Counted absence: no menin-inhibitor trial has ever reported a response RATE in NUP98-rearranged patients

All four records were fetched from the raw ClinicalTrials.gov v2 API in this session (https://clinicaltrials.gov/api/v2/studies/<NCT>), not through the MCP tool, so whyStopped and hasResults were readable.

hasResults is false and no resultsSection is present for any of the four, so no NUP98 subgroup result can exist on the registry for any of them. No whyStopped string exists on any record, so no closure reason is being hidden from the literature here — the reason none of these has reported NUP98 data is simply that none has reported. 1236

AUGMENT-101 (NCT04065399), general inclusion:

"...or acute leukemia harboring KMT2A rearrangement, NUP98 rearrangement, or NPM1 mutation that have detectable disease in the bone marrow."

and the cohort built for it:

"Cohort 2D: Documented R/R acute leukemia with a genetic mutation expected to lead to HOX/MEIS upregulation (for example, KMT2Ar, NPM1m, and NUP98r), including participants who are MRD-positive by multiparametric flow cytometry or molecular methods only, and including participants with isolated extramedullary disease."

cAMeLot-1 (NCT04811560), note the phase 1 → phase 2 narrowing:

"Phase 1: ... Acute leukemia harboring histone-lysine N-methyltransferase 2A (KMT2A), nucleophosmin 1 gene (NPM1) or nucleoporin 98 gene or nucleoporin 214 gene (NUP98 or NUP214) alterations"

"Phase: 2 ... AML harboring KMT2A-r (gene rearrangement/translocation) or NPM1 mutations only"

KOMET-001 (NCT04067336): there is nothing to quote. The strings NUP98 and NUP214 do not occur.

TACL T2023-004 (NCT07686107):

"Patients must have NUP98-r AML (according to WHO Classification of Hematolymphoid Tumors (5th Edition)) that is either refractory to or is in first relapse following initial therapy." "Patients must be > 1 year and < 22 years of age at time of enrollment."

NCT04067336's acronym field is null and its orgStudyIdInfo.id is "KO-MEN-001", not "KOMET-001". The registry record alone therefore does not confirm that this NCT is KOMET-001. The identification comes from the publication instead: the Lancet Oncology phase 1 report is titled "...(KOMET-001)..." and registers itself as NCT04067336 3. An agent matching trials to publications by acronym field would have failed here.

Separately, a published review calls this study "the phase I/II COMET-001 trial". That spelling is wrong and should not be propagated; it is recorded here without citation because the point is the name, not the review's other contents.

PubMed counts, run in this beat's research pass. Short or word-like terms were title-checked before counting, per the project's alias-precision rule.

Of the 31 hits for NUP98 AND (menin OR MEN1), each was title-checked: exactly three touch clinical response in NUP98 patients — the SAVE trial, the icovamenib single-patient report, and a paediatric case letter. The rest are preclinical, reviews, or false positives (one 2010 LEDGF/p75 chromatin paper matched on "MEN1" alone). The nine hits of the drug-name query contain zero primary clinical trial reports of a NUP98 cohort other than SAVE.

What this count does not cover, stated so it is not mistaken for coverage: Europe PMC queries (NUP98 AND revumenib 132 hits; NUP98 AND ziftomenib 84; NUP98 AND bleximenib 40) returned no ASH or EHA meeting abstracts — the conference-abstract layer was not indexed by any query run. That is the single most likely place for an unpublished NUP98 response rate to exist, and this absence claim is explicitly not a claim about it.

REVUFORJ (revumenib) 4:

"REVUFORJ is a menin inhibitor indicated for: the treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation as determined by an FDA-authorized test in adult and pediatric patients 1 year and older. ... the treatment of relapsed or refractory acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation in adult and pediatric patients 1 year and older who have no satisfactory alternative treatment options."

KOMZIFTI (ziftomenib) 5:

"KOMZIFTI is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation who have no satisfactory alternative treatment options."

A programmatic string count over both complete label JSON documents returns NUP98 = 0 and NUP214 = 0. The practical consequence for a clinician is direct: prescribing a menin inhibitor for a NUP98-rearranged child is off-label under both labels, and the on-label KMT2A indication requires an FDA-authorized test for a KMT2A translocation, which a NUP98-rearranged patient does not have.

AUGMENT-101 wrote NUP98 into its eligibility in 2019 and has been recruiting for nearly seven years. Cohort 2D exists specifically for the HOX/MEIS genotypes. Bleximenib admitted NUP98 and NUP214 in phase 1 and then dropped them from phase 2. Two readings compete and this entry does not adjudicate between them:

• Accrual. NUP98 rearrangements are rare and skew paediatric; adult-weighted registrational cohorts may simply never have accumulated enough patients to report.
• Signal. A sponsor that saw a convincing NUP98 response rate in cohort 2D would plausibly have reported it, and bleximenib's phase 2 narrowing is at least consistent with a phase 1 NUP98 signal that did not justify carrying the genotype forward.

The discriminating observation would be an accrual number for AUGMENT-101 cohort 2D, which is not posted. The reopening condition is simple: if cohort 2D reports, or if the bleximenib phase 1 NUP98/NUP214 subgroup is published, this entry must be rewritten.