What side effects are associated with this type of intervention?

The most common treatments for Idiopathic Pulmonary Fibrosis (IPF) are nintedanib and pirfenidone. Nintedanib is associated with side effects such as diarrhea, nausea, abdominal pain, liver enzyme elevation, and weight loss. Pirfenidone commonly causes nausea, rash, fatigue, diarrhea, and photosensitivity. While ABT-199 (Venetoclax) is being studied for its potential in IPF by inducing apoptosis in monocyte-derived macrophages, its known side effects from other uses include neutropenia, infections, and gastrointestinal issues. Both nintedanib and pirfenidone aim to slow disease progression but carry significant side effects.

What prior FDA approvals exist?

The FDA has approved two primary antifibrotic medications for the treatment of Idiopathic Pulmonary Fibrosis (IPF): nintedanib and pirfenidone. Nintedanib is a tyrosine kinase inhibitor that targets multiple pathways involved in fibrosis, while pirfenidone is an antifibrotic agent that reduces fibroblast proliferation and collagen synthesis. Both drugs have been shown to slow the progression of IPF and reduce the frequency of acute exacerbations. Unlike ABT-199 (Venetoclax), which is being studied for its ability to induce apoptosis in monocyte-derived macrophages by inhibiting Bcl-2, nintedanib and pirfenidone do not directly target apoptotic pathways.

What other types of research exist?

Promising novel alternatives to ABT-199 (Venetoclax) for IPF patients include antifibrotic medications such as nintedanib and pirfenidone. Both have been shown to slow disease progression and reduce the frequency of acute exacerbations. Additionally, ongoing research into telomerase inhibitors like imetelstat and JAK inhibitors such as ruxolitinib and fedratinib may offer new therapeutic options. These alternatives target different mechanisms involved in fibrosis and inflammation, providing potential benefits for IPF patients.

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BH3 Mimetic

Venetoclax for Idiopathic Pulmonary Fibrosis

Phase < 1

Waitlist Available

Research Sponsored by University of Alabama at Birmingham

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

A diagnosis of IPF that fulfills current ATS/ERS Consensus Criteria (1)

Age between 40-85 years old, male and female

Must not have

Evidence of cardiac conducting abnormalities, defined as second- or third-degree AV block not successfully treated with a pacemaker, or a personal or family history of long QT syndrome (QTc interval >450 msec for males or 470 msec for females)

A history of bone marrow disorder including aplastic anemia, or marked anemia defined as hemoglobin < 10.0 g/dL (or 6.2 mmol/L)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 3 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing ABT-199, a drug that helps certain immune cells die, in patients with idiopathic pulmonary fibrosis (IPF). IPF is a serious lung disease with no cure. The drug works by blocking a protein that prevents cell death, potentially reducing lung damage. This could offer significant benefits for IPF patients. ABT-199 has shown promise in reducing lung inflammation and fibrosis in animal models of pulmonary fibrosis.

Who is the study for?

This trial is for adults aged 40-85 with a confirmed diagnosis of idiopathic pulmonary fibrosis (IPF) that's been known for less than 5 years and have at least moderate lung function. Participants must be able to consent, follow the study plan, and not be on certain other drugs or have major health issues like severe liver problems, heart conditions, recent infections, kidney disease requiring dialysis, or a history of cancer in the last 5 years.

What is being tested?

The trial tests Venetoclax's safety and effectiveness in treating IPF. Researchers believe it may help by causing problematic cells in the lungs to self-destruct. The drug has worked well before in leukemia patients without serious side effects.

What are the potential side effects?

Venetoclax could potentially cause side effects such as digestive issues, fatigue, low blood cell counts leading to increased infection risk or bleeding problems. It might also affect liver enzymes and interact with other medications.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with idiopathic pulmonary fibrosis.

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I am between 40 and 85 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have untreated heart rhythm problems or a family history of long QT syndrome.

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I have a history of severe anemia or bone marrow disorders.

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I haven't taken high-dose steroids or immunosuppressants in the last month.

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I haven't needed antibiotics for an infection in the last 2 weeks.

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I am not taking strong drugs that affect liver enzyme activity during my treatment.

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I am on dialysis for end-stage kidney disease.

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I haven't had cancer in the last 5 years, except for skin cancer.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 3 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~3 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 3 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of participants with treatment-related adverse events as assessed by measuring blood counts.

Number of participants with treatment-related adverse events as assessed by measuring liver function.

Secondary study objectives

Percentage of monocyte-derived macrophages that undergoing apoptosis.

Side effects data

From 2022 Phase 3 trial • 389 Patients • NCT02005471

33%

Neutropenia

11%

SARS-CoV-2 test positive

11%

Sepsis

11%

Abdominal pain

11%

Pneumonia

11%

Rhinovirus infection

11%

COVID-19

11%

Gastroenteritis

11%

Pneumonia pseudomonal

11%

Electrocardiogram QT prolonged

11%

Anaemia

11%

Neutrophil count decreased

11%

Hypokalaemia

11%

Febrile neutropenia

11%

Supraventricular tachycardia

11%

Blood creatinine increased

11%

White blood cell count decreased

11%

Dermatitis

100%

80%

60%

40%

20%

Study treatment Arm

Bendamustine + Rituximab Crossover Substudy

Venetoclax + Rituximab Re-Treatment Substudy

Venetoclax + Rituximab Main Study

Bendamustine + Rituximab Main Study

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: treatmentExperimental Treatment1 Intervention

Venetoclax 100 mg daily for 3 weeks

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Venetoclax

2019

Completed Phase 3

~2240

0 / 9Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Idiopathic Pulmonary Fibrosis (IPF) include nintedanib and pirfenidone, which work by slowing disease progression and reducing acute exacerbations. Nintedanib is a tyrosine kinase inhibitor that targets multiple growth factors involved in fibrosis, inhibiting fibroblast proliferation and collagen deposition. Pirfenidone has antifibrotic and anti-inflammatory properties, reducing fibroblast activity and collagen synthesis. Additionally, ABT-199 (Venetoclax) is being studied for its ability to induce apoptosis in monocyte-derived macrophages (MDMs) by inhibiting Bcl-2, a protein that prevents cell death. This mechanism is significant as it targets the cellular components contributing to fibrosis, potentially reversing established fibrosis. Understanding these mechanisms is crucial for IPF patients as it highlights the therapeutic strategies aimed at slowing disease progression and improving survival.