Trial Outcomes & Findings for Gamma-Secretase/Notch Signalling Pathway Inhibitor RO4929097 in Treating Patients With Recurrent or Progressive Glioblastoma (NCT NCT01122901)

NCT ID: NCT01122901

Last Updated: 2017-05-03

Results Overview

Time to event endpoints for Group A will be measured from the date of first dose. All patients who receive at least one dose of study drug will be included in the analysis. Time to event endpoints for Group B will be measured from the first post-surgical date (this will be considered start date of treatment). Progression defined as: \>25% increase sum of products of perpendicular diameters (bi-dimensional measurements) of enhancing lesions (over baseline (BL) if no decrease) on stable or increasing doses of corticosteroids. and/or b) Significant increase in T2/FLAIR nonenhancing lesion on stable or increasing doses of corticosteroids compared to BL scan or best response following initiation of therapy c) Any new lesion. d) Clear clinical deterioration not attributable to other causes apart from the tumor e) Failure to return for evaluation due to death or deteriorating condition.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

47 participants

Primary outcome timeframe

At 6 months

Results posted on

2017-05-03

Participant Flow

Patients enrolled between April 2011 and May 2012 Patients enrolled in outpatient setting

Participant milestones

Participant milestones
Measure
Group A RO4929097 PO
Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days 1-3, 8-10, 15-17, and 22-24. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO pharmacological study: Correlative studies
Group B RO4929097 Pre Surgery
Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days -6 to -1. Patients undergo surgical resection on day 0. Within 30 days after surgical resection, patients receive gamma-secretase inhibitor RO4929097 as in group A. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO therapeutic conventional surgery: Undergo surgery pharmacological study: Correlative studies laboratory biomarker analysis: Correlative studies
Overall Study
STARTED
40
7
Overall Study
COMPLETED
40
7
Overall Study
NOT COMPLETED
0
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Gamma-Secretase/Notch Signalling Pathway Inhibitor RO4929097 in Treating Patients With Recurrent or Progressive Glioblastoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Group A RO4929097 PO
n=40 Participants
Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days 1-3, 8-10, 15-17, and 22-24. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO pharmacological study: Correlative studies
Group B RO4929097 Pre Surgery
n=7 Participants
Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days -6 to -1. Patients undergo surgical resection on day 0. Within 30 days after surgical resection, patients receive gamma-secretase inhibitor RO4929097 as in group A. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO therapeutic conventional surgery: Undergo surgery pharmacological study: Correlative studies laboratory biomarker analysis: Correlative studies
Total
n=47 Participants
Total of all reporting groups
Age, Continuous
58 years
n=5 Participants
56 years
n=7 Participants
57 years
n=5 Participants
Sex: Female, Male
Female
13 Participants
n=5 Participants
2 Participants
n=7 Participants
15 Participants
n=5 Participants
Sex: Female, Male
Male
27 Participants
n=5 Participants
5 Participants
n=7 Participants
32 Participants
n=5 Participants
Karnofsky Performance Status Scale
80 units on a scale
n=5 Participants
80 units on a scale
n=7 Participants
80 units on a scale
n=5 Participants
Debulking
Yes
30 Participants
n=5 Participants
6 Participants
n=7 Participants
36 Participants
n=5 Participants
Debulking
No
10 Participants
n=5 Participants
1 Participants
n=7 Participants
11 Participants
n=5 Participants

PRIMARY outcome

Timeframe: At 6 months

Time to event endpoints for Group A will be measured from the date of first dose. All patients who receive at least one dose of study drug will be included in the analysis. Time to event endpoints for Group B will be measured from the first post-surgical date (this will be considered start date of treatment). Progression defined as: \>25% increase sum of products of perpendicular diameters (bi-dimensional measurements) of enhancing lesions (over baseline (BL) if no decrease) on stable or increasing doses of corticosteroids. and/or b) Significant increase in T2/FLAIR nonenhancing lesion on stable or increasing doses of corticosteroids compared to BL scan or best response following initiation of therapy c) Any new lesion. d) Clear clinical deterioration not attributable to other causes apart from the tumor e) Failure to return for evaluation due to death or deteriorating condition.

