Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| R01HD097328 | U.S. NIH Grant/Contract | View source |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | NIH |
| Washington State University | OTHER |
| Oregon State University | OTHER |
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| CoA-Z dose 1 | Experimental | 6 months of CoA-Z at the highest assigned dose followed by 18 months of CoA-Z at dose 2 |
|
| CoA-Z dose 2 | Experimental | 6 months of CoA-Z at the medium assigned dose followed by 18 months of CoA-Z at dose 2 |
|
| CoA-Z dose 3 | Experimental | 6 months of CoA-Z at the lowest assigned dose followed by 18 months of CoA-Z at dose 2 |
|
| Placebo | Placebo Comparator | 6 months of placebo, followed by 18 months of CoA-Z at dose 2 (medium dose) |
|
| Open-label arm | Experimental | Up to 24 months of CoA-Z at dose 2 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CoA-Z | Other | 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. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Treatment-emergent Adverse Events Assessed Using CTCAE v4.0 | Safety will be measured using the number of treatment-emergent adverse events (both AE and SAE) by treatment arm. Counts are adjusted for number of prescription medications at baseline as a measure of baseline disease severity. | 6 months following first dose in double-blind phase |
| Number of Treatment-emergent Clinically Significant Laboratory Abnormalities on Complete Blood Count. | Safety will be assessed by measuring the number of treatment-emergent clinically significant laboratory abnormalities on Complete Blood Count. Clinical significance will be determined by Medical Safety Monitor and Clinical Investigators. | 6-month randomized, double-blind, placebo-controlled phase |
| Number of Treatment-emergent Clinically Significant Laboratory Abnormalities on Comprehensive Metabolic Profile. | Safety will be assessed by measuring the number of treatment-emergent clinically significant laboratory abnormalities on Comprehensive Metabolic Profile by collection month. Clinical significance will be determined by Medical Safety Monitor and Clinical Investigators. | 6-month randomized, double-blind, placebo-controlled phase |
| Number of Participants Retained in Each Arm. | Tolerability will be assessed by measuring the number of participants retained in each arm over the course of the study. | 6 month randomized, double-blind, placebo-controlled period |
| Mean Percent of Study Product Consumed. | Tolerability will be assessed by adherence to the study product regimen arm at each follow-up time point. | 6-month randomized, double-blind, placebo-controlled phase |
| Measure | Description | Time Frame |
|---|---|---|
| CoASY mRNA Expression | Average relative CoASY gene expression, measured as the ratio to baseline of 1 / (2^[COASY Ct - 18s Ct]), where Ct = cycle time and 18s is a housekeeping gene. Values >1 reflect higher expression. | Up to 6 months after first dose |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Penelope Hogarth, M.D. | Oregon Health and Science University | Principal Investigator |
| Susan J Hayflick, MD | Oregon Health and Science University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
Not provided
| Label | URL |
|---|---|
| Trial information website | View source |
Not provided
IPD will be entered in a stand-alone repository that will allow for future sharing with other researchers. At this time we have not yet determined what IPD are to be shared.
Not provided
Data will not be available until after September, 2024
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | CoA-Z Group 1 | Double blind CoA-Z 20mg/m^2 (Dose 1) Open label CoA-Z 15mg/m^2 (Dose 2) |
| FG001 | CoA-Z Group 2 | Double blind CoA-Z 15mg/m^2 (Dose 2) Open label CoA-Z 15mg/m^2 (Dose 2) |
| FG002 | CoA-Z Group 3 | Double blind CoA-Z 5mg/m^2 (Dose 3) Open label CoA-Z 15mg/m^2 |
| FG003 | Placebo Group | Double blind CoA-Z Placebo Open label CoA-Z 15mg/m^2 |
| FG004 | Open-label arm | Open-label Arm CoA-Z 15mg/m^2 |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Double-Blind Period, initial 6 months |
|
| ||||||||||||||||||
| Open-Label Period, subsequent 18 months |
|
Note that participants first complete the double-blind period, then crossover to open label, and additional subjects were able to enroll directly into open label. Therefore, the 12 open label participants at baseline are ones who enrolled directly into open label. The others crossed over to open label after the first 6 months and are captured in their original arms.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | CoA-Z dose 1 | Double blind CoA-Z 20mg/m^2 (Dose 1) Open label CoA-Z 15mg/m^2 (Dose 2) |
| BG001 | CoA-Z dose 2 | Double blind CoA-Z 15mg/m^2 (Dose 2) Open label CoA-Z 15mg/m^2 (Dose 2) |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Treatment-emergent Adverse Events Assessed Using CTCAE v4.0 | Safety will be measured using the number of treatment-emergent adverse events (both AE and SAE) by treatment arm. Counts are adjusted for number of prescription medications at baseline as a measure of baseline disease severity. | All randomized participants. | Posted | Geometric Least Squares Mean | 90% Confidence Interval | Events per person | 6 months following first dose in double-blind phase |
|
The 24-month study period for each participant
We included all treatment-emergent adverse events
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | CoA-Z dose 1 -- double-blind period | Double blind CoA-Z 20mg/m^2 (Dose 1) | 0 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bone fracture | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Boil | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Non-systematic Assessment |
December 2019, approximately 4 months before the onset of COVID-19 shutdowns. We therefore operated almost entirely during the pandemic, which led to certain challenges. We had to shorten the open-label period for 10 participants due to study product shortage. Additionally, some data presented here are from the 6-month, double-blind, placebo-controlled section of the study, the most important time period.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Allison Gregory, MS CGC, Project Manager | Oregon Health & Science University | 503-494-4344 | gregorya@ohsu.edu |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Nov 29, 2024 | Jun 23, 2025 | Prot_SAP_000.pdf |
Not provided
| ID | Term |
|---|---|
| D006211 | Pantothenate Kinase-Associated Neurodegeneration |
| ID | Term |
|---|---|
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
Not provided
Not provided
| Spoonbill Foundation |
| UNKNOWN |
| Spoonbill | UNKNOWN |
An initial 6-month, dose-ranging, parallel-group, randomized, double-blind, placebo-controlled phase is followed by an 18-month, single-dose, open-label phase--these arms met enrollment goals. A direct-to-open-label arm of the study was opened to allow for additional enrollment.
