OF PARTICIPANTS with at least one such finding, by treatment arm.
Time frame: Day 1 through Day 8
Number of Participants With Treatment-Emergent Clinically Significant Physical Examination Findings
A physical examination is performed at screening and predose on Day 1. A limited physical examination is performed at 24 hours postdose on Day 2 and on Day 8, at the Investigator's discretion. A value or finding is counted as clinically significant where it is newly present after dosing and the Investigator records it as clinically significant, applying the protocol definition: a finding that meets serious adverse event criteria, necessitates specific corrective therapy, or is deemed clinically significant by the Investigator. The single reported value is the NUMBER OF PARTICIPANTS with at least one such finding, by treatment arm.
Time frame: Day 1 through Day 8
Number of Participants With Treatment-Emergent Clinically Significant 12-Lead Electrocardiogram Abnormalities, Including QT Interval Corrected by Fridericia's Formula (QTcF)
Triplicate 12-lead electrocardiograms are recorded after the participant has rested supine for at least 10 minutes, at screening, predose on Day 1, within one hour before the 2-hour and 4-hour postdose pharmacokinetic blood draws on Day 1, at 24 hours postdose on Day 2, on Day 8 and on Day 28. The first recording is interpreted and the remaining two are checked for consistency and quality. The QT interval is corrected by Fridericia's formula and reported in milliseconds. A QTcF above 500 ms, or an increase from baseline above 60 ms, meets a protocol-defined dose-limiting toxicity criterion. A value or finding is counted as clinically significant where it is newly present after dosing and the Investigator records it as clinically significant, applying the protocol definition: a finding that meets serious adverse event criteria, necessitates specific corrective therapy, or is deemed clinically significant by the Investigator. The single reported value is the NUMBER OF PARTICIPANTS wit
Time frame: Day 1 through Day 28
Number of Participants With Treatment-Emergent Clinically Significant Hematology Abnormalities
Hematology comprises a complete blood count with differential, platelet count and absolute neutrophil count, collected at screening, predose on Day 1, at 24 hours postdose on Day 2, on Day 8 and on Day 28, and analyzed by a designated central laboratory. Coagulation testing (platelet count, international normalized ratio, prothrombin time and activated partial thromboplastin time) is performed at screening only. A laboratory value is counted as clinically significant where, per the protocol, it meets serious adverse event criteria, necessitates specific corrective therapy, or is deemed clinically significant by the Investigator. The single reported value is the number of participants with at least one such value, by treatment arm.
Time frame: Day 1 through Day 28
Number of Participants With Treatment-Emergent Clinically Significant Serum Chemistry Abnormalities
Serum chemistry comprises electrolytes, total protein, albumin, creatinine, blood urea nitrogen, uric acid, total and direct bilirubin, alkaline phosphatase, alanine aminotransferase, aspartate aminotransferase, gamma-glutamyl transferase, glucose, calcium, phosphorus, bicarbonate, chloride, sodium and potassium, collected at screening, predose on Day 1, at 24 hours postdose on Day 2, on Day 8 and on Day 28, and analyzed by a designated central laboratory. A laboratory value is counted as clinically significant where, per the protocol, it meets serious adverse event criteria, necessitates specific corrective therapy, or is deemed clinically significant by the Investigator. The single reported value is the number of participants with at least one such value, by treatment arm.
Time frame: Day 1 through Day 28
Number of Participants With Treatment-Emergent Clinically Significant Urinalysis Abnormalities
Urinalysis is performed by dipstick testing for blood, protein and glucose, with microscopic examination where an abnormality is detected, at screening, predose on Day 1, at 24 hours postdose on Day 2, on Day 8 and on Day 28, analyzed by a designated central laboratory. A finding is counted as clinically significant where, per the protocol, it meets serious adverse event criteria, necessitates specific corrective therapy, or is deemed clinically significant by the Investigator. The single reported value is the number of participants with at least one such finding, by treatment arm.
Time frame: Day 1 through Day 28
Number of Participants With discontinuations due to adverse events
Number and percentage of participants discontinuing study intervention or withdrawing from the study because of an adverse event.
Time frame: Day 1 through Day 28
Maximum tolerated dose of single-dose NI112
The maximum tolerated dose identified by the Safety Review Board applying the pre-specified dose-escalation and stopping criteria together with clinical judgment.
