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

77 Participants Needed

Trial Summary

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.

How does the drug CoA-Z for PKAN differ from other treatments?

CoA-Z is unique because it aims to restore levels of coenzyme-A (CoA) in the brain by delivering phosphopantothenic acid (PPA), which is crucial for CoA production. This approach is novel as it directly targets the underlying cause of PKAN, a genetic disorder, by reactivating CoA biosynthesis, unlike other treatments that may not address this specific biochemical pathway. ^{1 2 3 4 5}

What is the purpose of this trial?

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.

Research Team

Penelope Hogarth, M.D.

Principal Investigator

Oregon Health and Science University

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

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+

References

1. United States pubmed.ncbi.nlm.nih.gov

Cyclic Phosphopantothenic Acid Prodrugs for Treatment of Pantothenate Kinase-Associated Neurodegeneration. [2021]

2. United States pubmed.ncbi.nlm.nih.gov

Modification of bovine kidney pyruvate dehydrogenase kinase activity by CoA esters and their mechanism of action. [2021]

3. United States pubmed.ncbi.nlm.nih.gov

Pantothenate kinase activation relieves coenzyme A sequestration and improves mitochondrial function in mice with propionic acidemia. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

A pantothenate kinase from Staphylococcus aureus refractory to feedback regulation by coenzyme A. [2021]

5. England pubmed.ncbi.nlm.nih.gov

Determination by photoaffinity labelling of the hydrophobic part of the binding site for acyl-CoA esters on acyl-CoA-binding protein from bovine liver. [2019]