Outcome measures

Outcome measures
Measure
Group A (Post Surgery) & Group B (Pre-surgery) RO4929097 PO
n=40 Participants
GROUP A Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days 1-3, 8-10, 15-17, and 22-24. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO pharmacological study: Correlative studies GROUP B Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days -6 to -1. Patients undergo surgical resection on day 0. Within 30 days after surgical resection, patients receive gamma-secretase inhibitor RO4929097 as in group A. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO therapeutic conventional surgery: Undergo surgery pharmacological study: Correlative studies laboratory biomarker analysis: Correlative studies
Group B RO4929097 Pre Surgery
n=7 Participants
Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days -6 to -1. Patients undergo surgical resection on day 0. Within 30 days after surgical resection, patients receive gamma-secretase inhibitor RO4929097 as in group A. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO therapeutic conventional surgery: Undergo surgery pharmacological study: Correlative studies laboratory biomarker analysis: Correlative studies
Group B RO4929097 Pre Surgery
Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days -6 to -1. Patients undergo surgical resection on day 0. Within 30 days after surgical resection, patients receive gamma-secretase inhibitor RO4929097 as in group A. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO therapeutic conventional surgery: Undergo surgery pharmacological study: Correlative studies laboratory biomarker analysis: Correlative studies
Overall Median Progression-free Survival (6-month PFS)
1.7 months
Interval 1.1 to 1.8
1.6 months
Interval 0.8 to 2.3

PRIMARY outcome

Timeframe: At time of surgery

Population: This outcome could not be analyzed as the n was too small. Only had 7 samples. The study was terminated prematurely by Hoffman-La Roche Company due to their decision to terminate drug supply for further development of this drug

This outcome could not be analyzed as the n was too small. The study was terminated prematurely by Hoffman-La Roche Company due to their decision to terminate drug supply for further development of this drug. All CTEP-sponsored trials using RO4929097 were closed to accrual and all patients had to be off treatment by 7/31/12.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 6 months after completion of treatment

RANO: Measurable: Bidimensionally measurable lesions w/ clearly defined margins by MRI Evaluable: Unidimensionally measurable lesions, masses w/margins not clearly defined. Complete Response (CR): Complete disappearance of all measurable/evaluable disease. No new lesions. No evidence of non-evaluable disease. Patients on minimal/no steroids. Partial Response (PR): \>/= to 50% decrease under baseline in the sum of products of perpendicular diameters of all measurable lesions. No progression of evaluable disease. Responders must be on same/decreasing doses of dexamethasone. Stable/No Response: Does not qualify for CR, PR, or progression. Progression: 25% increase in the sum of products of all measurable lesions over smallest sum observed (over BL if no decrease), OR clear worsening of any evaluable disease, OR appearance of any new lesion/site, OR failure to return for evaluation due to death or deteriorating condition (unless clearly unrelated to this cancer).

Outcome measures

Outcome measures
Measure
Group A (Post Surgery) & Group B (Pre-surgery) RO4929097 PO
n=40 Participants
GROUP A Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days 1-3, 8-10, 15-17, and 22-24. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO pharmacological study: Correlative studies GROUP B Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days -6 to -1. Patients undergo surgical resection on day 0. Within 30 days after surgical resection, patients receive gamma-secretase inhibitor RO4929097 as in group A. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO therapeutic conventional surgery: Undergo surgery pharmacological study: Correlative studies laboratory biomarker analysis: Correlative studies
Group B RO4929097 Pre Surgery
n=7 Participants
Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days -6 to -1. Patients undergo surgical resection on day 0. Within 30 days after surgical resection, patients receive gamma-secretase inhibitor RO4929097 as in group A. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO therapeutic conventional surgery: Undergo surgery pharmacological study: Correlative studies laboratory biomarker analysis: Correlative studies
Group B RO4929097 Pre Surgery
Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days -6 to -1. Patients undergo surgical resection on day 0. Within 30 days after surgical resection, patients receive gamma-secretase inhibitor RO4929097 as in group A. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO therapeutic conventional surgery: Undergo surgery pharmacological study: Correlative studies laboratory biomarker analysis: Correlative studies
Radiographic Response Rate According to the Radiographic Assessment in Neuro-Oncology Criteria (Group A) and Group (B)
Complete Response
1 Participants
0 Participants
Radiographic Response Rate According to the Radiographic Assessment in Neuro-Oncology Criteria (Group A) and Group (B)
Partial Response
0 Participants
0 Participants
Radiographic Response Rate According to the Radiographic Assessment in Neuro-Oncology Criteria (Group A) and Group (B)
Stable Disease
3 Participants
0 Participants
Radiographic Response Rate According to the Radiographic Assessment in Neuro-Oncology Criteria (Group A) and Group (B)
Progression
33 Participants
5 Participants
Radiographic Response Rate According to the Radiographic Assessment in Neuro-Oncology Criteria (Group A) and Group (B)
Not Evaluable
3 Participants
2 Participants

SECONDARY outcome

Timeframe: Up to 30 days after completion of study treatment

assessed by the National Cancer Institute CTCAE version 4.0 assessed if patient received at least one dose of study drug Grade 3-5 toxicities grade 3 -severe grade 4 - life threatening grade 5 - death

