NCT04859829

Autoimmune Gastrointestinal Dysmotility: Symptoms, Pathogenesis and Treatment

Terminated Last updated 13 March 2023

What this trial tests

trial testing Current Intravenous Immunoglobulin (IVIG) treatment in Dysmotility Syndrome in 1 participant. Terminated before completion.

Timeline

22 April 2021

Primary endpoint
29 September 2022

29 September 2022

Quick facts

Lead sponsor	Johns Hopkins University
Status	Terminated
Study type	OBSERVATIONAL
Enrollment	1
Start date	22 April 2021
Primary completion	29 September 2022
Estimated completion	29 September 2022
Sites	1 location across United States

Drugs / interventions tested

• Current Intravenous Immunoglobulin (IVIG) treatment
• No Intravenous Immunoglobulin (IVIG) treatment

Conditions studied

• Dysmotility Syndrome — all drugs for Dysmotility Syndrome →
• Autoimmunity — all drugs for Autoimmunity →

Sponsor

Johns Hopkins University

Who can join

15 and older, any sex, with Dysmotility Syndrome or Autoimmunity. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Autoimmune gastrointestinal dysmotility syndromes are poorly understood, and often difficult to treat because the underlying pathogenesis is unclear. Refractory symptoms result in an impaired quality of life. The presence of positive serum autoantibodies to peripheral nervous system gangliosides and glycoproteins is suggestive of a possible mechanism. Immunomodulator treatments have shown benefit in case reports and case series but standardized data for treatment response is lacking. Therefore, our primary aims are to further characterize this syndrome in terms of symptoms, laboratory testing, pathology, and assess treatment response of immunomodulator therapy. Our research plan involves identifying this subset of patients with autoimmune gastrointestinal dysmotility and dysautonomia, and studying them as they are managed by their gastroenterologists.The study team will administer symptom-based questionnaires in a systematic manner to assess the clinical trajectory of this population and treatment response. The investigators will also analyze laboratory values (antibody titers, tilt testing, inflammatory markers) and study pathology specimens (enteric and skin biopsies) obtained from this cohort to gain a deeper understanding of the pathogenesis of their disease.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Joint Hypermobility, Autonomic Dysfunction, Gastrointestinal Dysfunction, and Autoimmune Markers: Clinical Associations and Response to Intravenous Immunoglobulin Therapy.
   Pasricha PJ, McKnight M, Villatoro L, Barahona G, et al · · 2024 · cited 7× · PMID 38912927 · DOI 10.14309/ajg.0000000000002910
2. A Syndrome of Joint Hypermobility, Autonomic Dysfunction, Gastrointestinal Dysfunction and Autoimmune markers (JAG-A): Clinical Associations and Response to Intravenous Immunoglobulin Therapy
   Pasricha PJ, McKnight M, Villatoro L, Barahona G, et al · · 2023 · DOI 10.1101/2023.10.01.23296388