Multiple System Atrophy - Cerebellar Subtype (MSA-C)Early Phase 1SPRITE-CNCT07514923
The Study of Safety and Preliminary Efficacy of ALT001 in Patients With MultIple System Atrophy-Cerebellar Type
A Early Phase 1 study testing ALT001 in people with Multiple System Atrophy - Cerebellar Subtype (MSA-C), run by yilong Wang at 2 sites in China.
Trial summary
This is a single-center, prospective, randomized, open-label, blinded outcome assessment (PROBE) study. At the end of the PROBE study, patients who have completed the study may opt to enter the open-label extension (OLE) study. Read more about this study
- Who is it for?
- Ages 30 to 75 years
- Conditions the study is for
- Multiple System Atrophy - Cerebellar Subtype (MSA-C)
- What is being tested?
- ALT001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 2 sitesChina
- Study dates
- 10 Jun 2026to 30 Oct 2027
- Phase
- Early 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 30 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 1. Age between 30 and 75 years inclusive, either sex;
- 2. Clinically established or clinically probable MSA-C as defined by the 2022 MDS diagnostic criteria for multiple system atrophy;
- 3. Duration of MSA-C-related motor symptoms, including ataxia, no longer than 5 years;
- 4. Score ≤ 3 on Item 14 of the Unified Multiple System Atrophy Rating Scale (UMSARS) Part II (motor examination);
- 5. Estimated life expectancy ≥ 1 year as assessed by the investigator;
- 6. Voluntary participation in this study and provision of written informed consent
Who cannot join
- 1. Presence of other diseases that may cause ataxia at screening (e.g., infectious diseases, immune-mediated diseases, tumors, paraneoplastic conditions, hereditary disorders, trauma, nutritional deficiencies, toxic exposure, or other neurodegenerative diseases);
- 2. Dementia indicated by the Mini-Mental State Examination (MMSE) at enrollment (score ≤17 for illiterate individuals, ≤20 for primary school education, or ≤24 for junior high school or above) or a prior definitive diagnosis of dementia;
- 3. Evidence of other central nervous system pathologies on brain MRI at screening suggesting a diagnosis of neurodegenerative diseases other than MSA; or other significant pathological findings on brain MRI at screening, including but not limited to: cerebral hemorrhage, acute cerebral infarction, aneurysm, vascular malformation, infectious lesions, brain tumors, or other space-occupying lesions (meningiomas or arachnoid cysts with a maximum diameter <1 cm do not require exclusion);
- 4. Presence of immune-mediated diseases that are inadequately controlled or require treatment with biologic agents;
- 5. Known history of allergies to biologic agents, such as proteins or cell-based products;
- 6. Receipt of any vaccination within the past 1 month;
Show all 21Show fewer
- 7. Patients with a prior definitive diagnosis of malignancy or those currently receiving anti-tumor drug therapy;
- 8. Patients with a prior definitive diagnosis of epilepsy or those currently taking anti-epileptic drugs;
- 9. Presence of lumbar spine diseases or deformities, or other contraindications to lumbar puncture;
- 10. Coagulation abnormalities at enrollment (e.g., platelet count <100 × 10⁹/L; prothrombin time [PT] >3 seconds), a prior diagnosis of coagulation disorders such as hemophilia, or current use of two or more antiplatelet agents;
- 11. Contraindications to MRI (e.g., claustrophobia, implanted cardiac pacemaker, or ferromagnetic metal);
- 12. Concurrent severe hepatic insufficiency, renal insufficiency, or severe cardiac insufficiency (severe hepatic insufficiency defined as ALT ≥1.5 times the upper limit of normal or AST ≥1.5 times the upper limit of normal; severe renal insufficiency defined as serum creatinine [CRE] ≥1.5 times the upper limit of normal or estimated glomerular filtration rate [eGFR] <40 mL/min/1.73 m²; severe cardiac insufficiency defined as NYHA class 3-4), or any significant abnormalities on physical examination, vital signs, laboratory tests, or electrocardiogram that, in the investigator's opinion, require further examination or treatment, or may interfere with the study procedures or safety;
