Cystic FibrosisPhase 1/2NCT03669614
SAD and MAD of Inhaled AR-501 in Health Adults and P. Aeruginosa Infected Cystic Fibrosis Subjects
A Phase 1/2 study testing Inhaled AR-501 in people with Cystic Fibrosis, run by Aridis Pharmaceuticals, Inc. at 25 sites in United States.
Trial summary
This is a Phase 1/2a randomized, double-blind, two-part, dose-ascending, multicenter study of AR-501 (gallium citrate) solution, administered via inhalation, in healthy adult and P. aeruginosa infected cystic fibrosis (CF) subjects. Read more about this study
- Who is it for?
- Ages 18 to 49 yearsHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Inhaled AR-501
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 102 participants
- Where
- 25 sitesUnited States
- Study dates
- 7 Dec 2018to Jul 2023
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 49 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Written informed consent given by the subject.
- At least ≥ 18 years old and < 50 years of age.
- Healthy with no acute medical condition for ≥ 2 weeks prior to screening and no known chronic medical condition requiring regular medical follow up and care.
- Body mass index (BMI) between 18 and 30 kg/m2, inclusive.
- Currently nonsmoking and no history of using nicotine/tobacco-containing products for ≥ 5 years prior to screening.
- Normal chest X-ray, per opinion of the Investigator.
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- FEV1 ≥ 80% of predicted values.
- No history or current illicit, pharmaceutical drug or alcohol abuse within ≤ 5 years prior to screening.
- A female subject must meet one of the following criteria:
- If of childbearing potential - agrees to use one of the accepted contraceptive regimens from at least 30 days prior to the first administration of the study medication, during the study, and for at least 90 days after the last dose of the study medication. An acceptable method of contraception includes one of the following:
- Abstinence from heterosexual intercourse
- Hormonal contraceptives (birth control pills, injectable/implant/insertable hormonal birth control products, transdermal patch)
- Intrauterine device (with or without hormones), OR
- Agrees to use a double barrier method (e.g., condom and spermicide) during the study and for at least 90 days after the last dose of the study medication
- If a female of non-childbearing potential, the subject should be surgically sterile (i.e., has undergone complete hysterectomy, bilateral oophorectomy, or tubal ligation) or in a menopausal state (at least 1 year without menses), as confirmed by follicle stimulating hormone (FSH) levels.
- A male subject must agree to use a double barrier method (e.g., condom and spermicide) during the study and for at least 90 days after the last dose of the study medication.
- Written informed consent given by the subject.
- At least18 years old
- Documentation of a CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:
- Sweat chloride equal to or greater than 60 mmol/liter by quantitative pilocarpine iontophoresis test (QPIT)
- Two well-characterized, disease-causing mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
- Confirmation of current colonization/infection with P. aeruginosa defined as: a positive sputum or oropharyngeal swab culture at screening.
- Respiratory symptoms and CF status are stable with no acute exacerbation at the time of randomization.
- BMI ≥ 18 kg/m2
- Currently non-smoking and no history of using nicotine/tobacco-containing products or smoking/vaping (inhaled tobacco products or other inhaled substances) for ≥ 1 year prior to screening.
- FEV1 ≥ 45% of predicted values.
- Serum creatinine and total bilirubin are both < 1.5 x upper limit of normal (ULN) range (isolated bilirubin > 1.5 x ULN range is acceptable if bilirubin is fractionated and direct bilirubin is < 35%).
- A female subject must meet one of the following criteria:
- If of childbearing potential - agrees to use one of the accepted contraceptive regimens from at least 21 days prior to the first administration of the study medication, during the study, and for at least 28 days after the last dose of the study medication. An acceptable method of contraception includes one of the following:
- Abstinence from heterosexual intercourse
- Hormonal contraceptives (birth control pills, injectable/implant/insertable hormonal birth control products, transdermal patch)
- Intrauterine device (with or without hormones), OR
- Agrees to use a double barrier method (e.g., condom and spermicide) during the study and for at least 28 days after the last dose of the study medication
- If a female of non-childbearing potential, the subject should be surgically sterile (i.e., has undergone complete hysterectomy, bilateral oophorectomy, or tubal ligation) or in a menopausal state (at least 1 year without menses), as confirmed by FSH levels.
