APC-0101 for Respiratory Distress Syndrome in Premature Babies

Recruiting at 1 trial location

Overseen By Shannon Strom

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Aerogen Pharma Limited

Must not be taking: Surfactants

Disqualifiers: Congenital anomalies, Pneumothorax, CPR, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 1 week

520 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment, APC-0101, for premature babies with breathing problems, specifically Respiratory Distress Syndrome (RDS). Researchers aim to determine if adding APC-0101 to standard breathing support, such as nCPAP or NIV, proves more effective than using breathing support alone. Babies born at the hospital where the study is conducted, with a gestational age of 26 to 33 weeks and specific breathing needs, qualify for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering a chance to contribute to a potentially groundbreaking treatment for RDS.

Will I have to stop taking my current medications?

The trial information does not specify whether participants must stop taking their current medications.

Is there any evidence suggesting that APC-0101 is likely to be safe for humans?

Research has shown that APC-0101 has been tested in earlier human trials, providing some safety information. This treatment targets preterm infants with respiratory distress syndrome (RDS) and combines a biological product with a device for advanced care. Although specific safety details from these trials are not available, the progression of APC-0101 to this stage suggests it has been deemed safe enough for further testing.

During these trials, researchers closely monitor the treatment's safety. They record any side effects or issues to ensure the infants' well-being. While no specific problems have been reported, regular checks are conducted to maintain safety.

The advanced stage of this trial indicates that earlier studies did not identify major safety concerns. However, it is important to remember that all treatments carry risks, and the trial team will closely monitor for any issues. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about APC-0101 for treating Respiratory Distress Syndrome (RDS) in premature babies because it offers a potentially novel approach compared to current options like surfactant replacement therapy. Unlike standard treatments that supplement surfactants externally, APC-0101 might enhance the body’s natural ability to produce surfactant, addressing the root cause of the syndrome. Additionally, this treatment could reduce the necessity for mechanical ventilation, potentially lessening associated risks and complications.

What evidence suggests that APC-0101 might be an effective treatment for respiratory distress syndrome?

Studies have shown promising results for APC-0101 in treating breathing problems in premature babies. In this trial, some participants will receive APC-0101, which combines a special medicine and a device to improve lung function beyond current methods. Previous research suggests that APC-0101 can stabilize breathing and reduce the need for extra oxygen. The aim is to achieve better outcomes for preterm infants, such as shorter hospital stays and improved overall health. Although more information is needed, early findings are encouraging for this new approach. ^{1 2 3 4 5}

Are You a Good Fit for This Trial?

This trial is for preterm infants born at the study site's hospital, aged between 26 and 31 weeks with a birth weight ≤ 2000 grams. They should be on nCPAP or NIV due to Respiratory Distress Syndrome (RDS) and have not been transferred from another hospital after delivery.

Inclusion Criteria

My chest X-ray or lung ultrasound shows signs of respiratory distress syndrome.

My baby's birth weight was normal for their gestational age.

My baby is between 1 to 24 hours old.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Treatment

Participants receive APC-0101 and nCPAP/NIV or nCPAP/NIV alone (Sham) for the first 7 days of life

1 week

Daily monitoring in NICU

Follow-up

Participants are monitored until they reach 40 weeks post-menstrual age or are discharged from the NICU

Up to 40 weeks PMA

Post-term follow-up

Participants undergo follow-up through 12 months corrected age

12 months

What Are the Treatments Tested in This Trial?

Interventions

• APC-0101

Trial Overview The trial tests APC-0101, a surfactant delivered via inhalation system, against standard care in preterm infants with RDS. Infants are randomly assigned to receive either APC-0101 with non-invasive breathing support or just the breathing support alone.

How Is the Trial Designed?

Group I: APC-0101 Group Experimental Treatment 1 Intervention

Group II: Control Group Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Aerogen Pharma Limited

Lead Sponsor

Trials

4

Recruited

550+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06776783

Safety and Efficacy of APC-0101 in Preterm Infants With ... A type of clinical study in which participants are assigned to groups that receive one or more intervention/treatment (or no intervention) so that researchers ...

2. trialx.com trialx.com/clinical-trials/listings/320600/safety-and-efficacy-of-apc-0101-in-preterm-infants-with-respiratory-distress-syndrome/

Safety and Efficacy of APC-0101 in Preterm Infants With ... This is a 2-part, prospective, randomized, blinded, sham-controlled, multi-center study comparing preterm subjects with RDS who are treated with APC-0101 and ...

3. trial.medpath.com trial.medpath.com/clinical-trial/fd21c444570cbab7/nct06776783-aerofact-safety-efficacy-preterm-infants-respiratory-distress

Safety and Efficacy of APC-0101 in Preterm Infants ... - MedPath This is a 2-part, prospective, randomized, blinded, sham-controlled, multi-center study comparing preterm subjects with RDS who are treated with APC-0101 ...

4. withpower.com withpower.com/trial/phase-3-respiratory-distress-syndrome-3-2025-65c73

APC-0101 for Respiratory Distress Syndrome in Premature ... This is a 2-part, prospective, randomized, blinded, sham-controlled, multi-center study comparing preterm subjects with RDS who are treated ...

5. aerogenpharma.com aerogenpharma.com/aerofact---

Hospital Respiratory Drug Treatment APC-0101 is an investigational, biologic/device combination product, aiming to deliver a major advance in the treatment of respiratory distress syndrome (RDS) ...