BIIB094 for Parkinson's Disease

(REASON Trial)

No longer recruiting at 21 trial locations

Overseen By Global Biogen Clinical Trial Center

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Ionis Pharmaceuticals, Inc.

Disqualifiers: Cognitive impairment, Brain surgery, Stroke, Heart issues, Diabetes, others

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety of BIIB094, a potential treatment for Parkinson's Disease (PD), administered through a spinal injection. The study also examines how the body processes this treatment. Participants will receive either a single dose or multiple doses, depending on their group within the trial. Individuals diagnosed with Parkinson's Disease within the last seven years and who have stable symptoms (without major movement problems) might be suitable for this trial. As a Phase 1 trial, participants will be among the first to help researchers understand how this new treatment works in people.

Do I have to stop taking my current medications for the BIIB094 trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

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

Research has shown that BIIB094 is generally safe for people. This treatment is being tested for Parkinson's Disease and has been tried on individuals from various genetic backgrounds. Importantly, these studies have not found any major safety issues.

BIIB094 is administered through an injection directly into the spinal canal, known as an intrathecal (IT) injection. While this can sometimes be uncomfortable, most participants have tolerated it well. The treatment may help by reducing certain proteins in the brain and spinal cord, potentially benefiting people with Parkinson's.

In summary, although all clinical trials carry risks, current research suggests that BIIB094 is safe enough for further testing. Participants in past studies have generally tolerated it without serious side effects. These findings may reassure those considering joining a trial about the treatment's safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Parkinson's Disease?

Unlike standard treatments for Parkinson's disease, which typically focus on managing symptoms with medications like levodopa or dopamine agonists, BIIB094 offers a novel approach by targeting the leucine-rich repeat kinase 2 (LRRK2) pathway. This pathway is believed to play a significant role in the disease's progression. What makes BIIB094 particularly exciting is its potential to modify the disease itself rather than just alleviating symptoms. Additionally, BIIB094 is delivered via an intrathecal (IT) injection, directly reaching the central nervous system, which could enhance its effectiveness. Researchers are hopeful that this unique mechanism and delivery method may offer a more powerful way to slow down or even halt the progression of Parkinson's disease.

What evidence suggests that BIIB094 might be an effective treatment for Parkinson's Disease?

Research has shown that BIIB094 could be a promising treatment for Parkinson's Disease due to its targeted approach. In this trial, participants will receive BIIB094 directly into the spinal fluid, potentially enhancing its ability to reach the brain. The treatment focuses on a gene called LRRK2, which is linked to Parkinson's and is believed to influence disease progression. Although detailed information on its effectiveness in humans is limited, this treatment targets a key genetic factor that might help slow or manage Parkinson's symptoms. Early trials primarily assess safety, but the unique targeting of LRRK2 offers hope for future effectiveness. ^{2 3 4 6 7}

Who Is on the Research Team?

Medical Director

Principal Investigator

Biogen

Are You a Good Fit for This Trial?

This trial is for adults with Parkinson's Disease diagnosed within the last 7 years, without severe motor issues or dyskinesia. Participants must understand study risks, give informed consent, and have a Modified Hoehn and Yahr Stage ≤ 3. Excluded are those with recent heart problems, uncontrolled diabetes, certain infections like hepatitis C or HIV, recent strokes or loss of consciousness, cognitive impairments (MoCA score <23), or brain surgery history for PD.

Inclusion Criteria

My Parkinson's disease is at an early or mid-stage.

I was diagnosed with Parkinson's disease within the last 7 years and do not have major movement issues.

You are able to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use confidential health information in accordance with national and local participant privacy regulations.

Exclusion Criteria

You have a history of hepatitis C or tested positive for hepatitis C virus.

I haven't had serious heart problems or a heart attack in the last year.

I have never had brain surgery or focused ultrasound for Parkinson's disease, nor any neuromodulation procedures.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

6 weeks

Treatment Part A (SAD)

Participants receive a single intrathecal injection of BIIB094 or placebo

12 weeks

Treatment Part B (MAD)

Participants receive multiple intrathecal injections of BIIB094 or placebo

25 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• BIIB094
• Placebo

Trial Overview The trial tests BIIB094 administered via spinal injection to see if it's safe and tolerable in single/multiple doses compared to a placebo. It also looks at how the body processes this drug. The study includes people with variations in the LRRK2 gene related to PD as well as those without genetic variants.

How Is the Trial Designed?

Group I: Part B (MAD): BIIB094 Dose 1 Experimental Treatment 1 Intervention

Participants will receive a single IT injection of BIIB094 on multiple days during Part B \[Multiple Ascending Dose (MAD)\].

Group II: Part B (MAD): BIIB094 (Non LRRK2) Dose 3 Experimental Treatment 1 Intervention

Participants (Non LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).

Group III: Part B (MAD): BIIB094 (Non LRRK2) Dose 2 Experimental Treatment 1 Intervention

Participants \[Non leucine-rich repeat kinase 2 (Non LRRK2)\] will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).

Group IV: Part B (MAD): BIIB094 (LRRK2) Dose 3 Experimental Treatment 1 Intervention

Participants (LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).

Group V: Part B (MAD): BIIB094 (LRRK2) Dose 2 Experimental Treatment 1 Intervention

Participants (LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).

