Amyotrophic Lateral SclerosisPhase 1PRO-101NCT05279755
A Study to Evaluate the Safety and Pharmacokinetics of Single and Multiple Doses of Prosetin in Healthy Volunteers and Participants With ALS
A Phase 1 study testing prosetin in people with Amyotrophic Lateral Sclerosis, run by ProJenX at 4 sites in 3 countries.
Trial summary
The primary purpose of this study is to evaluate the safety and tolerability of prosetin in healthy volunteers and participants with ALS. 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?
- prosetin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 72 participants
- Where
- 4 sitesin 3 countries
- Study dates
- 26 Feb 2022to 31 Oct 2026
- Phase
- Phase 1
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adults ≥18 years of age
- Diagnosis of ALS based on the Gold Coast diagnostic criteria
- Slow Vital Capacity (SVC) >50% predicted
- If being concomitantly treated with riluzole and/or locally approved standard of care treatments, the participant must be on a stable dose for at least 30 days prior to screening and throughout the study
- In the opinion of the Investigator, participant is able to swallow liquid in order to ingest the study medication.
- Participants who meet all of the following criteria may be included in Part D of the study:
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- Participants must have completed 14 days of blinded treatment in Part C.
- Participants taking approved ALS standard-of-care medications must remain on stable doses through Day 28 of open-label treatment.
- In the judgment of the Investigator, the participant's participation in the open-label portion of the study is medically appropriate
Who cannot join
- Active dementia, neurologic diseases other than ALS, or psychiatric illness that in the opinion of the investigator would affect participation in the current study.
- Significant history or clinical manifestation of comorbid disease in any organ system that currently requires active treatment or is likely to require treatment during the study.
- Any episodes of vertigo in the previous 12 months prior to screening.
- Any medical history of seizures, or any clinically significant EEG finding at Screening or at Day -1.
- A diagnosis of cancer or evidence of continued disease within five years before screening. Protocol-specified exceptions may be considered with approval from the Sponsor's Medical Monitor.
- Participation in any other investigational study drug trial in which receipt of an investigational study drug occurred within 30 days prior to the first dose of study medication.
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- Prior exposure to any stem cell or gene therapies (investigational or off-label) for the treatment of ALS.
- Treatment with any other investigational drug or device throughout the duration of the study is excluded, with the exception of any COVID-19 vaccine or treatment with an emergency use authorization.
- NOTE: Other protocol-defined Inclusion/Exclusion Criteria may apply. Please contact trials@projenx.com with any questions about eligibility criteria.
Trial locations and contacts
4 sites in 3 countries.
4 sites in 3 countries
United States2 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Worldwide Clinical Trials Early Phase Services
San Antonio, Texas, 78217
Canada1 site
The Neuro - Montréal Neurological Institute-Hospital
Montreal, Quebec, H3A 2B4
Netherlands1 site
University Medical Center Utrecht
Utrecht, Utrecht, 3584 CX
About this study
Official title: A Phase 1, Randomized, Double-Blind, Placebo-Controlled Dose Escalating Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending and Multiple Ascending Doses of Prosetin in Healthy Volunteers and Participants With Amyotrophic Lateral Sclerosis (ALS) With an Optional Open-Label Extended Treatment Period for ALS Participants Who Complete 14 Days of Blinded Treatment
The primary purpose of this study is to evaluate the safety and tolerability of prosetin in healthy volunteers and participants with ALS.
Read the detailed description
PRO-101 is a four-part study. Parts A and B, which respectively evaluated the safety, tolerability, and PK of single and multiple ascending doses of prosetin in 48 healthy volunteers, have been completed.
Parts C and D, which are ongoing, will evaluate the effects of prosetin on safety, tolerability, PK, and biomarkers in 24 participants with ALS. Part C is a double-blind, placebo-controlled, multiple ascending dose component of the study, and Part D is an optional 52-week open-label extension available to ALS participants who complete 14 days of dosing in Part C.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- prosetin, placebo
- Keywords
- ALS, Amyotrophic lateral sclerosis, MAP4K, prosetin
- 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.
