Ended Therapeutic exploratory (Phase II) Cystic Fibrosis

A Phase 2, Open-label, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability and Efficacy of ARCT-032 in People with Cystic Fibrosis

EU CTIS ID: 2024-517663-23-01

What this study is testing

To assess the safety and tolerability of ARCT-032 in people with CF.

Therapeutic exploratory (Phase II)

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

1. Able to comprehend and willing to voluntarily sign and date an informed consent form (ICF) approved by an independent ethics committee (EC)/institutional review board (IRB) before initiation of any screening or study-specific procedures; willing and able to comply with study procedures (including ability to perform acceptable and reproducible/repeatable spirometry) and complete all study visits.
10. Confirm that the subject had a standard CF clinic visit with routine clinical screening exams/tests (including sputum culture) according to the latest clinical guidelines (Goetz 2024) within 3 months of the Screening visit. If the subject has not performed the routine screening, this shall be completed during Screening and prior to dosing Day 1.
7. Screening test results must fall within these limits: a. Liver chemistries (aspartate aminotransferase [AST], alanine aminotransferase [ALT], alkaline phosphatase) <3 × the upper limit of normal (ULN), and total bilirubin <1.5 × ULN. b. Estimated glomerular filtration rate (eGFR) >45 L/min/1.73m2 calculated by Modification to Diet in Renal Disease [MDRD] study equation. c. Hemoglobin ≥10 g/dL. d. Absence of clinically significant abnormality on ECG regarding rate, rhythm, or conduction.
8. Females must be non-pregnant and non-lactating and if a woman of child-bearing potential (WOCBP) engaged in heterosexual relations, agree to use a highly effective contraceptive method from the time of signing the ICF until at least 30 days after the last dose of study drug. Females who are not WOCBP do not require contraception.
2. Adult male or female, at least 18 years old on the day of signing the ICF.
3. Confirmed diagnosis of CF documented in subject’s medical record or patient registry (2 CF-causing mutations and/or sweat chloride value ≥60 mmol/L with history of chronic sinopulmonary disease).

You likely can't join if

1. Presence of any co-morbidities or medical conditions that, in the opinion of the investigator, might pose an additional risk by administering study drug to the participant or may confound the results of the study. This includes but is not limited to the following: a. Uncontrolled asthma or allergic bronchopulmonary aspergillosis (ABPA) within a year of screening. b. Cirrhosis with portal hypertension and known esophageal varices. c. History of massive hemoptysis [CCI] within 3 months of screening. d. Major surgery within 3 months of screening. e. Prior pulmonary malignancy, any active malignancy, current chemotherapy, or any malignancy with a natural history or treatment that has the potential to interfere with safety or efficacy assessments. f. Solid organ or hematologic transplant. g. Known history of or positive test for human immunodeficiency virus (HIV) or chronic hepatitis B. History of hepatitis C is permitted if the subject was treated and there is documentation of a cure. h. History of known sensitivity to [CCI] or liposomal products. i. Must not require supplemental oxygen while awake, or >2 L per minute while sleeping.
2. History of drug or alcohol dependence within 6 months prior to Screening that may preclude adherence to the protocol, in the opinion of the investigator.
3. Smoking or vaping tobacco or cannabis products within [CCI] of Screening; must be willing to abstain throughout the study period"
4. Treatment with another investigational drug or biologic agent within 30 days of screening or 5 halflives of investigational drug, whichever is longer
5. Prior treatment with gene therapy for CF. Individual exceptions may be made in the case of investigational gene therapies that have only temporary benefit.
6. Chronic maintenance systemic corticosteroid use is permitted but doses must not exceed the equivalent of 15 mg oral prednisone daily or 30 mg every other day. No corticosteroid bursts for at least 2 months prior to [CCI].

