Safety, Tolerability, and Pharmacokinetics of RCS-21 in Healthy Volunteers.

NCT06752122 | Recruiting Phase 1

• 2 other identifiers: RNATICS-24012024-512351-21-00
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this clinical trial is to evaluate the safety and tolerability of RCS-21 in healthy volunteers. Participants will be asked to inhale a single dose of RCS-21 and their health status will be constantly monitored.

Conditions

Healthy Volunteers; Safety; Tolerability; Pharmacokinetics

Keywords

RCS-21; Phase I; Healthy volunteers; Inhalation

Outcome Measures

Primary Outcomes (1)

• SAD: Frequency and severity of AEs, frequency and severity of ARs.

  Through study completion, an average of 1 year

Secondary Outcomes (7)

• SAD: Cmax (maximum observed plasma concentration)

  up to 3 days after dosing

• SAD: tmax (time of Cmax after dosing)

  up to 3 days after dosing

• SAD: AUC0-t last (area under the time course of the plasma concentrations up to the last quantifiable plasma concentration)

  up to 3 days after dosing

• SAD: AUC0-24 (area under the time course of the plasma concentrations up to 24 h after dosing, i.e. over the course of the intended dosage interval for future repeated dosing)

  up to 24 h after dosing

• SAD: AUC0-inf (total area under the time course of the analyte in plasma concentrations extrapolated to infinity)

  up to 3 days after dosing

Study Arms (2)

Single ascending dose (SAD) - Placebo

PLACEBO COMPARATOR

In the single ascending dose (SAD) study, healthy volunteers receive a single inhaled dose of placebo, of the same volume as the active treatment.

Drug: Placebo (SAD)

Single ascending dose (SAD) - RCS-21

EXPERIMENTAL

In the single ascending dose (SAD) study, healthy volunteers will receive a single inhaled dose of 0.5 mg/participant in dose group (DG) I, 1.5 mg/participant in DG II, 4.5 mg/participant in DG III. In DG IV, the dose will be 0.5, 1.5 or 4.5 mg, depending on the safety and tolerability data obtained from DGs I-III.

Drug: RCS-21 (SAD)

Interventions

Placebo (SAD) DRUG

Inhalation of a single dose.

Also known as: 0.9% saline solution

Single ascending dose (SAD) - Placebo

RCS-21 (SAD) DRUG

Inhalation of a single dose.

Single ascending dose (SAD) - RCS-21

Eligibility Criteria

• Age: 18 Years - 64 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Able and willing to give written informed consent.
• Male or female aged 18 to 64 years (inclusive).
• Not pregnant, as confirmed by pregnancy test (see assess- ment schedule), and not breastfeeding. AND
• WOCBP must use one of the following highly effective methods of birth control that result in a low failure rate of less than 1% per year when used consistently and correctly
• according to recommendations by the European Heads of Medicines Agencies - from at least 14 days before the first administration of study medication until 30 days after the last administration of study medication:
• combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation:
• oral
• intravaginal
• transdermal
• progestogen-only hormonal contraception associated with inhibition of ovulation:
• oral
• injectable
• implantable
• intrauterine device (IUD)
• intrauterine hormone-releasing system (IUS)

You may not qualify if:

• Any clinically relevant abnormal findings in physical examination, clinical chemistry, hematology, urinalysis, vital signs, lung function or ECG at screening visit, which, in the opinion of the investigator, may either put the participant at risk because of participation in the study or may influence the results of the study, or the participant's ability to participate in the study.
• Past or present disease, which as judged by the investigator, may affect the outcome of this study. These diseases include, but are not limited to, cardiovascular disease, malignancy, he- patic disease (asymptomatic Gilbert syndrome is allowed), renal disease, hematological disease, neurological disease, endo- crine disease (stable and asymptomatic hypothyroidism with or without Hormone Replacement Therapy (HRT) is allowed) or pulmonary disease (including but not confined to chronic bronchitis, emphysema, tuberculosis, bronchiectasis or cystic fibrosis).
• Having received any vaccination within the last 2 weeks before the first screening visit.
• History or current evidence of clinically relevant allergies or idiosyncrasy to any drug or food.
• History of allergic reactions to any active or inactive component of the study medication (including medication for bronchoscopy, e.g. salbutamol, lidocaine, midazolam or propofol).
• Repeated measurement of systolic blood pressure outside the range of 90 to 140 mmHg, diastolic blood pressure outside the range of 50 to 90 mmHg, or pulse rate outside the range of 50 to 90 beats per minute (bpm).
• Proneness to orthostatic dysregulation, fainting, or blackouts.
• History or presence of any malignancy except for basalioma.
• Chronic or acute infections or history of an acute infection during the four weeks before the first screening visit.
• Positive results in any of the following virology tests: human im- munodeficiency virus (HIV) antibodies and antigen, Anti-hepati- tis B-core antibody (HBc-Ab), hepatitis B-surface antigen (HBs- Ag) and anti-hepatitis C virus antibody (HCV-Ab).
• Positive drug screen (amphetamines, barbiturates, benzodiaze- pines, cannabinoids, cocaine, methadone, methamphetamine, opiates, phencyclidine, or tricyclic antidepressants).
• History of previous administration of any registered or investiga- tional oligonucleotide-based drug.
• History or presence of alcohol or drug abuse.
• Use of any medication (including over-the-counter medication, herbal products) except allowed concomitant medication within 2 weeks (for biologics: 6 months) before administration of IMP or within \< 10 times the elimination half-life of the respective drug, or the duration of the pharmacodynamic effect, whatever is longer.
• Positive breath alcohol test.

Sponsors & Collaborators

• RNATICS GmbH: lead
• LungenClinic Grosshansdorf: collaborator

Study Sites (1)

Fraunhofer Institute for Toxicology and Experimental Medicine (ITEM)

Hanover, 30625, Germany

RECRUITING

Related Publications (1)

• Beck C, Ramanujam D, Vaccarello P, Widenmeyer F, Feuerherd M, Cheng CC, Bomhard A, Abikeeva T, Schadler J, Sperhake JP, Graw M, Safi S, Hoffmann H, Staab-Weijnitz CA, Rad R, Protzer U, Frischmuth T, Engelhardt S. Trimannose-coupled antimiR-21 for macrophage-targeted inhalation treatment of acute inflammatory lung damage. Nat Commun. 2023 Jul 28;14(1):4564. doi: 10.1038/s41467-023-40185-1.

  PMID: 37507393 BACKGROUND

MeSH Terms

Conditions

Respiratory Aspiration

Interventions

Sagittal Abdominal Diameter; Saline Solution

Condition Hierarchy (Ancestors)

Respiration Disorders; Respiratory Tract Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Body Size; Body Weights and Measures; Body Constitution; Physical Examination; Diagnostic Techniques and Procedures; Diagnosis; Anthropometry; Investigative Techniques; Physiological Phenomena; Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Central Study Contacts

Christina Beck, Dr.

CONTACT

+49 89 200006040; amir-21@rnatics.com

Thomas Frischmuth, Dr.

CONTACT

+49 89 200006040; amir-21@rnatics.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: A double blind, randomized, placebo controlled phase 1 study

Study Record Dates

• First Submitted: December 19, 2024
• First Posted: December 30, 2024
• Study Start: February 18, 2025
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): August 1, 2026
• Last Updated: November 20, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will share
• Time Frame: The data will be available for 5 years after publication.
• Access Criteria: Interested researchers must submit a methodologically sound proposal to amir-21@rnatics.com. Proposals will be reviewed by the sponsor to ensure that they comply with confidentiality obligations. Data will be made available within 3 months of approval of the proposal in a deidentified format in accordance with applicable privacy laws, data protection standards and consent requirements. Data requestors will need to sign a data access agreement and data will be made available through a dedicated platform.

Locations

DE (1)