NeurofibromatosisPhase 2NCT00754780
Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1
A Phase 2 study testing Pirfenidone in people with Neurofibromatosis, run by Mayo Clinic.
Trial summary
The study is a phase II, open label trial of oral Pirfenidone in 24 adult patients with neurofibromatosis type 1. Pirfenidone is a new, broad-spectrum anti-fibrotic drug, with proven in vitro and in vivo negative effects on fibroblast growth and collagen matrix synthesis. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Neurofibromatosis
- What is being tested?
- Pirfenidone
- Type of treatment
- Pill or oral medicine
- How many people
- 24 participants
- Where
- No sites listed yet
- Study dates
- Sep 2000to Aug 2004
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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
- Diagnosis of neurofibromatosis type I, based on clinical criteria (NIH Consensus Development Conference, 1988). Tumors will not be confirmed histologically, since the biopsy could cause a change in tumor growth and such interfere with effect of Pirfenidone treatment.
- Male or female patients
- Age 18 years old
- All patients should be mentally capable of signing the consent form or should have a legal guardian to provide consent
- Patients who are experiencing symptoms from neurofibromatous lesions and who refuse surgery or are not good surgical candidates, such as those with plexiform neurofibroma who are experiencing significant discomfort, disfigurement or nerve compression or
- Presence of multiple spinal neurofibromas in which the surgical removal would carry a major risk for spinal cord damage.
Who cannot join
- Tumors for which surgical removal could lead to permanent (or long-term) relief of symptoms
- Patients with open skin lesions and patients for whom surgery is being contemplated or who had surgery less than 4 weeks from starting treatment
- Patients for whom biopsy is warranted for suspected malignancies
- Individuals younger than 18 years
- Pregnant and lactating women
- Inability to have MR imaging (e.g. claustrophobia, pacemaker or allergy to contrast dye, if administration is needed for neurofibroma imaging)
About this study
Official title: Phase II Clinical Trial of Pirfenidone for the Treatment of Patients With Neurofibromatosis Type I
The study is a phase II, open label trial of oral Pirfenidone in 24 adult patients with neurofibromatosis type 1.
Pirfenidone is a new, broad-spectrum anti-fibrotic drug, with proven in vitro and in vivo negative effects on fibroblast growth and collagen matrix synthesis. Human studies indicate promising therapeutic effects in arresting and reversing fibrosis in a variety of different conditions, where the excessive formation of fibrous tissue is a major pathogenic mechanism. Since the fibrous tissue is a significant component of neurofibroma, reduction of fibrosis could diminish tumor progression and lead to tumor shrinkage. Therefore, Pirfenidone is an excellent candidate for the treatment of plexiform neurofibromas and surgically unresectable tumors in patients with NF1.
Read the detailed description
Specific aims of this study are:
- To evaluate efficacy of Pirfenidone in NF1 patients with disfiguring or disabling plexiform neurofibroma (PN) and spinal neurofibromas (SN)
- To determine the acute, subacute and chronic toxicity of Pirfenidone in patients with NF1.
- Conditions
- Neurofibromatosis
- Being tested
- Pirfenidone
- Keywords
- NF1, plexiform neurofibroma
- Medical subject headings
- Neurofibromatoses, Neurofibroma, Plexiform
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PirfenidoneDrug
Capsule 800 mg TID, oral
What is being measured
The primary outcome is the main question the study is designed to answer.
tumor volume
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Sep 2008 (estimated)
- Study startSep 2000
- Primary completionAug 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeurofibromatosisStudies 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Investigators
- Dusica Babovic-Vuksanovic, M.D., Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00754780
- Sponsor's protocol ID
- 1835-99
- Other IDs
- MC0077, Mayo Pirfenidone Study
- Status last verified
- Mar 2012
- First submitted
- 16 Sep 2008
- Last updated
- 19 Mar 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Mar 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Clinical Trial of Pirfenidone for the Treatment of Patients With Neurofibromatosis Type I
- ClinicalTrials.gov ID
- NCT00754780
- Acronym / sponsor ID
- 1835-99
- Conditions
- Neurofibromatosis
- Keywords
- NF1, plexiform neurofibroma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- Sep 2000
- Primary completion
- Aug 2004 (actual)
- First posted
- 18 Sep 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study is a phase II, open label trial of oral Pirfenidone in 24 adult patients with neurofibromatosis type 1.
Pirfenidone is a new, broad-spectrum anti-fibrotic drug, with proven in vitro and in vivo negative effects on fibroblast growth and collagen matrix synthesis. Human studies indicate promising therapeutic effects in arresting and reversing fibrosis in a variety of different conditions, where the excessive formation of fibrous tissue is a major pathogenic mechanism. Since the fibrous tissue is a significant component of neurofibroma, reduction of fibrosis could diminish tumor progression and lead to tumor shrinkage. Therefore, Pirfenidone is an excellent candidate for the treatment of plexiform neurofibromas and surgically unresectable tumors in patients with NF1.
Detailed description
Specific aims of this study are:
- To evaluate efficacy of Pirfenidone in NF1 patients with disfiguring or disabling plexiform neurofibroma (PN) and spinal neurofibromas (SN)
- To determine the acute, subacute and chronic toxicity of Pirfenidone in patients with NF1.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Diagnosis of neurofibromatosis type I, based on clinical criteria (NIH Consensus Development Conference, 1988). Tumors will not be confirmed histologically, since the biopsy could cause a change in tumor growth and such interfere with effect of Pirfenidone treatment.
- Male or female patients
- Age 18 years old
- All patients should be mentally capable of signing the consent form or should have a legal guardian to provide consent
- Patients who are experiencing symptoms from neurofibromatous lesions and who refuse surgery or are not good surgical candidates, such as those with plexiform neurofibroma who are experiencing significant discomfort, disfigurement or nerve compression or
Key exclusion criteria (6 listed)
- Tumors for which surgical removal could lead to permanent (or long-term) relief of symptoms
- Patients with open skin lesions and patients for whom surgery is being contemplated or who had surgery less than 4 weeks from starting treatment
- Patients for whom biopsy is warranted for suspected malignancies
- Individuals younger than 18 years
- Pregnant and lactating women
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 |
|---|---|---|
| PirfenidoneCapsule 800 mg TID, oral | Drug | – |
Primary outcome
tumor volume
Time frame: 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Mar 2012.
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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.
Dusica Babovic-Vuksanovic, M.D. principal investigator
Mayo Clinic
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pirfenidone in people with Neurofibromatosis, run by Mayo Clinic.
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 6 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 19 Mar 2012. Status can change between updates, so confirm with the study contact before making plans.
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
tumor volume (time frame: 24 months). 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 19 Mar 2012.