prosetinDrug
oral solution
placeboDrug
oral solution
| Study group | Role | What they receive |
|---|---|---|
| Part A - single dose of placebo | Placebo comparator | Healthy volunteers were administered a single dose of prosetin-matched placebo oral solution. Receives: Drug: placebo |
| Part A - single ascending doses of prosetin | Experimental | Healthy volunteers were administered a single dose of prosetin oral solution at 0.03, 0.06, 0.12, or 0.24 mg/kg. Receives: Drug: prosetin |
| Part B - multiple doses of placebo | Placebo comparator | Healthy volunteers were administered a once-daily dose of prosetin-matched placebo for 14 days. Receives: Drug: placebo |
| Part B - multiple ascending doses of prosetin | Experimental | Healthy volunteers were administered a once-daily dose of prosetin at 0.06 or 0.10 mg/kg for 14 days. Receives: Drug: prosetin |
| Part C - multiple doses of placebo in participants with ALS | Placebo comparator | Participants are administered a once-daily dose of prosetin-matched placebo for 14 days. Receives: Drug: placebo |
| Part C - multiple ascending doses of prosetin in participants with ALS | Experimental | Participants will be administered a once-daily dose of prosetin at multiple ascending dose levels for 14 days. Receives: Drug: prosetin |
| Part D - open-label administration of prosetin in participants with ALS | Experimental | Participants will be administered a once-daily dose of prosetin for up to 52 weeks. Receives: Drug: prosetin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Parts A, B, C, D: Number of Participants Experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts A, B, C, D: Number of Participants with Clinically Significant Laboratory Test Abnormalities
Measured: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts A, B, C, D: Number of Participants with Clinically Significant Vital Signs Abnormalities
Measured: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts A, B, C, and D: Number of Participants with Clinically Significant Electrocardiogram (ECG) Abnormalities
Measured: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts A, B, C, and D: Number of Participants with Clinically Significant Physical Examination Abnormalities
Measured: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts A, B, C, and D: Number of Participants with Clinically Significant Neurological Examination Abnormalities
Measured: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts A, B, C, and D: Number of Participants with Clinically Significant Ophthalmic Examination Abnormalities
Measured: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts C and D: Number of Participants with Clinically Significant Electroencephalogram (EEG) Abnormalities
Measured: Part C: Up to 28 days; Part D: Up to 54 weeks
Secondary outcomes (5)
Parts A, B, C, and D: Maximum Observed Concentration (Cmax) of Prosetin in Plasma
Measured: Part A: Up to 28 days Part B: Up to 42 days Part C: Up to 28 days Part D: Up to 54 weeks
Parts A, B, C, and D: Time to Reach Maximum Observed Concentration (Tmax) of Prosetin in Plasma
Measured: Part A: Up to 28 days Part B: Up to 42 days Part C: Up to 28 days Part D: Up to 54 weeks
Parts A, B, C, and D: Area Under the Concentration-Time Curve (AUC) of Prosetin in Plasma
Measured: Part A: Up to 28 days Part B: Up to 42 days Part C: Up to 28 days Part D: Up to 54 weeks
Parts A, B, C, and D: Apparent Terminal Elimination Half-life (t1/2) of Prosetin in Plasma
Measured: Part A: Up to 28 days Part B: Up to 42 days Part C: Up to 28 days Part D: Up to 54 weeks
Parts A and D: Measure of Concentration of Prosetin in CSF
Measured: Part A: Day 1; Part D: Up to 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Mar 2022
- Study start26 Feb 2022
- Primary completion30 Jun 2026 (estimated)
- Study completion31 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- 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
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 72 participants (estimated)
Sponsor and investigators
- Lead sponsor
- ProJenX
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Congressionally Directed Medical Research Programs
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05279755
- Sponsor's protocol ID
- PRO-101
- Other IDs
- CDMRP-AL240175, 2023-507363-20-00, 2023
- Acronym
- PRO-101
- Status last verified
- Apr 2026
- First submitted
- 23 Feb 2022
- Last updated
- 27 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Thams S, Lowry ER, Larraufie MH, Spiller KJ, Li H, Williams DJ, Hoang P, Jiang E, Williams LA, Sandoe J, Eggan K, Lieberam I, Kanning KC, Stockwell BR, Henderson CE, Wichterle H. A Stem Cell-Based Screening Platform Identifies Compounds that Desensitize Motor Neurons to Endoplasmic Reticulum Stress. Mol Ther. 2019 Jan 2;27(1):87-101. doi: 10.1016/j.ymthe.2018.10.010. Epub 2018 Oct 19.