- 13. Active hepatitis B infection (positive for hepatitis B surface antigen, recurrent/persistent ALT elevation, positive serum HBV DNA, or serum HBV DNA >2 × 10⁵ IU/mL);
- 14. Positive for hepatitis C virus antibody or a history of positive test results;
- 15. Positive for HIV or a history of positive test results;
- 16. Patients with a history of alcohol or substance abuse, or alcohol or substance dependence within the past 2 years;
- 17. Diagnosis of psychiatric disorders, severe anxiety, severe depression, or obvious suicidal ideation according to DSM-V diagnostic criteria;
- 18. Patients who are pregnant, lactating, or have the potential to become pregnant, or those planning a pregnancy;
- 19. Current participation in other interventional trials or use of other investigational biologic agents, drugs, or devices, or use of other investigational drugs within the past 1 month or within 5 half-lives of the drug;
- 20. Inability to comply with follow-up assessments due to other reasons;
- 21. Patients deemed by the investigator to be unsuitable for participation in this study.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Yahui Zhu contact
China2 sites, 2 recruiting
- Recruiting
Beijing Tiantan Hospital
Beijing, Beijing Municipality, 100070
- Recruiting
Beijing Tiantan Hospital
Beijing, Beijing Municipality, 100070
Zhu Yahui, +8613439182912, zhuyahuidr@sina.com
About this study
This is a single-center, prospective, randomized, open-label, blinded outcome assessment (PROBE) study. At the end of the PROBE study, patients who have completed the study may opt to enter the open-label extension (OLE) study. The objective of the study is to evaluate the safety, tolerability and potential preliminary efficacy of ALT001 in the treatment of patients with multiple system atrophy-cerebellar type (MSA-C).
Read the detailed description
Multiple system atrophy (MSA) is a progressive neurodegenerative disorder characterized by a blend of autonomic dysfunction, Parkinson's syndrome, and cerebellar syndrome. The incidence of MSA ranges from 1.9 to 4.9 per 100,000 individuals, with an average age of onset of 56.2 years. Among individuals aged 50 years and older, the prevalence stands at 3.0 per 100,000. The mean age of MSA onset is 56.2 years, and the median survival ranges from 6.2 to 7.5 years. The MSA-C subtype is associated with a poorer prognosis due to its predominant involvement of the cerebellum and brainstem. In patients with MSA-C, gait and balance disturbances manifest early, leading to nearly 50% of patients requiring a walking aid or physical assistance for ambulation within three years of motor symptom onset. Within five years, 60% of patients become wheelchair-dependent, and after six to eight years, most are completely bedridden, severely impacting their quality of life. Treating MSA-C, a rare neurodegenerative condition, remains a significant challenge. Current symptomatic and supportive therapies fall short of meeting the treatment requirements of MSA-C patients. Furthermore, potential adverse effects and disease progression factors restrict the use of certain drugs, highlighting the critical need for the development of disease-modifying or neuroprotective agents to decelerate disease advancement. "ALT001, a nerve repair protein created by Darwin Origin (Hubei) Biopharmaceutical Co.LTD, is derived from cellular exosomes, a set of specific microenvironmental protein polymers secreted by stem cells under emergency conditions. It boasts selective assembly, targeted delivery, highly efficient tissue repair, exceptional safety, chemical stability, and convenient storage. Previous basic research has indicated ALT001's potential for promoting endogenous neural tissue repair, exhibiting significant neuroprotective and neurorestorative effects in animal models. Therefore, building upon the