Amyotrophic Lateral Sclerosis (ALS)Phase 1NCT06769620
A First in Human Study of ORT247 in Healthy Volunteers
A Phase 1 study testing ORT247 in people with Amyotrophic Lateral Sclerosis (ALS), run by Orthogonal Neuroscience Inc. at 1 site in United States.
Trial summary
This is a single center, double-blinded, randomized, placebo controlled single ascending dose clinical study, with the primary purpose of evaluating the safety, tolerability, pharmacokinetics (PK), and immunohistochemistry of escalating intravenous doses of ORT247 in healthy volunteers. Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- ORT247
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 44 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Jun 2022to 24 Oct 2023
- 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 to 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Subject voluntarily consents to participate in this study and provides written informed consent before the start of any study-specific procedures
- Male and females, 18 to 65 years of age at time of screening
- Female subjects of childbearing potential must not be breastfeeding and must have no plans to become pregnant during the course of the study through 120 days after infusion of study drug. Female subjects of childbearing potential who are heterosexual must agree to use a method of contraception considered to be highly effective (i.e., results in <1% failure rate when used consistently and correctly) from screening through 120 days after the last dose of study drug
- Female subjects of non-childbearing potential must have evidence from their medical history indicating that they are not of childbearing potential and must not currently be breastfeeding.
- Any non-vasectomized male subjects must have agreed to use barrier contraceptives plus spermicide for 200 days after dosing.
- Male subjects must agree not to donate sperm for 200 days after dosing
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- Female subjects must agree not to preserve eggs (ova) for 120 days after dosing
- Has not participated in a clinical drug study within 30 days of study start, or within 5 half-lives, unless study blind has been broken and the subject was known to be on placebo
- Body mass index of 18-32
Who cannot join
- Contraindication to undergo LP including international normalized ratio (INR) >1.4 or other coagulopathy, platelet cell count of <120,000/μL, infection at the desired LP site, current use of anti-coagulant medication except for low dose aspirin, degenerative arthritis, spinal scoliosis, back surgery, suspected increased intracranial pressure on history or neurologic exam, non-communicating hydrocephalus or intracranial mass, or prior history of spinal mass or trauma
- Any significant acute or chronic medical illness
- Any history of cancer within 5 years of enrollment with the exception of resected skin basal cell carcinoma
- Any major surgery within 4 weeks of study drug administration
- Donation of blood or serum >500 mL to a blood bank or in a clinical study (except screening visit) within 3 months of study drug administration
- Inability to undergo venipuncture or tolerate venous access
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- Has smoked or used tobacco products within 3 months before study drug administration
- Positive drug screen for alcohol, drugs of abuse, or tobacco
- Recent (within 6 months of study drug administration) drug or alcohol abuse as defined in Diagnostic and Statistical Manual of Mental Disorders, 5th edition (DSM-V), Diagnostic Criteria for Drug and Alcohol Abuse
- Evidence of any clinically significant neurological or psychiatric disorder that could interfere with study assessments as determined by investigator and sponsor
- History of or currently has schizophrenia, schizoaffective disorder or bipolar disorder, untreated major depression (DSM-V or International Statistical Classification of Diseases and Related Health Problems, 10th edition [ICD-10] criteria)
- Significant illness or infection requiring intervention within the prior 30 days as determined by investigator and sponsor (must test negative for active coronavirus disease 2019 [COVID-19])
- Indication of potential suicidality risk
- Any of the following abnormalities at screening: serum creatinine > upper limit of normal (ULN), hepatic transaminases (aspartate aminotransferase or alanine aminotransferase) > ULN, abnormal blood pressure based on the clinical judgment of the investigator, QTcF >470 msec
- Known history of hypersensitivity to any component of the ORT247 drug product or placebo
- Currently taking, or planning to take, any medication (prescription or over-the-counter) that would potentially affect the assessment of pharmacokinetics (PK), pharmacodynamics (PD), or immunogenicity of ORT247.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1, Randomized, Double-blind, Placebo-Controlled, Single-Ascending-Dose Study to Evaluate the Safety, Tolerability, Immunogenicity, and Pharmacokinetics of Intravenous ORT247 in Healthy Volunteers
This is a single center, double-blinded, randomized, placebo controlled single ascending dose clinical study, with the primary purpose of evaluating the safety, tolerability, pharmacokinetics (PK), and immunohistochemistry of escalating intravenous doses of ORT247 in healthy volunteers.