- A male subject must agree to use a double barrier method (e.g., condom and spermicide) during the study and for at least 28 days after the last dose of the study medication.
- Ability to produce an oropharyngeal sample (e.g., Expectorated Sputum or throat swab).
Who cannot join
- None of the following criteria can be met.
- Female subjects who are currently pregnant or lactating.
- Oral temperature above 37.5ºC at the time of screening or prior to randomization.
- Clinically abnormal renal function, evidenced by serum creatinine > 1.5 mg/dL.
- Need for using any nephrotoxic agents during the study.
- Known allergy or hypersensitivity to albuterol.
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- Significantly abnormal liver function:
- Total bilirubin >1.5 x upper limit of the normal range (ULN),
- Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) > 3 x ULN and alkaline phosphatase (ALP) > 2 x ULN
- Hemoglobin <10 g/dL
- Abnormal corrected serum calcium concentration prior to enrollment.
- History or current use of illicit, pharmaceutical drug or alcohol abuse within 5 years prior to screening.
- Positive urine screen for alcohol, cotinine and/or drugs of abuse at screening and admission.
- Positive test results for Human Immunodeficiency Virus (HIV)-1/HIV-2 antibodies, Hepatitis B surface antigen (HBsAg) or Hepatitis C virus antibody (HCVAb).
- Inability to comply with any study requirements based on judgement of the Investigator.
- Any medical, psychological, cognitive, social or legal conditions that would interfere in the ability to give an informed consent and/or participate fully in the study.
- Participation in another clinical trial involving receipt of an investigative product within 30 days before screening.
- Any other reason as determined by an Investigator.
- None of the following criteria can be met.
- Female subjects who are currently pregnant or lactating.
- Oral temperature above 37.5ºC (or temporal temperature above 38.0ºC) at the time of screening or prior to randomization.
- Serum creatinine > 1.5 mg/dL or known significant kidney disease.
- Significantly abnormal liver function:
- Total bilirubin > 1.5 x ULN range,
- ALT and/or AST > 3 x ULN range and ALP > 2 x ULN range.
- History of medically attended hemoptysis < 1 year (small amount of blood streaking in sputum is acceptable).
- Pulmonary exacerbations within 3 months prior to randomization (defined as requiring IV antibiotics), in the hospital or at home.
- Hemoglobin < 10 g/dL.
- Abnormal corrected serum calcium concentration prior to randomization (normal range is typically 8.5-10.2 mg/dL).
- Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or re-initiation) in a chronic treatment/prophylaxis regimen for CF or CF-related conditions within 2 weeks prior to randomization.
- Known allergy or hypersensitivity to albuterol or any component of the study drug or placebo
- Use of illicit, pharmaceutical drug or alcohol abuse within 6 months prior to screening.
- Positive urine screen for alcohol, cotinine and/or drugs of abuse at screening (positive results for a subject on Orkambi should have a confirmatory test for cannabinoids performed, e.g., reflex testing, to rule out cross reaction with Orkambi).
- History of positive test result for human immunodeficiency virus (HIV) HIV-1/HIV-2 antibodies, HBsAg or chronic hepatitis C virus infection.
- Inability to comply with any study requirements based on judgement of the Investigator.
- Any medical, psychological, cognitive, social or legal conditions that would interfere in the ability to give an informed consent and/or participate fully in the study.
- Participation in another clinical trial involving receipt of an investigative product within 30 days before randomization.
- Suspected or confirmed acute respiratory infection (Examples: bacterial pneumonia, influenza, COVID-19).
- Any other reason as determined by an Investigator.
Trial locations and contacts
25 sites in 1 country.