Not provided
Not provided
Masking was used for the initial 6-month randomized, double-blind phase, placebo-controlled.
| Placebo | Other | The placebo is a strawberry-flavored syrup that looks and tastes like CoA-Z but has no active CoA-Z in it. |
|
| Death |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
| BG002 | CoA-Z dose 3 | Double blind CoA-Z 5mg/m^2 (Dose 3) Open label CoA-Z 15mg/m^2 |
| BG003 | Placebo | Double blind CoA-Z Placebo Open label CoA-Z 15mg/m^2 |
| BG004 | Open Label | Open-label Arm CoA-Z 15mg/m^2 |
| BG005 | Total | Total of all reporting groups |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Type of pantothenate kinase-associated neurodegeneration (PKAN) | Classic disease is defined as onset of motor symptoms by age 5 and loss of ambulation by age 10. Atypical PKAN encompasses all participants who are not considered classic. | Count of Participants | Participants |
|
| Mean number of baseline medications | Mean | Standard Deviation | medications |
|
| OG002 | CoA-Z dose 3 | Double blind CoA-Z 5mg/m^2 (Dose 3) Open label CoA-Z 15mg/m^2 |
| OG003 | Placebo | Double blind CoA-Z Placebo Open label CoA-Z 15mg/m^2 |
|
|
|
| Primary | Number of Treatment-emergent Clinically Significant Laboratory Abnormalities on Complete Blood Count. | Safety will be assessed by measuring the number of treatment-emergent clinically significant laboratory abnormalities on Complete Blood Count. Clinical significance will be determined by Medical Safety Monitor and Clinical Investigators. | Posted | Number | Abnormal CBC event | 6-month randomized, double-blind, placebo-controlled phase |
|
|
|
| Primary | Number of Treatment-emergent Clinically Significant Laboratory Abnormalities on Comprehensive Metabolic Profile. | Safety will be assessed by measuring the number of treatment-emergent clinically significant laboratory abnormalities on Comprehensive Metabolic Profile by collection month. Clinical significance will be determined by Medical Safety Monitor and Clinical Investigators. | Posted | Number | Abnormal CMP event | 6-month randomized, double-blind, placebo-controlled phase |
|
|
|
| Primary | Number of Participants Retained in Each Arm. | Tolerability will be assessed by measuring the number of participants retained in each arm over the course of the study. | All participants who completed Screening, Informed Consent, and enrolled in the study. | Posted | Count of Participants | Participants | 6 month randomized, double-blind, placebo-controlled period |
|
|
|
| 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. | Population of all subjects who returned partial or complete dosing diaries. | Posted | Mean | Standard Deviation | percent of doses taken | 6-month randomized, double-blind, placebo-controlled phase |
|
|
|
| Secondary | CoASY mRNA Expression | Average relative CoASY gene expression, measured as the ratio to baseline of 1 / (2^[COASY Ct - 18s Ct]), where Ct = cycle time and 18s is a housekeeping gene. Values >1 reflect higher expression. | All randomized participants | Posted | Geometric Least Squares Mean | 90% Confidence Interval | Ratio to baseline | Up to 6 months after first dose |
|
|
|
|
| 15 |
| 8 |
| 15 |
| 10 |
| 15 |
| EG001 | CoA-Z dose 2 -- double-blind period | Double blind CoA-Z 15mg/m^2 (Dose 2) | 1 | 17 | 7 | 17 | 15 | 17 |
| EG002 | CoA-Z dose 3 -- double-blind period | Double blind CoA-Z 5mg/m^2 (Dose 3) | 0 | 17 | 5 | 17 | 9 | 17 |
| EG003 | Placebo -- double-blind period | Double blind CoA-Z Placebo | 0 | 16 | 3 | 16 | 8 | 16 |
| EG004 | CoA-Z dose 1 -- open-label period | Open label CoA-Z 15mg/m^2 (Dose 2) | 3 | 15 | 7 | 15 | 9 | 15 |
| EG005 | CoA-Z dose 2 -- open-label period | Open label CoA-Z 15mg/m^2 (Dose 2) | 2 | 16 | 7 | 16 | 10 | 16 |
| EG006 | CoA-Z dose 3 -- open-label period | Open label CoA-Z 15mg/m^2 (Dose 2) | 2 | 17 | 7 | 17 | 10 | 17 |
| EG007 | Placebo -- open-label period | Open label CoA-Z 15mg/m^2 (Dose 2) | 0 | 16 | 3 | 16 | 10 | 16 |
| EG008 | Open-label period only | Open label CoA-Z 15mg/m^2 (Dose 2) | 1 | 12 | 3 | 12 | 2 | 12 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Excessive sleep | General disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| G-tube placement/surgery | Gastrointestinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Hip dislocation | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Hospitalization | General disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Mouth injury | Gastrointestinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Orthopedic surgery | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Pneumonia | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Respiratory infection | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Appendix rupture | Gastrointestinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Septic shock | Blood and lymphatic system disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Surgery for excessive salivation | Gastrointestinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Cardiopulmonary arrest | Cardiac disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Hypoxic encephalopathy | Nervous system disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Ileus | Gastrointestinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Deep brain stimulator placement | Nervous system disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Bursitis | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Cellulitis | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Chest pain | Cardiac disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Diarrhea | Gastrointestinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Ear infection | Ear and labyrinth disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Ear tube placement | Ear and labyrinth disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Elevated AST | Investigations | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Elevated LFT | Investigations | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Elevated CK | Investigations | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Fever | General disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| G-tube surgery (outpatient) | Gastrointestinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Head laceration | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Low platelet count | Investigations | MedDRA (Unspecified) | Non-systematic Assessment |
