Phase 2 Study of PEG-Intron in Hereditary Hemorrhagic Telangiectasia

Stopped early · Phase 2

Conditions studied: Anemia, Liver Disease, Hypoxemia

In brief

The purpose of the study is to evaluate the safety and tolerability of pegylated interferon alpha-2b (PEG-Intron) in patients with severe complications related to Hereditary hemorrhagic telangiectasia (HHT). Funding Source - FDA Office of Orphan Products Development (OOPD)

Key facts

Study ID

NCT00588146

Run by

Mayo Clinic

People needed

10

Starts

2007-01-01

Expected to finish

2011-09-01

Last updated by the study team

2013-02-22

Who can join

Age: 18 and older, up to 70. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Definite diagnosis of HHT by clinical criteria or genetic diagnosis. For the clinical diagnosis, 3 of the 4 following criteria1 must be present:
• Epistaxis: spontaneous, recurrent
• Telangiectases: multiple at characteristic sites
• Visceral lesions including telangiectases and/or arteriovenous malformations (AVM) (pulmonary, hepatic, gastrointestinal, cerebral, spinal)
• Family history of a first degree relative with HHT
• Transfusion-dependent anemia from HHT-related bleeding (epistaxis from nasal mucosal telangiectases or gastrointestinal bleeding from gastrointestinal telangiectases) defined as a hemoglobin (Hb) < 9g/dL with transfusion of at least one unit of packed red blood cells within the past 6 months or Hb < 11g/dL in females or < 13g/dL in males with transfusion of at least 5 units of blood within the past 6 months. Patients must be on a stable dose of iron or intolerant of iron. Patients must have failed traditional treatment options.
• Clinically stable outpatient
• Able and willing to return for outpatient visits
• Ability to perform subcutaneous injections
• Adult (Age 18 - 70 years)
• Presence of the following laboratory results at entry:
• White blood cell count ≥ 2000/mm\^3
• Neutrophil count ≥ 1000/mm\^3
• Platelet count ≥ 80,000/mm\^3
• Thyroid stimulating hormone within normal limits (Minimal abnormalities of the sensitive thyroid stimulating hormone may be allowed provided that the free thyroxin is normal and the patient is clinically euthyroid)
• Negative pregnancy test at enrollment, if applicable
• If the participant is a sexually active woman of childbearing potential, evidence that she is practicing adequate contraception during the treatment period. Adequate contraception includes use of an intrauterine device, oral contraceptives, progesterone implanted rods, medroxyprogesterone acetate, surgical sterilization, barrier method (diaphragm + spermicide), a monogamous relationship with a male partner who has had a vasectomy or is using a condom + spermicide or a birth control method acceptable to the study physicians. Participants and/or their partners must agree to continue the use of adequate contraception for at least 6 months following completion of treatment.
• Written informed consent specific for this protocol obtained prior to entry
• Patients agree to take study medication as directed and follow all study related procedures until the conclusion of their protocol participation
• Hepatic involvement by HHT characterized by high output heart failure due to hepatic vascular malformations (symptoms of heart failure including edema, ascites, S3 gallop, orthopnea, or jugular venous pressure > 10 cm H_2O) plus cardiac index (CI) measured at right heart catheterization > 4.4 L/min/m\^2. Patients must have failed traditional treatment options.
• Computed tomography scanning (CT) of the liver documenting vascular abnormalities consistent with HHT
• Child-Pugh category A
• Diffuse pulmonary telangiectases or AVMs documented by pulmonary angiography not amenable to treatment with embolization techniques. Patients must have failed traditional treatment options.
• Positive contrast echocardiography documenting right to left intrapulmonary shunt
• Resting or exercise-induced hypoxemia defined as a partial pressure of oxygen (PaO_2) < 70 mmHg at rest or an oxygen saturation (SpO_2) < 85% with exercise.

You may not qualify if…

• Anemia from any other cause than that due to HHT-related bleeding
• Hypersensitivity to PEG-Intron or any other component of the product
• Decompensated liver disease
• Chronic active Hepatitis B infection
• Child-Pugh category B or C
• History of severe psychiatric disease
• Prior suicide attempt
• Hospitalization for psychiatric disease
• Period of disability due to a psychiatric disease
• Current episode of moderate to severe depression not responsive to treatment
• History of immunologically mediated disease
• Inflammatory bowel disease
• Idiopathic thrombocytopenic purpura
• Systemic lupus erythematosus
• Autoimmune hemolytic anemia
• Scleroderma
• Sarcoidosis
• Multiple sclerosis
• Severe psoriasis
• Clinical evidence of rheumatoid arthritis
• Autoimmune hepatitis
• History of clinically significant cardiovascular disease
• Positive stress test
• Clinically significant arrhythmia
• Congestive heart failure

Where it is running

• Mayo Clinic — Rochester, Minnesota, United States

Full record on ClinicalTrials.gov

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