Trial · NCT04195399
A Study of a New Drug, Nirogacestat, for Treating Desmoid Tumors That Cannot be Removed by Surgery
NCT04195399, Phase 2, Active not recruiting, Children's Oncology Group
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Study of a New Drug, Nirogacestat, for Treating Desmoid Tumors That Cannot be Removed by Surgery. Trials, record NCT04195399. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04195399
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of a New Drug, Nirogacestat, for Treating Desmoid Tumors That Cannot be Removed by Surgery
- NCT number
- NCT04195399
- Status
- Active not recruiting
- Phases
- Phase 2
- Enrollment
- 31 participants, actual
- Lead sponsor
- Children's Oncology Group
- Conditions
- Desmoid Fibromatosis, Recurrent Desmoid Fibromatosis, Unresectable Desmoid Fibromatosis
- Interventions
- Biospecimen Collection, Computed Tomography, Echocardiography Test, Magnetic Resonance Imaging, Nirogacestat Hydrobromide, Quality-of-Life Assessment, Questionnaire Administration, X-Ray Imaging
- First posted
- 2019-12-11
- Last updated
- 2026-10-01
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Progression-free survival (PFS) | Up to 2 years from study entry | The Kaplan-Meier method will be used to estimate the 2-year PFS defined as the time from study entry until disease progression or death from any cause. |
| Primary | Percentage of patients experiencing grade 3+ adverse events (AE) | Up to 6 years from study entry | Percentage of patients experiencing grade 3 or higher AEs will be evaluated according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. All grade 1 and 2 toxicities observed in \> 5% of participants and deemed related to study drug will be reported. |
| Primary | Pharmacokinetic (PK) parameter: systemic exposure | Up to Cycle 3 (each cycle lasts 28 days) | PK parameters of nirogacestat will be defined to quantify systemic exposure, drug clearance, terminal half-life and other pharmacokinetic characteristics. These PK parameters will be summarized with descriptive statistics, including means, medians, ranges, and standard deviations. |
| Primary | PK parameter: drug clearance | Up to Cycle 3 (each cycle lasts 28 days) | PK parameters of nirogacestat will be defined to quantify systemic exposure, drug clearance, terminal half-life and other pharmacokinetic characteristics. These PK parameters will be summarized with descriptive statistics, including means, medians, ranges, and standard deviations. |
| Primary | PK parameter: half-life | Up to Cycle 3 (each cycle lasts 28 days) | PK parameters of nirogacestat will be defined to quantify systemic exposure, drug clearance, terminal half-life and other pharmacokinetic characteristics. These PK parameters will be summarized with descriptive statistics, including means, medians, ranges, and standard deviations. |
| Secondary | Objective response rate | Up to 24 months | Defined by the rate of a complete response or partial response by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1. |
| Other | CTNNB1 and APC gene mutations and genomic signatures | Up to 2 years | The chi-square test will be used to assess the correlation of CTNNB1 and APC gene mutations and genomic signatures with tumor response, and the long-rank test will be used to assess the correlation of CTNNB1 and APC gene mutations and genomic signatures with PFS. |
| Other | Changes in the levels of each of the lymphocyte subsets and immunoglobulins | Up to 2 years | Lymphocyte subsets include CD3 (total T cells), CD3+CD4+ (T helper cells), CD3+CD8+ (T cytotoxic cells), CD3+HLA-DR+ (activated T cells) percentages and absolute counts and CD4:CD8 ratio. The chi-square test will be used to assess the correlation of the levels of lymphocyte and immunoglobulin with tumor response, and the Cox regression will be used to assess the correlation of the levels of lymphocyte and immunoglobulin with PFS. |
| Other | Response assessments by the RECIST criteria | Up to 2 years | Assesses tumor response based primarily on changes in measurable tumor lesions on imaging using RECIST 1.1. |
| Other | Response assessment by the World Health Organization (WHO) criteria | Up to cycle 2 years | Assesses tumor response using the product of two perpendicular tumor measurements according to the WHO criteria. |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants1
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| U10CA180886 | COG NCTN Network Group Operations Center | Hawkins, Douglas S. | COG RESEARCH FOUNDATION, LLC | NATIONAL CANCER INSTITUTE | 2014 to 2026 |
Source NIH RePORTER
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04195399
GET https://conductscience.org/api/trials/v1/NCT04195399/registered