ongoing study in patients with the MSA-parkinsonian subtype, this project further initiates a single-center, prospective, randomized, open-label, blinded endpoint evaluation (PROBE) study, followed by an open-label extension (OLE) study, specifically targeting patients with the MSA-C subtype. The objectives are to evaluate the safety, tolerability, and potential preliminary efficacy of ALT001 in treating MSA-C patients. This study aims to recruit 20 MSA-C subtype patients aged between 30 and 75 years. The visit content at each stage of this study includes vital signs, neurological examination, laboratory tests (such as routine blood test, blood biochemical examination, coagulation test, etc.), imaging examinations (such as MRI, bladder ultrasound), neurological assessments (such as unified multiple system atrophy rating scale [UMSARS], composite autonomic symptom score [COMPASS], scale for the assessment and rating of ataxia[SARA], quick MSA quality of life questionnaire [MSA-QoL], mini-mental state examination [MMSE]), and cerebrospinal fluid collection. Different stages of the study focus on monitoring patients' concomitant medications, adverse events, and serious adverse events. Detailed follow-up is conducted at the end of each treatment period, with face-to-face visits at specific time points. Additionally, researchers are required to promptly report and manage events when patients develop new neurological symptoms or suspicious events. The follow-up content of the study includes treatment and follow-up assessments at multiple stages. Initially, comprehensive physical examinations, laboratory tests (including blood, urine tests, etc.), multidimensional scoring assessments (such as UMSARS, COMPASS, SARA, MSA-QoL), and brain examinations through imaging techniques such as 3T MRI are performed on subjects at baseline. Subsequently, subjects enter three treatment periods and follow-up stages lasting a total of 90 days, with daily monitoring of vital signs, cerebrospinal fluid collection, laboratory tests, and adverse event documentation in each stage. Follow-up during the Open-Label Extension (OLE) phase (from day 90 to day 165 post-randomization) continues monitoring of vital signs, laboratory tests, and adverse event recording. At the follow-up visits on day 90, day 180, and day 360, comprehensive physical examinations, laboratory tests, gait analysis, bladder ultrasound, MRI, and repeat UMSARS, COMPASS, SARA, MSA-QoL scoring are conducted. Additionally, if patients experience new neurological symptoms or suspicious events, additional visits will be carried out, and researchers are required to submit and interpret relevant data within 72 hours of the event occurrence.The protocol of this study has been approved by the Ethics Committee of Beijing Tiantan Hospital. All participants will provide written informed consents before entering the study.
- Being tested
- ALT001
- Keywords
- Multiple system atrophy
- Medical subject headings
- Multiple System Atrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALT001Drug
Intrathecal administration combined with intravenous administration of ALT001 was given to each MSA patient in the intervention group, with intrathecal administration on days 1, 31 and 61 and intravenous administration on days 2 to 14, 32 to 44±3 and 62 to 74±5, and treatment was given once a day. intrathecal administration: 8 mL sodium chloride injection + ALT001 (130 μg/branch) for intrathecal administration, which was completed in about 5 minutes; intravenous administration: ALT001 (130 μg/branch) was dissolved in 100 ml sodium chloride injection, which was completed in about 30-40 minutes. After completion of the above treatments and 90±7 days of follow-up, all subjects entered the OLE study phase. During this period, all participants were given the option of continuing to receive 3 phases (on Days 91 to 104±7, Days 121 to 134±7, and Days 151 to 164±7) of intravenous ALT001 administration (14 consecutive days of ALT001 130 μg qd i.v. in each phase).
