A Study to Evaluate the Feasibility of a Physiologic Biomarker to Assess Pain and Other Sensory Problems Using Pupillometry in Participants With Neurofibromatosis Type 1 (NF1)

NCT ID: NCT06507748

Last Updated: 2025-12-26

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

NOT_YET_RECRUITING

Clinical Phase

NA

Total Enrollment

70 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-12-30

Study Completion Date

2026-05-01

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

Background:

Neurofibromatosis type 1 (NF1) is a genetic condition that causes tumors to grow along the nerves in the skin, brain, and other parts of the body. People with NF1 often have pain and may experience other abnormal sensations like itching, numbness, or tingling. These symptoms can affect their daily life. Researchers want to learn more about these symptoms and find better ways to measure pain in people with NF1.

Objective:

To learn if a device called the AlgometRx Nociometer(Registered trademark) is effective in measuring pain or other abnormal sensations in people with NF1.

Eligibility:

People aged 1 year and older with NF1.

Design:

Individuals can have up to 3 assessments completed in person. Each assessment may last up to 1.0 to 1.5 hours.

Individuals will be screened. They will complete questionnaires about their health and how bad their pain is. If participants are having blood drawn for other reasons, some additional samples may be used in this study.

The AlgometRx Nociometer includes an electrode that will be placed onto a finger or a toe. The electrode will send non-painful electrical signals to activate nerves in the finger or toe. At the same time, a camera will be used to record changes in the pupil of the eye. The test will be done on all 4 of the participant s limbs; however, researchers may skip 1 or more limbs for various reasons. This test takes about 10 seconds to complete with at least a one-minute rest between testing different limbs.

Individuals will be asked to do a 2nd assessment with the AlgometRx Nociometer that may be done 1 hour later but no more than 72 hours after the first assessment. Participants who will be returning for another visit can opt to do a 3rd assessment that will be done at least 4 weeks but not more than 18 months after the 1st....

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Background:

* Chronic pain is a common and often debilitating symptom of neurofibromatosis 1 (NF1) that is related to multiple tumor (e.g., plexiform neurofibromas; PN) and non-tumor (e.g., headaches) manifestations.
* Patient-reported outcome (PRO) measures have been used to assess pain in PN trials; however, few are validated for NF populations. In addition, the subjective measurement of pain symptoms can be difficult particularly in young children, those with learning disabilities, and due to patients having various types of NF-related pain and other sensory problems.
* There is a critical unmet need for a simple, non-invasive, and objective measure of pain and other sensory problems for individuals with NF1.
* AlgometRx Nociometer (Registered Trademark) is a novel integration of infrared video pupillometry and neuroselective stimulation, and the outcome measure obtained from an assessment with this device is the Neuropathy Index. The use of the AlgometRx Nociometer (Registered Trademark) in studies was determined to be non-significant risk (NSR) by the Children s National Hospital (CNH) Institutional Review Board (IRB) based on similar technologies that have been defined by the Food and Drug Administration (FDA) to be NSR.
* In addition to the early detection and targeted clinical management and monitoring of pain and nociceptive processing in patients with NF1, this device also may help us learn more about the underlying mechanisms of their pain, as well as their symptoms of numbness and tingling (paresthesia), muscle weakness, loss of reflexes/hyperreflexia, and altered sensation.
* This novel biomarker could fill a critical gap for an objective, simple, and sensitive assessment to quantify and monitor NF-related pain as a physiologic measure for clinical monitoring and as potential surrogate marker for response in treatment trials.

Objective:

-To describe the feasibility of using the AlgometRx Nociometer (Registered Trademark) device to conduct a physiologic assessment of pain in different age groups of participants with NF1.

Eligibility:

* Participants must have a history of clinical or genetic diagnosis of NF1
* Age \>= 1 year
* At least one eye with an intact pupillary reflex
* At least one digit (finger or toe) without open wounds for application of the device
* Must understand English or Spanish
* Participants who are \< 18 years must have a caregiver willing to help the child engage in study procedures, assist with fitting the AlgometRx Nociometer (Registered Trademark) device, and complete the observer reported (ObsRO) measures. Note: the caregiver of a child participant \>= 5 years old must be able to understand English or Spanish, the caregiver of a child participant 1-4 years old must be able to understand English (to help complete the observational pain measure for the younger children that is only available in English).

Design:

* The design will be a multi-site diagnostic pilot study to describe the feasibility and acceptability and to evaluate the initial psychometric properties (reliability, validity, sensitivity to change) of a novel objective pain measurement device \[AlgometRx Nociometer (Registered Trademark)\] for use in future clinical trials for participants with NF1. NCI is the lead site and Children's National Hospital (CNH) is the participating site; both sites will enroll participants with NF1 in this study.
* A cross-sectional design will be used to describe the acceptability of the assessment with the AlgometRx Nociometer (Registered Trademark) device as well as construct validity of the Neuropathy Index. A longitudinal design will be used to assess test-retest reliability and sensitivity to change of the Neuropathy Index.
* The sample will consist of 55 evaluable participants who will be placed into 5 cohorts defined by age (1-4; 5-7; 8-12; 13-17; \>= 18 years) with approximately 11 participants in each group (plus/minus 3) and total enrollment of 70 participants.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Neurofibromatosis Type 1

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

Neurofibromatosis Type 1 Plexiform Neurofibromas Pain Sensation Clinical outcomes Objective measurement Biomarker

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

DEVICE_FEASIBILITY

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

1/Feasibility and longitudinal assessments

Evaluation with the AlgometRx device and patient- and observer-reported outcome measures

Group Type OTHER

AlgometRx Nociometer

Intervention Type DEVICE

Novel objective pain measurement device

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

AlgometRx Nociometer

Novel objective pain measurement device

Intervention Type DEVICE

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* History of clinical or genetic diagnosis of NF1 as per the 2021 revised diagnostic criteria
* Age \>= 1 year
* At least one digit (finger or toe) without open wounds for application of the device
* Individuals must understand English or Spanish
* Individuals who are \< 18 years must have a caregiver willing to help the child engage in study procedures, assist with fitting the AlgometRx Nociometer (Registered Trademark) device, and complete the observer reported (ObsRO) measures. Note: the caregiver of a child participant \>= 5 years old must be able to understand English or Spanish, the caregiver of a child participant 1-4 years old must be able to understand English (to help complete the observational pain measure for the younger children that is only available in English)
* Ability of individual or parent/guardian to understand and the willingness to sign a written informed consent document

Exclusion Criteria

* History of eye pathology which precludes pupillometry, such as problems with pupillary reflex, blindness or inability to open at least one eye fully for evaluation
* Individuals with chronic use of medication that specifically affects their pupillary response, such as atropine-containing eye drops
* Uncontrolled intercurrent illness evaluated by medical history that would potentially increase in risk of participant
Minimum Eligible Age

1 Year

Maximum Eligible Age

120 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

National Cancer Institute (NCI)

NIH

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Pamela L Wolters, Ph.D.

Role: PRINCIPAL_INVESTIGATOR

National Cancer Institute (NCI)

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

National Institutes of Health Clinical Center

Bethesda, Maryland, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Pamela L Wolters, Ph.D.

Role: CONTACT

Phone: (240) 760-6035

Email: [email protected]

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

National Cancer Institute Referral Office

Role: primary

Related Links

Access external resources that provide additional context or updates about the study.

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

001826-C

Identifier Type: -

Identifier Source: secondary_id

10001826

Identifier Type: -

Identifier Source: org_study_id