Time frame: From the first dose of the first cohort through the Day 28 visit of the last subject in the highest cohort evaluated, approximately 13 months
Peak Plasma Concentration (Cmax) of NI112
NI112 concentration in plasma is measured by a validated analytical assay in samples collected predose on Day 1, at 1, 2, 4 and 6 hours postdose on Day 1, at 24 hours postdose on Day 2, on Day 8 and on Day 28. Cmax is the maximum observed plasma concentration, reported as a geometric mean with geometric coefficient of variation by dose cohort.
Time frame: Predose and 1, 2, 4, 6 and 24 hours postdose, and on Day 8 and Day 28
Time to Peak Plasma Concentration (Tmax) of NI112
NI112 concentration in plasma is measured by a validated analytical assay in samples collected predose on Day 1, at 1, 2, 4 and 6 hours postdose on Day 1, at 24 hours postdose on Day 2, on Day 8 and on Day 28. Tmax is the time at which the maximum observed plasma concentration occurs, reported as a median with range by dose cohort.
Time frame: Predose and 1, 2, 4, 6 and 24 hours postdose, and on Day 8 and Day 28
Area Under the Plasma Concentration Versus Time Curve From Time Zero to the Last Measurable Concentration (AUC0-last) of NI112
NI112 concentration in plasma is measured by a validated analytical assay in samples collected predose on Day 1, at 1, 2, 4 and 6 hours postdose on Day 1, at 24 hours postdose on Day 2, on Day 8 and on Day 28. AUC0-last is calculated by the trapezoidal method to the last measurable concentration, reported as a geometric mean with geometric coefficient of variation by dose cohort.
Time frame: Predose and 1, 2, 4, 6 and 24 hours postdose, and on Day 8 and Day 28
Area Under the Plasma Concentration Versus Time Curve From Time Zero Extrapolated to Infinity (AUC0-inf) of NI112
NI112 concentration in plasma is measured by a validated analytical assay in samples collected predose on Day 1, at 1, 2, 4 and 6 hours postdose on Day 1, at 24 hours postdose on Day 2, on Day 8 and on Day 28. AUC0-inf is AUC0-last plus the extrapolated terminal area, reported as a geometric mean with geometric coefficient of variation by dose cohort, and is calculated where the terminal phase is adequately characterized.
Time frame: Predose and 1, 2, 4, 6 and 24 hours postdose, and on Day 8 and Day 28
Apparent Terminal Elimination Half-Life (t1/2) of NI112 in Plasma
NI112 concentration in plasma is measured by a validated analytical assay in samples collected predose on Day 1, at 1, 2, 4 and 6 hours postdose on Day 1, at 24 hours postdose on Day 2, on Day 8 and on Day 28. t1/2 is the natural logarithm of 2 divided by the terminal elimination rate constant, reported as a geometric mean with geometric coefficient of variation by dose cohort.
Time frame: Predose and 1, 2, 4, 6 and 24 hours postdose, and on Day 8 and Day 28
Cerebrospinal fluid concentration of NI112
Cerebrospinal fluid is collected by lumbar puncture predose on Day 1 and 24 hours postdose on Day 2, and NI112 concentration is measured by a validated analytical assay. The concentration is reported as a geometric mean with geometric coefficient of variation by dose cohort.
Time frame: Day 1 predose and Day 2, 24 hours postdose
Cerebrospinal fluid to plasma partition coefficient
Cerebrospinal fluid is collected by lumbar puncture predose on Day 1 and 24 hours postdose on Day 2, and NI112 concentration is measured by a validated analytical assay. The partition coefficient is the NI112 concentration in cerebrospinal fluid divided by the NI112 concentration in the matched plasma sample, reported as a dimensionless ratio, geometric mean with geometric coefficient of variation by dose cohort.
Time frame: Day 2, 24 hours postdose
Plan to share: No — Individual participant data will not be shared. This is a first-in-human study of up to 40 participants with a rare disease at three named sites, with dose level, visit dates and detailed demographic data recorded for each participant. A dataset of that size and specificity cannot be reliably de-identified, and the risk of re-identifying individual participants outweighs the value of release. Summary results will be posted on ClinicalTrials.gov.
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Amyotrophic Lateral Sclerosis →