|
| MSSA blood infection | Infections and infestations | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Mouth sores | Gastrointestinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Nail infection | Infections and infestations | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Respiratory infection | Infections and infestations | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Rhinovirus | Infections and infestations | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Scratch | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Sinuplasty | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Skin infection | Infections and infestations | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Swimmer's ear | Infections and infestations | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Tachycardia | Cardiac disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Tinnitus | Ear and labyrinth disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Urinary incontinence | Renal and urinary disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Urinary tract infection | Renal and urinary disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Whitlow finger | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Wound infection | Infections and infestations | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Hypoxemia | Vascular disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Coronavirus | Infections and infestations | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Avascular necrosis | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Foreign body | Gastrointestinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Pain | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Sleep apnea | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Aortic dilatation | Cardiac disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Barretts esophagus | Gastrointestinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Farsighted | Eye disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Foot infection | Infections and infestations | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Deep vein thrombosis | Blood and lymphatic system disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Salivation, excessive | Gastrointestinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Skin irritation | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| ADHD | Nervous system disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Urinary retention | Renal and urinary disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Tooth extraction | Surgical and medical procedures | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Pressure sore | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Pneumonia | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Sinus infection | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Seizure | Nervous system disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Overdose | Social circumstances | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Dental abscess | Infections and infestations | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Bacterial vaginosis | Reproductive system and breast disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Acne vulgaris | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Hypothyroidism | Endocrine disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Baclofen pump | Surgical and medical procedures | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Anemia | Blood and lymphatic system disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Tooth damage | Injury, poisoning and procedural complications | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Cauliflower ear | Ear and labyrinth disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Malabsorption | Gastrointestinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Hematoma drain | Surgical and medical procedures | MedDRA (Unspecified) | Non-systematic Assessment |
|
| c. diff infection | Infections and infestations | MedDRA (Unspecified) | Non-systematic Assessment |
|
| GERD | Gastrointestinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Dislocation | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Bone fracture | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Bone fragment removal | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Chest pain | Cardiac disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Vision loss | Eye disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Dysautonomia | Nervous system disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Anosmia | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Hyperopia | Eye disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Pes planus | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Sty | Eye disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Hyperventilation | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Sialorrhea | Gastrointestinal disorders | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Hyponatremia | Blood and lymphatic system disorders | MedDRA (Unspecified) | Systematic Assessment |
|
Not provided
Not provided
Not provided
| D019150 | Neuroaxonal Dystrophies |
| D009069 | Movement Disorders |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| 6 months after first dose |
|
| Test that high > med > low > placebo vs the null that all groups are equal at 6 months after first dose. PKAN type (classical or atypical) was included because randomization was stratified on type. The study hypothesis was that expression levels would not vary significantly at this time point. | Linear contrast / test for trend | Contrast after GLM with log link, treatment, and PKAN type. | 0.55 | Two-sided threshold of p<.10 | Superiority |