| Study group | Role | What they receive |
|---|---|---|
| Intervention group | Experimental | Intrathecal administration combined with intravenous administration of ALT001 was given to each MSA patient in the intervention group, with intrathecal administration on days 1, 31 and 61 and intravenous administration on days 2 to 14, 32 to 44±3 and 62 to 74±5, and treatment was given once a day. intrathecal administration: 8 mL sodium chloride injection + ALT001 (130 μg/branch) for intrathecal administration, which was completed in about 5 minutes; intravenous administration: ALT001 (130 μg/branch) was dissolved in 100 ml sodium chloride injection, which was completed in about 30-40 minutes. After completion of the above treatments and 90±7 days of follow-up, all subjects entered the OLE study phase. During this period, all participants were given the option of continuing to receive 3 phases (on Days 91 to 104±7, Days 121 to 134±7, and Days 151 to 164±7) of intravenous ALT001 administration (14 consecutive days of ALT001 130 μg qd i.v. in each phase). Receives: Drug: ALT001 |
| No intervention group | No intervention | Each MSA-C patient in the blank control group received only basic treatment with no trial-related treatment. After completion of the above treatments and 90±7 days of follow-up, all subjects entered the OLE study phase. During this period, all participants were given the option of continuing to receive 3 phases (on Days 91 to 104±7, Days 121 to 134±7, and Days 151 to 164±7) of intravenous ALT001 administration (14 consecutive days of ALT001 130 μg qd i.v. in each phase). |
What is being measured
The primary outcome is the main question the study is designed to answer.
The incidence of investigator-reported adverse events (AEs) and serious adverse events (SAEs)
The incidence of investigator-reported adverse events (AEs) and serious adverse events (SAEs)
Measured: Day 90±7 after randomization
The incidence of changes in clinical laboratory test parameters, changes in vital signs, neurological examination abnormalities and ECG abnormalities
Laboratory tests include: routine blood test, coagulation test, blood biochemical examination, urinalysis (note that abnormal changes in laboratory test results may need to be reviewed to further determine and assess their relevance to the study). Vital signs include: abnormal body temperature (≥38℃ or ≤35℃), blood pressure (systolic blood pressure ≥180mmHg or <90mmHg), respiration (>24 beats/min and other rhythm abnormalities), and heart rate (>100 beats/min or <60 beats/min).
Measured: Day 90±7 after randomization
Secondary outcomes (7)
Changes in the unified multiple system atrophy rating scale (UMSARS) part scores, sum of part 1 and 2 scores
The Unified Multiple System Atrophy Rating Scale (UMSARS) comprises four sections. Part I is the "Historical Review," consisting of 12 items that evaluate impairment in activities of daily living as reported by the patient. Each item is scored from a minimum of 0 points (normal) to a maximum of 4 points (severe disability), resulting in a total score range for this section of 0 to 48. Part II is the "Motor Examination," which assesses motor impairment based on clinical examination. It contains 14 items, each also scored from a minimum of 0 (normal) to a maximum of 4 (severe disability), yielding a total score range of 0 to 56. Part III is the "Autonomic Examination." Part IV is the "Global Disability Scale," a single-item scale used to assess the patient's overall level of disability, with scores ranging from a minimum of 1 (completely independent) to a maximum of 5 (totally bedridden).
Measured: Day 15, 45±3, 75±5, 90±7, 180±14, and 360±14 after randomization
Changes in the scale for the assessment and rating of ataxia (SARA)
The Scale for the Assessment and Rating of Ataxia (SARA) is a semi-quantitative clinical instrument designed to quantify the severity of cerebellar ataxia, with total scores ranging from 0 (no ataxia) to 40 (most severe ataxia). The scale comprises eight items that reflect progressively increasing functional impairment, assessing gait (0-8 points), stance (0-6 points), sitting (0-4 points), speech disturbance (0-6 points), finger chase (0-4 points), nose-finger test (0-4 points), fast alternating hand movements (0-4 points), and heel-knee-shin slide (0-4 points). For the upper and lower limb items, both sides are assessed separately, and the mean score is calculated for inclusion in the total score. SARA demonstrates good reliability and validity and is commonly used in clinical practice to evaluate the severity of ataxia and monitor disease progression.
Measured: Day 15, 45±3, 75±5, 90±7, 180±14, and 360±14 after randomization
Changes in the composite autonomic symptom score (COMPASS) scores
The Composite Autonomic Symptom Score (COMPASS) is a revised version of the 169-item Autonomic Symptom Profile assessing 11 domains of autonomic function, to the 31-item COMPASS, now assessing six domains: orthostatic hypotension (maximum score 10), vasomotor (maximum score 6), secretomotor (maximum score 7), gastrointestinal (maximum score 28), bladder (maximum score 9), and pupillomotor functions (maximum score 15). Scores from each component are recorded and a weighted total score is calculated. Higher scores on this scale indicate more severe clinical symptoms related to autonomic dysfunction. It is a validated, easy-to-use self-assessment instrument designed for clinical autonomic research and practice.
