Amyotrophic Lateral Sclerosis (ALS)Phase 2ALSNCT04505358
Evaluate PU-AD in Subjects With Amyotrophic Lateral Sclerosis
A Phase 2 study testing PU-AD in people with Amyotrophic Lateral Sclerosis (ALS), run by Samus Therapeutics, Inc..
Trial summary
This is a multicenter, Phase 2a, randomized, double-blind, placebo-controlled pilot study to assess the biological activity, safety and pharmacokinetics of PU-AD compared to placebo in ALS. It will be conducted in approximately 20 sites in the US. Read more about this study
- Who is it for?
- Ages 20 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- PU-AD
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Jan 2023to Jan 2025
- Phase
- Phase 2
- 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 20 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of ALS classified as Clinically Probable ALS or Clinically Definite ALS according to the El Escorial Revised Criteria4
- ALS onset ≤ 18 months from Screening
- Male or female aged 20 to 80 years old (inclusive)
- ALSFRS-R ≥ 30
- SVC ≥ 60% of predicted at Screening
- Willing and able to provide informed consent
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- Concurrent medications riluzole and edaravone are permitted as long as the regimen is stable for at least 4 weeks prior to randomization and is expected to remain unchanged during the course of the study
- Able to swallow tablets
- Has been stable on medications that affect the CNS, for at least 4 weeks (including antidepressants, hypnotics and antipsychotics) and dose is not expected to change during the trial
- Willing to abstain from benzodiazepine or other CNS depressant treatment for 24 hours prior to each clinic visit and night-time hypnotics for 8 hours prior to each clinic visit
- Negative serum pregnancy test at Screening, for female subjects of childbearing potential
- Male subjects:
- Male subjects with female partners of childbearing potential are eligible to participate if they agree to ONE of the following methods of contraception from 21 days before the first dose of IMP through 4 months after the last dose of the IMP:
- Abstinence from penile-vaginal intercourse as their usual and preferred lifestyle (abstinent on a long term and persistent basis) and agree to remain abstinent
- Use of a male condom plus partner use of a contraceptive method with a failure rate of < 1% per year when having penile-vaginal intercourse with a woman of childbearing potential who is not currently pregnant.
- In addition male subjects must refrain from donating sperm for the duration of the study and for 4 months after the last dose of the IMP.
- Female subjects:
- A female subject is eligible to participate if she is not pregnant and/or breastfeeding. Females of childbearing potential must agree to use a highly effective method of contraception consistently and correctly during the treatment period and for at least 3 months after the last dose of IMP.
- Highly effective methods of contraception include, combined hormonal (estrogen and progestogen containing) contraception, which may be oral, intravaginal, or transdermal, progestogen-only hormonal contraception associated with inhibition of ovulation, which may be oral, injectable, or implantable, placement of an intrauterine device, placement of an intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomized partner and true sexual abstinence, if it is the chosen life style of the subject.
- Periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only and the lactational amenorrhea method are not acceptable methods of contraception. Female condom and male condom should not be used together
Who cannot join
- Dependence on invasive or non-invasive mechanical ventilation (excluding continuous positive airway pressure for sleep apnea)
- Has current serious or unstable illnesses including cardiovascular, hepatic, renal, gastroenterological, respiratory, endocrinologic, neurologic (other than ALS), psychiatric, infectious, immunologic or hematologic disease and other conditions that, in the investigator's opinion, could interfere with the analyses of safety and pharmacologic effect in this study
- Has a life expectancy of <1 years
- Has had a history within the last 5 years of a serious infectious disease affecting the brain or head trauma resulting in protracted loss of consciousness
- Has a history within the last 5 years of a primary or recurrent malignant disease with the exception of resected cutaneous squamous cell carcinoma in situ, basal cell carcinoma, cervical carcinoma in situ or in situ prostate cancer with a normal prostate specific antigen post treatment
- Has a known history of human immunodeficiency virus, clinically significant multiple or severe drug allergies or severe post treatment hypersensitivity reactions
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- Has a "yes" answer to Columbia Suicide Severity Rating Scale (C-SSRS) suicidal ideation item 4 or 5, or any suicidal behavior assessment within 6 months of Screening, or has been hospitalized or treated for suicidal behavior in the past 5 years before Screening