PubMed 30446391Bos PH, Lowry ER, Costa J, Thams S, Garcia-Diaz A, Zask A, Wichterle H, Stockwell BR. Development of MAP4 Kinase Inhibitors as Motor Neuron-Protecting Agents. Cell Chem Biol. 2019 Dec 19;26(12):1703-1715.e37. doi: 10.1016/j.chembiol.2019.10.005. Epub 2019 Oct 31.
PubMed 31676236
Study at a glance
From the registry record, last updated by the sponsor on 27 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Randomized, Double-Blind, Placebo-Controlled Dose Escalating Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending and Multiple Ascending Doses of Prosetin in Healthy Volunteers and Participants With Amyotrophic Lateral Sclerosis (ALS) With an Optional Open-Label Extended Treatment Period for ALS Participants Who Complete 14 Days of Blinded Treatment
- ClinicalTrials.gov ID
- NCT05279755
- Acronym / sponsor ID
- PRO-101 / PRO-101
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS, Amyotrophic lateral sclerosis, MAP4K, prosetin
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 72 participants (estimated)
- Lead sponsor
- ProJenXIndustry (pharma, biotech, devices)
- Collaborators
- Congressionally Directed Medical Research Programs
- Study start
- 26 Feb 2022 (actual)
- Primary completion
- 30 Jun 2026 (estimated)
- Study completion
- 31 Oct 2026 (estimated)
- First posted
- 15 Mar 2022 (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.
The primary purpose of this study is to evaluate the safety and tolerability of prosetin in healthy volunteers and participants with ALS.
Detailed description
PRO-101 is a four-part study. Parts A and B, which respectively evaluated the safety, tolerability, and PK of single and multiple ascending doses of prosetin in 48 healthy volunteers, have been completed.
Parts C and D, which are ongoing, will evaluate the effects of prosetin on safety, tolerability, PK, and biomarkers in 24 participants with ALS. Part C is a double-blind, placebo-controlled, multiple ascending dose component of the study, and Part D is an optional 52-week open-label extension available to ALS participants who complete 14 days of dosing in Part C.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 7
| Arm | Type | Receives |
|---|---|---|
| Part A - single dose of placebo | Placebo comparator | Drug: placeboHealthy volunteers were administered a single dose of prosetin-matched placebo oral solution. |
| Part A - single ascending doses of prosetin | Experimental | Drug: prosetinHealthy volunteers were administered a single dose of prosetin oral solution at 0.03, 0.06, 0.12, or 0.24 mg/kg. |
| Part B - multiple doses of placebo | Placebo comparator | Drug: placeboHealthy volunteers were administered a once-daily dose of prosetin-matched placebo for 14 days. |
| Part B - multiple ascending doses of prosetin | Experimental | Drug: prosetinHealthy volunteers were administered a once-daily dose of prosetin at 0.06 or 0.10 mg/kg for 14 days. |
| Part C - multiple doses of placebo in participants with ALS | Placebo comparator | Drug: placeboParticipants are administered a once-daily dose of prosetin-matched placebo for 14 days. |
| Part C - multiple ascending doses of prosetin in participants with ALS | Experimental | Drug: prosetinParticipants will be administered a once-daily dose of prosetin at multiple ascending dose levels for 14 days. |
| Part D - open-label administration of prosetin in participants with ALS | Experimental | Drug: prosetinParticipants will be administered a once-daily dose of prosetin for up to 52 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Adults ≥18 years of age
- Diagnosis of ALS based on the Gold Coast diagnostic criteria
- Slow Vital Capacity (SVC) >50% predicted
- If being concomitantly treated with riluzole and/or locally approved standard of care treatments, the participant must be on a stable dose for at least 30 days prior to screening and throughout the study
- In the opinion of the Investigator, participant is able to swallow liquid in order to ingest the study medication.
Key exclusion criteria (9 listed)
- Active dementia, neurologic diseases other than ALS, or psychiatric illness that in the opinion of the investigator would affect participation in the current study.
- Significant history or clinical manifestation of comorbid disease in any organ system that currently requires active treatment or is likely to require treatment during the study.
