CompletedNCT04705415Updated Feb 3, 2025Results posted

A Single and Multiple Ascending Dose Study of Niclosamide in Healthy Volunteers

A Phase 1 interventional study of Niclosamide and Placebo in Healthy, sponsored by NeuroBo Pharmaceuticals Inc.. Completed at 1 site in United States. Open to participants aged 18 Years to 65 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2025-02-03.

Sponsored by NeuroBo Pharmaceuticals Inc. · Phase 1, Interventional, and Treatment

Phase: Phase 1
Study type: Interventional
Enrollment: 54
Allocation: Randomized
Ages: 18 Years to 65 Years
Sex: All

Study summary

A single and multiple ascending dose study of ANA001 in healthy adults to assess the safety and pharmacokinetics

Read the detailed description

This is a Phase 1, single center, randomized, double blind, single ascending dose (SAD) and multiple ascending dose (MAD) study to assess the safety and pharmacokinetics of ANA001 in healthy adult subjects. In the single ascending dose portion of the study, subjects in 3 cohorts of 10 subjects each will be randomized to receive a single daily oral dose of ANA001 or matching placebo. In the multiple ascending dose portion of the study, subjects in 3 cohorts of 12 subjects each will be randomized to receive twice or thrice daily oral dose of ANA001 or matching placebo.

Conditions studied

Healthy

In context

Lead sponsor

NeuroBo Pharmaceuticals Inc. is the lead sponsor of 15 studies on the registry; 1 is open to participants now.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 18 Years to 65 Years
Sexes eligible: All
Accepts healthy volunteers: Yes

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

Sign the study informed consent form
Man or woman, 18 to 65 years of age inclusive at the time of signing the informed consent form
Overtly healthy as determined by medical evaluation
Body mass index (BMI) within 18 to 30.0 kg/m2 (inclusive) and body weight not less than 50 kg
Blood pressure at Screening and Day -1 between 90 and 140 mmHg systolic, inclusive, and no higher than 90 mmHg diastolic.
A 12-lead electrocardiogram (ECG) at Screening consistent with normal cardiac conduction and function, including:
- Sinus rhythm
- Pulse rate between 50 and 100 beats per minute (bpm)
- QTc interval 450 milliseconds (QT interval corrected using Fridericia correction method [QTcF])
- QRS interval of \<120 milliseconds
- PR interval \<200 milliseconds
- Morphology consistent with healthy cardiac conduction and function
Non-smoker or ex-smoker for >12 months
If male, must agree to use contraception methods outlined for the study during the treatment period and for at least 30 days (a spermatogenesis cycle) after the last dose of study treatment and refrain from donating sperm during this period
If female, is not pregnant, not breastfeeding, and meets at least one of the following conditions:

Not a woman of childbearing potential (WOCBP)

A WOCBP who agrees to follow the contraceptive guidance during the treatment period and for at least 30 days (one menstrual cycle) after the last dose of study treatment.

Exclusion criteria

Exclusion Criteria:

Has a history of or current clinically significant medical illness including but not limited to, cardiac arrhythmias or other cardiac disease; hematologic disease; coagulation disorders (including any abnormal bleeding or blood dyscrasias); lipid abnormalities; significant pulmonary disease, including bronchospastic respiratory disease; diabetes mellitus; hepatic or renal insufficiency (creatinine clearance below 60 mL/min); thyroid disease; neurologic or psychiatric disease; infection; or any other illness that the Investigator considers should exclude the subject or that could interfere with the interpretation of the study results.
Has known allergy to niclosamide or salicylate-containing medications.
Clinically significant abnormal values for hematology, clinical chemistry, or urinalysis at screening.
Clinically significant abnormal physical examination, vital signs or 12 lead ECG at screening.
Has a history of human immunodeficiency virus (HIV) antibody positive, or tests positive for HIV; has a history of hepatitis B surface antigen (HBsAg) or hepatitis C antibody (anti-HCV) positive, or other clinically active liver disease, or tests positive for HBsAg or anti-HCV at Screening.
History of drug or alcohol abuse according to Diagnostic and Statistical Manual of Mental Disorders (4th edition) criteria within 5 years before screening or positive test result(s) for alcohol and/or drugs of abuse at screening and admission
Has received an investigational drug or used an invasive investigational medical device within 1 month or within a period less than 10 times the drug's half-life, whichever is longer, before Day 1.
Has preplanned surgery or procedures that would interfere with the conduct of the study
Is an employee of the Investigator or study site, with direct involvement in the proposed study or other studies under the direction of that Investigator or study site, as well as family members of the employees or the Investigator

