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Condition (MeSH)

Histiocytosis, Non-Langerhans-Cell

ClinicalTrials.gov lists 202 trials for Histiocytosis, Non-Langerhans-Cell or a narrower condition beneath it. Swedish Orphan Biovitrum has the most among industry and government lead sponsors, with 8.

MeSH D015616 Hemic and Lymphatic Diseases C15 Listed by Orphanet as a rare disease
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Phase 1 Phase 1 18 Phase 1/2 Phase 1/2 16 Phase 2 Phase 2 39 Phase 2/3 Phase 2/3 17 Phase 3 Phase 3 19 Phase 4 Phase 4 7 Not applicable Not applicable 20 Observational Observational 64 Phase 1 Phase 1 18 Phase 1/2 Phase 1/2 16 Phase 2 Phase 2 39 Phase 2/3 Phase 2/3 17 Phase 3 Phase 3 19 Phase 4 Phase 4 7 Not applicable Not applicable 20 Observational Observational 64

Trials per phase

1 more section with rows

2 of the 202 trials are tagged with Histiocytosis, Non-Langerhans-Cell itself; the rest are tagged with a narrower condition beneath it.

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The PDF carries up to 50 rows of each section, where the page shows the first few, with the sources listed. Enter a work email and confirm it from the message we send, and the report follows by email.

When did Histiocytosis, Non-Langerhans-Cell trials start? (26)

Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.

Trials over time, by phase

  • Phase 1
  • Phase 1/2
  • Phase 2
  • Phase 2/3
  • Phase 3
  • Phase 4
  • Not applicable
  • Observational

Top of scale: 18

1994: 1 trials (Observational 1) 1995: 1 trials (Phase 2/3 1) 1999: 1 trials (Observational 1) 2000: 5 trials (Observational 1, Not applicable 2, Phase 2/3 1, Phase 2 1) 2002: 3 trials (Phase 2 2, Phase 1 1) 2004: 1 trials (Phase 3 1) 2006: 3 trials (Observational 1, Phase 3 1, Phase 1 1) 2008: 4 trials (Phase 3 1, Phase 2 1, Phase 1/2 1, Phase 1 1) 2009: 2 trials (Phase 2 1, Phase 1/2 1) 2010: 4 trials (Observational 3, Phase 2 1) 2011: 7 trials (Observational 3, Phase 4 1, Phase 2/3 1, Phase 1/2 1, Phase 1 1) 2012: 6 trials (Observational 1, Not applicable 2, Phase 2 2, Phase 1 1) 2013: 11 trials (Observational 1, Not applicable 1, Phase 2/3 1, Phase 2 4, Phase 1/2 2, Phase 1 2) 2014: 10 trials (Observational 3, Phase 2/3 1, Phase 2 3, Phase 1/2 1, Phase 1 2) 2015: 12 trials (Observational 2, Phase 3 1, Phase 2/3 4, Phase 2 2, Phase 1/2 2, Phase 1 1) 2016: 5 trials (Observational 2, Phase 3 1, Phase 2/3 1, Phase 2 1) 2017: 8 trials (Observational 3, Phase 2 1, Phase 1/2 2, Phase 1 2) 2018: 10 trials (Observational 5, Phase 3 3, Phase 2 1, Phase 1/2 1) 2019: 13 trials (Observational 4, Not applicable 1, Phase 3 3, Phase 2/3 1, Phase 2 2, Phase 1/2 1, Phase 1 1) 2020: 13 trials (Observational 3, Not applicable 2, Phase 4 1, Phase 2/3 1, Phase 2 3, Phase 1/2 2, Phase 1 1) 2021: 18 trials (Observational 5, Not applicable 4, Phase 4 1, Phase 3 1, Phase 2 5, Phase 1/2 1, Phase 1 1) 2022: 13 trials (Observational 7, Not applicable 1, Phase 4 1, Phase 3 2, Phase 1 2) 2023: 18 trials (Observational 8, Not applicable 5, Phase 4 1, Phase 3 1, Phase 2/3 1, Phase 2 2) 2024: 13 trials (Observational 7, Not applicable 1, Phase 4 1, Phase 2 4) 2025: 11 trials (Observational 3, Not applicable 1, Phase 4 1, Phase 3 2, Phase 2/3 2, Phase 2 1, Phase 1 1)

Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.

