Axatilimab for Pulmonary Fibrosis

Recruiting at 78 trial locations

Overseen By Syndax Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Syndax Pharmaceuticals

Must not be taking: Nintedanib, Pirfenidone, Corticosteroids, others

Disqualifiers: Emphysema, Connective tissue disease, others

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

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 13

Duration: 26 weeks

135 Participants Needed

Trial Summary

What is the purpose of this trial?

The study will evaluate the efficacy and safety of axatilimab in participants with IPF.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications, such as nintedanib with pirfenidone, systemic corticosteroids over 10 mg/day, and several other specific drugs, at least 4 weeks before screening. It's best to discuss your current medications with the trial team to see if any need to be stopped.

What makes the drug Axatilimab unique for treating pulmonary fibrosis?

Axatilimab is unique because it targets specific pathways involved in pulmonary fibrosis, potentially offering a new approach compared to existing treatments like bosentan and pirfenidone, which have shown limited effectiveness or are still under investigation for this condition. ^{1 2 3 4 5}

Eligibility Criteria

This trial is for people with Idiopathic Pulmonary Fibrosis (IPF), a type of lung scarring. Participants must have an FVC (a measure of lung function) at or above 45% of the expected value, a confirmed diagnosis following specific guidelines, and recent chest scans that meet study standards. They should also have certain levels of other breathing measures within set limits.

Inclusion Criteria

I have been diagnosed with IPF according to the 2018 guidelines.

FVC ≥45% of predicted normal at Screening Visits

Forced expiratory volume in 1 second (FEV1)/FVC ≥0.7 at Screening Visits

Exclusion Criteria

I am currently taking nintedanib and pirfenidone together.

I am currently pregnant or breastfeeding.

Participants who cannot meet protocol-specified baseline stability criteria

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive axatilimab or placebo every 2 weeks during the 26-week Treatment Period

26 weeks

13 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Axatilimab

Trial Overview The MAXPIRe study is testing Axatilimab against a placebo to see if it's effective and safe for treating IPF. Participants will be randomly assigned to receive either the actual medication or a placebo in order to compare outcomes between the two groups.

Participant Groups

Group I: Axatilimab Experimental Treatment 1 Intervention

Participants will receive axatilimab every 2 weeks during the 26-week Treatment Period.

Group II: Placebo Placebo Group 1 Intervention

Participants will receive placebo every 2 weeks during the 26-week Treatment Period.

Axatilimab is already approved in United States for the following indications:

Approved in United States as Axatilimab for:

• Chronic Graft-Versus-Host Disease

Who Is Running the Clinical Trial?

Syndax Pharmaceuticals

Lead Sponsor

Trials

49

Recruited

2,700+

DevPro Biopharma

Collaborator

Trials

5

Recruited

800+

DevPro Biopharma

Industry Sponsor

Trials

5

Recruited

800+

Findings from Research

The BUILD-1 study suggests that bosentan, an oral dual endothelin receptor antagonist, may help delay disease progression and improve quality of life in patients with idiopathic pulmonary fibrosis (IPF), particularly in those with biopsy-confirmed diagnoses.

While bosentan showed promising trends in efficacy, it is important to note that IPF currently has no known effective therapy, and further investigation is ongoing in the BUILD-3 trial to confirm these findings.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Bosentan for idiopathic pulmonary fibrosis. King, TE.[2018]

Idiopathic pulmonary fibrosis (IPF) is a severe lung disease with a median survival of only about 3 years after diagnosis, highlighting the urgent need for effective treatments.

Bosentan, an endothelin receptor antagonist, did not show overall benefit in a recent trial for IPF, but a post hoc analysis suggests it may be effective in a specific subgroup of patients with biopsy-proven IPF and minimal honeycombing, warranting further investigation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Evaluation of bosentan for idiopathic pulmonary fibrosis. Swigris, JJ., Brown, KK.[2010]

In a phase II trial involving 178 adults with idiopathic pulmonary fibrosis, macitentan did not show a significant improvement in forced vital capacity compared to placebo after 12 months, indicating it may not be effective for this condition.

Both macitentan and placebo groups had similar rates of elevated liver enzymes, suggesting that macitentan is well tolerated and has a safety profile comparable to placebo.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Macitentan for the treatment of idiopathic pulmonary fibrosis: the randomised controlled MUSIC trial. Raghu, G., Million-Rousseau, R., Morganti, A., et al.[2014]

Astilbin has shown promising antifibrotic effects by inhibiting the proliferation and migration of cells involved in pulmonary fibrosis, as demonstrated in both in vitro and in vivo studies.

The mechanism of action for astilbin involves blocking the Hedgehog signaling pathway, which is linked to the regulation of fibrosis-associated genes, suggesting its potential as a therapeutic option for treating lung fibrosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Astilbin ameliorates pulmonary fibrosis via blockade of Hedgehog signaling pathway. Zhang, J., Liu, H., Song, C., et al.[2019]

Pirfenidone is an oral antifibrotic medication that has been shown to slow the progression of idiopathic pulmonary fibrosis (IPF), a serious and often fatal lung disease.

This treatment improves progression-free survival for patients with IPF, providing a new therapeutic option in a condition that previously had very limited treatment options.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pirfenidone Initiates a New Era in the Treatment of Idiopathic Pulmonary Fibrosis. Selvaggio, AS., Noble, PW.[2016]

References

1. England pubmed.ncbi.nlm.nih.gov

Bosentan for idiopathic pulmonary fibrosis. [2018]

2. England pubmed.ncbi.nlm.nih.gov

Evaluation of bosentan for idiopathic pulmonary fibrosis. [2010]

3. England pubmed.ncbi.nlm.nih.gov

Macitentan for the treatment of idiopathic pulmonary fibrosis: the randomised controlled MUSIC trial. [2014]

4. England pubmed.ncbi.nlm.nih.gov

Astilbin ameliorates pulmonary fibrosis via blockade of Hedgehog signaling pathway. [2019]

5. United States pubmed.ncbi.nlm.nih.gov

Pirfenidone Initiates a New Era in the Treatment of Idiopathic Pulmonary Fibrosis. [2016]