Read the detailed description
This is a phase 1, first in human, study of ORT247
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- ORT247, Placebo
- 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.
ORT247Drug
ORT247 will be provided to study sites in single-use, sterile vials for infusion. Each dose will be prepared with normal saline for infusion.
PlaceboDrug
Placebo consists of normal saline
| Study group | Role | What they receive |
|---|---|---|
| ORT247 | Experimental | Single ascending dose of intravenous ORT247 administered as a infusion over 60 minutes. Subjects enrolled into 1 of 5 planned cohorts will receive 75mg, 150mg, 300mg, 600mg, and 1200mg with dosing being conducted in a staggered fashion, separated by at least 30 minutes of an infusion in one subject and the start of an infusion in another subject. Receives: Drug: ORT247 |
| Placebo | Placebo comparator | Single intravenous dose of vehicle with dosing being conducted in a staggered fashion, separated by at least 30 minutes of an infusion in one subject and the start of an infusion in another subject. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-emergent adverse events assessed by severity, frequency and causality
Measured: From enrollment to day 85 or early termination
Secondary outcomes (9)
Time to maximum observed serum concentration (Tmax)
Measured: Before and at end of infusion (0 hours), and 0.5, 1, 2, 4, 8, and 12, 24, 36 and 48 hours after infusion. Additional samples for assessment of serum concentrations were collected on Day 8, 15, 29, 43, 57, 71 and 85/early termination.
Maximum observed serum concentration (Cmax)
Measured: Before and at end of infusion (0 hours), and 0.5, 1, 2, 4, 8, and 12, 24, 36 and 48 hours after infusion. Additional samples for assessment of serum concentrations were collected on Day 8, 15, 29, 43, 57, 71 and 85/early termination.
Number of subjects developing anti-drug antibodies
Titer and neutralizing activity of anti-drug antibodies will be evaluated
Measured: From enrollment to day 85 or early termination
Area under the curve from Time 0 to 168 hours (AUC0-168)
Measured: Before and at end of infusion (0 hours), and 0.5, 1, 2, 4, 8, and 12, 24, 36 and 48 hours after infusion. Additional samples for assessment of serum concentrations were collected on Day 8, 15, 29, 43, 57, 71 and 85/early termination.
Area under the curve from Time 0 to last sampling time (AUC0-t)
Measured: Before and at end of infusion (0 hours), and 0.5, 1, 2, 4, 8, and 12, 24, 36 and 48 hours after infusion. Additional samples for assessment of serum concentrations were collected on Day 8, 15, 29, 43, 57, 71 and 85/early termination.
Area under the curve from Time 0 to infinity (AUC0-∞)
Measured: Before and at end of infusion (0 hours), and 0.5, 1, 2, 4, 8, and 12, 24, 36 and 48 hours after infusion. Additional samples for assessment of serum concentrations were collected on Day 8, 15, 29, 43, 57, 71 and 85/early termination.
Time to clearance (CL) following single i.v. infusion of ORT247
Measured: Before and at end of infusion (0 hours), and 0.5, 1, 2, 4, 8, and 12, 24, 36 and 48 hours after infusion. Additional samples for assessment of serum concentrations were collected on Day 8, 15, 29, 43, 57, 71 and 85/early termination.
Volume of distribution (Vd) following single i.v. infusion of ORT247
Measured: Before and at end of infusion (0 hours), and 0.5, 1, 2, 4, 8, and 12, 24, 36 and 48 hours after infusion. Additional samples for assessment of serum concentrations were collected on Day 8, 15, 29, 43, 57, 71 and 85/early termination.