Measured: Day 15, 45±3, 75±5, 90±7, 180±14, and 360±14 after randomization
Changes in the incidence of orthostatic hypotension
Changes in the incidence of orthostatic hypotension in patients on days 15, 45±3, 75±5, 90±7, 180±14, and 360±14 after randomization as measured by recumbent-upright blood pressure monitoring/24-hour ambulatory blood pressure monitoring/head-upright tilt test.
Measured: Day 15, 45±3, 75±5, 90±7, 180±14, and 360±14 after randomization
Changes in quick MSA quality of life questionnaire (MSA-QoL)
The Quick MSA Quality of Life questionnaire (Quick MSA-QoL) is a concise, patient-reported outcome measure derived and validated from the original 40-item MSA-QoL scale. It is designed to reduce the completion burden for patients with multiple system atrophy (MSA) while enhancing data quality. This unidimensional scale comprises 15 items covering core domains, including motor function, non-motor symptoms, and emotional/social functioning. Each item offers five response options: no problem, mild problem, moderate problem, severe problem, and extreme problem. The total score of the scale ranges from 0 (minimum) to 60 (maximum). A higher total score indicates greater impairment in health-related quality of life.
Measured: Day 90±7, 180±14, and 360±14 after randomization
Changes in immunological indices (lymphocyte subpopulation)
Changes in immunological indices (lymphocyte subpopulation) on days 15, 30±3, 45±3, 60±5, 75±5, 105±7, 135±7, and 165±7 after randomization. Lymphocyte subsets include CD3+%, CD3+CD4+%, and CD3+CD8+%, among others.
Measured: Day 15, 30±3, 45±3, 60±5, 75±5, 105±7, 135±7, and 165±7 after randomization
Variation of three-dimensional gait analysis parameters under multitasking
Changes in three-dimensional gait analysis parameters under multitasking in patients on day 90±7, 180±14, and 360±14 after randomization. The main content of three-dimensional gait analysis is gait speed (m/s). The gait speed of participants was measured using a three-dimensional gait analyzer at various follow-up time points.
Measured: Day 90±7, 180±14, and 360±14 after randomization
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Apr 2026
- Study start10 Jun 2026
- Primary completion31 Jan 2027 (estimated)
- Study completion30 Oct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple System Atrophy - Cerebellar Subtype (MSA-C)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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- yilong Wang
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, yilong Wang, Beijing Tiantan Hospital
- Investigators
- Yilong Wang, Principal investigator, Beijing Tiantan Hospital, Capital Medical University, Beijing, ChinaTao Feng, Principal investigator, Beijing Tiantan Hospital, Capital Medical University, Beijing, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07514923
- Sponsor's protocol ID
- KY2026-049-01
- Other IDs
- HX-A-2025121
- Acronym
- SPRITE-C
- Status last verified
- Sep 2026
- First submitted
- 12 Mar 2026
- Last updated
- 9 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Study of Safety and Preliminary Efficacy of ALT001 in Patients With MultIple System Atrophy-Cerebellar Type
- ClinicalTrials.gov ID
- NCT07514923
- Acronym / sponsor ID
- SPRITE-C / KY2026-049-01
- Keywords
- Multiple system atrophy
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- yilong WangUniversity, hospital or other
- Study start
- 10 Jun 2026 (actual)
- Primary completion
- 31 Jan 2027 (estimated)
- Study completion
- 30 Oct 2027 (estimated)
- First posted
- 7 Apr 2026 (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, prospective, randomized, open-label, blinded outcome assessment (PROBE) study. At the end of the PROBE study, patients who have completed the study may opt to enter the open-label extension (OLE) study. The objective of the study is to evaluate the safety, tolerability and potential preliminary efficacy of ALT001 in the treatment of patients with multiple system atrophy-cerebellar type (MSA-C).