Study design

Phase: Phase 1
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel assignment
Masking: Triple (Participant, Care provider, Investigator)
Enrollment: 54 participants (actual)

Study arms

Experimental
SAD Cohort 1: ANA001 1000 mg po
Subjects will receive a 1000 mg single oral dose of ANA001 with a standardized light meal presented at 4 x 250 mg capsules. Each capsule is 250 mg.
Drug: Niclosamide
Experimental
SAD Cohort 2: ANA001 2000 mg po
Subjects will a 2000 mg single oral dose of ANA001 with a standardized light meal presented at 8 x 250 mg capsules. Each capsule is 250 mg.
Drug: Niclosamide
Experimental
SAD Cohort 3: ANA001 3000 mg po
Subjects will a 3000 mg single oral dose of ANA001 with a standardized light meal presented at 12 x 250 mg capsules. Each capsule is 250 mg.
Drug: Niclosamide
Placebo comparator
SAD Cohorts 1, 2 & 3: Pooled Matching Placebo
Subjects will receive matching placebo hydroxypropylmethylcellulose (HPMC) as a single oral dose (4, 8, or 12 capsules) with a standardized light meal.
Drug: Placebo
Experimental
MAD Cohort 1: ANA001 1000 mg po BID
Subjects will receive an oral dose of 1000mg ANA001 twice daily (BID) with a standardized light meal for 7 consecutive days. Each dose will be presented as 4 250mg capsules.
Drug: Niclosamide
Experimental
MAD Cohort 2: ANA001 1000 mg po TID
Subjects will receive an oral dose of 1000mg ANA001 three times daily (TID) with a standardized light meal for 7 consecutive days. Each dose will be presented as 4 250mg capsules.
Drug: Niclosamide
Placebo comparator
MAD Cohorts 1 & 2: Pooled Matching Placebo
Subjects will receive an oral dose of matching Placebo to ANA001 two (BID) three times daily (TID) with a standardized light meal for 7 consecutive days. Each dose will be presented as 4 250mg capsules of hydroxypropylmethylcellulose (HPMC)
Drug: Placebo

Interventions

DrugNiclosamide
Niclosamide is an antihelmintic with in-vitro antiviral activity
Also known as: ANA001
DrugPlacebo
Matching hydroxypropylmethylcellulose HPMC capsules with no active ingredients
Also known as: Matching Placebo to ANA001

What researchers measure

Primary outcomes

SAD: Number of Subjects Reporting TEAEs and STEAEs
Number of Subjects Reporting treatment-emergent AEs (TEAEs) and Serious treatment-emergent AEs (STEAEs) in the SAD
Time frame: Baseline to Day 7.
MAD: Number of Subjects Reporting TEAEs and STEAEs
Number of Subjects Reporting treatment-emergent AEs (TEAEs) and Serious treatment-emergent AEs (STEAEs) in the MAD
Time frame: Baseline to Day 14.
SAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in ECG Parameters
Number of Subjects with Clinically Significant changes in ECG parameters from Baseline (PR, QRS, QT, and QTc intervals)
Time frame: Baseline to Day 7
MAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in ECG Parameters
Number of Subjects with Clinically Significant changes in ECG parameters from Baseline (PR, QRS, QT, and QTc intervals)
Time frame: Baseline to Day 14
SAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Haematology
Number of Subjects with Clinically Significant changes in Haematology (Basophils (%),Eosinophils (%, Ery. Mean Corpuscular Hemoglobin, Ery. Mean Corpuscular Volume, Erythrocytes (10\^12/L), Hematocrit (%), Hemoglobin (g/dL). Hemoglobin (g/dL), Lymphocytes (%), Monocytes (%), Neutrophils (%)' Platelets (10\^9/L), Reticulocytes (%))
Time frame: Baseline to Day 7
MAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Haematology
Number of Subjects with Clinically Significant changes in Haematology (Basophils (%),Eosinophils (%, Ery. Mean Corpuscular Hemoglobin, Ery. Mean Corpuscular Volume, Erythrocytes (10\^12/L), Hematocrit (%), Hemoglobin (g/dL). Hemoglobin (g/dL), Lymphocytes (%), Monocytes (%), Neutrophils (%)' Platelets (10\^9/L), Reticulocytes (%))
Time frame: Baseline to Day 14
SAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Serum Chemistry
Number of Subjects with Clinically Significant changes in Serum Chemistry (Alanine Aminotransferase (U/L), Alkaline Phosphatase (U/L), Aspartate Aminotransferase (U/L), Bilirubin (mg/dL), Calcium (mg/dL), Creatinine (mg/dL), Direct Bilirubin (mg/dL, Glucose (mg/dL), Potassium (mmol/L), Protein (g/dL), Sodium (mmol/L), Urea Nitrogen (mg/dL)
Time frame: Baseline to Day 7
MAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Serum Chemistry
Number of Subjects with Clinically Significant changes in Serum Chemistry (Alanine Aminotransferase (U/L), Alkaline Phosphatase (U/L), Aspartate Aminotransferase (U/L), Bilirubin (mg/dL), Calcium (mg/dL), Creatinine (mg/dL), Direct Bilirubin (mg/dL, Glucose (mg/dL), Potassium (mmol/L), Protein (g/dL), Sodium (mmol/L), Urea Nitrogen (mg/dL)
Time frame: Baseline to Day 14
SAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Urinalysis
Number of Subjects with Clinically Significant changes in Urinalysis (Specific Gravity, Urobilinogen (EU), pH)
Time frame: Baseline to Day 7
MAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Urinalysis
Number of Subjects with Clinically Significant changes in Urinalysis (Specific Gravity, Urobilinogen (EU), pH)
Time frame: Baseline to Day 14
SAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Vital Signs
Number of Subjects with Clinically Significant changes in Vital Signs from Baseline (Diastolic Blood Pressure, Systolic Blood Pressure (mmHg), Pulse Rate (beats/min), Respiratory Rate (breaths/min))
Time frame: Baseline to Day 7
MAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Vital Signs
Number of Subjects with Clinically Significant changes in Vital Signs from Baseline (Diastolic Blood Pressure, Systolic Blood Pressure (mmHg), Pulse Rate (beats/min), Respiratory Rate (breaths/min))
Time frame: Baseline to Day 14

