A Study Evaluating the Safety and Efficacy of ENV-101 in Subjects With Idiopathic Pulmonary Fibrosis (IPF)

NCT04968574 | Completed Phase 2 FDA Drug

• 1 other identifier: ENV-IPF-101
• Study Type: interventional
• Target: 41
• Locations: 5 countries
• Sites: 24

Brief Summary

This is a Phase 2, randomized, placebo controlled, multi-center study in subjects with mild to moderate IPF. Eligible subjects will be randomized to receive placebo or ENV-101 as a daily oral dose for 12 consecutive weeks of treatment. Following treatment, subjects will be observed for an additional 6 weeks.

Conditions

Idiopathic Pulmonary Fibrosis

Keywords

hedgehog; smoothened; pulmonary fibrosis

Outcome Measures

Primary Outcomes (10)

• Change from baseline in frequency of adverse events (AEs)

  An AE is any untoward medical occurrence in a study subject administered a pharmaceutical product and which does not necessarily have a causal relationship with the treatment under investigation. Frequency of a given AE is determined by dividing the total number of that AE observed during the study by the total number of subjects in the study.

  Baseline to Week 18

• Change from baseline in severity of AEs

  Severity of AEs are categorized as mild, moderate or severe as described below: * Mild - Events require minimal or no treatment and do not interfere with the subject's daily activities. * Moderate - Events result in a low level of inconvenience or concern with the therapeutic measures. Moderate events may cause some interference with functioning. * Severe - Events interrupt a subject's usual daily activity and may require systemic drug therapy or other treatment. Severe events are usually potentially life-threatening or incapacitating.

  Baseline to Week 18

• Change from baseline in vital sign measurements - pulse

  Comparison of a subject's pulse rate at the beginning of the study to that subject's pulse rate at the completion of the study.

  Baseline to Week 18

• Change from baseline in vital sign measurements - blood pressure

  Comparison of a subject's blood pressure at the beginning of the study to that subject's blood pressure at the completion of the study.

  Baseline to Week 18

• Change from baseline in vital sign measurements - respiration rate

  Comparison of a subject's respiration rate (number of breaths taken per minute while at rest) at the beginning of the study to that subject's respiration rate at the completion of the study.

  Baseline to Week 18

• Change from baseline in vital sign measurements - temperature

  Comparison of a subject's body temperature at the beginning of the study to that subject's body temperature at the completion of the study.

  Baseline to Week 18

• Change from baseline in blood oxygen saturation level

  Comparison of a subject's blood oxygen saturation level (measured at rest using a pulse oximeter) at the beginning of the study to that subject's blood oxygen saturation level at the completion of the study.

  Baseline to Week 18

• Incidence of clinical laboratory abnormalities

  Assessment of the clinical laboratory measurements (chemistry, hematology, urinalysis parameters) that are above or below the laboratory normal ranges. Incidence of clinical laboratory abnormalities is determined by dividing the total number of clinical laboratory abnormalities by the total number of subjects in the study.

  Baseline to Week 18

• Severity of clinical laboratory abnormalities

  Assessment of the severity (defined as either clinically significant or not clinically significant) for the clinical laboratory abnormalities observed during the study.

  Baseline to Week 18

• Number of hospitalizations

  Assessment of the number of hospitalizations for any reason observed among all subjects from the beginning of the study to the completion of the study.

  Baseline to Week 18

Secondary Outcomes (3)

• Change from baseline of FVC (forced vital capacity)

  Baseline and Week 12

• Change from baseline of DLCO (diffusing capacity of the lungs for carbon monoxide)

  Baseline and Week 12

• Change from baseline of patient reported outcomes by the University of California-San Diego (UCSD) Shortness of Breath Questionnaire (SOBQ)

  Baseline and Week 12

Other Outcomes (6)

• Change from baseline of FVC

  Baseline and Week 6

• Change from baseline of FVC

  Baseline and Week 18

• Change from baseline of DLCO

  Baseline and Week 6

Study Arms (2)

ENV-101

EXPERIMENTAL

taladegib, 200 mg tablet, once daily for 12 weeks

Drug: taladegib

placebo

PLACEBO COMPARATOR

placebo, tablet, once daily for 12 weeks

Drug: placebo

Interventions

taladegib DRUG

hedgehog pathway inhibitor dosed once daily

ENV-101

placebo DRUG

identical tablets to the experimental arm with no active ingredient

placebo

Eligibility Criteria

• Age: 40 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• IPF diagnosis based upon American Thoracic Association, Japanese Respiratory Society, European Respiratory Society, Latin American Thoracic Association guidelines within the last 7 years. Diagnosis will be confirmed to be consistent with IPF by centrally read high resolution computed tomography (HRCT).
• Ability to successfully perform lung function tests.
• Subjects are willing to remain on study treatment for the duration of the study.
• Subjects have a full understanding of the informed consent.