See the full eligibility criteria

Who can join

1. Able to comprehend and willing to voluntarily sign and date an informed consent form (ICF) approved by an independent ethics committee (EC)/institutional review board (IRB) before initiation of any screening or study-specific procedures; willing and able to comply with study procedures (including ability to perform acceptable and reproducible/repeatable spirometry) and complete all study visits.
10. Confirm that the subject had a standard CF clinic visit with routine clinical screening exams/tests (including sputum culture) according to the latest clinical guidelines (Goetz 2024) within 3 months of the Screening visit. If the subject has not performed the routine screening, this shall be completed during Screening and prior to dosing Day 1.
7. Screening test results must fall within these limits: a. Liver chemistries (aspartate aminotransferase [AST], alanine aminotransferase [ALT], alkaline phosphatase) <3 × the upper limit of normal (ULN), and total bilirubin <1.5 × ULN. b. Estimated glomerular filtration rate (eGFR) >45 L/min/1.73m2 calculated by Modification to Diet in Renal Disease [MDRD] study equation. c. Hemoglobin ≥10 g/dL. d. Absence of clinically significant abnormality on ECG regarding rate, rhythm, or conduction.
8. Females must be non-pregnant and non-lactating and if a woman of child-bearing potential (WOCBP) engaged in heterosexual relations, agree to use a highly effective contraceptive method from the time of signing the ICF until at least 30 days after the last dose of study drug. Females who are not WOCBP do not require contraception.
2. Adult male or female, at least 18 years old on the day of signing the ICF.
3. Confirmed diagnosis of CF documented in subject’s medical record or patient registry (2 CF-causing mutations and/or sweat chloride value ≥60 mmol/L with history of chronic sinopulmonary disease).
4. Not eligible for CFTR modulator therapy (e.g., 2 null mutations) or not taking CFTR modulators for at least [CCI] days prior to [CCI] (e.g., due to drug intolerance, poor response, or lack of access to modulators).
5. Forced expiratory volume in 1 second (FEV1) at screening between 40% to 100% (inclusive) of predicted value for age, sex, and height (equations of the current Global Lung Function Initiative standards) at Screening.
6. Stable pulmonary status, specifically no acute upper or lower respiratory tract infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within [CCI] days of [CCI].
9. Males who are engaged in sexual relations with a nonpregnant WOCBP must agree to use condoms from Day 1 through 90 days after the last dose of ARCT-032, and have their female partner use a highly effective contraceptive method (see Section 4.1.2) from the signing of the ICF until at least 90 days after the last dose of study drug. Males who are incapable of fathering a child (documented bilateral absence of the vas deferens or bilateral vasectomy with confirmation of aspermia or bilateral orchiectomy) or their female partner will not be required to use birth control during the study. However, all males with partners who are pregnant must use condoms from [CCI] through [CCI] to ensure that the fetus is not exposed to the study drug.

What rules you out

1. Presence of any co-morbidities or medical conditions that, in the opinion of the investigator, might pose an additional risk by administering study drug to the participant or may confound the results of the study. This includes but is not limited to the following: a. Uncontrolled asthma or allergic bronchopulmonary aspergillosis (ABPA) within a year of screening. b. Cirrhosis with portal hypertension and known esophageal varices. c. History of massive hemoptysis [CCI] within 3 months of screening. d. Major surgery within 3 months of screening. e. Prior pulmonary malignancy, any active malignancy, current chemotherapy, or any malignancy with a natural history or treatment that has the potential to interfere with safety or efficacy assessments. f. Solid organ or hematologic transplant. g. Known history of or positive test for human immunodeficiency virus (HIV) or chronic hepatitis B. History of hepatitis C is permitted if the subject was treated and there is documentation of a cure. h. History of known sensitivity to [CCI] or liposomal products. i. Must not require supplemental oxygen while awake, or >2 L per minute while sleeping.
2. History of drug or alcohol dependence within 6 months prior to Screening that may preclude adherence to the protocol, in the opinion of the investigator.
3. Smoking or vaping tobacco or cannabis products within [CCI] of Screening; must be willing to abstain throughout the study period"
4. Treatment with another investigational drug or biologic agent within 30 days of screening or 5 halflives of investigational drug, whichever is longer
5. Prior treatment with gene therapy for CF. Individual exceptions may be made in the case of investigational gene therapies that have only temporary benefit.
6. Chronic maintenance systemic corticosteroid use is permitted but doses must not exceed the equivalent of 15 mg oral prednisone daily or 30 mg every other day. No corticosteroid bursts for at least 2 months prior to [CCI].
7. Unable to tolerate [CCI] (also known as [CCI]).

The study team makes the final eligibility decision.

Where it's taking place

United States
Turkey
Israel

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include United States; Turkey; Israel. Enter your location above to see the nearest site and check your eligibility.

Explore other conditions

Obesity Type 2 diabetes Type 1 diabetes Prediabetes Weight loss Metabolic syndrome High cholesterol High triglycerides High blood pressure PCOS Hypothyroidism Thyroid disease Gout Fatty liver disease (NAFLD/NASH)Heart failure Coronary artery disease

BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.