Secondary outcomes

SAD: Tmax
Time to reach the maximum plasma concentration (SAD)
Time frame: PK samples were collected before dosing and at 0.5, 1, 2, 4, 6, 8, 10, 12 and 24 hours. A Follow-up sample was taken Day 7 (±2) after dosing.
SAD: Cmax
Maximum plasma concentration during a dosing interval (SAD)
Time frame: PK samples were collected before dosing and at 0.5, 1, 2, 4, 6, 8, 10, 12 and 24 hours. A Follow-up sample was taken Day 7 (±2) after dosing.
SAD: AUC0-t
Area under the plasma concentration-time curve from time 0 to time the last quantifiable concentration (SAD)
Time frame: PK samples were collected before dosing and at 0.5, 1, 2, 4, 6, 8, 10, 12 and 24 hours. A Follow-up sample was taken Day 7 (±2) after dosing.
SAD: AUC0-∞
Area under the plasma concentration time curve from time 0 extrapolated to infinity, calculated as AUC0-last + Clast/λ, where Clast is the last quantifiable concentration at time t (SAD)
Time frame: PK samples were collected before dosing and at 0.5, 1, 2, 4, 6, 8, 10, 12 and 24 hours. A Follow-up sample was taken Day 7 (±2) after dosing.
SAD: t1/2
Elimination half-life associated with the terminal slope (λ) of the semilogarithmic drug concentration-time curve, calculated as 0.693/λ (SAD)
Time frame: PK samples were collected before dosing and at 0.5, 1, 2, 4, 6, 8, 10, 12 and 24 hours. A Follow-up sample was taken Day 7 (±2) after dosing.
SAD: CL/F
Systemic Clearance (SAD)
Time frame: PK samples were collected before dosing and at 0.5, 1, 2, 4, 6, 8, 10, 12 and 24 hours. A Follow-up sample was taken Day 7 (±2) after dosing.
SAD: Vz/F
Volume of distribution (SAD)
Time frame: PK samples were collected before dosing and at 0.5, 1, 2, 4, 6, 8, 10, 12 and 24 hours. A Follow-up sample was taken Day 7 (±2) after dosing.
MAD: Tmax Day 1
Time to reach the maximum plasma concentration Day 1
Time frame: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
MAD: Tmax Day 7
Time to reach the maximum plasma concentration (Day 7)
Time frame: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
MAD: Cmax Day 1
Maximum plasma concentration during a dosing interval (Day 1)
Time frame: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: For BID but omitting samples at 10, and 12 hours after dosing.
MAD: Cmax Day 7
Maximum plasma concentration during a dosing interval (Day 7)
Time frame: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
MAD: AUC0-t Day 1
Area under the plasma concentration-time curve from time 0 to time the last quantifiable concentration (Day 1)
Time frame: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
MAD: AUC0-t Day 7
Area under the plasma concentration-time curve from time 0 to time the last quantifiable concentration (Day 7)
Time frame: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
MAD: AUC0-tau Day 1
Area under the plasma concentration-time curve over the dosing interval at steady-state, calculated from the dosing interval (Day 1)
Time frame: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
MAD: AUC0-tau Day 7
Area under the plasma concentration-time curve over the dosing interval at steady-state, calculated from the dosing interval (Day 7)
Time frame: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
MAD: t1/2 Day 1
Elimination half-life associated with the terminal slope (λ) of the semilogarithmic drug concentration-time curve, calculated as 0.693/λ (Day 1)
Time frame: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
MAD: t1/2 Day 7
Elimination half-life associated with the terminal slope (λ) of the semilogarithmic drug concentration-time curve, calculated as 0.693/λ (Day 7)
Time frame: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
MAD: CLss Day 1
Systemic Clearance at steady state (Day 1)
Time frame: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
MAD: CLss Day 7
Systemic Clearance at steady state (Day 7)
Time frame: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
MAD: Vdss Day 1
Volume of distribution at steady state (Day 1)
Time frame: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
MAD: Vdss Day 7
Volume of distribution at steady state (Day 7)
Time frame: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.