Group VI: Part A (SAD): BIIB094 Dose 6 Experimental Treatment 1 Intervention

Participants will receive a single IT injection of BIIB094 during Part A (SAD).

Group VII: Part A (SAD): BIIB094 Dose 5 Experimental Treatment 1 Intervention

Participants will receive a single IT injection of BIIB094 during Part A (SAD).

Group VIII: Part A (SAD): BIIB094 Dose 4 Experimental Treatment 1 Intervention

Participants will receive a single IT injection of BIIB094 during Part A (SAD).

Group IX: Part A (SAD): BIIB094 Dose 3 Experimental Treatment 1 Intervention

Participants will receive a single IT injection of BIIB094 during Part A (SAD).

Group X: Part A (SAD): BIIB094 Dose 2 Experimental Treatment 1 Intervention

Participants will receive a single IT injection of BIIB094 during Part A (SAD).

Group XI: Part A (SAD): BIIB094 Dose 1 Experimental Treatment 1 Intervention

Participants will receive a single IT injection of BIIB094 during Part A \[Single Ascending Dose (SAD)\].

Group XII: Part A (SAD): Matching Placebo Placebo Group 1 Intervention

Participants will receive matching placebo during Part A \[Single Ascending Dose (SAD)\].

Group XIII: Part B (MAD): Matching Placebo Placebo Group 1 Intervention

Participants will receive matching placebo on multiple days during Part B (MAD).

Who Is Running the Clinical Trial?

Ionis Pharmaceuticals, Inc.

Lead Sponsor

Trials

151

Recruited

27,800+

Dr. Brett P. Monia

Ionis Pharmaceuticals, Inc.

Chief Executive Officer since 2020

PhD in Pharmacology from the University of Pennsylvania, BSc in Molecular Biology and Analytical Chemistry from Stockton State College

Dr. Eric Bastings

Ionis Pharmaceuticals, Inc.

Chief Medical Officer

MD

Biogen

Lead Sponsor

Trials

655

Recruited

468,000+

Daniel Quirk

Biogen

Chief Medical Officer

MD

Christopher A. Viehbacher

Biogen

Chief Executive Officer since 2022

Graduated from Queen's University, Kingston, Ontario, Canada

Published Research Related to This Trial

Current dopamine replacement therapies can alleviate motor symptoms of Parkinson's disease, but there is a significant need for improved treatments that also address non-motor symptoms and provide neuroprotection.

Research is focusing on identifying promising biological targets and therapeutic agents, with an emphasis on understanding genetic factors and enhancing the translation of findings from animal models to human clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Priorities in Parkinson's disease research. Meissner, WG., Frasier, M., Gasser, T., et al.[2022]

In a study of 20 patients with advanced Parkinson's disease, deep brain stimulation (DBS) of the subthalamic nucleus (STN) led to significant improvements in motor symptoms, with a 49.3% reduction in the Unified Parkinson's Disease Rating Scale (UPDRS) scores in the 'off' medication state after one year.

DBS also enhanced daily living activities by 73.8%, reduced the need for medication by 54.1%, and decreased complications like dyskinesia and motor fluctuations, indicating its efficacy in managing Parkinson's disease symptoms.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Improvement of Advanced Parkinson's Disease Manifestations with Deep Brain Stimulation of the Subthalamic Nucleus: A Single Institution Experience. Rabie, A., Verhagen Metman, L., Fakhry, M., et al.[2020]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03976349

NCT03976349 | A Study to Evaluate the Safety, Tolerability ... The primary objective of this study is to evaluate the safety and tolerability of single and multiple doses of BIIB094 administered via intrathecal (IT) ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12263715/

Investigational Gene Therapies for Parkinson's Disease - PMC Safety of AADC gene therapy for moderately advanced Parkinson disease: three-year outcomes from the PD-1101 Trial. Neurology. 2022;98(1):e40 ...

3. withpower.com withpower.com/trial/phase-1-parkinson-disease-7-2019-374ab

BIIB094 for Parkinson's Disease (REASON Trial) This trial tests the safety and behavior of a drug called BIIB094 in patients with Parkinson's Disease, including those with different genetic backgrounds.

4. biogentrialtransparency.com biogentrialtransparency.com/content/dam/global-development/general/biogen-trial-link/educational/en-us/pdf/lls/english-master/NCT03976349-LLS.pdf

A Study to Learn About the Safety of BIIB094 in Adults With ... This study helped researchers learn more about the safety of BIIB094 in humans for the first time and the potential to help people with Parkinson's disease (PD) ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10001341/

Recent Advances in the Treatment of Genetic Forms of ... Unfortunately, a randomized, double-blind, placebo-controlled study (SPARK) that evaluated the efficacy and safety of BIIB054 in PD patients did ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT03976349

NCT03976349 | A Study to Evaluate the Safety, Tolerability ... The primary objective of this study is to evaluate the safety and tolerability of single and multiple doses of BIIB094 administered via intrathecal (IT) ...

7. sigma.larvol.com sigma.larvol.com/product.php?e1=788528&tab=newstrac

BIIB094 News BIIB094 / Ionis, Biogen ION859, Which Targets LRRK2 RNA to Reduce CNS LRRK2 Protein Production, is Well-tolerated and Potently Reduces CSF LRRK2 Protein and ...