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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
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 moreSecondary 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)
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.
Find a Location
Who is running the clinical trial?
Washington State UniversityOTHER
109 Previous Clinical Trials
57,602 Total Patients Enrolled
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)NIH
2,061 Previous Clinical Trials
2,745,071 Total Patients Enrolled
Oregon Health and Science UniversityLead Sponsor
1,006 Previous Clinical Trials
7,414,035 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have been diagnosed with PKAN through genetic testing showing specific mutations or through typical findings on examination and brain imaging.You must be between 3 months and 89 years old.You have taken a specific type of medication for a certain condition within the 30 days before screening.
Research Study Groups:
This trial has the following groups:- Group 1: CoA-Z dose 3
- Group 2: Placebo
- Group 3: CoA-Z dose 1
- Group 4: CoA-Z dose 2
- Group 5: Open-label arm
Awards:
This trial has 0 awards, including:Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.