Results

Posted Feb 3, 2025

Participant flow

Participant flow — Overall Study
Milestone	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po	SAD Cohorts 1, 2 & 3: Pooled Matching Placebo	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID	MAD Cohorts 1 & 2: Pooled Matching Placebo
Started	8	8	8	6	9	9	6
Completed	7	7	8	5	8	9	5
Not completed	1	1	0	1	1	0	1

Outcome measures

PrimarySAD: Number of Subjects Reporting TEAEs and STEAEs

Number of Subjects Reporting treatment-emergent AEs (TEAEs) and Serious treatment-emergent AEs (STEAEs) in the SAD

Time frame:: Baseline to Day 7.
Reported as:: Number · participants

SAD: Number of Subjects Reporting TEAEs and STEAEs
participants	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po	SAD Cohorts 1, 2 & 3: Pooled Matching Placebo
Number of Subjects Reporting treatment-emergent AEs (TEAEs)	0	0	0	1
Number of Subjects Reporting treatment-emergent Serious AEs (STEAEs)	0	0	0	0

PrimaryMAD: Number of Subjects Reporting TEAEs and STEAEs

Number of Subjects Reporting treatment-emergent AEs (TEAEs) and Serious treatment-emergent AEs (STEAEs) in the MAD

Time frame:: Baseline to Day 14.
Reported as:: Number · participants

MAD: Number of Subjects Reporting TEAEs and STEAEs
participants	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID	MAD Cohorts 1 & 2: Pooled Matching Placebo
Number of Subjects Reporting TEAEs	3	3	1
Number of Subjects Reporting STEAEs	0	0	0

PrimarySAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in ECG Parameters

Number of Subjects with Clinically Significant changes in ECG parameters from Baseline (PR, QRS, QT, and QTc intervals)

Time frame:: Baseline to Day 7
Reported as:: Number · participants

SAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in ECG Parameters
participants	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po	SAD Cohorts 1, 2 & 3: Pooled Matching Placebo
SAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in ECG Parameters	0	0	0	0

PrimaryMAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in ECG Parameters

Number of Subjects with Clinically Significant changes in ECG parameters from Baseline (PR, QRS, QT, and QTc intervals)

Time frame:: Baseline to Day 14
Reported as:: Number · participants

MAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in ECG Parameters
participants	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID	MAD Cohorts 1 & 2: Pooled Matching Placebo
MAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in ECG Parameters	0	0	0

PrimarySAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Haematology

Number of Subjects with Clinically Significant changes in Haematology (Basophils (%),Eosinophils (%, Ery. Mean Corpuscular Hemoglobin, Ery. Mean Corpuscular Volume, Erythrocytes (10\^12/L), Hematocrit (%), Hemoglobin (g/dL). Hemoglobin (g/dL), Lymphocytes (%), Monocytes (%), Neutrophils (%)' Platelets (10\^9/L), Reticulocytes (%))

