CoA-Z for PKAN

Overseen By Allison Gregory, M.S.

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Oregon Health and Science University

Must not be taking: PANK2 bypass agents

Disqualifiers: Other trials, Confounding conditions, others

Compensation: Varies

Number of Visits: Unknown

Duration: 24 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial focuses on how individuals with pantothenate kinase-associated neurodegeneration (PKAN) respond to a study product called CoA-Z. Researchers aim to determine CoA-Z's safety, identify its effects, and assess its impact on PKAN symptoms. Participants will receive varying doses of CoA-Z or a placebo (a dummy treatment with no active ingredients), with some switching to CoA-Z after six months. Suitable candidates include those with PKAN who can take the treatment orally or via feeding tube and reside in North America. As an unphased trial, this study offers patients the chance to contribute to groundbreaking research that could lead to new insights and potential treatments for PKAN.

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. However, you cannot have taken a specific type of medication called a 'putative PANK2 bypass therapeutic agent' in the 30 days before the trial.

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

Research has shown that CoA-Z is under study for safety in people with pantothenate kinase-associated neurodegeneration (PKAN). Earlier trials suggested that researchers are evaluating CoA-Z for safety and tolerability. Evidence from similar studies indicates that treatments like CoA-Z, such as pantethine, have been tested for safety in children with PKAN.

Submitting CoA-Z for FDA review indicates data supporting its safety. Typically, reaching this stage means the treatment has undergone sufficient testing to suggest it is well-tolerated, though more information is still being gathered. Participants in such trials contribute valuable data on safety and potential side effects. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Most treatments for Pantothenate Kinase-Associated Neurodegeneration (PKAN) focus on managing symptoms rather than addressing the root cause. CoA-Z is unique because it targets the underlying biochemical deficiency in PKAN by replenishing Coenzyme A levels, which are crucial for cellular energy production and metabolism. Researchers are excited about CoA-Z because it offers a novel way to potentially modify the disease's progression, rather than just alleviating symptoms. Additionally, CoA-Z is administered in a range of doses, allowing researchers to determine the most effective and safe dosing regimen for long-term use.

What evidence suggests that this trial's treatments could be effective for PKAN?

Research has shown that CoA-Z might help treat pantothenate kinase-associated neurodegeneration (PKAN) by boosting certain brain chemicals. This is important because PKAN is linked to issues like muscle stiffness and movement problems. Some studies suggest that treatments like CoA-Z can pass through the blood-brain barrier, a protective layer around the brain, to provide benefits where needed. Early evidence from similar treatments has shown promise in improving symptoms. However, more research is needed to confirm how well CoA-Z works specifically for PKAN. Participants in this trial will receive different doses of CoA-Z or a placebo to evaluate its effectiveness. ^{1 3 4 6 7}

Who Is on the Research Team?

Susan J Hayflick, MD

Principal Investigator

Oregon Health and Science University

Penelope Hogarth, M.D.

Principal Investigator

Oregon Health and Science University

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

Be willing and able to complete study procedures, telephone visits, and blood draws independently, OR have a caregiver or parent willing and able to assist with these tasks

Be enrolled or willing to enroll in the PKANready natural history study (eIRB 10832)

Be resident in North America (US or Canada) for the duration of the trial

Exclusion Criteria

Be concurrently enrolled in another interventional clinical trial

Have concurrent medical or other condition expected to preclude completion of study procedures or confound the assessment of clinical and laboratory measures of safety

You have taken a specific type of medication for a certain condition within the 30 days before screening.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive CoA-Z or placebo for 6 months, followed by 18 months of CoA-Z at dose 2

24 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of CoA-Z treatment for up to 24 months

Up to 24 months

What Are the Treatments Tested in This Trial?

Interventions

• CoA-Z
• Placebo

Trial Overview 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.

How Is the Trial Designed?

Group I: Open-label arm Experimental Treatment 1 Intervention

Up to 24 months of CoA-Z at dose 2

Group II: CoA-Z dose 3 Experimental Treatment 1 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 2 Experimental Treatment 1 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 1 Experimental Treatment 1 Intervention

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

Group V: Placebo Placebo Group 2 Interventions

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

Who Is Running the Clinical Trial?

Oregon Health and Science University

Lead Sponsor

Trials

1,024

Recruited

7,420,000+

Spoonbill

Collaborator

Washington State University

Collaborator

Trials

114

Recruited

58,800+

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Collaborator

Trials

2,103

Recruited

2,760,000+

Oregon State University

Collaborator

Trials

51

Recruited

8,300+

Spoonbill Foundation

Collaborator

Trials

1

Recruited

80+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04182763

NCT04182763 | CoA-Z in Pantothenate Kinase-associated ... The purpose of this study is to learn more about how people with the condition pantothenate kinase-associated neurodegeneration (PKAN) respond to a ...

2. ncbi.nlm.nih.gov ncbi.nlm.nih.gov/books/NBK430689/

Pantothenate Kinase-Associated Neurodegeneration (PKAN) Long-term outcomes in atypical PKAN vary, with some individuals maintaining functional abilities for extended periods. PKAN progresses ...

3. ojrd.biomedcentral.com ojrd.biomedcentral.com/articles/10.1186/s13023-020-01530-5

Pilot trial on the efficacy and safety of pantethine in children ... This study aimed to explore the efficacy and safety of pantethine in children with pantothenate kinase-associated neurodegeneration (PKAN).

4. sciencedirect.com sciencedirect.com/science/article/pii/S0022356524171479

Pantothenate Kinase Activation Restores Brain Coenzyme ... Pantothenate kinase-associated neurodegeneration (PKAN) is characterized by a motor disorder with combinations of dystonia, parkinsonism, and spasticity, ...

5. nature.com nature.com/articles/s41467-018-06703-2

A therapeutic approach to pantothenate kinase associated ... The potential therapeutic benefit of a treatment for PKAN neurodegeneration rests on its ability to cross the blood–brain barrier and increase ...

6. nbiadisorders.org nbiadisorders.org/news-events/newsroom/506-nbiacure-prepares-to-submit-for-fda-approval-of-coa-z-compound

NBIAcure Prepares to Submit for FDA Approval of CoA-Z ... The FDA will evaluate data from the clinical trial to assess the safety and tolerability of CoA-Z and its effect in altering a biological marker of PKAN in the ...

7. withpower.com withpower.com/trial/phase-nerve-degeneration-11-2019-29137

CoA-Z for PKAN · Info for Participants 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 ...