RECRUITING

Evaluating Efgartigimod in Patients With Guillain-Barré Syndrome

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The goal of this clinical trial is to evaluate the safety and effectiveness of Efgartigimod in patients with Guillain-Barre syndrome (GBS). The main questions it aims to answer are: * Is Efgartigimod a safe treatment option for GBS patients? * Does treatment with Efgartigimod improve patient outcomes? In addition to standard-of-care procedures and assessments, participants will: * Undergo seven blood draws during hospitalization and in four follow-up study visits to evaluate the concentration of neurofilament light chain, a protein that is elevated in patients with Guillain-Barré syndrome. The presence of neurofilament light chain is believed to be indicative of damage to the nervous system, with higher levels resulting from greater damage. * Complete the Columbia Suicide Severity Rating Scale (C-SSRS) to monitor any suicidal ideation or behaviors during the course of the study.

Official Title

Phase 2, Randomized, Patient- and Rater-blinded Single-site Trial Evaluating Safety and Efficacy of Efgartigimod in Patients With Guillain-Barré Syndrome.

Quick Facts

Study Start:2024-09-10

Study Completion:2027-05

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05701189

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
* Provision of signed and dated informed consent form * Stated willingness to comply with all study procedures and availability for the duration of the study * Male or female, aged 18 years or older * Have a diagnosis of GBS according to the National Institute of Neurological Disorders and Stroke Diagnostic Criteria for Guillain-Barré Syndrome * Onset of GBS-related weakness ≤14 days prior to infusion * GBS-DS score of 3, 4, or 5	* Pregnant and lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing. Women of childbearing potential should have a negative serum pregnancy test at Screening and a negative urine pregnancy test at Baseline prior to administration of IMP. Note: Women of childbearing potential should use a highly effective method of contraception (i.e., pregnancy rate of less than 1% per year) during the trial and for 90 days after the last administration of the IMP. They must be on a stable regimen, for at least 1 month, of combined estrogen and progestogen hormonal contraception with inhibition of ovulation, progestogen-only hormonal contraception associated with inhibition of ovulation, intrauterine device (IUD), intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomized partner, or agree upon continuous abstinence from heterosexual sexual contact. * Male patients who are sexually active and do not intend to use effective methods of contraception (as mentioned above) during the trial or within 90 days after the last dosing or male patients who plan to donate sperm during the trial or within 90 days after the last dosing. Note: Sterilized male patients who have had vasectomy with documented aspermia post-procedure, or male patients who have a partner of non-childbearing potential, can be included. * GBS DS of 2 or less. * Patients with any known severe bacterial, viral or fungal infection or any major episode of infection that required hospitalization or injectable antimicrobial therapy in the last 8 weeks prior to Screening. * Patients with more than 14 days after onset of symptoms. * Patients with known IgG deficiency. * Patients with recurrent GBS. * Use of investigational drug within 3 months or 5 half-lives of the drug (whichever is longer) prior to Screening. * Patients who have a history of malignancy, including malignant thymoma, or myeloproliferative or lymphoproliferative disorders, unless deemed cured by adequate treatment with no evidence of recurrence for ≥ 3 years before Screening. Patients with completely excised non-melanoma skin cancer (such as basal cell carcinoma or squamous cell carcinoma) or cervical carcinoma in situ would be permitted at any time. * Patients with clinical evidence of other significant serious disease or patients who underwent a recent major surgery, which could confound the results of the trial or put the patient at undue risk. Patients with renal/hepatic function impairment can be included.

Inclusion Criteria

Exclusion Criteria

* Provision of signed and dated informed consent form
* Stated willingness to comply with all study procedures and availability for the duration of the study
* Male or female, aged 18 years or older
* Have a diagnosis of GBS according to the National Institute of Neurological Disorders and Stroke Diagnostic Criteria for Guillain-Barré Syndrome
* Onset of GBS-related weakness ≤14 days prior to infusion
* GBS-DS score of 3, 4, or 5

* Pregnant and lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing. Women of childbearing potential should have a negative serum pregnancy test at Screening and a negative urine pregnancy test at Baseline prior to administration of IMP. Note: Women of childbearing potential should use a highly effective method of contraception (i.e., pregnancy rate of less than 1% per year) during the trial and for 90 days after the last administration of the IMP. They must be on a stable regimen, for at least 1 month, of combined estrogen and progestogen hormonal contraception with inhibition of ovulation, progestogen-only hormonal contraception associated with inhibition of ovulation, intrauterine device (IUD), intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomized partner, or agree upon continuous abstinence from heterosexual sexual contact.
* Male patients who are sexually active and do not intend to use effective methods of contraception (as mentioned above) during the trial or within 90 days after the last dosing or male patients who plan to donate sperm during the trial or within 90 days after the last dosing. Note: Sterilized male patients who have had vasectomy with documented aspermia post-procedure, or male patients who have a partner of non-childbearing potential, can be included.
* GBS DS of 2 or less.
* Patients with any known severe bacterial, viral or fungal infection or any major episode of infection that required hospitalization or injectable antimicrobial therapy in the last 8 weeks prior to Screening.
* Patients with more than 14 days after onset of symptoms.
* Patients with known IgG deficiency.
* Patients with recurrent GBS.
* Use of investigational drug within 3 months or 5 half-lives of the drug (whichever is longer) prior to Screening.
* Patients who have a history of malignancy, including malignant thymoma, or myeloproliferative or lymphoproliferative disorders, unless deemed cured by adequate treatment with no evidence of recurrence for ≥ 3 years before Screening. Patients with completely excised non-melanoma skin cancer (such as basal cell carcinoma or squamous cell carcinoma) or cervical carcinoma in situ would be permitted at any time.
* Patients with clinical evidence of other significant serious disease or patients who underwent a recent major surgery, which could confound the results of the trial or put the patient at undue risk. Patients with renal/hepatic function impairment can be included.

Contacts and Locations

Study Contact

Kelsey Moulton

CONTACT

267-593-9459

kelsey.moulton@pennmedicine.upenn.edu

Principal Investigator

Madeline Singer, MD

PRINCIPAL_INVESTIGATOR

Staff Physician and Neuromuscular Research Fellow

Chafic Karam, MD

PRINCIPAL_INVESTIGATOR

Staff Physician and Associate Professor of Clinical Neurology

Colin Quinn, MD

STUDY_DIRECTOR

Staff Physician and Associate Professor of Clinical Neurology

Study Locations (Sites)

Hospital of University of Pennsylvania

Philadelphia, Pennsylvania, 19104

United States

Collaborators and Investigators

Sponsor: Chafic Karam

Madeline Singer, MD, PRINCIPAL_INVESTIGATOR, Staff Physician and Neuromuscular Research Fellow
Chafic Karam, MD, PRINCIPAL_INVESTIGATOR, Staff Physician and Associate Professor of Clinical Neurology
Colin Quinn, MD, STUDY_DIRECTOR, Staff Physician and Associate Professor of Clinical Neurology

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-09-10

Study Completion Date2027-05

Study Record Updates

Study Start Date2024-09-10

Study Completion Date2027-05

Terms related to this study

Additional Relevant MeSH Terms

Guillain-Barre Syndrome