Time frame:: Baseline to Day 7
Reported as:: Number · participants

SAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Haematology
participants	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po	SAD Cohorts 1, 2 & 3: Pooled Matching Placebo
SAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Haematology	0	0	0	0

PrimaryMAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Haematology

Time frame:: Baseline to Day 14
Reported as:: Number · participants

MAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Haematology
participants	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID	MAD Cohorts 1 & 2: Pooled Matching Placebo
MAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Haematology	0	0	0

PrimarySAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Serum Chemistry

Number of Subjects with Clinically Significant changes in Serum Chemistry (Alanine Aminotransferase (U/L), Alkaline Phosphatase (U/L), Aspartate Aminotransferase (U/L), Bilirubin (mg/dL), Calcium (mg/dL), Creatinine (mg/dL), Direct Bilirubin (mg/dL, Glucose (mg/dL), Potassium (mmol/L), Protein (g/dL), Sodium (mmol/L), Urea Nitrogen (mg/dL)

Time frame:: Baseline to Day 7
Reported as:: Number · participants

SAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Serum Chemistry
participants	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po	SAD Cohorts 1, 2 & 3: Pooled Matching Placebo
SAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Serum Chemistry	0	0	0	0

PrimaryMAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Serum Chemistry

Time frame:: Baseline to Day 14
Reported as:: Number · participants

MAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Serum Chemistry
participants	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID	MAD Cohorts 1 & 2: Pooled Matching Placebo
MAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Serum Chemistry	0	0	0

PrimarySAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Urinalysis

Number of Subjects with Clinically Significant changes in Urinalysis (Specific Gravity, Urobilinogen (EU), pH)

Time frame:: Baseline to Day 7
Reported as:: Number · participants

SAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Urinalysis
participants	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po	SAD Cohorts 1, 2 & 3: Pooled Matching Placebo
SAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Urinalysis	0	0	0	0

PrimaryMAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Urinalysis

Number of Subjects with Clinically Significant changes in Urinalysis (Specific Gravity, Urobilinogen (EU), pH)

Time frame:: Baseline to Day 14
Reported as:: Number · participants

MAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Urinalysis
participants	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID	MAD Cohorts 1 & 2: Pooled Matching Placebo
MAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Urinalysis	0	0	0

PrimarySAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Vital Signs

Number of Subjects with Clinically Significant changes in Vital Signs from Baseline (Diastolic Blood Pressure, Systolic Blood Pressure (mmHg), Pulse Rate (beats/min), Respiratory Rate (breaths/min))

Time frame:: Baseline to Day 7
Reported as:: Number · participants

SAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Vital Signs
participants	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po	SAD Cohorts 1, 2 & 3: Pooled Matching Placebo
SAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Vital Signs	0	0	0	0

PrimaryMAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Vital Signs

Number of Subjects with Clinically Significant changes in Vital Signs from Baseline (Diastolic Blood Pressure, Systolic Blood Pressure (mmHg), Pulse Rate (beats/min), Respiratory Rate (breaths/min))

Time frame:: Baseline to Day 14
Reported as:: Number · participants

MAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Vital Signs
participants	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID	MAD Cohorts 1 & 2: Pooled Matching Placebo
MAD: Safety and Tolerability of ANA001 as Measured by Clinically Significant Changes in Vital Signs	0	0	0

SecondarySAD: Tmax

Time to reach the maximum plasma concentration (SAD)

Time frame:: PK samples were collected before dosing and at 0.5, 1, 2, 4, 6, 8, 10, 12 and 24 hours. A Follow-up sample was taken Day 7 (±2) after dosing.
Reported as:: Median · h

SAD: Tmax
h	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po
SAD: Tmax	4 (4 to 8)	4 (4 to 10)	4 (2 to 4.02)

SecondarySAD: Cmax

Maximum plasma concentration during a dosing interval (SAD)

Time frame:: PK samples were collected before dosing and at 0.5, 1, 2, 4, 6, 8, 10, 12 and 24 hours. A Follow-up sample was taken Day 7 (±2) after dosing.
Reported as:: Mean · ng/mL

SAD: Cmax
ng/mL	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po
SAD: Cmax	328 ± 230	647 ± 306	347 ± 124

SecondarySAD: AUC0-t

Area under the plasma concentration-time curve from time 0 to time the last quantifiable concentration (SAD)

Time frame:: PK samples were collected before dosing and at 0.5, 1, 2, 4, 6, 8, 10, 12 and 24 hours. A Follow-up sample was taken Day 7 (±2) after dosing.
Reported as:: Mean · h*ng/mL