- Has a history of chronic alcohol or drug abuse/dependence within the past 5 years
- Transient ischemic attack or stroke or any unexplained loss of consciousness within 1 year prior to Screening
- History of bleeding disorder or predisposing conditions, blood clotting or clinically significant abnormal results on coagulation profile at Screening, as determined by the investigator
- History of unstable angina, myocardial infarction, chronic heart failure (New York Heart Association Class III or IV) or clinically significant conduction abnormalities (e.g., unstable atrial fibrillation) within 1 year prior to Screening
- Clinically significant 12 lead electrocardiogram (ECG) abnormalities, as determined by the investigator
- Indication of impaired liver function as shown by an abnormal liver function profile at Screening (e.g., repeated values of aspartate aminotransferase [AST] and alanine aminotransferase [ALT] ≥ 2 × the upper limit of normal [ULN] and/or indication of impaired renal function at Screening) (e.g., repeated values of creatinine and blood urea nitrogen [BUN] ≥ 1.5 × ULN or estimated glomerular filtration rate [GFR] < 45 mL/minute/1.73 m2 and corroborating medical history and physical examination)
- Any contraindications to lumbar puncture (LP), e.g., increased bleeding risk (platelet count < 100,000/µL, coagulopathies, anticoagulant drugs, antiplatelet therapy), lumbar spine deformity that might interfere with the procedure, evidence on magnetic resonance imaging (MRI) contraindicating LP, risk for cerebral herniation, space occupying lesion with mass effect, abnormal intracranial pressure due to increased CSF pressure, Arnold Chiari malformation, local infections at the puncture site and subject refusal of LP; abnormalities in the Screening CSF profile that are considered by the investigator to be clinically significant are exclusionary
- Any major surgery or trauma within 12 weeks of Screening or during the Screening Period or any surgery planned during the study
- Has active ocular condition, that in the opinion of the investigator, may alter visual acuity during the course of the study
- Use of any drugs that are strong inhibitors of cytochrome (CYP) 450 (2D6 or 2C19) within 7 days or 5 half-lives of the inhibitor (whichever is longer), prior to administration of the first dose of IMP and/or plan to use throughout the study
- Has taken other investigational drugs or participated in any clinical study within 30 days or 5 half-lives (if known) of the investigational drug, whichever is longer, prior to first dose of IMP in this study or is currently participating in another clinical study
- Other unspecified reasons that, in the opinion of the investigator or Samus and/or its delegated medical monitor, place the subject at risk or make the subject unsuitable for the study or unable or unwilling to comply with the requirements of the study
- Female subject of childbearing potential with positive pregnancy test or who is lactating
- History or presence of conditions, which in the judgment of the investigator, are known to interfere with the absorption distribution, metabolism or excretion of drugs, such as prior surgery or gastrointestinal dysfunction that may affect drug absorption (e.g., gastric bypass surgery, gastrectomy)
- History of serious head injuries, unexplained syncope, loss of consciousness, CNS disease or dementia
- Prior exposure to PU-AD or related compounds
- Significant blood loss (> 450 mL) or has donated one or more units of blood or plasma within 6 weeks prior to study participation
About this study
Official title: A Randomized, Double-blind, Placebo-controlled Pilot Study to Evaluate the Biological Activity, Safety, and Pharmacokinetics of PU-AD in Subjects With Amyotrophic Lateral Sclerosis (ALS)
This is a multicenter, Phase 2a, randomized, double-blind, placebo-controlled pilot study to assess the biological activity, safety and pharmacokinetics of PU-AD compared to placebo in ALS. It will be conducted in approximately 20 sites in the US. Approximately 30 subjects will be enrolled in this study; subjects will be randomized 3:2 to receive either PU-AD 30 mg or matching placebo qd, added onto any current stable background treatment.
Read the detailed description
This is a multicenter, Phase 2a, randomized, double-blind, placebo-controlled pilot study to assess the biological activity, safety and pharmacokinetics of PU-AD compared to placebo in ALS. It will be conducted in approximately 20 sites in the US. Approximately 30 subjects will be enrolled in this study; subjects will be randomized 3:2 to receive either PU-AD 30 mg or matching placebo qd, added onto any current stable background treatment. Subjects who meet all inclusion criteria and none of the exclusion criteria will be eligible to participate in this study.