Detailed description
Multiple system atrophy (MSA) is a progressive neurodegenerative disorder characterized by a blend of autonomic dysfunction, Parkinson's syndrome, and cerebellar syndrome. The incidence of MSA ranges from 1.9 to 4.9 per 100,000 individuals, with an average age of onset of 56.2 years. Among individuals aged 50 years and older, the prevalence stands at 3.0 per 100,000. The mean age of MSA onset is 56.2 years, and the median survival ranges from 6.2 to 7.5 years. The MSA-C subtype is associated with a poorer prognosis due to its predominant involvement of the cerebellum and brainstem. In patients with MSA-C, gait and balance disturbances manifest early, leading to nearly 50% of patients requiring a walking aid or physical assistance for ambulation within three years of motor symptom onset. Within five years, 60% of patients become wheelchair-dependent, and after six to eight years, most are completely bedridden, severely impacting their quality of life. Treating MSA-C, a rare neurodegenerative condition, remains a significant challenge. Current symptomatic and supportive therapies fall short of meeting the treatment requirements of MSA-C patients. Furthermore, potential adverse effects and disease progression factors restrict the use of certain drugs, highlighting the critical need for the development of disease-modifying or neuroprotective agents to decelerate disease advancement. "ALT001, a nerve repair protein created by Darwin Origin (Hubei) Biopharmaceutical Co.LTD, is derived from cellular exosomes, a set of specific microenvironmental protein polymers secreted by stem cells under emergency conditions. It boasts selective assembly, targeted delivery, highly efficient tissue repair, exceptional safety, chemical stability, and convenient storage. Previous basic research has indicated ALT001's potential for promoting endogenous neural tissue repair, exhibiting significant neuroprotective and neurorestorative effects in animal models. Therefore, building upon the ongoing study in patients with the MSA-parkinsonian subtype, this project further initiates a single-center, prospective, randomized, open-label, blinded endpoint evaluation (PROBE) study, followed by an open-label extension (OLE) study, specifically targeting patients with the MSA-C subtype. The objectives are to evaluate the safety, tolerability, and potential preliminary efficacy of ALT001 in treating MSA-C patients. This study aims to recruit 20 MSA-C subtype patients aged between 30 and 75 years. The visit content at each stage of this study includes vital signs, neurological examination, laboratory tests (such as routine blood test, blood biochemical examination, coagulation test, etc.), imaging examinations (such as MRI, bladder ultrasound), neurological assessments (such as unified multiple system atrophy rating scale [UMSARS], composite autonomic symptom score [COMPASS], scale for the assessment and rating of ataxia[SARA], quick MSA quality of life questionnaire [MSA-QoL], mini-mental state examination [MMSE]), and cerebrospinal fluid collection. Different stages of the study focus on monitoring patients' concomitant medications, adverse events, and serious adverse events. Detailed follow-up is conducted at the end of each treatment period, with face-to-face visits at specific time points. Additionally, researchers are required to promptly report and manage events when patients develop new neurological symptoms or suspicious events. The follow-up content of the study includes treatment and follow-up assessments at multiple stages. Initially, comprehensive physical examinations, laboratory tests (including blood, urine tests, etc.), multidimensional scoring assessments (such as UMSARS, COMPASS, SARA, MSA-QoL), and brain examinations through imaging techniques such as 3T MRI are performed on subjects at baseline. Subsequently, subjects enter three treatment periods and follow-up stages lasting a total of 90 days, with daily monitoring of vital signs, cerebrospinal fluid collection, laboratory tests, and adverse event documentation in each stage. Follow-up during the Open-Label Extension (OLE) phase (from day 90 to day 165 post-randomization) continues monitoring of vital signs, laboratory tests, and adverse event recording. At the follow-up visits on day 90, day 