25 sites in 1 country
United States25 sites, 24 recruiting
- Recruiting
Research Site
Baltimore, Maryland, 21204
- Recruiting
Research Site
Chicago, Illinois, 60093
- Recruiting
Research Site
Cleveland, Ohio, 44106
- Recruiting
Research Site
Columbus, Ohio, 43205
- Recruiting
Research Site
Denver, Colorado, 80206
- Recruiting
Research Site
Detroit, Michigan, 48201
- Recruiting
Research Site
Gainesville, Florida, 32610
- Recruiting
Research Site
Hollywood, Florida, 33021
- Recruiting
Research Site
Iowa City, Iowa, 55242
- Recruiting
Research Site
Long Beach, California, 90806
- Recruiting
Research Site
Louisville, Kentucky, 40202
- Recruiting
Research Site
Miami, Florida, 33136
- Recruiting
Research Site
New York, New York, 10532
- Recruiting
Research Site
Oklahoma City, Oklahoma, 73104
- Recruiting
Research Site
Omaha, Nebraska, 68198
- Recruiting
Research Site
Orlando, Florida, 32803
- Completed
Research Site
Overland Park, Kansas, 66212
- Recruiting
Research Site
Philadelphia, Pennsylvania, 19104
- Recruiting
Research Site
Portland, Maine, 04102
- Recruiting
Research Site
Salt Lake City, Utah, 84132
- Recruiting
Research Site
Seattle, Washington, 98195
- Recruiting
Research Site
Spokane, Washington, 99204
- Recruiting
Research Site
Tampa, Florida, 33606
- Recruiting
Research Site
Tucson, Arizona, 85724
- Recruiting
Research Site
Tyler, Texas, 75708
About this study
Official title: A Phase 1/2a Randomized, Double-Blind, Two-Part, Dose-Ascending, Multicenter Study of the Safety and PK of AR-501 (Gallium Citrate), Administered Via Inhalation, in Healthy Adult and P. Aeruginosa Infected Cystic Fibrosis Subjects
This is a Phase 1/2a randomized, double-blind, two-part, dose-ascending, multicenter study of AR-501 (gallium citrate) solution, administered via inhalation, in healthy adult and P. aeruginosa infected cystic fibrosis (CF) subjects. Phase 1 of the study in HV subjects will consist of a single-ascending-dose (SAD) cohort, followed by the HV multiple-ascending-dose (MAD) cohort. Phase 2a of the study in CF subjects will consist of a MAD study design. The study will evaluate the safety and pharmacokinetic (PK) profile of single and repeat administrations of inhaled AR-501 solution in healthy adults, and the safety, PK and efficacy of repeat administrations of inhaled AR-501 solution in P. aeruginosa infected CF subjects.
Read the detailed description
Three dose levels (low, medium and high) will be assessed in succession, first in healthy volunteer (HV) subjects, then four ascending doses will be assessed in cystic fibrosis (CF) subjects. The study will be performed in 2 parts: Phase 1 part of the study in HV subjects will consist of a single-ascending-dose (SAD) cohort, followed by the HV multiple-ascending-dose (MAD) cohort. Phase 2a part of the study in CF subjects will consist of a MAD study design.
The HV cohort will include up to 48 subjects. The CF cohort will have 54 subjects. Thus, the total number of subjects is 102.
The Phase 1 HV study will be performed at a Phase 1 Clinical Study Unit and the Phase 2a will be performed at approximately 24 clinical trial sites located in the United States and possibly in Europe, some of which may be part of the Cystic Fibrosis Foundation (CFF)-accredited Therapeutic Development Network (TDN) or the European Cystic Fibrosis Society Clinical Trial Network (ECFS-CTN). Subjects who meet all eligibility criteria, including giving informed consent, will be enrolled and undergo a screening period of 28 days for HV cohorts and 42 days for CF Cohorts.
The HV cohort will include up to 24 adult subjects in 3 dose groups (8 per dose group [Low, Medium and High]) for the SAD phase of the study. In each dose group, subjects will be randomly assigned in a 3:1 ratio to the active drug or placebo, resulting in 6 subjects receiving inhaled AR-501 and 2 receiving inhaled placebo in a double-blind manner. The HV MAD phase of the study will include 24 adult subjects in 3 dose groups (8 per dose group [Low, Medium and High]). In each dose group, subjects will be randomly assigned in a 3:1 ratio to active study drug or placebo, resulting in 6 subjects receiving inhaled AR-501 and 2 receiving inhaled placebo in a double-blind manner. All subjects in HV MAD cohorts will receive once weekly inhaled study drug (either AR-501 or matching placebo) for 4 weeks for a total of 5 doses.