The study consists of a Screening Period (including background treatment stabilization, if required) (4 weeks), Treatment Period (24 weeks) and a safety Follow up Visit (4 weeks [±1 week] after the last dose of investigational medicinal product [IMP]). The expected study duration is up to 32 weeks.
The Screening visit will take place within 4 weeks prior to dosing, to assess eligibility of subjects. Eligible subjects will be randomized and then return to the site for baseline assessments at Week 1. During the 24 week Treatment Period, subjects will be administered 30 mg PU AD or matching placebo qd, orally, on an empty stomach (1 hour prior to food or 2 hours after), at about the same time each day.
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- PU-AD, 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.
PU-ADDrug
Also called active
active vs placebo
PlaceboDrug
Also called non active
active vs placebo
| Study group | Role | What they receive |
|---|---|---|
| 30 mg PU AD 3:2 ratio | Experimental | will be administered orally, as 30 mg active dose strength tablets qd on an empty stomach (1 hour prior to food or 2 hours after), at about the same time each day, via standard of care procedures at the site or at home. All subjects will have their first dose administered in clinic following completion of all baseline assessments Receives: Drug: PU-AD |
| 30 mg Placebo 3:2 ratio | Placebo comparator | will be administered orally, as 30 mg placebo tablets (placebo has no active ingredients) qd on an empty stomach (1 hour prior to food or 2 hours after), at about the same time each day, via standard of care procedures at the site or at home. All subjects will have their first dose administered in clinic following completion of all baseline assessments Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
Clinical effect outcome assessments change from baseline (CFB)
Measured: 12 weeks
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
Clinical effect outcome assessments change from baseline (CFB)
Measured: 24 weeks
Slow Vital Capacity (SVC)
Clinical effect outcome assessments change from baseline (CFB)
Measured: 12 weeks
Slow Vital Capacity (SVC)
Clinical effect outcome assessments change from baseline (CFB)
Measured: 24 weeks
Handgrip strength using hand-held dynamometry
Clinical effect outcome assessments change from baseline (CFB)
Measured: 12 weeks
Handgrip strength using hand-held dynamometry
Clinical effect outcome assessments change from baseline (CFB)
Measured: 24 weeks
6 Minute Walk Test (6MWT)
Clinical effect outcome assessments change from baseline (CFB)
Measured: 12 weeks
6 Minute Walk Test (6MWT)
Clinical effect outcome assessments change from baseline (CFB)
Measured: 24 weeks
Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale (ROADS)
Clinical effect outcome assessments change from baseline (CFB)
Measured: 12 weeks
Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale (ROADS)
Clinical effect outcome assessments change from baseline (CFB)
Measured: 24 weeks
Secondary outcomes (10)
Blood biomarkers (neurofilament light chain [NfL], phosphorylated neurofilament heavy chain [pNfH], Super oxide dismutase 1 (SOD1)
Biomarker change from baseline (CFB)
Measured: 12 weeks
Blood biomarkers (neurofilament light chain [NfL], phosphorylated neurofilament heavy chain [pNfH], Super oxide dismutase 1 (SOD1)
Biomarker change from baseline (CFB)
Measured: 24 weeks
CSF biomarkers (total tau [t-tau], phosphorylated tau231 [p-tau231], Neurofilament light chain (NfL), phosphorylated neurofilament heavy chain (pNfH), glial fibrillary acidic protein [GFAP], Transactive DNA binding protein 43 (TDP 43)
Biomarker change from baseline (CFB)
Measured: 12 weeks
CSF biomarkers (total tau [t-tau], phosphorylated tau231 [p-tau231], NfL, pNfH, glial fibrillary acidic protein [GFAP], TDP 43
Biomarker change from baseline (CFB)
Measured: 24 weeks
Urinary biomarker (P75 extracellular domain [p75ECD])
Biomarker change from baseline (CFB)
Measured: 12 weeks
Urinary biomarker (P75 extracellular domain [p75ECD])
Biomarker change from baseline (CFB)
Measured: 24 weeks
Super oxide dismutase 1 (SOD1) (in subjects with known gene mutations: SOD1) in CSF and blood
Biomarker change from baseline (CFB)
Measured: 12 weeks
Super oxide dismutase 1 (SOD1) (in subjects with known gene mutations: SOD1) in CSF and blood
Biomarker change from baseline (CFB)
Measured: 24 weeks
Poly(GP) (in subjects with known gene mutations: Chromosome 9 open reading frame 72 (C9orf72) ) in CSF
Biomarker change from baseline (CFB)
Measured: 12 weeks
