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SchwannomatosisPhase 2NCT04163419

Phase 2 Study of Tanezumab in Subjects With Moderate to Severe Pain Due to Schwannomatosis

A Phase 2 study testing Tanezumab in people with Schwannomatosis and Pain, run by Massachusetts General Hospital at 1 site in United States.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

The primary objective of this study is to determine whether the administration of tanezumab, an anti-nerve growth factor (NGF) antibody, improves pain relief in schwannomatosis patients receiving background non-NSAID therapy. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Schwannomatosis, Pain
What is being tested?
Tanezumab
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
9 participants
Where
1 siteUnited States
Study dates
30 Apr 2020to 30 Sep 2024
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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patients must have a confirmed diagnosis schwannomatosis by fulfilling either clinical or molecular diagnosis.
  • Age ≥ 18 years. Patients < 18 years are excluded since the safety profile of tanezumab in this population has not been determined.
  • ECOG performance status ≤2 or Karnofsky ≥60%
  • Participants must have normal organ and marrow function as defined per the full protocol
  • The subject's weight must be≥ 45 kg at Screening.
  • The subject must be willing to avoid prohibited pain medications (including non-steroidal anti-inflammatory drugs) throughout the duration of the study except as permitted per Protocol.
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  • Subject must have moderate to severe pain secondary to schwannomatosis, defined as Score ≥5 on the Numeric Rating Scale-11 (NRS-11) at Screening.
  • Subject must have failure, intolerance, or contraindication to at least three standard of care therapies:
  • Documented history indicating that NSAID therapy has not provided adequate pain relief or subject is unable to take NSAIDs due to contraindication or inability to tolerate.
  • Documented history indicating that opioid treatment has not provided adequate pain relief or subject is unwilling to take opioids, or unable to take opioids due to contraindication or inability to tolerate
  • Documented history indicating that neuropathic pain medications, such as gabapentin, pregabalin, or others, have not provided adequate pain relief or subject is unable to take these treatments due to contraindication or inability to tolerate.
  • Female subjects of childbearing potential and at risk for pregnancy (e.g., not abstinent) must agree to use 2 highly effective methods of contraception throughout the study and for 112 days (16 weeks) after the last dose of assigned subcutaneous study medication.

Who cannot join

  • Subjects with any of the following criteria: evidence of bilateral vestibular schwannomas on imaging, a known germline pathogenic NF2 mutation, a first-degree relative who meets diagnostic criteria for NF2, or have schwannomas limited to a previous radiation field.
  • Subjects with intracranial meningioma associated with cerebral edema on neuroimaging. Note: presence of intracranial meningioma itself is not an exclusion criterion.
  • Subjects who have had surgery, chemotherapy or radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) for treatment of a painful schwannomatosis-related tumor prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier.
  • Participation in other studies involving investigational drug(s) (Phases 1-4) within 30 days (or 90 days for biologics) before Screening and/or during study participation.
  • Subjects receiving anticoagulation to treat an underlying medical condition.
  • Subject has a history of allergic or anaphylactic reaction to a therapeutic or diagnostic monoclonal antibody or IgG fusion protein.
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  • The subject's pain is related to a non-schwannomatosis cause such as prior cancer therapy, infection, bowel obstruction/perforation, spinal cord compression, or fracture or impending fracture of weight bearing bone.
  • The subject has a diagnosis of malignancy in the last 3 years (except for Gleason 6 prostate cancer, basal cell carcinoma or carcinoma in situ).
  • Use of concurrent adjuvant analgesics such as serotonin norepinephrine reuptake inhibitors (SNRI), tricyclic antidepressants, anticonvulsant medication, or muscle relaxants (unless the drugs were started at least 30 days prior to Screening and are maintained at a stable dose).
  • Use of concurrent analgesic non-steroidal anti-inflammatory drugs (NSAIDs, including selective COX-2 inhibitors) unless the subject is expected to be able to discontinue these medications at least 2 weeks prior to treatment. Note: Subjects who take daily low dose aspirin (≤ 325 mg as per local prescribing practice) therapy for cardiovascular prophylaxis are not excluded from participation.
  • Diagnosis of osteoarthritis of the knee or hip as defined by the American College of Rheumatology (ACR) combined clinical and radiographic criteria; Radiographic criteria will be assessed by the Central Reader.
  • Use of concurrent corticosteroids (except for inhaled or topical corticosteroids as needed for management of ongoing pulmonary or dermatologic conditions)
  • Subjects considered unfit for surgery, defined as Grade >3 on the American Society of Anesthesiologists (ASA) physical classification system for surgery or subjects who would not be willing to undergo joint replacement surgery if required.
  • Subjects with symptoms and radiographic findings (i.e. joint space narrowing, osteophytes) consistent with osteoarthritis in the shoulder.
  • History of significant trauma or surgery to a major joint (e.g. hip, knee or shoulder) within one year prior to Screening.
  • A history of osteonecrosis or osteoporotic fracture (i.e., a subject with a history of osteoporosis and a minimally traumatic or atraumatic fracture).
  • Radiographic (x-ray) evidence of any of the following conditions as determined by the central radiology reviewer at Screening: 1) rapidly progressive osteoarthritis, 2) atrophic or hypotrophic osteoarthritis, 3) subchondral insufficiency fracture, 4) spontaneous osteonecrosis of the knee (SPONK), 5) osteonecrosis, or 6) pathologic fracture.
  • Subjects who have evidence of orthostatic hypotension based upon replicate orthostatic blood pressure measurements at Screening.
  • Subjects with a total impact score of >7 on the Survey of Autonomic Symptoms (SAS) at Screening.
  • Diagnosis of a transient ischemic attack in the 6 months prior to Screening or diagnosis of stroke with residual deficits (e.g., aphasia, substantial motor or sensory deficits), that would preclude completion of required study activities.
  • History, diagnosis, or signs and symptoms of clinically significant neurological
  • Subjects with a past history of carpal tunnel syndrome (CTS) with signs or symptoms of CTS in the one year prior to Screening.
  • Subject with a history of significant alcohol, analgesic, or narcotic substance abuse within the six months prior to Screening.
  • Subject who, in the judgement of the investigator, is expected to require a surgical procedure during the duration of the study.
  • Previous exposure to exogenous nerve growth factor or to an anti-nerve growth factor antibody.
  • Subjects who are investigational site staff members directly involved in the conduct of the trial and their family members, site staff members otherwise supervised by the Investigator, or subjects who are Pfizer employees directly involved in the conduct of the trial
  • Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
  • Pregnant females; breastfeeding females; females of childbearing potential not using two (2) methods of highly effective contraception or not agreeing to continue two (2) methods of highly effective contraception for 112 days (16 weeks) after last dose of investigational product.
  • Any subject who, in the judgement of the investigator, is deemed inappropriate for participation in the study.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

