Study of Safety and Efficacy of ALT-100mAb in Participants With Moderate/Severe ARDS

NCT05938036 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: ALT-100-002
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

A Phase 2a, multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy and safety of ALT-100mAb in patients with moderate to severe ARDS.

Conditions

Acute Respiratory Distress Syndrome (ARDS)

Outcome Measures

Primary Outcomes (1)

• To evaluate the Safety and tolerability of a single intravenously (IV) infused dose of ALT-100 in participants with moderate to severe ARDS: Incidence and severity of treatment-emergent adverse events (TEAEs) from Day 1 to end of study (EOS).

  Assessment will be done by measuring the following parameter: • Incidence and severity of treatment-emergent adverse events (TEAEs) from Day 1 to end of study (EOS).

  Up to 60 days

Secondary Outcomes (19)

• To evaluate the impact of a single IV infusion of ALT-100 on respiratory support.

  Up to 29 Days

• To assess the effect of ALT-100 on duration of hospitalization: Time to hospital discharge based on days since admission to discharge.

  Up to 29 Days

• To assess the effect of ALT-100 on duration of hospitalization: Hospital Free Days (HFD) to Day 29.

  Up to 29 Days

• To assess the effect of ALT-100mAb as measured by the Sequential Organ Failure Assessment (SOFA) score.

  Up to 29 Days

• To assess the effect of ALT-100 mAb on oxygen-related parameters :Changes as measured by change from baseline in plethysmographic pulse oximetry derived oxygen saturation / fraction of inspired oxygen and ROX Index

  Up to 29 Days

Other Outcomes (13)

• Exploratory: detection of NAMPT genetic variants in blood: Determination of ARDS-associated NAMPT promoter SNP expression in baseline blood samples from all participants using a NAMPT genotyping platform.

  Up to 60 days

• Exploratory: detection of NAMPT genetic variants in blood: Assessment of predictive capacity of NAMPT SNPs and plasma eNAMPT levels to identify participants who respond to single dose treatment with ALT-100 mAb.

  Up to 60 days

• Exploratory - To explore the effect of ALT-100 on other measurements of lung injury that may be performed during standard care: Change from baseline in LIS (if performed) daily while hospitalized.

  Up to 60 days

Study Arms (3)

Part A : ALT-100 mAB (Dose Escalation)

EXPERIMENTAL

90 eligible participants will be randomized at a 2:1 ratio to receive a single dose of ALT-100 mAb. Part A will assess 2 doses of ALT-100 mAb in sequentially enrolled cohorts of up to 9 participants in each cohort. (Randomised, Double-blind) Drug: ALT-100 mAb Dosage Form: Sterile liquid, pH 5.5, Dosage: 0.4 mg/kg (Cohort 1a) and 1.0 mg/kg (Cohort 2a) Dosage Form \& Route of Admin: Solution for IV Infusion

Drug: ALT-100 mAb

Part A Placebo

PLACEBO COMPARATOR

Part A participants with acute respiratory distress syndrome (ARDS) (Randomised, Double-blind) Dosage Form \& Route of Admin: Normal Saline Solution for IV Infusion

Drug: ALT-100 (Placebo)

Part B (Dose Expansion) ALT-100 mAb

EXPERIMENTAL

Approximately 9 participants in each cohort in Part A, additional participants (up to 36 per dose cohort) may be enrolled into 2 dose expansion cohorts, the dose of which will be determined by the SRC. Drug: AT-02 Dosage: Will be decided by the SCR Route of Admin: Solution for IV Infusion

Drug: ALT-100 mAb

Interventions

ALT-100 mAb DRUG

Experimental: Part A : ALT-100 mAB (Dose Escalation) 90 eligible participants will be randomized at a 2:1 ratio to receive a single dose of ALT-100 mAb.