Poly(GP) (in subjects with known gene mutations: Chromosome 9 open reading frame 72 (C9orf72) ) in CSF
Biomarker change from baseline (CFB)
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Aug 2020
- Study startJan 2023 (estimated)
- Primary completionDec 2024 (estimated)
- Study completionJan 2025 (estimated)
Why it stopped: Samus Therapeutics company closure
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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- 3:2 randomization
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Samus Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Michael Silverman, MD, Study director, Samus Therapeutics Consultant
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04505358
- Sponsor's protocol ID
- PU-AD-SD-0202
- Acronym
- ALS
- Status last verified
- Nov 2022
- First submitted
- 23 Jul 2020
- Last updated
- 17 Nov 2022
- 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 17 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled Pilot Study to Evaluate the Biological Activity, Safety, and Pharmacokinetics of PU-AD in Subjects With Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT04505358
- Acronym / sponsor ID
- ALS / PU-AD-SD-0202
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Samus Therapeutics company closure
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Samus Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- Jan 2023 (estimated)
- Primary completion
- Dec 2024 (estimated)
- Study completion
- Jan 2025 (estimated)
- First posted
- 10 Aug 2020 (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 multicenter, Phase 2a, randomized, double-blind, placebo-controlled pilot study to assess the biological activity, safety and pharmacokinetics of PU-AD compared to placebo in ALS. It will be conducted in approximately 20 sites in the US. Approximately 30 subjects will be enrolled in this study; subjects will be randomized 3:2 to receive either PU-AD 30 mg or matching placebo qd, added onto any current stable background treatment.
Detailed description
This is a multicenter, Phase 2a, randomized, double-blind, placebo-controlled pilot study to assess the biological activity, safety and pharmacokinetics of PU-AD compared to placebo in ALS. It will be conducted in approximately 20 sites in the US. Approximately 30 subjects will be enrolled in this study; subjects will be randomized 3:2 to receive either PU-AD 30 mg or matching placebo qd, added onto any current stable background treatment. Subjects who meet all inclusion criteria and none of the exclusion criteria will be eligible to participate in this study.
The study consists of a Screening Period (including background treatment stabilization, if required) (4 weeks), Treatment Period (24 weeks) and a safety Follow up Visit (4 weeks [±1 week] after the last dose of investigational medicinal product [IMP]). The expected study duration is up to 32 weeks.
The Screening visit will take place within 4 weeks prior to dosing, to assess eligibility of subjects. Eligible subjects will be randomized and then return to the site for baseline assessments at Week 1. During the 24 week Treatment Period, subjects will be administered 30 mg PU AD or matching placebo qd, orally, on an empty stomach (1 hour prior to food or 2 hours after), at about the same time each day.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- 3:2 randomization
| Arm | Type | Receives |
|---|---|---|
| 30 mg PU AD 3:2 ratio | Experimental | Drug: PU-ADwill be administered orally, as 30 mg active dose strength tablets qd on an empty stomach (1 hour prior to food or 2 hours after), at about the same time each day, via standard of care procedures at the site or at home. All subjects will have their first dose administered in clinic following completion of all baseline assessments |
| 30 mg Placebo 3:2 ratio | Placebo comparator | Drug: Placebowill be administered orally, as 30 mg placebo tablets (placebo has no active ingredients) qd on an empty stomach (1 hour prior to food or 2 hours after), at about the same time each day, via standard of care procedures at the site or at home. All subjects will have their first dose administered in clinic following completion of all baseline assessments |
Who may qualify
- Ages
- 20 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (20 listed)
- Diagnosis of ALS classified as Clinically Probable ALS or Clinically Definite ALS according to the El Escorial Revised Criteria4
- ALS onset ≤ 18 months from Screening
- Male or female aged 20 to 80 years old (inclusive)
- ALSFRS-R ≥ 30
- SVC ≥ 60% of predicted at Screening
Key exclusion criteria (24 listed)
