HistiocytosisNCT05915208
Histiocytic Disorder Follow-up Study
An observational study of Histiocytosis and Langerhans Cell Histiocytosis, run by University of Alabama at Birmingham at 1 site in United States.
Trial summary
The purpose of the study is to describe the burden of chronic health conditions, psychological dysfunction, chronic pain, healthcare utilization, worse health-related quality of life, overall mortality, and cause-specific mortality among individuals with histiocytic disorders Read more about this study
- Who is it for?
- Ages 0 to 89 years
- Conditions the study is for
- Histiocytosis, Langerhans Cell Histiocytosis, Erdheim-Chester Disease
- How many people
- 6,000 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Sep 2022to Dec 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 0 to 89 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Individuals with diagnosis of histiocytic disorder are eligible to participate, including family members (next of kin) of deceased individuals.
Who may be able to join
- diagnosis of histiocytic disorder at any age
- Langerhans cell histiocytosis,
- Erdheim-Chester disease,
- Rosai-Dorfman disease,
- Xanthogranuloma,
- Mixed histiocytosis
- Malignant histiocytosis (Histiocytic sarcoma, langerhans cell sarcoma, interdigitating cell sarcoma)
- Hemophagocytic lymphohistiocytosis
Who cannot join
- None
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of Alabama at Birmingham
Birmingham, Alabama, 35233
Gaurav Goyal, MD, 1-866-438-1640, histio@uabmc.edu
About this study
The purpose of the study is to describe the burden of chronic health conditions, psychological dysfunction, chronic pain, healthcare utilization, worse health-related quality of life, overall mortality, and cause-specific mortality among individuals with histiocytic disorders
Read the detailed description
Background
Over the last decade, major advances have occurred in histiocytic disorders with the discovery of MAPK-ERK pathway mutations leading to targeted therapeutics using BRAF- and MEK-inhibitors. However, there is a lack of large studies informing the burden of morbidity and mortality among people with histiocytosis. Institutional studies in pediatric LCH suggest that survivors suffer from long-term impairment of health related quality of life, cognitive dysfunction, pituitary dysfunction, and hearing difficulties in 20-50% cases. Studies from the investigative team and others have also shown a high incidence of acute myeloid leukemia and other second primary malignancies in pediatric and adult LCH. Chronic medical conditions may arise as a function of the disease biology or due to cancer therapy, as seen in other hematologic malignancies.
Design
Retrospective cohort study aimed at determining the risk of chronic health conditions and cause-specific mortality in pediatric and adult patients with histiocytic disorders. The study will include patients from the Histiocytosis Association registry and other institutions including UAB (n~6000). The participants will complete a validated questionnaire capturing details of chronic health conditions, health related quality of life, cognitive/psychological function, and healthcare utilization.
Future directions
The results from our study will be instrumental in formulating follow-up guidelines for histiocytic disorders and developing targeted survivorship programs to improve overall outcomes.
- Conditions
- Histiocytosis, Langerhans Cell Histiocytosis, Erdheim-Chester Disease, Rosai Dorfman Disease, Xanthogranuloma, Malignant Histiocytoses
- Keywords
- survivorship, long-term, targeted therapy, second cancer
- Medical subject headings
- Histiocytosis, Histiocytosis, Langerhans-Cell, Erdheim-Chester Disease, Histiocytosis, Sinus, Histiocytic Sarcoma, Neoplasms, Second Primary
What is being measured
The primary outcome is the main question the study is designed to answer.
Chronic health conditions diagnosed after LCH diagnosis graded using the Common Terminology Criteria for Adverse Events (CTCAE), v5.0 (https://ctep.cancer.gov)
The CTCAE criteria for CHCs distinguishes each acute and chronic condition (including SPMs) in persons with cancer from grades 1 through 4 with unique clinical descriptions of severity (grade 1, mild; grade 2, moderate; grade 3, severe; grade 4, life-threatening/disabling). The main outcome will be the number of distinct CTCAE grade 3-4 conditions among LCH cases compared with unaffected (non-cancer) controls.
Measured: From diagnosis of histiocytic disorder to age at completion of survey, assessed up to 200 months
All-cause and cause-specific mortality
Causes of death will be grouped into primary cancer (LCH)-related mortality (PCRM) and non-primary cancer-related mortality (NPCRM - death from all causes other than the index LCH diagnosis) by two independent investigators.
Measured: 2 years preceding death
Secondary outcomes (1)
Factors associated with new-onset morbidity
We will build a prediction model that has a good discrimination for predicting CHC, SPMs, and psychological outcomes separately using data from the LCH (case) cohort.
Measured: From diagnosis to age at completion of survey, assessed up to 200 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2023
- Study start1 Sep 2022
- Primary completionJul 2027 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HistiocytosisStudies of the same condition, open and under way.
