UB-312 Vaccine for Parkinson's & Multiple System Atrophy

Overseen By

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: NYU Langone Health

Must be taking: Antiparkinsonian medications

Must not be taking: Immunosuppressants, others

Disqualifiers: Substance use disorder, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 12

Duration: 97 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the safety and effectiveness of a new vaccine, UB-312, for people with Parkinson's disease (PD) and multiple system atrophy (MSA). Both conditions involve problems with nerve cell proteins and can lead to movement issues. Participants will receive a mix of the actual vaccine and placebo shots to evaluate the vaccine's effectiveness. Suitable candidates have either PD or MSA, with stable treatment plans and no recent history of other major health issues. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new vaccine.

Will I have to stop taking my current medications?

The trial requires that participants have stable treatment of permitted antiparkinsonian medications for at least 30 days before the first study drug administration, or 60 days for MAO-B inhibitors, and remain stable throughout the study. Some medications are prohibited, so you may need to adjust your current medications based on the trial's requirements.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that UB-312 is generally safe for people with Parkinson's disease (PD) and multiple system atrophy (MSA). Most side effects were mild to moderate, similar to those seen with a placebo. No serious side effects related to the treatment emerged. Earlier studies showed that UB-312 was well tolerated in doses up to 300 micrograms, with no deaths or serious side effects. This suggests that UB-312 is safe based on data from previous trials. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatment?

Researchers are excited about the UB-312 vaccine because it offers a novel approach to treating Parkinson's disease (PD) and Multiple System Atrophy (MSA). Unlike current treatments that mainly manage symptoms, UB-312 targets alpha-synuclein, a protein associated with the progression of these diseases. This vaccine is designed to stimulate the body's immune system to specifically target and reduce alpha-synuclein, potentially slowing the progression of PD and MSA. This mechanism is different from the standard medications like Levodopa or dopamine agonists that primarily focus on symptom relief. If successful, UB-312 could change how we approach treatment for these conditions, offering hope for disease modification rather than just symptom management.

What evidence suggests that this trial's treatments could be effective for Parkinson's and multiple system atrophy?

Research has shown that UB-312 may help treat Parkinson's disease (PD) and multiple system atrophy (MSA). In earlier studies, UB-312 safely aided the immune system in producing antibodies against harmful proteins in the brain called alpha-synuclein, which are linked to PD and MSA. Participants generally tolerated the vaccine well, experiencing mostly mild or moderate side effects. This trial will have separate treatment arms for PD and MSA, with some participants receiving UB-312 from the start and others starting with a placebo before transitioning to UB-312. These early results suggest that UB-312 could effectively manage symptoms or slow the progression of these diseases. ^{1 2 3 6 7}

Who Is on the Research Team?

Horacio Kaufmann, MD

Principal Investigator

NYU Langone Health

Are You a Good Fit for This Trial?

This trial is for adults aged 40-75 with Parkinson's Disease or Multiple System Atrophy, confirmed by specific criteria. Participants must have a certain cognitive score, be within a specified weight range, and agree to use contraception if of childbearing potential. They should not have had recent investigational drug use, significant allergies, autoimmune disorders, or conditions that could interfere with the study.

Inclusion Criteria

I am between 40 and 75 years old.

I have been diagnosed with Parkinson's disease or Multiple System Atrophy.

Expected to be able to undergo all study procedures.

Exclusion Criteria

Receipt of an investigational product or device, or participation in a drug research study within 90 days before baseline at V1.

Participants who are currently breastfeeding or intend to breastfeed during the study. Participants should not be willing to get pregnant and breastfeed till 24 weeks after the last injection.

You have had problems with drug or alcohol addiction in the past 2 years.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive active treatment and placebo injections over a period of 97 weeks

97 weeks

12 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Placebo Injection
• UB-312 Injection

Trial Overview The trial tests UB-312 Injection against a placebo in patients with synucleinopathies like Parkinson's Disease and Multiple System Atrophy. UB-312 is an experimental vaccine aimed at providing active immunotherapy for these conditions. The study will assess safety, tolerability, and immune response.

How Is the Trial Designed?

Group I: Treatment First - PD Experimental Treatment 2 Interventions

Includes participants with PD only. Patients in the treatment-first arm will receive active treatment at weeks 1, 5, 13, 25, 37, 49, 73, and 97; and placebo doses at weeks 17, 61, 85, and 109. Participants will be followed up for 24 weeks after their last dose. All participants will receive three priming doses of UB-312 300 µg, followed by 5 booster doses of UB-312 300 µg. To keep the blind, both treatment arms will receive active treatment and placebo injections for a total of 12 injections (8 active treatment injections + 4 placebo injections).

Group II: Treatment First - MSA Experimental Treatment 2 Interventions

Includes participants with MSA only. Patients in the treatment-first arm will receive active treatment at weeks 1, 5, 13, 25, 37, 49, 73, and 97; and placebo doses at weeks 17, 61, 85, and 109. Participants will be followed up for 24 weeks after their last dose. All participants will receive three priming doses of UB-312 300 µg, followed by 5 booster doses of UB-312 300 µg. To keep the blind, both treatment arms will receive active treatment and placebo injections for a total of 12 injections (8 active treatment injections + 4 placebo injections).

Group III: Delayed Start - PD Experimental Treatment 2 Interventions

Includes participants with PD only. Patients in the delayed-start arm will receive placebo injections at weeks 1, 5, 73, and 97; and active treatment at weeks 13, 17, 25, 37, 49, 61, 85, and 109. Participants will be followed up for 24 weeks after their last dose. All participants will receive three priming doses of UB-312 300 µg, followed by 5 booster doses of UB-312 300 µg. To keep the blind, both treatment arms will receive active treatment and placebo injections for a total of 12 injections (8 active treatment injections + 4 placebo injections).

