Letrozole in Patients With Hepatopulmonary Syndrome

NCT04577001 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: 19-005779
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to assess the safety, tolerability and possible benefit of letrozole compared to placebo in patients with Hepatopulmonary Syndrome.

Conditions

Hepatopulmonary Syndrome

Outcome Measures

Primary Outcomes (1)

• Alveolar-Arterial Oxygen

  To determine whether letrozole affects alveolar-arterial oxygen gradient (AaPO2) at 6 months in patients with HPS.

  6 months

Secondary Outcomes (3)

• Hormone Levels

  6 months

• Oxygen Saturation

  3 and 6 months

• Side Effects

  6 months

Study Arms (2)

Letrozole Group

EXPERIMENTAL

Subjects with hepatopulmonary syndrome will get the study drug letrozole

Drug: Letrozole

Placebo Group

PLACEBO COMPARATOR

Subjects with hepatopulmonary syndrome will get the study placebo

Drug: Placebo

Interventions

Letrozole DRUG

2.5 mg orally daily for 6 months

Letrozole Group

Placebo DRUG

No active ingredient taken orally daily for 6 months

Placebo Group

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of moderate to very severe hepatopulmonary syndrome which consists of having all 3 of the following conditions:
• Presence of liver disease or portal hypertension
• Intrapulmonary shunting on contrast-enhanced echocardiogram
• Hypoxemia \[A-a gradient ≥15mmHg (or ≥20mmHg if age \>64) and PaO2\<80mmHg on arterial blood gas testing\]
• Child-Pugh class A or B liver disease
• MELD score \< 20
• ≥ 18 years old
• Female subjects must be post-menopausal (defined as 12 months of spontaneous amenorrhea or 6 weeks postsurgical bilateral oophorectomy without or without hysterectomy)
• Ability to provide informed consent

You may not qualify if:

• Enrollment in a clinical trial or concurrent use of another investigational drug or device therapy (i.e., outside of study treatment) during, or within 28 days of baseline
• Current hepatic encephalopathy
• Expectation of liver transplant within six months of randomization
• Concomitant lung disease defined as restriction (TLC \< 70%) or obstruction (FEV1 \< 80% \& FEV1/FVC \< 70%)
• Inability to comply with the study protocol
• Osteoporosis
• Premenopausal women (those who have not reached 1 year absence of menarche)
• Vulnerable study population, including imprisoned individuals, or those who cannot consent on their own.

Sponsors & Collaborators

• Mayo Clinic: lead
• University of California, San Francisco: collaborator

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55902, United States

Location

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Conditions

Hepatopulmonary Syndrome

Interventions

Letrozole

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases

Intervention Hierarchy (Ancestors)

Nitriles; Organic Chemicals; Triazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Limitations and Caveats

Terminated study due to low recruitment numbers.

Results Point of Contact

• Title: Dr. Hilary DuBrock
• Organization: Mayo Clinic

DuBrock.Hilary@mayo.edu

(507) 284-2957

Study Officials

• Hilary M DuBrock, MD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: September 30, 2020
• First Posted: October 6, 2020
• Study Start: February 18, 2021
• Primary Completion: March 26, 2021
• Study Completion: March 26, 2021
• Last Updated: May 14, 2024
• Results First Posted: May 14, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)