Outcome measures

Outcome measures
Measure
Group A (Post Surgery) & Group B (Pre-surgery) RO4929097 PO
n=40 Participants
GROUP A Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days 1-3, 8-10, 15-17, and 22-24. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO pharmacological study: Correlative studies GROUP B Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days -6 to -1. Patients undergo surgical resection on day 0. Within 30 days after surgical resection, patients receive gamma-secretase inhibitor RO4929097 as in group A. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO therapeutic conventional surgery: Undergo surgery pharmacological study: Correlative studies laboratory biomarker analysis: Correlative studies
Group B RO4929097 Pre Surgery
n=7 Participants
Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days -6 to -1. Patients undergo surgical resection on day 0. Within 30 days after surgical resection, patients receive gamma-secretase inhibitor RO4929097 as in group A. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO therapeutic conventional surgery: Undergo surgery pharmacological study: Correlative studies laboratory biomarker analysis: Correlative studies
Group B RO4929097 Pre Surgery
Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days -6 to -1. Patients undergo surgical resection on day 0. Within 30 days after surgical resection, patients receive gamma-secretase inhibitor RO4929097 as in group A. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO therapeutic conventional surgery: Undergo surgery pharmacological study: Correlative studies laboratory biomarker analysis: Correlative studies
Number of Participants With Toxicities Associated With Study Drug (Group A and Group B)
Cognitive Disturbance gr 3
1 Participants
0 Participants
Number of Participants With Toxicities Associated With Study Drug (Group A and Group B)
Fatigue gr 3
1 Participants
0 Participants
Number of Participants With Toxicities Associated With Study Drug (Group A and Group B)
Hypophosphatemia gr 3
4 Participants
0 Participants
Number of Participants With Toxicities Associated With Study Drug (Group A and Group B)
Skin Infection gr 3
1 Participants
0 Participants
Number of Participants With Toxicities Associated With Study Drug (Group A and Group B)
AST increase gr 3
1 Participants
0 Participants

SECONDARY outcome

Timeframe: 2 years

time to event endpoints for Group A will be measured from date of first dose. All patients who receive at least one dose will be included in analysis. Time to event endpoints for Group B will be measured from the first post-surgical date (this will be considered start date of treatment).

Outcome measures

Outcome measures
Measure
Group A (Post Surgery) & Group B (Pre-surgery) RO4929097 PO
n=40 Participants
GROUP A Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days 1-3, 8-10, 15-17, and 22-24. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO pharmacological study: Correlative studies GROUP B Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days -6 to -1. Patients undergo surgical resection on day 0. Within 30 days after surgical resection, patients receive gamma-secretase inhibitor RO4929097 as in group A. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO therapeutic conventional surgery: Undergo surgery pharmacological study: Correlative studies laboratory biomarker analysis: Correlative studies
Group B RO4929097 Pre Surgery
n=7 Participants
Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days -6 to -1. Patients undergo surgical resection on day 0. Within 30 days after surgical resection, patients receive gamma-secretase inhibitor RO4929097 as in group A. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO therapeutic conventional surgery: Undergo surgery pharmacological study: Correlative studies laboratory biomarker analysis: Correlative studies
Group B RO4929097 Pre Surgery
Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days -6 to -1. Patients undergo surgical resection on day 0. Within 30 days after surgical resection, patients receive gamma-secretase inhibitor RO4929097 as in group A. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO therapeutic conventional surgery: Undergo surgery pharmacological study: Correlative studies laboratory biomarker analysis: Correlative studies
Overall Survival
6.6 months
Interval 5.3 to 10.5
6.7 months
Interval 5.1 to 12.0

SECONDARY outcome

Timeframe: At the time of surgery

Population: This outcome could not be analyzed as the n was too small. The study was terminated prematurely by Hoffman-La Roche Company due to their decision to terminate drug supply for further development of this drug.

This outcome could not be analyzed as the n was too small. The study was terminated prematurely by Hoffman-La Roche Company due to their decision to terminate drug supply for further development of this drug. All CTEP-sponsored trials using RO4929097 were closed to accrual and all patients had to be off treatment by 7/31/12.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: At the time of surgery

Population: This outcome could not be analyzed as the n was too small. The study was terminated prematurely by Hoffman-La Roche Company due to their decision to terminate drug supply for further development of this drug.

This outcome could not be analyzed as the n was too small. The study was terminated prematurely by Hoffman-La Roche Company due to their decision to terminate drug supply for further development of this drug. All CTEP-sponsored trials using RO4929097 were closed to accrual and all patients had to be off treatment by 7/31/12.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Time to event endpoints for Group A will be measured from the date of first dose. All patients who receive at least one dose of study drug will be included in the analysis. Time to event endpoints for Group B will be measured from the first post-surgical date (this will be considered start date of treatment). Progression defined as: \>25% increase sum of products of perpendicular diameters (bi-dimensional measurements) of enhancing lesions (over baseline (BL) if no decrease) on stable or increasing doses of corticosteroids. and/or b) Significant increase in T2/FLAIR nonenhancing lesion on stable or increasing doses of corticosteroids compared to BL scan or best response following initiation of therapy c) Any new lesion. d) Clear clinical deterioration not attributable to other causes apart from the tumor e) Failure to return for evaluation due to death or deteriorating condition.