Other trials in United States

About this study

Official title: A Phase 2 Randomized, Double-blind, Placebo-Controlled Study of the Analgesic Efficacy and Safety of the Subcutaneous Administration of the Anti-NGF Antibody Tanezumab in Subjects With Moderate to Severe Pain Due to Schwannomatosis

The primary objective of this study is to determine whether the administration of tanezumab, an anti-nerve growth factor (NGF) antibody, improves pain relief in schwannomatosis patients receiving background non-NSAID therapy.

Read the detailed description

Schwannomatosis is characterized by the predisposition to develop multiple schwannomas and, less commonly, meningiomas. Pain is the most frequent symptom reported by these patients, with 68% experiencing chronic pain.

The investigators propose to test the efficacy and tolerability of tanezumab as a treatment for schwannomatosis patients with chronic pain who have had inadequate pain relief in a randomized, placebo-controlled trial, which could form the basis of a larger, randomized controlled trial in the future.

The study is designed with a total duration of 281 days (40 weeks) and will consist of four periods: Pre-treatment, Double-Blind Treatment, Single Arm Treatment, and 24-Week Safety Follow-up. The Pre-Treatment Period lasting up to 30 days, followed by a Double-Blind Treatment Period lasting up to 8 weeks, followed by a Single Arm Treatment Period lasting up to 8 weeks, and a 24-Week Safety Follow-Up Period ending with the End of Study Visit at Week 40.

Being tested
Tanezumab, Placebo
Keywords
schwannoma, nerve growth factor, antibody, NGF
Medical subject headings
Schwannomatosis, Pain, Neurilemmoma, Hereditary Sensory and Autonomic Neuropathies

What participants receive

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

  • TanezumabDrug

    Also called PF-04383119

    Tanezumab 10 mg SC

  • PlaceboDrug

    Placebo 10 mg SC

Study groups in NCT04163419
Study groupRoleWhat they receive
Arm A (treatment)ExperimentalTanezumab 10 mg SC administered on day 1 and Day 57 (± 4 days)
Receives: Drug: Tanezumab
Arm B (placebo then treatment)Placebo comparatorPlacebo SC (to match tanezumab SC) administered on Day 1 and tanezumab 10 mg SC on Day 57 (± 4 days)
Receives: Drug: Tanezumab; Drug: Placebo

What is being measured

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

  1. Change in pain level

    Change in worst pain level during past week from baseline to week 8 measured by NRS-11

    Measured: 8 weeks

Secondary outcomes (2)

  1. Frequency of AEs

    Frequency of AEs related to treatment with Tanezumab

    Measured: 40 weeks

  2. Incidence of orthostatic hypotension

    Incidence of orthostatic hypotension using postural changes in blood pressure in addition to mean changes in postural blood pressure

    Measured: 40 weeks

Timeline

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

  1. First posted14 Nov 2019
  2. Study start30 Apr 2020
  3. Primary completion30 Sep 2022
  4. Study completion30 Sep 2024 (estimated)

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
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
9 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04163419
Sponsor's protocol ID
2019P003201
Status last verified
Mar 2024
First submitted
12 Nov 2019
Last updated
13 Mar 2024
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Da JLW, Merker VL, Jordan JT, Ly KI, Muzikansky A, Parsons M, Wolters PL, Xu L, Styren S, Brown MT, Plotkin SR. Design of a randomized, placebo-controlled, phase 2 study evaluating the safety and efficacy of tanezumab for treatment of schwannomatosis-related pain. Contemp Clin Trials. 2022 Oct;121:106900. doi: 10.1016/j.cct.2022.106900. Epub 2022 Aug 26.

    PubMed 36038003

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Tanezumab in people with Schwannomatosis and Pain, run by Massachusetts General Hospital 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 12 inclusion and 29 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: status not confirmed, as of the sponsor's last update on 13 Mar 2024. 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. Some participants in this study may receive a placebo. 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?

Results are not available in the source record.

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 pain level (time frame: 8 weeks). 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 NCT04163419, 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 13 Mar 2024.

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