- Dependence on invasive or non-invasive mechanical ventilation (excluding continuous positive airway pressure for sleep apnea)
- Has current serious or unstable illnesses including cardiovascular, hepatic, renal, gastroenterological, respiratory, endocrinologic, neurologic (other than ALS), psychiatric, infectious, immunologic or hematologic disease and other conditions that, in the investigator's opinion, could interfere with the analyses of safety and pharmacologic effect in this study
- Has a life expectancy of <1 years
- Has had a history within the last 5 years of a serious infectious disease affecting the brain or head trauma resulting in protracted loss of consciousness
- Has a history within the last 5 years of a primary or recurrent malignant disease with the exception of resected cutaneous squamous cell carcinoma in situ, basal cell carcinoma, cervical carcinoma in situ or in situ prostate cancer with a normal prostate specific antigen post treatment
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 |
|---|---|---|
| PU-ADAlso: activeactive vs placebo | Drug | 30 mg PU AD 3:2 ratio |
| PlaceboAlso: non activeactive vs placebo | Drug | 30 mg Placebo 3:2 ratio |
Primary outcome
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
Time frame: 12 weeks
Clinical effect outcome assessments change from baseline (CFB)
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
Time frame: 24 weeks
Clinical effect outcome assessments change from baseline (CFB)
Slow Vital Capacity (SVC)
Time frame: 12 weeks
Clinical effect outcome assessments change from baseline (CFB)
Slow Vital Capacity (SVC)
Time frame: 24 weeks
Clinical effect outcome assessments change from baseline (CFB)
Handgrip strength using hand-held dynamometry
Time frame: 12 weeks
Clinical effect outcome assessments change from baseline (CFB)
Handgrip strength using hand-held dynamometry
Time frame: 24 weeks
Clinical effect outcome assessments change from baseline (CFB)
6 Minute Walk Test (6MWT)
Time frame: 12 weeks
Clinical effect outcome assessments change from baseline (CFB)
6 Minute Walk Test (6MWT)
Time frame: 24 weeks
Clinical effect outcome assessments change from baseline (CFB)
Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale (ROADS)
Time frame: 12 weeks
Clinical effect outcome assessments change from baseline (CFB)
Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale (ROADS)
Time frame: 24 weeks
Clinical effect outcome assessments change from baseline (CFB)
10 secondary outcomes
Blood biomarkers (neurofilament light chain [NfL], phosphorylated neurofilament heavy chain [pNfH], Super oxide dismutase 1 (SOD1)
Time frame: 12 weeks
Blood biomarkers (neurofilament light chain [NfL], phosphorylated neurofilament heavy chain [pNfH], Super oxide dismutase 1 (SOD1)
Time frame: 24 weeks
CSF biomarkers (total tau [t-tau], phosphorylated tau231 [p-tau231], Neurofilament light chain (NfL), phosphorylated neurofilament heavy chain (pNfH), glial fibrillary acidic protein [GFAP], Transactive DNA binding protein 43 (TDP 43)
Time frame: 12 weeks
CSF biomarkers (total tau [t-tau], phosphorylated tau231 [p-tau231], NfL, pNfH, glial fibrillary acidic protein [GFAP], TDP 43
Time frame: 24 weeks
Urinary biomarker (P75 extracellular domain [p75ECD])
Time frame: 12 weeks
Urinary biomarker (P75 extracellular domain [p75ECD])
Time frame: 24 weeks
Super oxide dismutase 1 (SOD1) (in subjects with known gene mutations: SOD1) in CSF and blood
Time frame: 12 weeks
Super oxide dismutase 1 (SOD1) (in subjects with known gene mutations: SOD1) in CSF and blood
Time frame: 24 weeks
Poly(GP) (in subjects with known gene mutations: Chromosome 9 open reading frame 72 (C9orf72) ) in CSF
Time frame: 12 weeks
Poly(GP) (in subjects with known gene mutations: Chromosome 9 open reading frame 72 (C9orf72) ) in CSF
Time frame: 24 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Nov 2022.
- 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.
Michael Silverman, MD study director
Samus Therapeutics Consultant
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PU-AD in people with Amyotrophic Lateral Sclerosis (ALS), run by Samus Therapeutics, Inc..
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 20 inclusion and 24 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: withdrawn, as of the sponsor's last update on 17 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
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 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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) (time frame: 12 weeks). The study lists 10 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 17 Nov 2022.