Outcome measures

Outcome measures
Measure
Group A (Post Surgery) & Group B (Pre-surgery) RO4929097 PO
n=47 Participants
GROUP A Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days 1-3, 8-10, 15-17, and 22-24. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO pharmacological study: Correlative studies GROUP B Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days -6 to -1. Patients undergo surgical resection on day 0. Within 30 days after surgical resection, patients receive gamma-secretase inhibitor RO4929097 as in group A. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO therapeutic conventional surgery: Undergo surgery pharmacological study: Correlative studies laboratory biomarker analysis: Correlative studies
Group B RO4929097 Pre Surgery
n=40 Participants
Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days -6 to -1. Patients undergo surgical resection on day 0. Within 30 days after surgical resection, patients receive gamma-secretase inhibitor RO4929097 as in group A. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO therapeutic conventional surgery: Undergo surgery pharmacological study: Correlative studies laboratory biomarker analysis: Correlative studies
Group B RO4929097 Pre Surgery
n=7 Participants
Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days -6 to -1. Patients undergo surgical resection on day 0. Within 30 days after surgical resection, patients receive gamma-secretase inhibitor RO4929097 as in group A. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO therapeutic conventional surgery: Undergo surgery pharmacological study: Correlative studies laboratory biomarker analysis: Correlative studies
Progression Free Survival
1.7 months
Interval 1.2 to 1.8
1.7 months
Interval 1.1 to 1.8
1.6 months
Interval 0.8 to 2.3

Adverse Events

Group A RO4929097 PO

Serious events: 0 serious events
Other events: 27 other events
Deaths: 0 deaths

Group B RO4929097 Pre Surgery

Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Group A RO4929097 PO
n=40 participants at risk
Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days 1-3, 8-10, 15-17, and 22-24. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO pharmacological study: Correlative studies
Group B RO4929097 Pre Surgery
n=7 participants at risk
Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO QD on days -6 to -1. Patients undergo surgical resection on day 0. Within 30 days after surgical resection, patients receive gamma-secretase inhibitor RO4929097 as in group A. gamma-secretase/Notch signalling pathway inhibitor RO4929097: Given PO therapeutic conventional surgery: Undergo surgery pharmacological study: Correlative studies laboratory biomarker analysis: Correlative studies
Investigations
white blood cell decrease
7.5%
3/40 • Number of events 3 • 2 year
14.3%
1/7 • Number of events 1 • 2 year
Investigations
Alanine aminotransferase increase
7.5%
3/40 • Number of events 3 • 2 year
0.00%
0/7 • 2 year
Blood and lymphatic system disorders
anemia
7.5%
3/40 • Number of events 3 • 2 year
0.00%
0/7 • 2 year
Metabolism and nutrition disorders
anorexia
7.5%
3/40 • Number of events 3 • 2 year
0.00%
0/7 • 2 year
Investigations
Aspartate aminotransferase increase
7.5%
3/40 • Number of events 3 • 2 year
0.00%
0/7 • 2 year
Gastrointestinal disorders
constipation
10.0%
4/40 • Number of events 4 • 2 year
0.00%
0/7 • 2 year
Gastrointestinal disorders
diarrhea
7.5%
3/40 • Number of events 3 • 2 year
0.00%
0/7 • 2 year
General disorders
edema face
7.5%
3/40 • Number of events 3 • 2 year
0.00%
0/7 • 2 year
General disorders
fatigue
27.5%
11/40 • Number of events 11 • 2 year
14.3%
1/7 • Number of events 1 • 2 year
Investigations
electrocardiogram QT corrected interval prolonged
5.0%
2/40 • Number of events 2 • 2 year
0.00%
0/7 • 2 year
Metabolism and nutrition disorders
Hypomagnesemia
12.5%
5/40 • Number of events 5 • 2 year
0.00%
0/7 • 2 year
Investigations
platelet count decrease
10.0%
4/40 • Number of events 4 • 2 year
14.3%
1/7 • Number of events 1 • 2 year
Skin and subcutaneous tissue disorders
Pruritus
5.0%
2/40 • Number of events 2 • 2 year
0.00%
0/7 • 2 year
Gastrointestinal disorders
vomiting
5.0%
2/40 • Number of events 2 • 2 year
0.00%
0/7 • 2 year
Investigations
weight loss
5.0%
2/40 • Number of events 2 • 2 year
0.00%
0/7 • 2 year

Additional Information

David Peereboom, MD

Adult Brain Tumor Consortium

Phone: 410-955-8837

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place

Restriction type: LTE60