CoA-Z in Pantothenate Kinase-associated Neurodegeneration (PKAN)

Pantothenate Kinase-Associated Neurodegeneration Clinical Trial

Official title:

A Phase 2 Study of a Vitamin Metabolite for PKAN

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04182763
• Other study ID #: IRB00018782
• Secondary ID: R01HD097328
• Status: Active, not recruiting
• Phase: N/A
• Start date: December 4, 2019
• Est. completion date: January 2024

Study information

• Verified date: January 2022
• Source: Oregon Health and Science University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to learn more about how people with the condition pantothenate kinase-associated neurodegeneration (PKAN) respond to a specialized study product. We are hoping to find out if the study product is safe, what effects-good and bad-the study product causes, and whether the study product changes certain measures of disease in PKAN.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 77
• Est. completion date: January 2024
• Est. primary completion date: January 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Months to 89 Years

Eligibility

Inclusion Criteria:

• Has a diagnosis of PKAN confirmed by: a) genetic testing confirming 2 pathogenic or likely pathogenic mutations, or (b) typical findings on exam and brain MR imaging with only one pathogenic mutation +/- a second likely pathogenic or VOUS in PANK2, or (c) typical findings on exam and brain MR imaging with a single likely pathogenic or VOUS in PANK2, or (d) be a symptomatic sibling of a proband subject meeting a, b or c.

• Be between 3 months old and 89 years old.

• Be able to take study product by mouth or feeding tube.

• Be willing and able to complete study procedures / telephone visits / blood draws independently, OR have a caregiver / 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:

• Have had exposure to a putative PANK2 bypass therapeutic agent in the 30 days prior to screening.

• Be concurrently enrolled in another interventional clinical trial.

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

Related Conditions & MeSH terms

• Nerve Degeneration
• Pantothenate Kinase-Associated Neurodegeneration

Intervention

Other:
• CoA-Z
People with PKAN lack a chemical to process or metabolize a certain vitamin in the brain. CoA-Z is designed to bypass this metabolic defect that causes PKAN.
• Placebo
The placebo is a strawberry-flavored syrup that looks and tastes like CoA-Z but has no active CoA-Z in it.

Locations

Country	Name	City	State
United States	Oregon Health & Science University	Portland	Oregon

Sponsors (5)

Lead Sponsor	Collaborator
Oregon Health and Science University	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Oregon State University, Spoonbill Foundation, Washington State University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants experiencing adverse events assessed by CTCAE v4.0	Safety will be measured by measuring the number of participants experiencing adverse events by study period and treatment arm	24 months
Primary	Percentage of participants experiencing adverse events assessed by CTCAE v4.0	Safety will be measured by measuring the percentage of participants experiencing adverse events by study period and treatment arm	24 months
Primary	Percentage of participants experiencing clinically significant laboratory abnormalities on Complete Blood Count.	Safety will be assessed by measuring the percentage of participants experiencing clinically significant laboratory abnormalities on Complete Blood Count by collection month. Clinical significance will be determined by Medical Safety Monitor and Clinical Investigators.	24 months
Primary	Number of participants experiencing clinically significant laboratory abnormalities on Complete Blood Count.	Safety will be assessed by measuring the number of participants experiencing clinically significant laboratory abnormalities on Complete Blood Count by collection month. Clinical significance will be determined by Medical Safety Monitor and Clinical Investigators.	24 months
Primary	Percentage of participants experiencing clinically significant laboratory abnormalities on Comprehensive Metabolic Profile.	Safety will be assessed by measuring the percentage of participants experiencing clinically significant laboratory abnormalities on Comprehensive Metabolic Profile by collection month. Clinical significance will be determined by Medical Safety Monitor and Clinical Investigators.	24 months
Primary	Number of participants experiencing clinically significant laboratory abnormalities on Comprehensive Metabolic Profile.	Safety will be assessed by measuring the number of participants experiencing clinically significant laboratory abnormalities on Comprehensive Metabolic Profile by collection month. Clinical significance will be determined by Medical Safety Monitor and Clinical Investigators.	24 months
Primary	Number of participants retained in each arm.	Tolerability will be assessed by measuring the number of participants retained in each arm at each follow up time point.	24 months
Primary	Mean percent of study product consumed.	Tolerability will be assessed by adherence to the study product regimen arm at each follow-up time point.	24 months
Secondary	Ratio of CoASY mRNA expression level to that of 18s, an internal control	The pharmacodynamic profile of a putative disease biomarker will be assessed by measuring the mean level of CoASY gene expression by treatment arm across study timepoints.	24 months

External Links

•   Trial information website