Trial · NCT01154452

Vismodegib and Gamma-Secretase/Notch Signalling Pathway Inhibitor RO4929097 in Treating Patients With Advanced or Metastatic Sarcoma

NCT01154452, Phase 1, Phase 2, Completed, National Cancer Institute (NCI); results first posted 2016-07-22

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Vismodegib and Gamma-Secretase/Notch Signalling Pathway Inhibitor RO4929097 in Treating Patients With Advanced or Metastatic Sarcoma. Trials, record NCT01154452. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01154452

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Vismodegib and Gamma-Secretase/Notch Signalling Pathway Inhibitor RO4929097 in Treating Patients With Advanced or Metastatic Sarcoma
NCT number: NCT01154452
Status: Completed
Phases: Phase 1, Phase 2
Enrollment: 78 participants, actual
Lead sponsor: National Cancer Institute (NCI)
Conditions: Adult Alveolar Soft Part Sarcoma, Adult Angiosarcoma, Adult Desmoplastic Small Round Cell Tumor, Adult Epithelioid Hemangioendothelioma, Adult Epithelioid Sarcoma, Adult Extraskeletal Myxoid Chondrosarcoma, Adult Extraskeletal Osteosarcoma, Adult Fibrosarcoma, Adult Leiomyosarcoma, Adult Liposarcoma, Adult Malignant Mesenchymoma, Adult Malignant Peripheral Nerve Sheath Tumor, Adult Rhabdomyosarcoma, Adult Synovial Sarcoma, Adult Unclassified Pleomorphic Sarcoma, Chondrosarcoma, Clear Cell Sarcoma of the Kidney, Conjunctival Kaposi Sarcoma, Dermatofibrosarcoma Protuberans, Gastrointestinal Stromal Tumor, Metastatic Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Metastatic Osteosarcoma, Ovarian Sarcoma, Recurrent Adult Soft Tissue Sarcoma, Recurrent Adult Unclassified Pleomorphic Sarcoma of Bone, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Kaposi Sarcoma, Recurrent Osteosarcoma, Recurrent Uterine Corpus Sarcoma, Small Intestine Leiomyosarcoma, Stage III Adult Soft Tissue Sarcoma, Stage III Uterine Sarcoma, Stage IV Adult Soft Tissue Sarcoma, Stage IV Uterine Sarcoma, Unclassified Pleomorphic Sarcoma of Bone
Interventions: Gamma-Secretase Inhibitor RO4929097, Laboratory Biomarker Analysis, Pharmacological Study, Vismodegib
First posted: 2010-06-30
Last updated: 2018-08-15
Results first posted: 2016-07-22
Limitations and caveats: Roche has discontinued further development of the Notch inhibitor (RO4929097) and this Phase II trial was prematurely closed.

Source ClinicalTrials.gov

Registered outcomes3

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Maximum-tolerated Dose of Gamma-secretase Inhibitor RO4929097, Defined as the Dose Level Where no More Than 1 Out of 6 Patients Experience DLT at the Highest Dose Level Below the MAD, Graded According to NCI-CTCAE Version 4.0 (Phase Ib)	Up to 28 days
Primary	Progression-free Survival (PFS)	6 weeks	Progression-free survival (PFS) of the combination of RO4929097 with and without GDC-0449 in patients with advanced sarcoma. (Phase II)
Secondary	Response Rate (CR + PR) as Assessed by RECIST 1.1 (Phase Ib and II)	Up to 4 months	Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions

Source ClinicalTrials.gov

Outcomes3

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Units	Measured as
PRIMARY	Maximum-tolerated Dose of Gamma-secretase Inhibitor RO4929097, Defined as the Dose Level Where no More Than 1 Out of 6 Patients Experience DLT at the Highest Dose Level Below the MAD, Graded According to NCI-CTCAE Version 4.0 (Phase Ib)	Up to 28 days	mg	NUMBER
PRIMARY	Progression-free Survival (PFS)	6 weeks	percentage of participants	NUMBER
SECONDARY	Response Rate (CR + PR) as Assessed by RECIST 1.1 (Phase Ib and II)	Up to 4 months	participants	NUMBER

