What is the mechanism of action of this treatment?

Common treatments for neurodegeneration work by slowing down neuronal loss, protecting neurons, or restoring their function. For instance, the Coenzyme A precursor being studied for PKAN aims to restore Coenzyme A levels, essential for energy metabolism and synthesis of vital molecules, thereby improving cellular function and potentially slowing disease progression. Other treatments include neurotrophic factors that support neuron survival, antioxidants that reduce oxidative stress, and anti-inflammatory agents that mitigate harmful inflammation. These mechanisms are crucial as they target the underlying processes of neurodegeneration, offering hope for better quality of life and disease outcomes for patients.

What side effects are associated with this type of intervention?

Common treatments for neurodegenerative diseases include cholinesterase inhibitors, antioxidants, and various experimental therapies such as stem cell treatments and enzyme inhibitors. Cholinesterase inhibitors, like rivastigmine and donepezil, often cause side effects such as nausea, vomiting, and worsened tremors. Antioxidants, while generally well-tolerated, can sometimes lead to gastrointestinal issues. Experimental therapies, including stem cell treatments, may have side effects like immune reactions or infections. Overall, while these treatments aim to manage symptoms or slow disease progression, they can come with a range of side effects that need to be carefully monitored.

What prior FDA approvals exist?

FDA-approved treatments for neurodegenerative diseases include several drugs that target metabolic pathways and offer neuroprotective effects. For Alzheimer's disease, acetylcholinesterase inhibitors like donepezil and NMDA receptor antagonists like memantine are approved. In ALS, riluzole and edaravone are used to slow disease progression. For multiple sclerosis, disease-modifying therapies such as dimethyl fumarate and glatiramer acetate are common. These treatments share a focus on modifying disease progression and protecting neural function, similar to the goals of the Coenzyme A Precursor being studied.

What other types of research exist?

Promising novel alternatives for neurodegeneration patients include: 1) Mesenchymal stem cells induced to secrete high levels of neurotrophic factors, which have shown efficacy in amyotrophic lateral sclerosis (ALS) (Cudkowicz et al., 2022). 2) Ultra-high-dose methylcobalamin, which has demonstrated neuroprotective effects in ALS (Kaji et al., 2019). 3) Antisense therapies targeting huntingtin expression for Huntington's disease, which have shown sustained therapeutic reversal (Pepinemab antibody blockade, RNA interference therapy). 4) CRISPR-Cas9 gene editing for transthyretin amyloidosis, which has shown promising results in early trials (Gillmore et al., 2021).

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CoA-Z for PKAN

N/A

Waitlist Available

Led By Penelope Hogarth, M.D.

Research Sponsored by Oregon Health and Science University

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 months

Summary

This trial is testing a new treatment to see if it helps people with PKAN, a rare condition. Researchers want to find out if the treatment is safe and effective in improving symptoms or slowing the disease.

Who is the study for?

This trial is for people with PKAN, a neurodegenerative condition. Participants can be from 3 months to 89 years old and must be able to take the study product orally or via feeding tube. They need a confirmed diagnosis of PKAN through genetic testing or brain imaging, live in North America, and commit to study procedures. Those on similar treatments within the last month or in another clinical trial are excluded.

What is being tested?

The trial is testing CoA-Z against a placebo in individuals with PKAN. The goal is to determine if CoA-Z is safe, its effects on patients, and whether it alters disease measures. Participants will randomly receive either CoA-Z or an inactive substance (placebo) for comparison.

What are the potential side effects?

While specific side effects of CoA-Z aren't listed here, common ones may include allergic reactions, gastrointestinal issues like nausea or diarrhea, headaches, fatigue, and potential interactions with other medications.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Mean percent of study product consumed.

Number of participants experiencing adverse events assessed by CTCAE v4.0

Number of participants experiencing clinically significant laboratory abnormalities on Complete Blood Count.

+5 more

Secondary study objectives

Ratio of CoASY mRNA expression level to that of 18s, an internal control

Trial Design

5Treatment groups

Experimental Treatment

Placebo Group

Group I: Open-label armExperimental Treatment1 Intervention

Up to 24 months of CoA-Z at dose 2

Group II: CoA-Z dose 3Experimental Treatment1 Intervention

6 months of CoA-Z at the lowest assigned dose followed by 18 months of CoA-Z at dose 2

Group III: CoA-Z dose 2Experimental Treatment1 Intervention

6 months of CoA-Z at the medium assigned dose followed by 18 months of CoA-Z at dose 2

Group IV: CoA-Z dose 1Experimental Treatment1 Intervention

6 months of CoA-Z at the highest assigned dose followed by 18 months of CoA-Z at dose 2

Group V: PlaceboPlacebo Group2 Interventions

6 months of placebo, followed by 18 months of CoA-Z at dose 2 (medium dose)

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for neurodegeneration work by slowing down neuronal loss, protecting neurons, or restoring their function. For instance, the Coenzyme A precursor being studied for PKAN aims to restore Coenzyme A levels, essential for energy metabolism and synthesis of vital molecules, thereby improving cellular function and potentially slowing disease progression. Other treatments include neurotrophic factors that support neuron survival, antioxidants that reduce oxidative stress, and anti-inflammatory agents that mitigate harmful inflammation. These mechanisms are crucial as they target the underlying processes of neurodegeneration, offering hope for better quality of life and disease outcomes for patients.