You may not qualify if:

• Evidence of other known causes of interstitial lung disease (ILD) (e.g., domestic, and occupational environmental exposures, connective tissue disease \[CTD\], and drug toxicity), lung transplant expected within 12 months of screening or evidence of clinically significant lung disease other than IPF including but not limited to asthma, chronic obstructive pulmonary disease (COPD), uncontrolled pulmonary hypertension and emphysema where computed tomography (CT)-assessed extent of emphysema is greater than extent of fibrosis.
• History of malignancy, including carcinoma during the preceding 5 years. With the following exceptions:
• Prior history of in situ basal or squamous cell skin cancer that was successfully treated with curative therapies.
• Subjects with other malignancies if they have been continuously disease free for at least 5 years prior to study start.
• Subjects with prostate cancer that are managed by surveillance are also eligible.
• Current use of supplemental oxygen for any condition unless prior approval is received from the Sponsor.
• Smoking within 6 months of study start, current smoker, or unwillingness to refrain from smoking during the clinical trial duration.
• Presence of active infection at study start or confirmed active human immunodeficiency virus (HIV), Hepatitis B virus (HBV) or Hepatitis C virus (HCV).
• Occurrence of serious illness requiring hospitalization within 90 days prior to study start.
• Current or previous use (within 30 days prior to study start) of the following:
• N-acetylcysteine
• endothelin receptor antagonist
• riociguat
• prostacyclin or prostacyclin analogue
• Warfarin for IPF

Sponsors & Collaborators

• Endeavor Biomedicines, Inc.: lead

Study Sites (24)

Research Site

Liverpool, New South Wales, 1871, Australia

Location

Research Site

Benowa, Queensland, 4217, Australia

Location

Research Site

Box Hill, Victoria, 3128, Australia

Location

Research Site

Clayton, Victoria, 3168, Australia

Location

Research Site

Vancouver, British Columbia, V5Z 1M9, Canada

Location

Research Site

Sherbrooke, Quebec, J1H 5N4, Canada

Location

Research Site

Batu Caves, 68100, Malaysia

Location

Research Site

Kota Bharu, 15200, Malaysia

Location

Research Site

Kuala Lumpur, 53000, Malaysia

Location

Research Sire

Kuala Lumpur, 56000, Malaysia

Location

Research Site

Kuala Lumpur, 59100, Malaysia

Location

Research Site

Monterrey, Nuevo León, 64060, Mexico

Location

Research Site

Monterrey, Nuevo León, 64718, Mexico

Location

Research Site

San Nicolás de los Garza, Nuevo León, 66465, Mexico

Location

Research Site

Chihuahua City, 31203, Mexico

Location

Research Site

Mexico City, 03100, Mexico

Location

Research Site

Mexico City, 14080, Mexico

Location

Research Site

Oaxaca City, 68000, Mexico

Location

Research Site

Puebla City, 72180, Mexico

Location

Research Site (Namdong District)

Incheon, South Korea

Location

Research Site (Bundang District)

Seongnam, South Korea

Location

Research Site (Gangnam District)

Seoul, South Korea

Location

Research Site (Seongbuk District)

Seoul, South Korea

Location

Research Site (Songpa District)

Seoul, South Korea

Location

Related Publications (1)

• Maher TM, Goldin JG, Hood J, Pitman J, de Los Rios M, Hobbs BP, Yu-Lin AB, Buendia-Roldan I, Thien F, Song JW, Perea PC, Ramirez-Rivera A, DiFrancesco A. Taladegib for the treatment of idiopathic pulmonary fibrosis (ENV-IPF-101): a multicentre, randomised, double-blind, placebo-controlled, phase 2a trial. Lancet Respir Med. 2025 Nov;13(11):1001-1010. doi: 10.1016/S2213-2600(25)00239-5. Epub 2025 Sep 30.

  PMID: 41043447 DERIVED

MeSH Terms

Conditions

Idiopathic Pulmonary Fibrosis; Pulmonary Fibrosis

Interventions

LY2940680

Condition Hierarchy (Ancestors)

Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases; Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• John Huetsch, M.D.

  Endeavor Biomedicines

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: June 29, 2021
• First Posted: July 20, 2021
• Study Start: August 26, 2021
• Primary Completion: November 30, 2023
• Study Completion: November 30, 2023
• Last Updated: December 2, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

AU (4); CA (2); ME (8); MY (5); KR (5)