Letrozole in Patients With Hepatopulmonary Syndrome

Mayo Clinic

Status and phase: Terminated; Phase 2

Conditions: Hepatopulmonary Syndrome

Treatments: Drug: Letrozole; Drug: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT04577001

19-005779

Details and patient eligibility

About

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

Full description

This study is a phase II randomized, double-blind, placebo-controlled parallel trial of 20 subjects with hepatopulmonary syndrome designed to assess the effect of letrozole 2.5 mg orally daily or placebo for 6 months on the alveolar-arterial oxygen gradient (AaPO2).

Subjects at each site will be screened at outpatient clinic visit appointments and interested qualified subjects will be consented and offered participation in this trial. Once consent has been obtained baseline values will be established and subjects will begin letrozole with follow-up clinic visits and testing at 3 months, and 6 months.

Enrollment: 1 patient

Sex: All

Ages: 18 to 99 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria

• 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

Exclusion Criteria

• 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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1 participants in 2 patient groups, including a placebo group

Letrozole Group

Experimental group

Description:

Subjects with hepatopulmonary syndrome will get the study drug letrozole

Treatment:

Drug: Letrozole

Placebo Group

Placebo Comparator group

Description:

Subjects with hepatopulmonary syndrome will get the study placebo

Treatment:

Drug: Placebo