- Any episodes of vertigo in the previous 12 months prior to screening.
- Any medical history of seizures, or any clinically significant EEG finding at Screening or at Day -1.
- A diagnosis of cancer or evidence of continued disease within five years before screening. Protocol-specified exceptions may be considered with approval from the Sponsor's Medical Monitor.
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 |
|---|---|---|
| prosetinoral solution | Drug | Part A - single ascending doses of prosetin, Part B - multiple ascending doses of prosetin, Part C - multiple ascending doses of prosetin in participants with ALS, Part D - open-label administration of prosetin in participants with ALS |
| placebooral solution | Drug | Part A - single dose of placebo, Part B - multiple doses of placebo, Part C - multiple doses of placebo in participants with ALS |
Primary outcome
Parts A, B, C, D: Number of Participants Experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts A, B, C, D: Number of Participants with Clinically Significant Laboratory Test Abnormalities
Time frame: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts A, B, C, D: Number of Participants with Clinically Significant Vital Signs Abnormalities
Time frame: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts A, B, C, and D: Number of Participants with Clinically Significant Electrocardiogram (ECG) Abnormalities
Time frame: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts A, B, C, and D: Number of Participants with Clinically Significant Physical Examination Abnormalities
Time frame: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts A, B, C, and D: Number of Participants with Clinically Significant Neurological Examination Abnormalities
Time frame: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts A, B, C, and D: Number of Participants with Clinically Significant Ophthalmic Examination Abnormalities
Time frame: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts C and D: Number of Participants with Clinically Significant Electroencephalogram (EEG) Abnormalities
Time frame: Part C: Up to 28 days; Part D: Up to 54 weeks
5 secondary outcomes
Parts A, B, C, and D: Maximum Observed Concentration (Cmax) of Prosetin in Plasma
Time frame: Part A: Up to 28 days Part B: Up to 42 days Part C: Up to 28 days Part D: Up to 54 weeks
Parts A, B, C, and D: Time to Reach Maximum Observed Concentration (Tmax) of Prosetin in Plasma
Time frame: Part A: Up to 28 days Part B: Up to 42 days Part C: Up to 28 days Part D: Up to 54 weeks
Parts A, B, C, and D: Area Under the Concentration-Time Curve (AUC) of Prosetin in Plasma
Time frame: Part A: Up to 28 days Part B: Up to 42 days Part C: Up to 28 days Part D: Up to 54 weeks
Parts A, B, C, and D: Apparent Terminal Elimination Half-life (t1/2) of Prosetin in Plasma
Time frame: Part A: Up to 28 days Part B: Up to 42 days Part C: Up to 28 days Part D: Up to 54 weeks
Parts A and D: Measure of Concentration of Prosetin in CSF
Time frame: Part A: Day 1; Part D: Up to 48 weeks
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 27 Apr 2026.
- Background: PubMed 30446391Thams S, Lowry ER, Larraufie MH, Spiller KJ, Li H, Williams DJ, Hoang P, Jiang E, Williams LA, Sandoe J, Eggan K, Lieberam I, Kanning KC, Stockwell BR, Henderson CE, Wichterle H. A Stem Cell-Based Screening Platform Identifies Compounds that Desensitize Motor Neurons to Endoplasmic Reticulum Stress. Mol Ther. 2019 Jan 2;27(1):87-101. doi: 10.1016/j.ymthe.2018.10.010. Epub 2018 Oct 19.
- Background: PubMed 31676236Bos PH, Lowry ER, Costa J, Thams S, Garcia-Diaz A, Zask A, Wichterle H, Stockwell BR. Development of MAP4 Kinase Inhibitors as Motor Neuron-Protecting Agents. Cell Chem Biol. 2019 Dec 19;26(12):1703-1715.e37. doi: 10.1016/j.chembiol.2019.10.005. Epub 2019 Oct 31.
- 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.
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing prosetin in people with Amyotrophic Lateral Sclerosis, run by ProJenX at 4 sites in 3 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 9 inclusion and 9 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: under way, not looking for participants, as of the sponsor's last update on 27 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, Canada, Netherlands. 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?
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?
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?
Parts A, B, C, D: Number of Participants Experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05279755, which also lists 2 linked publications.
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 Apr 2026.