The CF MAD cohort will evaluate 4 different dose levels for a total of 54 adult CF. Of the 54 subjects, 40 will be randomized to receive one of the three ascending doses of AR-501, while 14 will be randomized to receive placebo. All subjects in CF cohorts will receive once weekly inhaled study drug (either AR-501 or matching placebo) for 2 weeks for a total of 3 doses.
The primary purpose of this study is to assess preliminary clinical safety of inhaled AR-501. The true frequency of AEs is unknown, and the sample size cannot be estimated accurately. For unknown AEs, the probabilities of observing at least 1 AE among 36 total healthy adult subjects and 40 adult CF subjects receiving any AR-501 dose are ≥ 80% if the true rate of such events are at least 4.4% and 4.8% respectively.
- Conditions
- Cystic Fibrosis
- Being tested
- Inhaled AR-501, Inhaled Placebo
- Keywords
- Cystic Fibrosis, Gallium Citrate, Pseudomonas Aeruginosa
- Medical subject headings
- Cystic Fibrosis, Pseudomonas Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Inhaled AR-501Drug
Also called Inhaled Gallium Citrate
single and multiple ascending doses of inhaled AR-501
Inhaled PlaceboDrug
Also called Control (inhaled placebo)
single and multiple ascending doses of inhaled placebo
| Study group | Role | What they receive |
|---|---|---|
| AR-501 inhaled | Experimental | Four doses (low, medium, high, top) of inhaled AR-501 will be used. Receives: Drug: Inhaled AR-501 |
| inhaled AR-501 Placebo | Placebo comparator | Four doses (low, medium, high, top) of inhaled placebo will be used Receives: Drug: Inhaled Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical safety profile (adverse events) - Single Ascending Dose
Evaluation of adverse events in HV subjects
Measured: 28 days following dose administration
Clinical safety profile (adverse events) - Multiple Ascending Dose
Evaluation of adverse events in HV and CF subjects
Measured: up to 28 days after last dose administration
Secondary outcomes (14)
Pharmacokinetics (PK) Profile - SAD Cmax
Pharmacokinetics Characteristics in Single Ascending Dose HV Subjects: Observed maximum concentration (Cmax)
Measured: 28 days following dose administration
Pharmacokinetics (PK) Profile - SAD Tmax
Pharmacokinetics Characteristics in Single Ascending Dose HV Subjects: time to reach maximum concentration (Tmax)
Measured: 28 days following dose administration
Pharmacokinetics (PK) Profile - SAD AUC0-inf
Pharmacokinetics Characteristics in Single Ascending Dose HV Subjects: area under the time-concentration curve (AUC) from time zero to infinity (AUC0-inf)
Measured: 28 days following dose administration
Pharmacokinetics (PK) Profile - SAD AUC0-last
Pharmacokinetics Characteristics in Single Ascending Dose HV Subjects: area under the time-concentration curve (AUC) from time zero to the last dose (AUC0-last)
Measured: 28 days following dose administration
Pharmacokinetics (PK) Profile - SAD λz
Pharmacokinetics Characteristics in Single Ascending Dose HV Subjects: terminal elimination rate (λz)
Measured: 28 days following dose administration
Pharmacokinetics (PK) Profile - SAD t½
Pharmacokinetics Characteristics in Single Ascending Dose HV Subjects: half-life (t½)
Measured: 28 days following dose administration
Pharmacokinetics (PK) Profile - SAD Clp
Pharmacokinetics Characteristics in Single Ascending Dose HV Subjects: plasma clearance (Clp)
Measured: 28 days following dose administration
Pharmacokinetics (PK) Profile - MAD Cmax
Pharmacokinetics Characteristics in Multiple Ascending Dose CF Subjects: Observed maximum concentration (Cmax)
Measured: up to 28 days after last dose administration
Pharmacokinetics (PK) Profile - MAD Tmax
Pharmacokinetics Characteristics in Multiple Ascending Dose CF Subjects: time to reach maximum concentration (Tmax)
Measured: up to 28 days after last dose administration
Pharmacokinetics (PK) Profile - MAD AUC0-inf
Pharmacokinetics Characteristics in Multiple Ascending Dose CF Subjects: area under the time-concentration curve (AUC) from time zero to infinity (AUC0-inf)
Measured: up to 28 days after last dose administration
Pharmacokinetics (PK) Profile - MAD AUC0-last
