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Plexiform NeurofibromaPhase 2NCT00396019

Study of PEG-Intron for Plexiform Neurofibromas

A Phase 2 study testing PEG-Intron in people with Plexiform Neurofibroma, run by University of Pittsburgh at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This study is for slow growing tumors called plexiform neurofibromas (PNF) which are a relatively common problem in people with neurofibromatosis type 1 (NF1). These tumors are benign but as they grow, they can become disfiguring as well as disabling or even life threatening. Read more about this study

Who is it for?
Ages 18 months to 21 years
Conditions the study is for
Plexiform Neurofibroma
What is being tested?
PEG-Intron
How many people
86 participants
Where
1 siteUnited States
Study dates
Dec 2006to Apr 2014
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 months to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Age > or equal to 18 months to 21 years
  • Diagnosis Patients with NF1 and progressive, symptomatic or life-threatening plexiform neurofibromas which are not surgically resectable. Patients without biopsy-proof of plexiform must have at least one other diagnostic criteria for NF1: 6 or more cafe-au-lait spots, freckling in the axilla or groin, optic glioma, 2 or more Lisch nodules, distinctive bony lesion, first degree relative with NF1.
  • Only eligible if complete tumor resection is not feasible, or if a patient with a surgical option refuses surgery.
  • Patients may be treated on this trial without having received prior therapy. If patients have received prior therapy, they must have recovered from all toxic effects prior to entering this study.
  • Must have life expectancy of at least 12 months and a performance score (Karnofsky or Lansky) of > or equal to 50.
  • Must have adequate liver, kidney and bone marrow function

Who cannot join

  • Clinically significant unrelated systemic illness.
  • Received an investigational agent within the past 30 days.
  • Evidence of active visual pathway glioma
  • History of malignant peripheral nerve sheath tumor or ther cancer other than surgically cured non-melanoma skin cancer or cervical carcinoma in situ
  • Ongoing radiation therapy, chemotherapy, hormonal therapy directed at the tumor, or immunotherapy.
  • Inability to return for follow-up visits or obtain follow-up studies
Show all 14Show fewer
  • Severe cardiovascular disease
  • Pre-existing sever psychiatric condition or history of psychiatric disorder requiring hospitalization or a history of suicidal ideation or attempt.
  • Thyroid dysfunction not responsive to therapy.
  • Uncontrolled diabetes mellitus
  • History of seropositivity for HIV
  • Subjects who are pregnant, lactating, or of reproductive potential and not practicing an effective means of contraception.
  • Any medical condition requiring chronic systemic corticosteroids.
  • Subjects who are known to be actively abusing alcohol or drugs.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Children's Hospital of Pittsburgh of UPMC

    Pittsburgh, Pennsylvania, 15224

Other trials in United States

About this study

Official title: A Phase II Trial of Peginterferon Alfa-2b (PEG-Intron) for Plexiform Neurofibromas

This study is for slow growing tumors called plexiform neurofibromas (PNF) which are a relatively common problem in people with neurofibromatosis type 1 (NF1). These tumors are benign but as they grow, they can become disfiguring as well as disabling or even life threatening. They often cause pain, difficulty using arms or legs because of spinal cord compression, and/or nerve damage. At present, the only available therapy for plexiform neurofibromas is to try to surgically remove as much of the tumor as is possible. Because these tumors grow into the surrounding areas, total surgical resection is often impossible. Most tumors will re-grow after surgery if the entire tumor cannot be removed. To date, other treatments including chemotherapy and radiotherapy have not been able to shrink these tumors.

Interferon is a drug that is used for different types of tumors as well as for hepatitis. It has been used in the treatment of plexiform neurofibromas (PNF) with some subjects showing improvement in symptoms and/or a decrease in the size of the tumor. Most subjects had no further growth of their tumor while on the PEG-Intron. The drug used in this study is PEG (pegylated)-Intron. PEG-Intron is a long acting form of interferon which keeps the drug from being broken down in the body for a longer period of time and potentially could be more effective than the short-acting interferon. PEG-Intron has been approved by the Food and Drug Administration (FDA) for the treatment of Hepatitis C.