Terminal half-life (t1/2) following single i.v. infusion of ORT247
Measured: Before and at end of infusion (0 hours), and 0.5, 1, 2, 4, 8, and 12, 24, 36 and 48 hours after infusion. Additional samples for assessment of serum concentrations were collected on Day 8, 15, 29, 43, 57, 71 and 85/early termination.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jan 2025
- Study start6 Jun 2022
- Primary completion24 Oct 2023
- Study completion24 Oct 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral Sclerosis (ALS)Studies 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
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- After screening, eligible subjects will be enrolled into 1 of 5 planned cohorts: 75 mg, 150 mg, 300 mg, 600 mg, and 1200 mg. The study will start with the lowest dose cohort, and each dose cohort will consist of 8 subjects (6 active, 2 placebo). A safety review committee consisting of the sponsor's medical director, the contract research organization's (CRO's) medical monitor, and the site principal investigator will review AE data collected after dosing to evaluate the following: 1. Acceptability of proceeding with enrollment of the full cohort of 8 subjects based on review of safety data of the initial 2 subjects (sentinels: 1 active, 1 placebo) through 48 hours after dosing 2. Acceptability of proceeding with enrollment of the subsequent cohort and studying ascending doses based on review of data for the full cohort of 8 subjects (6 active, 2 placebo) through 7 days after dosing and ongoing data from prior cohorts.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- Orthogonal Neuroscience Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06769620
- Sponsor's protocol ID
- ORT247-001
- Status last verified
- Jan 2025
- First submitted
- 6 Jan 2025
- Last updated
- 15 Jan 2025
- Results posted
- No
- Data monitoring committee
- No
- 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 15 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Randomized, Double-blind, Placebo-Controlled, Single-Ascending-Dose Study to Evaluate the Safety, Tolerability, Immunogenicity, and Pharmacokinetics of Intravenous ORT247 in Healthy Volunteers
- ClinicalTrials.gov ID
- NCT06769620
- Acronym / sponsor ID
- ORT247-001
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 44 participants (actual)
- Lead sponsor
- Orthogonal Neuroscience Inc.Industry (pharma, biotech, devices)
- Study start
- 6 Jun 2022 (actual)
- Primary completion
- 24 Oct 2023 (actual)
- Study completion
- 24 Oct 2023 (actual)
- First posted
- 10 Jan 2025 (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 is a single center, double-blinded, randomized, placebo controlled single ascending dose clinical study, with the primary purpose of evaluating the safety, tolerability, pharmacokinetics (PK), and immunohistochemistry of escalating intravenous doses of ORT247 in healthy volunteers.
Detailed description
This is a phase 1, first in human, study of ORT247
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
- Model description
- After screening, eligible subjects will be enrolled into 1 of 5 planned cohorts: 75 mg, 150 mg, 300 mg, 600 mg, and 1200 mg. The study will start with the lowest dose cohort, and each dose cohort will consist of 8 subjects (6 active, 2 placebo). A safety review committee consisting of the sponsor's medical director, the contract research organization's (CRO's) medical monitor, and the site principal investigator will review AE data collected after dosing to evaluate the following: 1. Acceptability of proceeding with enrollment of the full cohort of 8 subjects based on review of safety data of the initial 2 subjects (sentinels: 1 active, 1 placebo) through 48 hours after dosing 2. Acceptability of proceeding with enrollment of the subsequent cohort and studying ascending doses based on review of data for the full cohort of 8 subjects (6 active, 2 placebo) through 7 days after dosing and ongoing data from prior cohorts.
| Arm | Type | Receives |
|---|---|---|
| ORT247 | Experimental | Drug: ORT247Single ascending dose of intravenous ORT247 administered as a infusion over 60 minutes. Subjects enrolled into 1 of 5 planned cohorts will receive 75mg, 150mg, 300mg, 600mg, and 1200mg with dosing being conducted in a staggered fashion, separated by at least 30 minutes of an infusion in one subject and the start of an infusion in another subject. |
| Placebo | Placebo comparator | Drug: PlaceboSingle intravenous dose of vehicle with dosing being conducted in a staggered fashion, separated by at least 30 minutes of an infusion in one subject and the start of an infusion in another subject. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Subject voluntarily consents to participate in this study and provides written informed consent before the start of any study-specific procedures
- Male and females, 18 to 65 years of age at time of screening
- Any non-vasectomized male subjects must have agreed to use barrier contraceptives plus spermicide for 200 days after dosing.