180, and day 360, comprehensive physical examinations, laboratory tests, gait analysis, bladder ultrasound, MRI, and repeat UMSARS, COMPASS, SARA, MSA-QoL scoring are conducted. Additionally, if patients experience new neurological symptoms or suspicious events, additional visits will be carried out, and researchers are required to submit and interpret relevant data within 72 hours of the event occurrence.The protocol of this study has been approved by the Ethics Committee of Beijing Tiantan Hospital. All participants will provide written informed consents before entering the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Intervention group | Experimental | Drug: ALT001Intrathecal administration combined with intravenous administration of ALT001 was given to each MSA patient in the intervention group, with intrathecal administration on days 1, 31 and 61 and intravenous administration on days 2 to 14, 32 to 44±3 and 62 to 74±5, and treatment was given once a day. intrathecal administration: 8 mL sodium chloride injection + ALT001 (130 μg/branch) for intrathecal administration, which was completed in about 5 minutes; intravenous administration: ALT001 (130 μg/branch) was dissolved in 100 ml sodium chloride injection, which was completed in about 30-40 minutes. After completion of the above treatments and 90±7 days of follow-up, all subjects entered the OLE study phase. During this period, all participants were given the option of continuing to receive 3 phases (on Days 91 to 104±7, Days 121 to 134±7, and Days 151 to 164±7) of intravenous ALT001 administration (14 consecutive days of ALT001 130 μg qd i.v. in each phase). |
| No intervention group | No intervention | –Each MSA-C patient in the blank control group received only basic treatment with no trial-related treatment. After completion of the above treatments and 90±7 days of follow-up, all subjects entered the OLE study phase. During this period, all participants were given the option of continuing to receive 3 phases (on Days 91 to 104±7, Days 121 to 134±7, and Days 151 to 164±7) of intravenous ALT001 administration (14 consecutive days of ALT001 130 μg qd i.v. in each phase). |
Who may qualify
- Ages
- 30 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 1. Age between 30 and 75 years inclusive, either sex;
- 2. Clinically established or clinically probable MSA-C as defined by the 2022 MDS diagnostic criteria for multiple system atrophy;
- 3. Duration of MSA-C-related motor symptoms, including ataxia, no longer than 5 years;
- 4. Score ≤ 3 on Item 14 of the Unified Multiple System Atrophy Rating Scale (UMSARS) Part II (motor examination);
- 5. Estimated life expectancy ≥ 1 year as assessed by the investigator;
Key exclusion criteria (21 listed)
- 1. Presence of other diseases that may cause ataxia at screening (e.g., infectious diseases, immune-mediated diseases, tumors, paraneoplastic conditions, hereditary disorders, trauma, nutritional deficiencies, toxic exposure, or other neurodegenerative diseases);
- 2. Dementia indicated by the Mini-Mental State Examination (MMSE) at enrollment (score ≤17 for illiterate individuals, ≤20 for primary school education, or ≤24 for junior high school or above) or a prior definitive diagnosis of dementia;
- 3. Evidence of other central nervous system pathologies on brain MRI at screening suggesting a diagnosis of neurodegenerative diseases other than MSA; or other significant pathological findings on brain MRI at screening, including but not limited to: cerebral hemorrhage, acute cerebral infarction, aneurysm, vascular malformation, infectious lesions, brain tumors, or other space-occupying lesions (meningiomas or arachnoid cysts with a maximum diameter <1 cm do not require exclusion);
- 4. Presence of immune-mediated diseases that are inadequately controlled or require treatment with biologic agents;
- 5. Known history of allergies to biologic agents, such as proteins or cell-based products;
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 |
|---|---|---|
