AnemiaPhase 2NCT00588146
Phase 2 Study of PEG-Intron in Hereditary Hemorrhagic Telangiectasia
A Phase 2 study testing Pegylated Interferon Alpha2b in people with Anemia and Liver Disease, run by Mayo Clinic at 1 site in United States.
Trial summary
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) Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Anemia, Liver Disease, Hypoxemia
- What is being tested?
- Pegylated Interferon Alpha2b
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2007to Sep 2011
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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
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- 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
- Uncontrolled hypertension
- Coronary artery bypass surgery within 24 weeks prior to entry
- Angina pectoris or myocardial infarction within 1 year prior to entry
- Seizure disorder uncontrolled by anticonvulsants (within the last 12 months)
- History of thyroid disease poorly controlled on prescribed medications
- History or evidence of retinopathy
- Patients on chronic anticoagulation
- History of chronic renal insufficiency (creatinine > 2.5 mg/dL)
- Patients who have received an investigational drug within 24 weeks of treatment assignment
- History or other evidence of severe illness or other comorbid condition which would make the patient unsuitable for participation in a research protocol
- Liver dysfunction from any other cause than that due to HHT (chronic active hepatitis B infection, hepatitis C infection, alcoholic cirrhosis, etc.)
- Cardiac index < 4.4 L/min/m^2
- Pulmonary AVMs with feeding arteries > 3 mm in diameter amenable to embolization techniques
- Other pulmonary diseases causing hypoxemia.
Trial locations and contacts
1 site in 1 country.
About this study
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)
Read the detailed description
The objective of this 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). Participants will be randomized to the treatment arm or control arm and then crossed over to the alternate arm at 6 months for the remainder of the 12-month study. Study treatment will consist of weekly subcutaneous injections of pegylated interferon alpha-2b (PEG-Intron), 1 microgram/kilogram/week. Adverse events as well as monitoring and treatment of toxicities will be followed as stated in the protocol. Adverse events will be graded according to the Modified NCI Common Toxicity Criteria. After every five participants have completed one month of treatment, an independent data safety monitoring board will review any adverse events.
- Conditions
- Anemia, Liver Disease, Hypoxemia
- Being tested
- Pegylated Interferon Alpha2b, Standard care
- Keywords
- anemia, HHT, Liver disease, Hypoxemia, Iron deficiency anemia, Liver disease with high cardiac output, Diffuse pulmonary AVMs with hypoxemia
- Medical subject headings
- Anemia, Liver Diseases, Hypoxia, Anemia, Iron-Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Pegylated Interferon Alpha2bDrug
Also called PEG-Intron
Weekly subcutaneous injection of 1 microgram/kg/week
Standard careOther
Standard care
| Study group | Role | What they receive |
|---|---|---|
| Pegylated Interferon Alpha2b, then Standard Care | Experimental | Weekly subcutaneous injection of pegylated interferon alpha2b 1 microgram/kg/week for 6 months, then standard care for 6 months. Receives: Drug: Pegylated Interferon Alpha2b; Other: Standard care |
| Standard Care, then Pegylated Interferon Alpha2b | Experimental | Standard care for 6 months, then weekly subcutaneous injection of pegylated interferon alpha2b 1 microgram/kg/week for 6 months. Receives: Drug: Pegylated Interferon Alpha2b; Other: Standard care |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Hemoglobin
The hemoglobin level is expressed as the amount of hemoglobin in grams (gm) per deciliter (dL) of whole blood.
Measured: baseline, one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jan 2008 (estimated)
- Study startJan 2007
- Primary completionSep 2011
- Study completionSep 2011
Why it stopped: Schering-Plough discontinued supplying study drug.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for AnemiaStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Collaborators
- Augusta University, Unity Health Toronto, Schering-Plough
- Investigators
- Karen L Swanson, DO, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00588146
- Sponsor's protocol ID
- 2400-05
- Other IDs
- R01FD003076-01
- Status last verified
- Feb 2013
- First submitted
- 26 Dec 2007
- Last updated
- 22 Feb 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 22 Feb 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Study of PEG-Intron in Hereditary Hemorrhagic Telangiectasia
- ClinicalTrials.gov ID
- NCT00588146
- Acronym / sponsor ID
- 2400-05
- Conditions
- Anemia, Liver Disease, Hypoxemia
- Keywords
- anemia, HHT, Liver disease, Hypoxemia, Iron deficiency anemia, Liver disease with high cardiac output, Diffuse pulmonary AVMs with hypoxemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Schering-Plough discontinued supplying study drug.
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Collaborators
- Augusta University, Unity Health Toronto, Schering-Plough
- Study start
- Jan 2007
- Primary completion
- Sep 2011 (actual)
- Study completion
- Sep 2011 (actual)
- First posted
- 8 Jan 2008 (estimated)
- Results
- Posted 28 Jan 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Detailed description
The objective of this 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). Participants will be randomized to the treatment arm or control arm and then crossed over to the alternate arm at 6 months for the remainder of the 12-month study. Study treatment will consist of weekly subcutaneous injections of pegylated interferon alpha-2b (PEG-Intron), 1 microgram/kilogram/week. Adverse events as well as monitoring and treatment of toxicities will be followed as stated in the protocol. Adverse events will be graded according to the Modified NCI Common Toxicity Criteria. After every five participants have completed one month of treatment, an independent data safety monitoring board will review any adverse events.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Pegylated Interferon Alpha2b, then Standard Care | Experimental | Drug: Pegylated Interferon Alpha2b, Other: Standard careWeekly subcutaneous injection of pegylated interferon alpha2b 1 microgram/kg/week for 6 months, then standard care for 6 months. |
| Standard Care, then Pegylated Interferon Alpha2b | Experimental | Drug: Pegylated Interferon Alpha2b, Other: Standard careStandard care for 6 months, then weekly subcutaneous injection of pegylated interferon alpha2b 1 microgram/kg/week for 6 months. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (25 listed)
- Definite diagnosis of HHT by clinical criteria or genetic diagnosis. For the clinical diagnosis, 3 of the 4 following criteria1 must be present:
- 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
Key exclusion criteria (39 listed)
- 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
- History of severe psychiatric disease
- History of immunologically mediated disease
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Pegylated Interferon Alpha2bAlso: PEG-IntronWeekly subcutaneous injection of 1 microgram/kg/week | Drug | Pegylated Interferon Alpha2b, then Standard Care, Standard Care, then Pegylated Interferon Alpha2b |
| Standard careStandard care | Other | Pegylated Interferon Alpha2b, then Standard Care, Standard Care, then Pegylated Interferon Alpha2b |
Primary outcome
Change in Hemoglobin
Time frame: baseline, one year
The hemoglobin level is expressed as the amount of hemoglobin in grams (gm) per deciliter (dL) of whole blood.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Feb 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Karen L Swanson, DO principal investigator
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pegylated Interferon Alpha2b in people with Anemia and Liver Disease, run by Mayo Clinic at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 25 inclusion and 39 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 22 Feb 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Jan 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Hemoglobin (time frame: baseline, one year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Feb 2013.