SAD: AUC0-t
h*ng/mL	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po
SAD: AUC0-t	1420 ± 889	4620 ± 2040	1780 ± 558

SecondarySAD: AUC0-∞

Area under the plasma concentration time curve from time 0 extrapolated to infinity, calculated as AUC0-last + Clast/λ, where Clast is the last quantifiable concentration at time t (SAD)

Time frame:: PK samples were collected before dosing and at 0.5, 1, 2, 4, 6, 8, 10, 12 and 24 hours. A Follow-up sample was taken Day 7 (±2) after dosing.
Reported as:: Mean · h*ng/mL

SAD: AUC0-∞
h*ng/mL	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po
SAD: AUC0-∞	1660 ± 985	4890 ± 2310	1860 ± 575

SecondarySAD: t1/2

Elimination half-life associated with the terminal slope (λ) of the semilogarithmic drug concentration-time curve, calculated as 0.693/λ (SAD)

Time frame:: PK samples were collected before dosing and at 0.5, 1, 2, 4, 6, 8, 10, 12 and 24 hours. A Follow-up sample was taken Day 7 (±2) after dosing.
Reported as:: Mean · h

SAD: t1/2
h	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po
SAD: t1/2	4.40 ± 2.30	3.30 ± 0.925	5.34 ± 2.24

SecondarySAD: CL/F

Systemic Clearance (SAD)

Time frame:: PK samples were collected before dosing and at 0.5, 1, 2, 4, 6, 8, 10, 12 and 24 hours. A Follow-up sample was taken Day 7 (±2) after dosing.
Reported as:: Mean · L/h

SAD: CL/F
L/h	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po
SAD: CL/F	940 ± 631	572 ± 397	1780 ± 705

SecondarySAD: Vz/F

Volume of distribution (SAD)

Time frame:: PK samples were collected before dosing and at 0.5, 1, 2, 4, 6, 8, 10, 12 and 24 hours. A Follow-up sample was taken Day 7 (±2) after dosing.
Reported as:: Mean · L

SAD: Vz/F
L	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po
SAD: Vz/F	8540 ± 9010	3210 ± 3190	15100 ± 9390

SecondaryMAD: Tmax Day 1

Time to reach the maximum plasma concentration Day 1

Time frame:: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
Reported as:: Median · h

MAD: Tmax Day 1
h	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID
MAD: Tmax Day 1	4 (2 to 6.05)	4 (2 to 6)

SecondaryMAD: Tmax Day 7

Time to reach the maximum plasma concentration (Day 7)

Time frame:: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
Reported as:: Median · h

MAD: Tmax Day 7
h	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID
MAD: Tmax Day 7	4 (2 to 6)	2 (2 to 6)

SecondaryMAD: Cmax Day 1

Maximum plasma concentration during a dosing interval (Day 1)

Time frame:: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: For BID but omitting samples at 10, and 12 hours after dosing.
Reported as:: Mean · ng/mL

MAD: Cmax Day 1
ng/mL	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID
MAD: Cmax Day 1	332 ± 110	259 ± 158

SecondaryMAD: Cmax Day 7

Maximum plasma concentration during a dosing interval (Day 7)

Time frame:: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
Reported as:: Mean · ng/mL

MAD: Cmax Day 7
ng/mL	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID
MAD: Cmax Day 7	234 ± 77.9	235 ± 70.9

SecondaryMAD: AUC0-t Day 1

Area under the plasma concentration-time curve from time 0 to time the last quantifiable concentration (Day 1)

Time frame:: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
Reported as:: Mean · h*ng/mL

MAD: AUC0-t Day 1
h*ng/mL	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID
MAD: AUC0-t Day 1	1630 ± 499	1360 ± 968

SecondaryMAD: AUC0-t Day 7

Area under the plasma concentration-time curve from time 0 to time the last quantifiable concentration (Day 7)

Time frame:: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
Reported as:: Mean · h*ng/mL

MAD: AUC0-t Day 7
h*ng/mL	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID
MAD: AUC0-t Day 7	1100 ± 481	993 ± 250

SecondaryMAD: AUC0-tau Day 1

Area under the plasma concentration-time curve over the dosing interval at steady-state, calculated from the dosing interval (Day 1)

Time frame:: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
Reported as:: Mean · h*ng/mL

MAD: AUC0-tau Day 1
h*ng/mL	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID
MAD: AUC0-tau Day 1	1630 ± 500	1680 ± 1810