Pharmacokinetics Characteristics in Multiple Ascending Dose CF Subjects: area under the time-concentration curve (AUC) from time zero to the last dose (AUC0-last)
Measured: up to 28 days after last dose administration
Pharmacokinetics (PK) Profile - MAD λz
Pharmacokinetics Characteristics in Multiple Ascending Dose CF Subjects: terminal elimination rate (λz)
Measured: up to 28 days after last dose administration
Pharmacokinetics (PK) Profile - MAD t½
Pharmacokinetics Characteristics in Multiple Ascending Dose CF Subjects: half-life (t½)
Measured: up to 28 days after last dose administration
Pharmacokinetics (PK) Profile - MAD Clp
Pharmacokinetics Characteristics in Multiple Ascending Dose CF Subjects: plasma clearance (Clp)
Measured: up to 28 days after last dose administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Sep 2018
- Study start7 Dec 2018
- Primary completionJul 2023 (estimated)
- Study completionJul 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This study is a dose-escalation, placebo-controlled study where subjects are randomized to receive study drug or placebo. SAD and MAD HV Phases: The HV SAD and MAD will each include up to 24 adult subjects in 3 groups (8 per dose group) in each the SAD and MAD phase . All subjects will be randomly assigned in a 3:1 ratio to receive a single administration of active drug (12 HVs) or placebo (4 HVs). The CF MAD will evaluate 4 different dose levels in a total of 54 adult CF subjects (10 per dose level). The first 3 subjects (sentinel subjects) in the low, medium and high dose level will be randomly assigned at a 2:1 ratio to receive either AR-501 or placebo, for a total of 9 CF subjects. An expanded cohort will randomize 30 adult subjects at a 2:3:3:2 ratio to three doses of inhaled AR-501 or placebo. Subsequently an 80mg Ga dose (top dose) cohort will randomize subjects at a 2:1 ratio to receive either AR 501 or placebo for a total of 15 subjects , including 6 sentinel subjects.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 102 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Aridis Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Hasan S Jafri, MD, FAAP, Study director, Aridis Pharmaceuticals, Inc.Alan H Cohen, MD, Study director, Aridis Pharmaceuticals, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03669614
- Sponsor's protocol ID
- AR-501-001
- Status last verified
- Mar 2023
- First submitted
- 30 Aug 2018
- Last updated
- 9 Mar 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: The Sponsor has not assessed whether to make individual participant data (IPD) available to other researchers.
Study at a glance
From the registry record, last updated by the sponsor on 9 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2a Randomized, Double-Blind, Two-Part, Dose-Ascending, Multicenter Study of the Safety and PK of AR-501 (Gallium Citrate), Administered Via Inhalation, in Healthy Adult and P. Aeruginosa Infected Cystic Fibrosis Subjects
- ClinicalTrials.gov ID
- NCT03669614
- Acronym / sponsor ID
- AR-501-001
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, Gallium Citrate, Pseudomonas Aeruginosa
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 102 participants (estimated)
- Lead sponsor
- Aridis Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 7 Dec 2018 (actual)
- Primary completion
- Jul 2023 (estimated)
- Study completion
- Jul 2023 (estimated)
- First posted
- 13 Sep 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase 1/2a randomized, double-blind, two-part, dose-ascending, multicenter study of AR-501 (gallium citrate) solution, administered via inhalation, in healthy adult and P. aeruginosa infected cystic fibrosis (CF) subjects. Phase 1 of the study in HV subjects will consist of a single-ascending-dose (SAD) cohort, followed by the HV multiple-ascending-dose (MAD) cohort. Phase 2a of the study in CF subjects will consist of a MAD study design. The study will evaluate the safety and pharmacokinetic (PK) profile of single and repeat administrations of inhaled AR-501 solution in healthy adults, and the safety, PK and efficacy of repeat administrations of inhaled AR-501 solution in P. aeruginosa infected CF subjects.