Group IV: Delayed Start - MSA Experimental Treatment 2 Interventions

Includes participants with MSA only Patients in the delayed-start arm will receive placebo injections at weeks 1, 5, 73, and 97; and active treatment at weeks 13, 17, 25, 37, 49, 61, 85, and 109. Participants will be followed up for 24 weeks after their last dose. All participants will receive three priming doses of UB-312 300 µg, followed by 5 booster doses of UB-312 300 µg. To keep the blind, both treatment arms will receive active treatment and placebo injections for a total of 12 injections (8 active treatment injections + 4 placebo injections).

Who Is Running the Clinical Trial?

NYU Langone Health

Lead Sponsor

Trials

1,431

Recruited

838,000+

Published Research Related to This Trial

Intrathecal baclofen (ITB) therapy showed positive effects on muscle tone and function in three patients with multiple system atrophy (MSA), as evidenced by improvements in the modified Ashworth scale.

None of the patients experienced expected progression in disability or ambulation, suggesting that ITB may help maintain quality of life and delay disease progression in MSA, warranting further research into its benefits.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intrathecal baclofen therapy slows progressive disability in multiple system atrophy. Madan, A., Schiess, MC.[2022]

Transplantation of human umbilical cord blood mononuclear cells (hUCB-MCs) via lateral atlanto-occipital space puncture (LASP) showed significant improvement in neurological function in seven patients with multiple system atrophy (MSA) over a follow-up period of 3 to 5 years, particularly noted at 3 to 6 months post-treatment.

The procedure was found to be safe, with no serious adverse events reported, indicating that hUCB-MC transplantation via LASP could be a viable long-term treatment option for MSA.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-Term Clinical Efficacy of Human Umbilical Cord Blood Mononuclear Cell Transplantation by Lateral Atlanto-Occipital Space Puncture (Gong's Puncture) for the Treatment of Multiple System Atrophy. Gong, D., Wang, W., Yuan, X., et al.[2022]

In a study of 14 patients with multiple system atrophy parkinsonism (MSA-P), high-dose intravenous amantadine for 5 days led to clinical improvement in 71.4% of subjects, with a significant decrease in disease severity as measured by the Unified Multiple System Atrophy Rating Scale (UMSARS).

The treatment was generally safe, with only mild and transient adverse effects reported, although one patient experienced acute psychosis, which resolved after stopping the treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of Parenteral Amantadine Therapy in the Treatment of Multiple System Atrophy With Predominant Parkinsonism. Friedberg, A., Erikh, I., Nassar, M., et al.[2018]

In a placebo-controlled trial involving 10 patients with probable multiple system atrophy (MSA), riluzole (100 mg twice daily) was found to be well tolerated but did not show significant anti-Parkinsonian effects based on the Unified Parkinson's Disease Rating Scale (UPDRS).

The results suggest that riluzole is unlikely to provide clinically meaningful benefits for Parkinsonian symptoms in MSA, prompting further investigation into its potential disease-modifying effects in ongoing trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Placebo-controlled trial of riluzole in multiple system atrophy. Seppi, K., Peralta, C., Diem-Zangerl, A., et al.[2015]

Citations

1. trialx.com trialx.com/clinical-trials/listings/265252/ub-312-in-patients-with-synucleinopathies/

UB-312 in Patients With Synucleinopathies This is a Phase 1b study to determine the safety, tolerability, and immunogenicity of UB-312 in participants with multiple system atrophy (MSA), ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11405261/

Target engagement and immunogenicity of an active ... Most TEAEs were considered either mild or moderate after administration of UB-312, comparable to placebo. Three SAEs were reported.

3. withpower.com withpower.com/trial/phase-2-parkinson-disease-11-2022-3a989

UB-312 Vaccine for Parkinson's & Multiple System Atrophy This trial tests a new vaccine called UB-312, which aims to help patients with MSA and PD by teaching their immune systems to attack harmful brain proteins.

4. movementdisorders.onlinelibrary.wiley.com movementdisorders.onlinelibrary.wiley.com/doi/10.1002/mds.29016

A Randomized First‐in‐Human Study With UB‐312, a UBITh ... UB-312 was generally safe, well tolerated, and induced anti-αSyn antibodies in serum and CSF of healthy participants.

5. pharmaceutical-technology.com pharmaceutical-technology.com/data-insights/ub-312-vaxxinity-multiple-system-atrophy-msa-or-shy-drager-syndrome-or-multi-system-degeneration-likelihood-of-approval/

UB-312 by Vaxxinity for Multiple System Atrophy (MSA or ... UB-312 is under development for the treatment of Parkinson's disease and multiple system atrophy (MSA) and for dementia with Lewy bodies. The ...

6. cdek.pharmacy.purdue.edu cdek.pharmacy.purdue.edu/trial/NCT05634876/

Trial | NCT05634876 This is a Phase 1b study to determine the safety, tolerability, and immunogenicity of UB-312 in participants with multiple system atrophy (MSA), ...

7. medscape.com medscape.com/viewarticle/early-promise-vaccine-parkinsons-and-multiple-system-atrophy-2024a1000im2

Early Promise for Vaccine in Parkinson's and MSA "UB-312 immunization in participants with multiple system atrophy and Parkinson's disease has been safe, well-tolerated, and is able to produce ...