SecondaryMAD: AUC0-tau Day 7

Area under the plasma concentration-time curve over the dosing interval at steady-state, calculated from the dosing interval (Day 7)

Time frame:: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
Reported as:: Mean · h*ng/mL

MAD: AUC0-tau Day 7
h*ng/mL	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID
MAD: AUC0-tau Day 7	1100 ± 480	992 ± 250

SecondaryMAD: t1/2 Day 1

Elimination half-life associated with the terminal slope (λ) of the semilogarithmic drug concentration-time curve, calculated as 0.693/λ (Day 1)

Time frame:: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
Reported as:: Mean · h

MAD: t1/2 Day 1
h	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID
MAD: t1/2 Day 1	1.8 ± 0.595	4.82 ± 2.94

SecondaryMAD: t1/2 Day 7

Elimination half-life associated with the terminal slope (λ) of the semilogarithmic drug concentration-time curve, calculated as 0.693/λ (Day 7)

Time frame:: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
Reported as:: Mean · h

MAD: t1/2 Day 7
h	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID
MAD: t1/2 Day 7	2.83 ± 1.33	2.16 ± 0.194

SecondaryMAD: CLss Day 1

Systemic Clearance at steady state (Day 1)

Time frame:: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
Reported as:: Mean · L/h

MAD: CLss Day 1
L/h	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID
MAD: CLss Day 1	677 ± 246	1200 ± 928

SecondaryMAD: CLss Day 7

Systemic Clearance at steady state (Day 7)

Time frame:: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
Reported as:: Mean · L/h

MAD: CLss Day 7
L/h	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID
MAD: CLss Day 7	1060 ± 410	1070 ± 287

SecondaryMAD: Vdss Day 1

Volume of distribution at steady state (Day 1)

Time frame:: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
Reported as:: Mean · L

MAD: Vdss Day 1
L	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID
MAD: Vdss Day 1	1920 ± 788	6200 ± 3750

SecondaryMAD: Vdss Day 7

Volume of distribution at steady state (Day 7)

Time frame:: BID: Samples on Days 1 and 7 before dosing and at 0.5, 1, 2, 4, 6, 8, 10, and 12 hours after dosing; Days 2, 4, 6, and 8 before dosing and before the 12 h. dose on Days 2, 4, and 6.TID: As BID but omitting samples at 10, and 12 hours after dosing.
Reported as:: Mean · L

MAD: Vdss Day 7
L	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID
MAD: Vdss Day 7	4080 ± 2010	3590 ± 874

Adverse events

Collected over SAD: Adverse Event Collected from Dosing to 7 days after dosing (8 days). MAD: Adverse Event Collected from Dosing on Day 1 to 7 days after final dosing day (15 days).. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
SAD Cohort 1: ANA001 1000 mg po	0/8 (0%)	0/8 (0%)	0/8 (0%)
SAD Cohort 2: ANA001 2000 mg po	0/8 (0%)	0/8 (0%)	0/8 (0%)
SAD Cohort 3: ANA001 3000 mg po	0/8 (0%)	0/8 (0%)	0/8 (0%)
SAD Cohorts 1, 2 & 3: Pooled Matching Placebo	0/6 (0%)	0/6 (0%)	1/6 (16.7%)
MAD Cohort 1: ANA001 1000 mg po BID	0/9 (0%)	0/9 (0%)	3/9 (33.3%)
MAD Cohort 2: ANA001 1000 mg po TID	0/9 (0%)	0/9 (0%)	3/9 (33.3%)
MAD Cohorts 1 & 2: Pooled Matching Placebo	0/6 (0%)	0/6 (0%)	1/6 (16.7%)

Most frequent other events

Most frequent other events
Event	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po	SAD Cohorts 1, 2 & 3: Pooled Matching Placebo	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID	MAD Cohorts 1 & 2: Pooled Matching Placebo
DizzinessNervous system disorders	—	—	—	1/6	0/9	1/9	0/6
Abdominal PainGastrointestinal disorders	—	—	—	0/6	0/9	0/9	1/6
AlopeciaSkin and subcutaneous tissue disorders	—	—	—	0/6	1/9	0/9	0/6
Rash maculo-papularSkin and subcutaneous tissue disorders	—	—	—	0/6	1/9	0/9	0/6
Headaches NECNervous system disorders	—	—	—	0/6	1/9	1/9	0/6
HaemorrhoidsGastrointestinal disorders	—	—	—	0/6	1/9	0/9	0/6
Skin LacerationInjury, poisoning and procedural complications	—	—	—	0/6	0/9	1/9	0/6
AcneRespiratory, thoracic and mediastinal disorders	—	—	—	0/6	0/9	1/9	0/6

