Enasidenib in IDH2-Mutated Malignant Sinonasal and Skull Base Tumors

Phase 2

Recruiting

• Conditions: Locally Advanced Chondrosarcoma; Locally Advanced Sinonasal Adenocarcinoma; Metastatic Olfactory Neuroblastoma; Locally Advanced Olfactory Neuroblastoma; Metastatic Sinonasal Undifferentiated Carcinoma; Metastatic Sinonasal Adenocarcinoma; Locally Advanced Large-cell Neuroendocrine Carcinoma; Metastatic Chondrosarcoma; Locally Advanced Sinonasal Undifferentiated Carcinoma; Metastatic Large-cell Neuroendocrine Carcinoma
• Interventions: Drug: Enasidenib

• Registration Number: NCT06176989
• Lead Sponsor: National Cancer Institute (NCI)

Brief Summary

Background:

Cancers of the nasal cavity or skull base are rare. They often are not diagnosed until they are at an advanced stage, and they often spread to other parts of the body. These cancers may have mutations in a gene called IDH2. Researchers want to find out if a drug (enasidenib) that targets the IDH2 mutation can help people with these cancers.
...

Detailed Description

Background:

* Sinonasal undifferentiated carcinoma (SNUC) and olfactory neuroblastoma (ONB) are rare malignant sinonasal and skull base tumors, a group that also includes sinonasal adenocarcinoma, squamous cell carcinoma, large cell neuroendocrine carcinoma, sinonasal papilloma, chondrosarcoma (CS), chordoma and others.
...

Basic Information
|
Recruitment & Eligibility
|
Study & Design
|
Trial Locations

Study Timeline

• Study First Submitted: 2023-12-19
• Study First Submitted QC: 2023-12-19
• Study First Posted: 2023-12-20
• Study Start: 2024-03-04
• Status Verified: 2024-11-20
• Last Update Submitted: 2024-11-22
• Last Update Posted: 2024-11-25
• Primary Completion: 2027-12-31
• Study Completion: 2030-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

Not provided

Read More

Exclusion Criteria

Not provided

Read More

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
1	Enasidenib	Participants with IDH2 mutated (R140/R172) malignant sinonasal and skull base tumors.

Primary Outcome Measures

Name	Time	Method
Progression Free Survival (PFS) in all study participants	up to 5 years post study treatment	The date of first treatment until the date of disease progression or death without progression

Secondary Outcome Measures

Name	Time	Method
safety	from study treatment initiation up to 28 days post study treatment	Adverse events assessed per CTCAE version 5
clinical benefit rate (CBR: CR+PR+SD>4 months) in participants with IDH2m SNUC	up to 5 years post study treatment	Fraction of participants experiencing CBR = CR+PR+ SD \> 16 weeks
PFS in non-SNUC IDH2m tumors	up to 5 years post study treatment	The date of first treatment until the date of disease progression or death without progression
Overall survival (OS) in non-SNUC IDH2m tumors	up to 5 years post study treatment	The date of first treatment until date of death or last follow-up
clinical benefit rate (CBR: CR+PR+SD>4 months) in participants with non-SNUC IDH2m tumors	up to 5 years post study treatment	Fraction of participants experiencing CBR = CR+PR+ SD \> 16 weeks
Overall survival (OS) in participants with IDH2m SNUC	up to 5 years post study treatment	The date of first treatment until date of death or last follow-up
PFS in participants with IDH2m SNUC	up to 5 years post study treatment	The date of first treatment until the date of disease progression or death without progression
Correlate UGTIA1 genotypes with toxicity	from study treatment initiation up to 28 days post study treatment	Descriptive statistics of AE occurence per UGTIA1 genotype

Trial Locations

Locations (1)

National Institutes of Health Clinical Center; 🇺🇸 Bethesda, Maryland, United States