Read more

The goals of this study are:

  1. To determine how your child's plexiform neurofibroma responds to PEG-Intron when given weekly.
  2. To determine the side effects of PEG-Intron when given weekly to participants with plexiform neurofibromas.
  3. To evaluate a new method of measuring changes in the size of tumors called volume analysis. This method measures the entire volume of a tumor in three dimensions. The standard method of measuring tumors uses only the length and/or width of the tumor. By studying the different ways of measuring tumors the investigators hope to be able to determine which method is the most accurate and useful.
Read the detailed description

PEG-Intron will be given every week through a small needle under the skin, in the same way that insulin is given to people with diabetes. Subjects will be taught to do this at home. As long as the tumor isn't growing and the side effects are tolerable, the injections will be given once a week for 2 years.

Children will be enrolled on the study into one of three strata (patient groups): strata 1 includes children who do not have any symptoms associated with their PNF and MRI scans over the past year may or may not show tumor growth, strata 2 includes children who have symptoms associated with the PNF but MRI scans over the past year have not shown growth and strata 3 includes children who have shown an increase in the size of the PNF on MRI scans over the past year, with or without any symptoms.

Being tested
PEG-Intron
Medical subject headings
Neurofibroma, Plexiform

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PEG-IntronDrug

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants With Imaging Response in Strata 1 and 2

    A response was defined as a ≥20% reduction in the sum of the volume of the "target" plexiform neurofibroma ( PN) within 12 months confirmed by a follow-up MRI after ≥4 weeks.

    Measured: MRI scans were performed at baseline and at months 4, 8, 12, 18, and 24 while on treatment and every 6 months thereafter until off study.

  2. Clinical Response in Stratum 2

    Clinical response was defined as a protocol-specified improvement in ophthalmologic evaluation, an improvement of at least one level in performance status (PS) ≥50% decrease in the amount of pain medications required per week compared with baseline or ability to change from a narcotic to a nonnarcotic analgesic, sustained for at least one month.

    Measured: baseline, week 6, months 4, 8, 12, 18, and 24 while on treatment and every 6 months thereafter until off study

  3. Time to Progression (TTP) in Stratum 3

    TTP was estimated by the Kaplan-Meter method

    Measured: baseline, week 6, months 4, 8, 12, 18, and 24 while on treatment and every 6 months thereafter until off study

Secondary outcomes (1)

  1. Number of Participants With Imaging Response in Stratum 3

    A Response was defined as a ≥ 20% reduction in the sum of the volume of the "target" plexiform neurofibroma (PN) within 12 months, confirmed by a follow-up MRI after ≥ 24 weeks

    Measured: MRI scans were performed at baseline and at months 4, 8, 12, 18, and 24 while on treatment and every 6 months thereafter until off study

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Nov 2006 (estimated)
  2. Study startDec 2006
  3. Primary completionApr 2014
  4. Study completionApr 2014

Resources

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
86 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00396019
Sponsor's protocol ID
UPCI 06-116
Status last verified
Oct 2017
First submitted
2 Nov 2006
Last updated
6 Nov 2017
Data monitoring committee
Yes

Publications

  • Jakacki RI, Dombi E, Steinberg SM, Goldman S, Kieran MW, Ullrich NJ, Pollack IF, Goodwin A, Manley PE, Fangusaro J, Allen R, Widemann BC. Phase II trial of pegylated interferon alfa-2b in young patients with neurofibromatosis type 1 and unresectable plexiform neurofibromas. Neuro Oncol. 2017 Feb 1;19(2):289-297. doi: 10.1093/neuonc/now158.

    PubMed 27510726

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing PEG-Intron in people with Plexiform Neurofibroma, run by University of Pittsburgh 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 6 inclusion and 14 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: finished, as of the sponsor's last update on 6 Nov 2017. 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 6 Nov 2017). 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?

Number of Participants With Imaging Response in Strata 1 and 2 (time frame: MRI scans were performed at baseline and at months 4, 8, 12, 18, and 24 while on treatment and every 6 months thereafter until off study.). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00396019, which also lists 1 linked publication.

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 6 Nov 2017.

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