Source ClinicalTrials.gov

Results by group11

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Units
Maximum-tolerated Dose of Gamma-secretase Inhibitor RO4929097, Defined as the Dose Level Where no More Than 1 Out of 6 Patients Experience DLT at the Highest Dose Level Below the MAD, Graded According to NCI-CTCAE Version 4.0 (Phase Ib)	All Phase Ib Participants		15	mg
Progression-free Survival (PFS)	Arm I (Gamma-secretase Inhibitor RO4929097)		60	percentage of participants
Progression-free Survival (PFS)	Arm II (Vismodegib and Gamma-secretase Inhibitor RO4929097)		60	percentage of participants
Response Rate (CR + PR) as Assessed by RECIST 1.1 (Phase Ib and II)	RO4929097 10mg	Stable Disease	2	participants
Response Rate (CR + PR) as Assessed by RECIST 1.1 (Phase Ib and II)	RO4929097 15 mg	Stable Disease	2	participants
Response Rate (CR + PR) as Assessed by RECIST 1.1 (Phase Ib and II)	ARM 1 - RO4929097: 15 mg PO QD	Stable Disease	17	participants
Response Rate (CR + PR) as Assessed by RECIST 1.1 (Phase Ib and II)	ARM 2: GDC-0449 150 mg PO QD and RO4929097 15 mg PO QD	Stable Disease	16	participants
Response Rate (CR + PR) as Assessed by RECIST 1.1 (Phase Ib and II)	RO4929097 10mg	Progression of Disease	1	participants
Response Rate (CR + PR) as Assessed by RECIST 1.1 (Phase Ib and II)	RO4929097 15 mg	Progression of Disease	1	participants
Response Rate (CR + PR) as Assessed by RECIST 1.1 (Phase Ib and II)	ARM 1 - RO4929097: 15 mg PO QD	Progression of Disease	13	participants

1 to 10 of 11Source ClinicalTrials.gov

See all 11 results by group

Participant flow24

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Phase Ib	STARTED	RO4929097 10mg	6
Phase Ib	STARTED	RO4929097 15 mg	4
Phase Ib	STARTED	ARM 1 - RO4929097: 15 mg PO QD	0
Phase Ib	STARTED	ARM 2: GDC-0449 150 mg PO QD and RO4929097 15 mg PO QD	0
Phase Ib	COMPLETED	RO4929097 10mg	3
Phase Ib	COMPLETED	RO4929097 15 mg	3
Phase Ib	COMPLETED	ARM 1 - RO4929097: 15 mg PO QD	0
Phase Ib	COMPLETED	ARM 2: GDC-0449 150 mg PO QD and RO4929097 15 mg PO QD	0
Phase Ib	NOT COMPLETED	RO4929097 10mg	3
Phase Ib	NOT COMPLETED	RO4929097 15 mg	1

1 to 10 of 24Source ClinicalTrials.gov

See all 24 participant flow

Adverse events228

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Activated partial thromboplastin time prolonged	Investigations	RO4929097 10mg	1	6	1
other	Activated partial thromboplastin time prolonged	Investigations	RO4929097 15 mg	1	4	2
other	Activated partial thromboplastin time prolonged	Investigations	ARM 1 - RO4929097: 15 mg PO QD	0	35	0
other	Activated partial thromboplastin time prolonged	Investigations	ARM 2: GDC-0449 150 mg PO QD and RO4929097 15 mg PO QD	0	33	0
other	Anemia	Blood and lymphatic system disorders	RO4929097 10mg	1	6	12
other	Anemia	Blood and lymphatic system disorders	RO4929097 15 mg	3	4	42
other	Anemia	Blood and lymphatic system disorders	ARM 1 - RO4929097: 15 mg PO QD	11	35	56
other	Anemia	Blood and lymphatic system disorders	ARM 2: GDC-0449 150 mg PO QD and RO4929097 15 mg PO QD	8	33	32
other	Blurred vision	Eye disorders	RO4929097 10mg	1	6	1
other	Blurred vision	Eye disorders	RO4929097 15 mg	0	4	0

1 to 10 of 228Source ClinicalTrials.gov

See all 228 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	RO4929097 10mg
Total, all-cause mortality	RO4929097 15 mg
Total, all-cause mortality	ARM 1 - RO4929097: 15 mg PO QD
Total, all-cause mortality	ARM 2: GDC-0449 150 mg PO QD and RO4929097 15 mg PO QD
Total, other adverse events	RO4929097 10mg	4	6
Total, other adverse events	RO4929097 15 mg	4	4
Total, other adverse events	ARM 1 - RO4929097: 15 mg PO QD	26	35
Total, other adverse events	ARM 2: GDC-0449 150 mg PO QD and RO4929097 15 mg PO QD	23	33
Total, serious adverse events	RO4929097 10mg	2	6
Total, serious adverse events	RO4929097 15 mg	2	4

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants2

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	Source
P30CA008748	Cancer Center Support Grant	Rudin, Charles M.	SLOAN-KETTERING INST CAN RESEARCH	NATIONAL CANCER INSTITUTE	1985 to 2026	NIH RePORTER
U01CA069856	Early Clinical Trials of New Anti-Cancer Agents with Phase 1 Emphasis	SPRIGGS, DAVID R	SLOAN-KETTERING INST CAN RESEARCH	NATIONAL CANCER INSTITUTE	1995 to 2013	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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT01154452

GET https://conductscience.org/api/trials/v1/NCT01154452/flow?page=2

API reference For AI assistants