- Male subjects must agree not to donate sperm for 200 days after dosing
- Female subjects must agree not to preserve eggs (ova) for 120 days after dosing
Key exclusion criteria (16 listed)
- Contraindication to undergo LP including international normalized ratio (INR) >1.4 or other coagulopathy, platelet cell count of <120,000/μL, infection at the desired LP site, current use of anti-coagulant medication except for low dose aspirin, degenerative arthritis, spinal scoliosis, back surgery, suspected increased intracranial pressure on history or neurologic exam, non-communicating hydrocephalus or intracranial mass, or prior history of spinal mass or trauma
- Any significant acute or chronic medical illness
- Any history of cancer within 5 years of enrollment with the exception of resected skin basal cell carcinoma
- Any major surgery within 4 weeks of study drug administration
- Donation of blood or serum >500 mL to a blood bank or in a clinical study (except screening visit) within 3 months of study drug administration
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 |
|---|---|---|
| ORT247ORT247 will be provided to study sites in single-use, sterile vials for infusion. Each dose will be prepared with normal saline for infusion. | Drug | ORT247 |
| PlaceboPlacebo consists of normal saline | Drug | Placebo |
Primary outcome
Number of participants with treatment-emergent adverse events assessed by severity, frequency and causality
Time frame: From enrollment to day 85 or early termination
9 secondary outcomes
Time to maximum observed serum concentration (Tmax)
Time frame: Before and at end of infusion (0 hours), and 0.5, 1, 2, 4, 8, and 12, 24, 36 and 48 hours after infusion. Additional samples for assessment of serum concentrations were collected on Day 8, 15, 29, 43, 57, 71 and 85/early termination.
Maximum observed serum concentration (Cmax)
Time frame: Before and at end of infusion (0 hours), and 0.5, 1, 2, 4, 8, and 12, 24, 36 and 48 hours after infusion. Additional samples for assessment of serum concentrations were collected on Day 8, 15, 29, 43, 57, 71 and 85/early termination.
Number of subjects developing anti-drug antibodies
Time frame: From enrollment to day 85 or early termination
Area under the curve from Time 0 to 168 hours (AUC0-168)
Time frame: Before and at end of infusion (0 hours), and 0.5, 1, 2, 4, 8, and 12, 24, 36 and 48 hours after infusion. Additional samples for assessment of serum concentrations were collected on Day 8, 15, 29, 43, 57, 71 and 85/early termination.
Area under the curve from Time 0 to last sampling time (AUC0-t)
Time frame: Before and at end of infusion (0 hours), and 0.5, 1, 2, 4, 8, and 12, 24, 36 and 48 hours after infusion. Additional samples for assessment of serum concentrations were collected on Day 8, 15, 29, 43, 57, 71 and 85/early termination.
Area under the curve from Time 0 to infinity (AUC0-∞)
Time frame: Before and at end of infusion (0 hours), and 0.5, 1, 2, 4, 8, and 12, 24, 36 and 48 hours after infusion. Additional samples for assessment of serum concentrations were collected on Day 8, 15, 29, 43, 57, 71 and 85/early termination.
Time to clearance (CL) following single i.v. infusion of ORT247
Time frame: Before and at end of infusion (0 hours), and 0.5, 1, 2, 4, 8, and 12, 24, 36 and 48 hours after infusion. Additional samples for assessment of serum concentrations were collected on Day 8, 15, 29, 43, 57, 71 and 85/early termination.
Volume of distribution (Vd) following single i.v. infusion of ORT247
Time frame: Before and at end of infusion (0 hours), and 0.5, 1, 2, 4, 8, and 12, 24, 36 and 48 hours after infusion. Additional samples for assessment of serum concentrations were collected on Day 8, 15, 29, 43, 57, 71 and 85/early termination.
Terminal half-life (t1/2) following single i.v. infusion of ORT247
Time frame: Before and at end of infusion (0 hours), and 0.5, 1, 2, 4, 8, and 12, 24, 36 and 48 hours after infusion. Additional samples for assessment of serum concentrations were collected on Day 8, 15, 29, 43, 57, 71 and 85/early termination.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jan 2025.
- 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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ORT247 in people with Amyotrophic Lateral Sclerosis (ALS), run by Orthogonal Neuroscience Inc. at 1 site in United States.
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 9 inclusion and 16 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: finished, as of the sponsor's last update on 15 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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 treatment-emergent adverse events assessed by severity, frequency and causality (time frame: From enrollment to day 85 or early termination). 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 15 Jan 2025.