Trial Outcomes & Findings for Phase 2 Study of PEG-Intron in Hereditary Hemorrhagic Telangiectasia (NCT NCT00588146)

NCT ID: NCT00588146

Last Updated: 2013-02-22

Results Overview

The hemoglobin level is expressed as the amount of hemoglobin in grams (gm) per deciliter (dL) of whole blood.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 10 participants
• Primary outcome timeframe: baseline, one year
• Results posted on: 2013-02-22

Participant Flow

Subjects were recruited from Mayo Clinic, Rochester, Minnesota from January 2007 through September 2010.

Participant milestones

Measure	Pegylated Interferon Alpha2b, Then Standard Care Weekly 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 Standard care for 6 months, then weekly subcutaneous injection of pegylated interferon alpha2b 1 microgram/kg/week for 6 months.
First Intervention (Baseline to 6 mo) STARTED	5	5
First Intervention (Baseline to 6 mo) COMPLETED	1	2
First Intervention (Baseline to 6 mo) NOT COMPLETED	4	3
Second Intervention (6 Months to 1 Year) STARTED	1	2
Second Intervention (6 Months to 1 Year) COMPLETED	1	2
Second Intervention (6 Months to 1 Year) NOT COMPLETED	0	0

Reasons for withdrawal

Measure	Pegylated Interferon Alpha2b, Then Standard Care Weekly 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 Standard care for 6 months, then weekly subcutaneous injection of pegylated interferon alpha2b 1 microgram/kg/week for 6 months.
First Intervention (Baseline to 6 mo) Adverse Event	4	3

Baseline Characteristics

Phase 2 Study of PEG-Intron in Hereditary Hemorrhagic Telangiectasia

Baseline characteristics by cohort

Measure	Entire Study Population n=10 Participants Includes groups randomized to pegylated interferon alpha-2b first and standard care first.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	6 Participants n=5 Participants
Age, Categorical >=65 years	4 Participants n=5 Participants
Age Continuous	56 years STANDARD_DEVIATION 15.8 • n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Region of Enrollment United States	10 participants n=5 Participants

PRIMARY outcome

Timeframe: baseline, one year

Population: Only 3 subjects completed the study, so the numbers were too low to analyze the study.

The hemoglobin level is expressed as the amount of hemoglobin in grams (gm) per deciliter (dL) of whole blood.

Outcome measures

Outcome data not reported

Adverse Events

Pegylated Interferon Alpha-2b

Serious events: 3 serious events

Other events: 2 other events

Deaths: 0 deaths

Standard Care

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Measure	Pegylated Interferon Alpha-2b n=10 participants at risk Weekly subcutaneous injection of pegylated interferon alpha2b 1 microgram/kg/week	Standard Care n=10 participants at risk Subjects received standard care for hereditary hemorrhagic telangiectasia.
Blood and lymphatic system disorders Neutropenia	30.0% 3/10 • Number of events 3 • Adverse events were collected over the 1-year study by telephone or direct interview. Adverse events were graded according to Modified NCI Common Toxicity Criteria. If a subject discontinued for an adverse event, they were observed for 12 weeks.	0.00% 0/10 • Adverse events were collected over the 1-year study by telephone or direct interview. Adverse events were graded according to Modified NCI Common Toxicity Criteria. If a subject discontinued for an adverse event, they were observed for 12 weeks.

Other adverse events

Measure	Pegylated Interferon Alpha-2b n=10 participants at risk Weekly subcutaneous injection of pegylated interferon alpha2b 1 microgram/kg/week	Standard Care n=10 participants at risk Subjects received standard care for hereditary hemorrhagic telangiectasia.
General disorders Seizure	0.00% 0/10 • Adverse events were collected over the 1-year study by telephone or direct interview. Adverse events were graded according to Modified NCI Common Toxicity Criteria. If a subject discontinued for an adverse event, they were observed for 12 weeks.	10.0% 1/10 • Number of events 1 • Adverse events were collected over the 1-year study by telephone or direct interview. Adverse events were graded according to Modified NCI Common Toxicity Criteria. If a subject discontinued for an adverse event, they were observed for 12 weeks.
Psychiatric disorders Depression	0.00% 0/10 • Adverse events were collected over the 1-year study by telephone or direct interview. Adverse events were graded according to Modified NCI Common Toxicity Criteria. If a subject discontinued for an adverse event, they were observed for 12 weeks.	10.0% 1/10 • Number of events 2 • Adverse events were collected over the 1-year study by telephone or direct interview. Adverse events were graded according to Modified NCI Common Toxicity Criteria. If a subject discontinued for an adverse event, they were observed for 12 weeks.
Musculoskeletal and connective tissue disorders Myalgias	10.0% 1/10 • Number of events 1 • Adverse events were collected over the 1-year study by telephone or direct interview. Adverse events were graded according to Modified NCI Common Toxicity Criteria. If a subject discontinued for an adverse event, they were observed for 12 weeks.	10.0% 1/10 • Number of events 1 • Adverse events were collected over the 1-year study by telephone or direct interview. Adverse events were graded according to Modified NCI Common Toxicity Criteria. If a subject discontinued for an adverse event, they were observed for 12 weeks.
Musculoskeletal and connective tissue disorders Arthralgias	10.0% 1/10 • Number of events 1 • Adverse events were collected over the 1-year study by telephone or direct interview. Adverse events were graded according to Modified NCI Common Toxicity Criteria. If a subject discontinued for an adverse event, they were observed for 12 weeks.	10.0% 1/10 • Number of events 1 • Adverse events were collected over the 1-year study by telephone or direct interview. Adverse events were graded according to Modified NCI Common Toxicity Criteria. If a subject discontinued for an adverse event, they were observed for 12 weeks.
Musculoskeletal and connective tissue disorders Right flank pain	10.0% 1/10 • Number of events 1 • Adverse events were collected over the 1-year study by telephone or direct interview. Adverse events were graded according to Modified NCI Common Toxicity Criteria. If a subject discontinued for an adverse event, they were observed for 12 weeks.	0.00% 0/10 • Adverse events were collected over the 1-year study by telephone or direct interview. Adverse events were graded according to Modified NCI Common Toxicity Criteria. If a subject discontinued for an adverse event, they were observed for 12 weeks.

Additional Information

Karen L. Swanson, D.O.

Mayo Clinic

Phone: 507-255-3214

Email: swanson.karen@mayo.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place