- More trials run by University of Alabama at BirminghamThe 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
- Retrospective
- Enrolment
- 6,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Alabama at Birmingham
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Gaurav Goyal, University of Alabama at Birmingham
- Investigators
- Gaurav Goyal, MD, Principal investigator, University of Alabama at Birmingham
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05915208
- Sponsor's protocol ID
- IRB-300008744
- Other IDs
- UAB
- Status last verified
- Jun 2026
- First submitted
- 25 May 2023
- Last updated
- 12 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 12 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Histiocytic Disorder Follow-up Study
- ClinicalTrials.gov ID
- NCT05915208
- Acronym / sponsor ID
- IRB-300008744
- Conditions
- Histiocytosis, Langerhans Cell Histiocytosis, Erdheim-Chester Disease, Rosai Dorfman Disease, Xanthogranuloma, Malignant Histiocytoses
- Keywords
- survivorship, long-term, targeted therapy, second cancer
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 6,000 participants (estimated)
- Lead sponsor
- University of Alabama at BirminghamUniversity, hospital or other
- Study start
- 1 Sep 2022 (actual)
- Primary completion
- Jul 2027 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 22 Jun 2023 (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.
The purpose of the study is to describe the burden of chronic health conditions, psychological dysfunction, chronic pain, healthcare utilization, worse health-related quality of life, overall mortality, and cause-specific mortality among individuals with histiocytic disorders
Detailed description
Background
Over the last decade, major advances have occurred in histiocytic disorders with the discovery of MAPK-ERK pathway mutations leading to targeted therapeutics using BRAF- and MEK-inhibitors. However, there is a lack of large studies informing the burden of morbidity and mortality among people with histiocytosis. Institutional studies in pediatric LCH suggest that survivors suffer from long-term impairment of health related quality of life, cognitive dysfunction, pituitary dysfunction, and hearing difficulties in 20-50% cases. Studies from the investigative team and others have also shown a high incidence of acute myeloid leukemia and other second primary malignancies in pediatric and adult LCH. Chronic medical conditions may arise as a function of the disease biology or due to cancer therapy, as seen in other hematologic malignancies.
Design
Retrospective cohort study aimed at determining the risk of chronic health conditions and cause-specific mortality in pediatric and adult patients with histiocytic disorders. The study will include patients from the Histiocytosis Association registry and other institutions including UAB (n~6000). The participants will complete a validated questionnaire capturing details of chronic health conditions, health related quality of life, cognitive/psychological function, and healthcare utilization.
Future directions
The results from our study will be instrumental in formulating follow-up guidelines for histiocytic disorders and developing targeted survivorship programs to improve overall outcomes.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Ages
- 0 to 89 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Individuals with diagnosis of histiocytic disorder are eligible to participate, including family members (next of kin) of deceased individuals.
Key inclusion criteria (8 listed)
- diagnosis of histiocytic disorder at any age
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Chronic health conditions diagnosed after LCH diagnosis graded using the Common Terminology Criteria for Adverse Events (CTCAE), v5.0 (https://ctep.cancer.gov)
Time frame: From diagnosis of histiocytic disorder to age at completion of survey, assessed up to 200 months
The CTCAE criteria for CHCs distinguishes each acute and chronic condition (including SPMs) in persons with cancer from grades 1 through 4 with unique clinical descriptions of severity (grade 1, mild; grade 2, moderate; grade 3, severe; grade 4, life-threatening/disabling). The main outcome will be the number of distinct CTCAE grade 3-4 conditions among LCH cases compared with unaffected (non-cancer) controls.
All-cause and cause-specific mortality
Time frame: 2 years preceding death
Causes of death will be grouped into primary cancer (LCH)-related mortality (PCRM) and non-primary cancer-related mortality (NPCRM - death from all causes other than the index LCH diagnosis) by two independent investigators.
1 secondary outcome
Factors associated with new-onset morbidity
Time frame: From diagnosis to age at completion of survey, assessed up to 200 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jun 2026.
- Individual participant dataSharing planned: Undecided
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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.
Gaurav Goyal, MD principal investigator
University of Alabama at Birmingham
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 Histiocytosis and Langerhans Cell Histiocytosis, run by University of Alabama at Birmingham 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 8 inclusion and 1 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: looking for participants now, as of the sponsor's last update on 12 Jun 2026. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Gaurav Goyal, MD (1-866-438-1640, histio@uabmc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Chronic health conditions diagnosed after LCH diagnosis graded using the Common Terminology Criteria for Adverse Events (CTCAE), v5.0 (https://ctep.cancer.gov) (time frame: From diagnosis of histiocytic disorder to age at completion of survey, assessed up to 200 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 12 Jun 2026.