Dysmotility SyndromeNCT04859829
Autoimmune Gastrointestinal Dysmotility: Symptoms, Pathogenesis and Treatment
An observational study of Dysmotility Syndrome and Autoimmunity, run by Johns Hopkins University at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 15 years and older
- Conditions the study is for
- Dysmotility Syndrome, Autoimmunity
- What is being tested?
- Current Intravenous Immunoglobulin (IVIG) treatment and No Intravenous Immunoglobulin (IVIG) treatment
- Type of treatment
- Injection or infusion
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Apr 2021to 29 Sep 2022
- How long will I take part?
- 2 Years
- Phase
- Observational
- 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 15 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with autoimmune autonomic ganglionopathy (AAG) or autoimmune autonomic neuropathy. Postural orthostatic tachycardia syndrome (POTS) and gastrointestinal (GI) dysmotility.
Who may be able to join
- Males and females, Aged>15
- Evidence of enteric dysmotility with documented abnormality on objective testing which may include high resolution esophageal manometry, gastric emptying scintigraphy, SmartPill Wireless Motility Capsule, Sitz marker studies, antroduodenal manometry or anorectal manometry.
- English proficiency and literacy sufficient to sign consent form and fill out questionnaires.
- Previous Mayo paraneoplastic panel testing.
- Referring gastroenterologist has recommended intravenous immunoglobulin (IVIg) therapy.
Who cannot join
- Non-autoimmune causes of enteric dysmotility (diabetes, adrenal insufficiency, Parkinson's, thyroid, electrolytes, drugs, malignancy).
- Pregnancy as documented in EPIC with serum or urine Human chorionic gonadotropin (hCG) testing
- If participants believe that they are pregnant, they will need to notify a study physician who will order a serum or urine hCG test for pregnancy within EPIC and remove them from the study if the test results come back positive.
- Previous treatment with IVIg.
Trial locations and contacts
1 site in 1 country.
United States1 site
Johns Hopkins Bayview Medical Center
Baltimore, Maryland, 21224
About this study
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.
- Conditions
- Dysmotility Syndrome, Autoimmunity
- Being tested
- Current Intravenous Immunoglobulin (IVIG) treatment, No Intravenous Immunoglobulin (IVIG) treatment
- Medical subject headings
- Ciliary Motility Disorders, Autoimmune Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Current Intravenous Immunoglobulin (IVIG) treatmentOther
Questionnaires every two months for patients with a diagnosis of autoimmune dysmotility that are receiving Intravenous Immunoglobulin (IVIG) treatment.
No Intravenous Immunoglobulin (IVIG) treatmentOther
Questionnaires every two months for patients with a diagnosis of autoimmune dismotility that are not receiving Intravenous Immunoglobulin (IVIG) treatment.
| Study group | Role | What they receive |
|---|---|---|
| Patients with autoimmune dysmotility receiving IVIG infusions | Not described Receives: Other: Current Intravenous Immunoglobulin (IVIG) treatment | |
| Patients with autoimmune dysmotility without IVIG infusions | Not described Receives: Other: No Intravenous Immunoglobulin (IVIG) treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in upper gastrointestinal symptom severity as assessed by the Patient Assessment of Gastrointestinal Disorders-Symptom Severity Index
The Patient Assessment of Gastrointestinal Disorders-Symptom Severity Index (PAGI-SYM) is composed of 20 items and 6 sub-scales: heartburn/regurgitation (7 items), nausea/vomiting (3 items), postprandial fullness/early satiety (4 items), bloating (2 items), upper abdominal pain (2 items), and lower abdominal pain (2 items). Sub-scale scores are calculated by averaging across items comprising the sub-scale; scores vary from 0 (none or absent) to 5 (very severe).
Measured: Every two months up to 2 years.
Secondary outcomes (1)
Change in Quality of Life as assessed by the Patient Assessment of Upper Gastrointestinal Disorders-Quality of Life scale
The effect of Autoimmune Dysmotility symptoms on quality of life assessed by the Patient Assessment of Upper Gastrointestinal Disorders-Quality of Life (PAGI-QoL). The PAGI-QOL instrument consists of 30 items, each with response options based on a 6-point scale and with a recall period of the previous 2 weeks. The items are grouped into 5 dimensions: Daily Activities, Clothing, Diet and Food Habits, Relationship and Psychological Well-being and Distress. A score per dimension as well as a total score can be calculated. The PAGI-QOL scores range from 0 (lowest QoL) to 5 (highest QoL).
Measured: Every two months up to 2 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Apr 2021
- Study start22 Apr 2021
- Primary completion29 Sep 2022
- Study completion29 Sep 2022
Why it stopped: PI moved to another institution and closed study.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Dysmotility SyndromeStudies of the same condition, open and under way.