Detailed description
Three dose levels (low, medium and high) will be assessed in succession, first in healthy volunteer (HV) subjects, then four ascending doses will be assessed in cystic fibrosis (CF) subjects. The study will be performed in 2 parts: Phase 1 part of the study in HV subjects will consist of a single-ascending-dose (SAD) cohort, followed by the HV multiple-ascending-dose (MAD) cohort. Phase 2a part of the study in CF subjects will consist of a MAD study design.
The HV cohort will include up to 48 subjects. The CF cohort will have 54 subjects. Thus, the total number of subjects is 102.
The Phase 1 HV study will be performed at a Phase 1 Clinical Study Unit and the Phase 2a will be performed at approximately 24 clinical trial sites located in the United States and possibly in Europe, some of which may be part of the Cystic Fibrosis Foundation (CFF)-accredited Therapeutic Development Network (TDN) or the European Cystic Fibrosis Society Clinical Trial Network (ECFS-CTN). Subjects who meet all eligibility criteria, including giving informed consent, will be enrolled and undergo a screening period of 28 days for HV cohorts and 42 days for CF Cohorts.
The HV cohort will include up to 24 adult subjects in 3 dose groups (8 per dose group [Low, Medium and High]) for the SAD phase of the study. In each dose group, subjects will be randomly assigned in a 3:1 ratio to the active drug or placebo, resulting in 6 subjects receiving inhaled AR-501 and 2 receiving inhaled placebo in a double-blind manner. The HV MAD phase of the study will include 24 adult subjects in 3 dose groups (8 per dose group [Low, Medium and High]). In each dose group, subjects will be randomly assigned in a 3:1 ratio to active study drug or placebo, resulting in 6 subjects receiving inhaled AR-501 and 2 receiving inhaled placebo in a double-blind manner. All subjects in HV MAD cohorts will receive once weekly inhaled study drug (either AR-501 or matching placebo) for 4 weeks for a total of 5 doses.
The CF MAD cohort will evaluate 4 different dose levels for a total of 54 adult CF. Of the 54 subjects, 40 will be randomized to receive one of the three ascending doses of AR-501, while 14 will be randomized to receive placebo. All subjects in CF cohorts will receive once weekly inhaled study drug (either AR-501 or matching placebo) for 2 weeks for a total of 3 doses.
The primary purpose of this study is to assess preliminary clinical safety of inhaled AR-501. The true frequency of AEs is unknown, and the sample size cannot be estimated accurately. For unknown AEs, the probabilities of observing at least 1 AE among 36 total healthy adult subjects and 40 adult CF subjects receiving any AR-501 dose are ≥ 80% if the true rate of such events are at least 4.4% and 4.8% respectively.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- This study is a dose-escalation, placebo-controlled study where subjects are randomized to receive study drug or placebo. SAD and MAD HV Phases: The HV SAD and MAD will each include up to 24 adult subjects in 3 groups (8 per dose group) in each the SAD and MAD phase . All subjects will be randomly assigned in a 3:1 ratio to receive a single administration of active drug (12 HVs) or placebo (4 HVs). The CF MAD will evaluate 4 different dose levels in a total of 54 adult CF subjects (10 per dose level). The first 3 subjects (sentinel subjects) in the low, medium and high dose level will be randomly assigned at a 2:1 ratio to receive either AR-501 or placebo, for a total of 9 CF subjects. An expanded cohort will randomize 30 adult subjects at a 2:3:3:2 ratio to three doses of inhaled AR-501 or placebo. Subsequently an 80mg Ga dose (top dose) cohort will randomize subjects at a 2:1 ratio to receive either AR 501 or placebo for a total of 15 subjects , including 6 sentinel subjects.
| Arm | Type | Receives |
|---|---|---|
| AR-501 inhaled | Experimental | Drug: Inhaled AR-501Four doses (low, medium, high, top) of inhaled AR-501 will be used. |
| inhaled AR-501 Placebo | Placebo comparator | Drug: Inhaled PlaceboFour doses (low, medium, high, top) of inhaled placebo will be used |
Who may qualify
- Ages
- 18 to 49 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (36 listed)
- Written informed consent given by the subject.