Part A : ALT-100 mAB (Dose Escalation); Part B (Dose Expansion) ALT-100 mAb

ALT-100 (Placebo) DRUG

Normal saline solution via IV solution

Part A Placebo

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• To be eligible for this study, a participant must meet all of the following criteria:
• Hospitalized (or documentation of a plan to admit to the hospital if the patient is in an emergency department) male or non-pregnant female 18 years and above of age at time of enrollment.
• Participant (or LAR) is able and willing to provide written informed consent, which includes compliance with study requirements and restrictions listed in the consent form.
• Participant has a diagnosis of moderate or severe ARDS:
• A participant with a diagnosis of moderate or severe ARDS according to the Berlin definition of ARDS:
• Acute onset of respiratory failure within 1 week of a known clinical insult or new or worsening respiratory symptoms.
• Respiratory failure associated with known ARDS risk factors and not fully explained by either cardiac failure or fluid overload (an objective assessment of cardiac failure or fluid overload is needed if no risk factors for ARDS are present).
• Radiological abnormalities on chest x-ray or computed tomography (CT) scan, ie, bilateral opacities that are not fully explained by effusions, nodules, masses, or lobar/lung collapse.
• Hypoxemia:
• i. Moderate ARDS: PaO2/FiO2 more than 100 mmHg (more than 13.3 kPa) to 200 mmHg and below (26.6 kPa and below) with PEEP 5 cmH2O and above, or imputed SpO2/FiO2 equivalent.
• ii. Severe ARDS: PaO2/FiO2 100 mmHg and below (13.3 kPa and below) with PEEP 5 cmH2O and above.
• OR Participant presents with acute respiratory failure phenotypically similar to ARDS in a setting demonstrating clinical risk for ARDS, whether or not they meet the Berlin criteria, and requiring heated and humidified HFNC 30 L/min and above and 100 percent FiO2, or NIPPV (ie, BiPAP/CPAP) for hypoxemia.
• OR Participant presents with acute respiratory failure phenotypically similar to ARDS in a setting demonstrating clinical risk for ARDS, do not meet the Berlin criteria, and initially treated with 12 continuous hours and above with HFNO using gas flow of 40 L/min and above or treated with non-invasive ventilation (NIV), and has a PEEP of 5 cm and above H2O and PaO2/FIO2 below 300 mm Hg.
• Administration of study treatment must be planned to occur within 12 hours of the participant's moderate or severe ARDS diagnosis and within 4 hours of initiation of MV (in the case of individuals requiring immediate MV).
• Females must be non-pregnant and non-lactating, and either surgically sterile (eg, tubal occlusion, hysterectomy, bilateral salpingectomy, bilateral oophorectomy), or use highly effective contraceptive method (oral contraceptive pills \[OCPs\], long-acting implantable hormones, injectable hormones, a vaginal ring or an intrauterine device \[IUD\]) from screening until study completion or be post-menopausal for 12 months and above. Post-menopausal status will be confirmed through testing of follicle-stimulating hormone (FSH) levels at screening for amenorrheic female participants, but the result is not required prior to enrollment. Female participants whose only partner has had a vasectomy, and female participants who are abstinent from heterosexual intercourse as part of their usual lifestyle will also be eligible for participation.

You may not qualify if:

• A participant who meets any of the following criteria must be excluded from the study:
• Participants with ARDS consequent to COVID-19 infection.
• Participants requiring immediate MV who have been intubated and on MV for more than 4 hours prior to the planned administration of study treatment on Day 1
• Moribund participant not expected to survive more 24 hours, in the opinion of the Investigator.
• Use of extracorporeal life support (eg, ECMO) or, in the opinion of the Investigator, there is a high likelihood that extracorporeal life support will be initiated within 48 hours after randomization.
• Participant has an underlying clinical condition where, in the opinion of the Investigator, it would be unlikely that the participant would be able to come off ventilation, eg, chronic progressive neuromuscular or respiratory disease.
• Severe chronic respiratory disease (eg, known chronic obstructive pulmonary disease \[COPD\], pulmonary arterial hypertension \[PAH\], idiopathic pulmonary fibrosis \[IPF\], interstitial lung disease \[ILD\]) requiring supplemental oxygen therapy or MV pre-hospitalization (eg, prior to ARDS diagnosis).
• Evidence of life-threatening dysrhythmia (eg, ventricular tachycardia, ventricular fibrillation) or cardiac arrest on presentation.
• Evidence of new or preexisting decompensated heart failure.
• Absolute neutrophil count lesser than1000 per mm3.
• Platelet count less than 50000 per mm3.
• Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) more than 5 × ULN.
• Estimated glomerular filtration rate (eGFR) less than 30 mL/min/1.73m2 (based on MDRD equation) or requiring hemofiltration or dialysis.
• Known or suspected active and untreated tuberculosis (TB), HIV, hepatitis B or C infection.
• Note: Results of TB, hepatitis B and C, and HIV tests are not required prior to enrollment if there is no suspicion of active infection.

Sponsors & Collaborators

• Aqualung Therapeutics Corp.: lead

Study Sites (1)

Banner University of Arizona

Tucson, Arizona, 85721, United States

Location

MeSH Terms

Conditions

Respiratory Distress Syndrome

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Respiration Disorders

Study Officials

• Stan Miele

  Aqualung Therapeutics Corp.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 17, 2023
• First Posted: July 10, 2023
• Study Start: December 1, 2023
• Primary Completion (Estimated): May 1, 2027
• Study Completion (Estimated): July 1, 2027
• Last Updated: October 23, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)