- More trials run by Johns Hopkins UniversityThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1 participants (actual)
- Target duration
- 2 Years
Sponsor and investigators
- Lead sponsor
- Johns Hopkins University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Pankaj J Pasricha, MD, Principal investigator, Johns Hopkins University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04859829
- Sponsor's protocol ID
- IRB00087869
- Status last verified
- Mar 2022
- First submitted
- 22 Apr 2021
- Last updated
- 13 Mar 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Pasricha PJ, McKnight M, Villatoro L, Barahona G, Brinker J, Hui K, Polydefkis M, Burns R, McMahan ZH, Gould N, Goodman B, Hentz J, Treisman G. Joint Hypermobility, Autonomic Dysfunction, Gastrointestinal Dysfunction, and Autoimmune Markers: Clinical Associations and Response to Intravenous Immunoglobulin Therapy. Am J Gastroenterol. 2024 Nov 1;119(11):2298-2306. doi: 10.14309/ajg.0000000000002910. Epub 2024 Jun 24.
PubMed 38912927
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Autoimmune Gastrointestinal Dysmotility: Symptoms, Pathogenesis and Treatment
- ClinicalTrials.gov ID
- NCT04859829
- Acronym / sponsor ID
- IRB00087869
- Conditions
- Dysmotility Syndrome, Autoimmunity
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: PI moved to another institution and closed study.
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Johns Hopkins UniversityUniversity, hospital or other
- Study start
- 22 Apr 2021 (actual)
- Primary completion
- 29 Sep 2022 (actual)
- Study completion
- 29 Sep 2022 (actual)
- First posted
- 26 Apr 2021 (actual)
- 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.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 2 Years
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients with autoimmune dysmotility receiving IVIG infusions | – | Other: Current Intravenous Immunoglobulin (IVIG) treatment |
| Patients with autoimmune dysmotility without IVIG infusions | – | Other: No Intravenous Immunoglobulin (IVIG) treatment |
Who may qualify
- Ages
- 15 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with autoimmune autonomic ganglionopathy (AAG) or autoimmune autonomic neuropathy. Postural orthostatic tachycardia syndrome (POTS) and gastrointestinal (GI) dysmotility.
Key inclusion criteria (5 listed)
- Males and females, Aged>15
- Evidence of enteric dysmotility with documented abnormality on objective testing which may include high resolution esophageal manometry, gastric emptying scintigraphy, SmartPill Wireless Motility Capsule, Sitz marker studies, antroduodenal manometry or anorectal manometry.
- English proficiency and literacy sufficient to sign consent form and fill out questionnaires.
- Previous Mayo paraneoplastic panel testing.
- Referring gastroenterologist has recommended intravenous immunoglobulin (IVIg) therapy.
Key exclusion criteria (4 listed)
- Non-autoimmune causes of enteric dysmotility (diabetes, adrenal insufficiency, Parkinson's, thyroid, electrolytes, drugs, malignancy).
- Pregnancy as documented in EPIC with serum or urine Human chorionic gonadotropin (hCG) testing
- Previous treatment with IVIg.
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 |
|---|---|---|
| Current Intravenous Immunoglobulin (IVIG) treatmentQuestionnaires every two months for patients with a diagnosis of autoimmune dysmotility that are receiving Intravenous Immunoglobulin (IVIG) treatment. | Other | Patients with autoimmune dysmotility receiving IVIG infusions |
| No Intravenous Immunoglobulin (IVIG) treatmentQuestionnaires every two months for patients with a diagnosis of autoimmune dismotility that are not receiving Intravenous Immunoglobulin (IVIG) treatment. | Other | Patients with autoimmune dysmotility without IVIG infusions |
Primary outcome
Change in upper gastrointestinal symptom severity as assessed by the Patient Assessment of Gastrointestinal Disorders-Symptom Severity Index
Time frame: Every two months up to 2 years.
The Patient Assessment of Gastrointestinal Disorders-Symptom Severity Index (PAGI-SYM) is composed of 20 items and 6 sub-scales: heartburn/regurgitation (7 items), nausea/vomiting (3 items), postprandial fullness/early satiety (4 items), bloating (2 items), upper abdominal pain (2 items), and lower abdominal pain (2 items). Sub-scale scores are calculated by averaging across items comprising the sub-scale; scores vary from 0 (none or absent) to 5 (very severe).
1 secondary outcome
Change in Quality of Life as assessed by the Patient Assessment of Upper Gastrointestinal Disorders-Quality of Life scale
Time frame: Every two months up to 2 years.
Results and safety
The study was stopped early (reason given: PI moved to another institution and closed study.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Mar 2023.
- Derived from this study: PubMed 38912927Pasricha PJ, McKnight M, Villatoro L, Barahona G, Brinker J, Hui K, Polydefkis M, Burns R, McMahan ZH, Gould N, Goodman B, Hentz J, Treisman G. Joint Hypermobility, Autonomic Dysfunction, Gastrointestinal Dysfunction, and Autoimmune Markers: Clinical Associations and Response to Intravenous Immunoglobulin Therapy. Am J Gastroenterol. 2024 Nov 1;119(11):2298-2306. doi: 10.14309/ajg.0000000000002910. Epub 2024 Jun 24.
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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.
Pankaj J Pasricha, MD principal investigator
Johns Hopkins University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Dysmotility Syndrome and Autoimmunity, run by Johns Hopkins University 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 5 inclusion and 4 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: stopped early, as of the sponsor's last update on 13 Mar 2023. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: PI moved to another institution and closed 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 upper gastrointestinal symptom severity as assessed by the Patient Assessment of Gastrointestinal Disorders-Symptom Severity Index (time frame: Every two months up to 2 years.). 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 NCT04859829, 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 2023.