| ALT001Intrathecal administration combined with intravenous administration of ALT001 was given to each MSA patient in the intervention group, with intrathecal administration on days 1, 31 and 61 and intravenous administration on days 2 to 14, 32 to 44±3 and 62 to 74±5, and treatment was given once a day. intrathecal administration: 8 mL sodium chloride injection + ALT001 (130 μg/branch) for intrathecal administration, which was completed in about 5 minutes; intravenous administration: ALT001 (130 μg/branch) was dissolved in 100 ml sodium chloride injection, which was completed in about 30-40 minutes. After completion of the above treatments and 90±7 days of follow-up, all subjects entered the OLE study phase. During this period, all participants were given the option of continuing to receive 3 phases (on Days 91 to 104±7, Days 121 to 134±7, and Days 151 to 164±7) of intravenous ALT001 administration (14 consecutive days of ALT001 130 μg qd i.v. in each phase). | Drug | Intervention group |
Primary outcome
The incidence of investigator-reported adverse events (AEs) and serious adverse events (SAEs)
Time frame: Day 90±7 after randomization
The incidence of investigator-reported adverse events (AEs) and serious adverse events (SAEs)
The incidence of changes in clinical laboratory test parameters, changes in vital signs, neurological examination abnormalities and ECG abnormalities
Time frame: Day 90±7 after randomization
Laboratory tests include: routine blood test, coagulation test, blood biochemical examination, urinalysis (note that abnormal changes in laboratory test results may need to be reviewed to further determine and assess their relevance to the study). Vital signs include: abnormal body temperature (≥38℃ or ≤35℃), blood pressure (systolic blood pressure ≥180mmHg or <90mmHg), respiration (>24 beats/min and other rhythm abnormalities), and heart rate (>100 beats/min or <60 beats/min).
7 secondary outcomes
Changes in the unified multiple system atrophy rating scale (UMSARS) part scores, sum of part 1 and 2 scores
Time frame: Day 15, 45±3, 75±5, 90±7, 180±14, and 360±14 after randomization
Changes in the scale for the assessment and rating of ataxia (SARA)
Time frame: Day 15, 45±3, 75±5, 90±7, 180±14, and 360±14 after randomization
Changes in the composite autonomic symptom score (COMPASS) scores
Time frame: Day 15, 45±3, 75±5, 90±7, 180±14, and 360±14 after randomization
Changes in the incidence of orthostatic hypotension
Time frame: Day 15, 45±3, 75±5, 90±7, 180±14, and 360±14 after randomization
Changes in quick MSA quality of life questionnaire (MSA-QoL)
Time frame: Day 90±7, 180±14, and 360±14 after randomization
Changes in immunological indices (lymphocyte subpopulation)
Time frame: Day 15, 30±3, 45±3, 60±5, 75±5, 105±7, 135±7, and 165±7 after randomization
Variation of three-dimensional gait analysis parameters under multitasking
Time frame: Day 90±7, 180±14, and 360±14 after randomization
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 9 Sep 2026.
- Individual participant dataSharing planned: No
Related trials for Multiple System Atrophy - Cerebellar Subtype (MSA-C)
Recruiting
Efficacy and Safety of Transcranial dIrect Current stiMulation in Multiple System Atrophy-Cerebellar Variant
Multiple System Atrophy - Cerebellar Subtype (MSA-C)
RecruitingObservational study
The CurePSP Genetics Program
PSP, PSP - Progressive Supranuclear Palsy
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.
Yahui Zhu contact
Yilong Wang principal investigator
Beijing Tiantan Hospital, Capital Medical University, Beijing, China
Tao Feng principal investigator
Beijing Tiantan Hospital, Capital Medical University, Beijing, China
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing ALT001 in people with Multiple System Atrophy - Cerebellar Subtype (MSA-C), run by yilong Wang at 2 sites in China.
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 6 inclusion and 21 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: looking for participants now, as of the sponsor's last update on 9 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in China. 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. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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?
Contact Weiqi Chen (8615652813380, weiqichen@aliyun.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The incidence of investigator-reported adverse events (AEs) and serious adverse events (SAEs) (time frame: Day 90±7 after randomization). The study lists 2 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 9 Sep 2026.