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po	SAD Cohorts 1, 2 & 3: Pooled Matching Placebo	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID	MAD Cohorts 1 & 2: Pooled Matching Placebo	Total
<=18 years	0	0	0	0	0	0	0	0
Between 18 and 65 years	8	8	8	6	9	9	6	54
>=65 years	0	0	0	0	0	0	0	0

Age, Continuous
Age, Continuous(Years)	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po	SAD Cohorts 1, 2 & 3: Pooled Matching Placebo	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID	MAD Cohorts 1 & 2: Pooled Matching Placebo	Total
Mean	44.3 ± 13.9	41.6 ± 16.3	45.8 ± 11.6	47.2 ± 12.8	45.8 ± 13.3	41.8 ± 12.4	38.2 ± 6	43.6 ± 12.5

Sex: Female, Male
Sex: Female, Male(Participants)	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po	SAD Cohorts 1, 2 & 3: Pooled Matching Placebo	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID	MAD Cohorts 1 & 2: Pooled Matching Placebo	Total
Female	1	4	4	3	5	4	3	24
Male	7	4	4	3	4	5	3	30

Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po	SAD Cohorts 1, 2 & 3: Pooled Matching Placebo	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID	MAD Cohorts 1 & 2: Pooled Matching Placebo	Total
Hispanic or Latino	3	4	2	1	3	2	1	16
Not Hispanic or Latino	5	4	6	5	6	7	5	38
Unknown or Not Reported	0	0	0	0	0	0	0	0

Race (NIH/OMB)
Race (NIH/OMB)(Participants)	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po	SAD Cohorts 1, 2 & 3: Pooled Matching Placebo	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID	MAD Cohorts 1 & 2: Pooled Matching Placebo	Total
American Indian or Alaska Native	0	0	0	0	0	0	0	0
Asian	2	1	1	2	0	0	0	6
Native Hawaiian or Other Pacific Islander	0	0	0	0	0	0	0	0
Black or African American	0	1	1	0	3	3	3	11
White	5	6	6	3	6	5	1	32
More than one race	0	0	0	0	0	1	1	2
Unknown or Not Reported	1	0	0	1	0	0	1	3

Region of Enrollment
Region of Enrollment(participants)	SAD Cohort 1: ANA001 1000 mg po	SAD Cohort 2: ANA001 2000 mg po	SAD Cohort 3: ANA001 3000 mg po	SAD Cohorts 1, 2 & 3: Pooled Matching Placebo	MAD Cohort 1: ANA001 1000 mg po BID	MAD Cohort 2: ANA001 1000 mg po TID	MAD Cohorts 1 & 2: Pooled Matching Placebo	Total
United States	8	8	8	6	9	9	6	54

Study locations

1 site

WCCT Global Inc.
Cypress, California 90630, United States

References and documents

Study documents

Study protocol · Jan 9, 2021
Statistical analysis plan · Oct 22, 2020

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Undecided

Timeline

Tracking since Sep 25, 2026

ElapsedEstimatedEstimated, not yet reached

Ran from Nov 2020 to primary completion in Oct 2021, 10 months.

Results were first posted in Feb 2025, 3 years 3 months after primary completion.

How these figures are worked out, and which studies are left out of them.

Registered: Dec 8, 2020
First posted: Jan 12, 2021
StartActual: Nov 17, 2020
Primary completionActual: Oct 13, 2021
CompletionActual: Oct 28, 2022
Results first posted: Feb 3, 2025
Last update: Feb 3, 2025

What these dates mean

Start.: When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.: When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.: When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.: Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.

No changes since tracking began. The registry record was last updated on Feb 3, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT04705415
Lead sponsor: NeuroBo Pharmaceuticals Inc.
Responsible party: Sponsor
First posted: Jan 12, 2021
Start date: Nov 17, 2020
Primary completion: Oct 13, 2021
Completion: Oct 28, 2022
Results posted: Feb 3, 2025
Last update: Feb 3, 2025

Study contacts

Doug Rank, MD

study director · NeuroBo Pharmaceuticals Inc.

Oversight

Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Jan 2025. You cannot join it, but the record below documents what was studied.

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Discussion

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Studies of the drug tested here: Niclosamide

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NeuroBo Pharmaceuticals Inc.

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