- At least ≥ 18 years old and < 50 years of age.
- Healthy with no acute medical condition for ≥ 2 weeks prior to screening and no known chronic medical condition requiring regular medical follow up and care.
- Body mass index (BMI) between 18 and 30 kg/m2, inclusive.
- Currently nonsmoking and no history of using nicotine/tobacco-containing products for ≥ 5 years prior to screening.
Key exclusion criteria (39 listed)
- None of the following criteria can be met.
- Female subjects who are currently pregnant or lactating.
- Oral temperature above 37.5ºC at the time of screening or prior to randomization.
- Clinically abnormal renal function, evidenced by serum creatinine > 1.5 mg/dL.
- Need for using any nephrotoxic agents during the study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Inhaled AR-501Also: Inhaled Gallium Citratesingle and multiple ascending doses of inhaled AR-501 | Drug | AR-501 inhaled |
| Inhaled PlaceboAlso: Control (inhaled placebo)single and multiple ascending doses of inhaled placebo | Drug | inhaled AR-501 Placebo |
Primary outcome
Clinical safety profile (adverse events) - Single Ascending Dose
Time frame: 28 days following dose administration
Evaluation of adverse events in HV subjects
Clinical safety profile (adverse events) - Multiple Ascending Dose
Time frame: up to 28 days after last dose administration
Evaluation of adverse events in HV and CF subjects
14 secondary outcomes
Pharmacokinetics (PK) Profile - SAD Cmax
Time frame: 28 days following dose administration
Pharmacokinetics (PK) Profile - SAD Tmax
Time frame: 28 days following dose administration
Pharmacokinetics (PK) Profile - SAD AUC0-inf
Time frame: 28 days following dose administration
Pharmacokinetics (PK) Profile - SAD AUC0-last
Time frame: 28 days following dose administration
Pharmacokinetics (PK) Profile - SAD λz
Time frame: 28 days following dose administration
Pharmacokinetics (PK) Profile - SAD t½
Time frame: 28 days following dose administration
Pharmacokinetics (PK) Profile - SAD Clp
Time frame: 28 days following dose administration
Pharmacokinetics (PK) Profile - MAD Cmax
Time frame: up to 28 days after last dose administration
Pharmacokinetics (PK) Profile - MAD Tmax
Time frame: up to 28 days after last dose administration
Pharmacokinetics (PK) Profile - MAD AUC0-inf
Time frame: up to 28 days after last dose administration
Pharmacokinetics (PK) Profile - MAD AUC0-last
Time frame: up to 28 days after last dose administration
Pharmacokinetics (PK) Profile - MAD λz
Time frame: up to 28 days after last dose administration
Pharmacokinetics (PK) Profile - MAD t½
Time frame: up to 28 days after last dose administration
Pharmacokinetics (PK) Profile - MAD Clp
Time frame: up to 28 days after last dose administration
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Mar 2023.
- Individual participant dataSharing planned: No. The Sponsor has not assessed whether to make individual participant data (IPD) available to other researchers.
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Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hasan S Jafri, MD, FAAP study director
Aridis Pharmaceuticals, Inc.
Alan H Cohen, MD study director
Aridis Pharmaceuticals, Inc.
25 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Inhaled AR-501 in people with Cystic Fibrosis, run by Aridis Pharmaceuticals, Inc. at 25 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 36 inclusion and 39 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 9 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Lynne M Deans, MT (4083851742, clinicaltrial@aridispharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinical safety profile (adverse events) - Single Ascending Dose (time frame: 28 days following dose administration). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Mar 2023.