Trials started per year
Year Trials started
2026 (partial year) 7
2025 11
2024 13
2023 18
2022 13
2021 18
2020 13
2019 13
2018 10
2017 8

Showing 10 of 26 earlier years.

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Forecast of the full-year count, planned starts included, not rows of this table: about 8 trials starting in 2026 and about 10 in 2027, the average of the 24 months through September, counted as registered and carried forward. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 28 percent and ran about 20 percent low against the count later registered; forecasts made before the year began were off by about 39 percent and ran about 37 percent low against the count later registered. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.

Prediction, not a registry value: median of 36 months from start to primary completion across 19 trials still running at the 2026-10-09 snapshot; registered median of 41 months across 92 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Histiocytosis, Non-Langerhans-Cell trials in? (9)

Shown in this order: phase order.

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Trials by phase
Phase Trials
Phase 1 18
Phase 1/2 16
Phase 2 39
Phase 2/3 17
Phase 3 19
Phase 4 7
Not applicable 20
Observational 64
Expanded access 2

How many Histiocytosis, Non-Langerhans-Cell trials are recruiting? (11)

Shown in this order: the order a trial moves through, from not yet recruiting to unknown.

Trials by recruitment status
Status Trials
Not yet recruiting 11
Recruiting 42
Enrolling by invitation 1
Active, not recruiting 14
Available 1

Showing 5 of 11 statuses.

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Status as registered on ClinicalTrials.gov. Each trial is counted once.

Which lead sponsors run the most Histiocytosis, Non-Langerhans-Cell trials? (8)

Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.

Lead sponsors
Sponsor Trials
Swedish Orphan Biovitrum 8
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 6
Sanofi 6
Azafaros B.V. 4
Cyclo Therapeutics, Inc. 4

Showing 5 of 8 sponsors.

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Which drugs are being tested for Histiocytosis, Non-Langerhans-Cell? (6)

Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.

Drugs tested
Drug Trials
Olipudase alfa 8
Cytoxan 8
VTS-270 7
Busulfan 6
METHYLPREDNISOLONE 6

Showing 5 of 6 drugs.

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Interventions as named in the registry (9)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
Etoposid 20
Dexamethasone/Dexamethasone acetate 14
Alemtuzumab s.c. 8
Cyclosporine 0.05% 8
Ruxolitinib IR 7

Showing 5 of 9 interventions.

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These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.

Which serious adverse events do Histiocytosis, Non-Langerhans-Cell trials report? (10)

Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.

Serious adverse events in this condition's trials
Adverse event Subjects affected Trials
Death 63 5
Condition aggravated 43 4
Pyrexia 42 10
Pneumonia 24 12
Gastroenteritis 22 7

Showing 5 of 10 adverse events.

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Which publications cover Histiocytosis, Non-Langerhans-Cell? (19)

Shown in this order: major-topic papers first, then newest, then most linked to trials.

Publications
Publication Journal Year Major topic
High prevalence of BRAF V600E mutations in Erdheim-Chester disease but not in other non-Langerhans cell histiocytoses. Blood 2012 Yes
Uncommon histiocytic disorders: the non-Langerhans cell histiocytoses. Pediatric blood & cancer 2005 Yes
Genetic subtypes of familial hemophagocytic lymphohistiocytosis: correlations with clinical features and cytotoxic T lymphocyte/natural killer cell functions. Blood 2005 Yes
Primary hemophagocytic syndromes point to a direct link between lymphocyte cytotoxicity and homeostasis. Immunological reviews 2005 Yes
Haematopoietic stem cell transplantation in haemophagocytic lymphohistiocytosis. British journal of haematology 2005 Yes

Showing 5 of 19 publications.

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Rare-disease identity (2)

Shown in this order: exact, broader, narrower, other.

Rare-disease identity
Orphanet disease ORPHA code Relation Source
Non-Langerhans cell histiocytosis 157987 Exact Orphanet Rare Disease Ontology (ORDO)
Non-Langerhans cell histiocytosis 157987 Exact Orphadata Science knowledge files, Product 1

Sub-conditions in MeSH (5)

MeSH descendants one level down with at least one trial, most trials first.

Not on this page

Not measured: delivery record.

Checked, with nothing recorded for this condition: prevalence.

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