Trial Outcomes & Findings for AZD2171 in Treating Patients With Neurofibromatosis Type 1 and Plexiform Neurofibroma and/or Neurofibroma Near the Spine (NCT NCT00326872)
NCT ID: NCT00326872
Last Updated: 2017-08-18
Results Overview
Complete Response (CR): Disappearance of all target lesions. Partial Response (PR): At least a 30% decrease in the volume of target lesions taking as reference the baseline volume.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
26 participants
Primary outcome timeframe
Baseline to end of treatment, maximum of 26 cycles (28 days/cycle).
Results posted on
2017-08-18
Participant Flow
From 8/29/2006 until 7/31/2009, 26 patients were accrued.
All 26 patients accrued were evaluable for the primary endpoint.
Participant milestones
| Measure |
Treatment (Cediranib Maleate)
Patients receive 30 mg oral AZD2171 once daily on days 1-28. Treatment repeats every 28 days for 26 courses in the absence of disease progression or unacceptable toxicity. Patients with responding or stable disease may continue treatment beyond 26 courses in the absence of disease progression or unacceptable toxicity.
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|---|---|
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Overall Study
STARTED
|
26
|
|
Overall Study
COMPLETED
|
26
|
|
Overall Study
NOT COMPLETED
|
0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
AZD2171 in Treating Patients With Neurofibromatosis Type 1 and Plexiform Neurofibroma and/or Neurofibroma Near the Spine
Baseline characteristics by cohort
| Measure |
Treatment (Cediranib Maleate)
n=26 Participants
Patients receive 30 mg oral AZD2171 once daily on days 1-28. Treatment repeats every 28 days for 26 courses in the absence of disease progression or unacceptable toxicity. Patients with responding or stable disease may continue treatment beyond 26 courses in the absence of disease progression or unacceptable toxicity.
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|---|---|
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Age, Continuous
|
34 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=5 Participants
|
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Sex: Female, Male
Male
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13 Participants
n=5 Participants
|
|
Region of Enrollment
United States
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26 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to end of treatment, maximum of 26 cycles (28 days/cycle).Complete Response (CR): Disappearance of all target lesions. Partial Response (PR): At least a 30% decrease in the volume of target lesions taking as reference the baseline volume.
Outcome measures
| Measure |
Treatment (Cediranib Maleate)
n=26 Participants
Patients receive 30 mg oral AZD2171 once daily on days 1-28. Treatment repeats every 28 days for 26 courses in the absence of disease progression or unacceptable toxicity. Patients with responding or stable disease may continue treatment beyond 26 courses in the absence of disease progression or unacceptable toxicity.
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|---|---|
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Proportion of Patients With Tumor Response (Complete Response [CR] or Partial Response [PR])
|
0.0384 Proportion of patients
Interval 0.002 to 0.216
|
SECONDARY outcome
Timeframe: From registration to death (due to any cause) max 51 monthsSurvival time is defined as the time from registration to death due to any cause.
Outcome measures
| Measure |
Treatment (Cediranib Maleate)
n=26 Participants
Patients receive 30 mg oral AZD2171 once daily on days 1-28. Treatment repeats every 28 days for 26 courses in the absence of disease progression or unacceptable toxicity. Patients with responding or stable disease may continue treatment beyond 26 courses in the absence of disease progression or unacceptable toxicity.
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|---|---|
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Survival Time as Measured Using Kaplan-Meier Method
|
NA Months
All 26 patients were alive at last follow-up. Median follow-up time is 24.4 months with a range of 1 to 51 months.
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SECONDARY outcome
Timeframe: From registration to documentation of disease progression up to 26 cycles (28 days/cycle).Progression (PD): At least a 20% increase in the sum of volumes of target lesions taking as reference the smallest volume recorded since the treatment started or the appearance of one or more new lesions. If a patient dies without documentation of disease progression, the patient will be considered to have had tumor progression at the time of their death unless there is sufficient documented evidence to conclude no progression occurred prior to death.
Outcome measures
| Measure |
Treatment (Cediranib Maleate)
n=26 Participants
Patients receive 30 mg oral AZD2171 once daily on days 1-28. Treatment repeats every 28 days for 26 courses in the absence of disease progression or unacceptable toxicity. Patients with responding or stable disease may continue treatment beyond 26 courses in the absence of disease progression or unacceptable toxicity.
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|---|---|
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Time to Disease Progression as Measured Using Kaplan-Meier Method
|
49.15 Months
Interval 45.76 to
The number of events (n=7) was insufficient to define the upper 95% confidence interval.
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SECONDARY outcome
Timeframe: From time of confirmed tumor objective response as CR or PR to the date of progression max 51 monthsPopulation: There was only 1 response and due to patient confidentiality we are not reporting this endpoint.
Duration of response is defined for all evaluable patients who have achieved a confirmed tumor objective response as the date at which the patient's objective status is first noted to be either a CR or PR to the date progression is documented. Duration of response will be estimated using the method of Kaplan-Meier.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From the date of randomization to the date at which the patient is removed from treatment due to progression, toxicity, or refusal up to 51 months.Time to treatment failure is defined to be the time from the date of randomization to the date at which the patient is removed from treatment due to progression, toxicity, or refusal. If the patient is considered to be a major treatment violation or is taken off study as a non-protocol failure, the patient will be censored on the date they are removed from treatment. Time to treatment failure will be estimated using the method of Kaplan-Meier.
Outcome measures
| Measure |
Treatment (Cediranib Maleate)
n=26 Participants
Patients receive 30 mg oral AZD2171 once daily on days 1-28. Treatment repeats every 28 days for 26 courses in the absence of disease progression or unacceptable toxicity. Patients with responding or stable disease may continue treatment beyond 26 courses in the absence of disease progression or unacceptable toxicity.
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|---|---|
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Time to Treatment Failure as Assessed Using the Method of Kaplan-Meier
|
5.9 Months
Interval 2.6 to 42.3
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SECONDARY outcome
Timeframe: At baseline, prior to each subsequent course (q 28+/- 3 days), and at end of treatment up to 51 monthsReduction in self reported worst pain per cycle as measured by the Worst Pain scale from the North Central Cancer Treatment Group Brief Pain Inventory (short form). The worst pain scale is from 0-10 (10 is worst pain possible). The per-cycle average reduction in worst pain will be analyzed using generalized linear models to account for repeated measures within patients.
Outcome measures
| Measure |
Treatment (Cediranib Maleate)
n=26 Participants
Patients receive 30 mg oral AZD2171 once daily on days 1-28. Treatment repeats every 28 days for 26 courses in the absence of disease progression or unacceptable toxicity. Patients with responding or stable disease may continue treatment beyond 26 courses in the absence of disease progression or unacceptable toxicity.
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|---|---|
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Reduction in Self Reported Worst Pain Per Cycle.
|
0.137 units on a scale of 0-10
Standard Error 0.0552
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Adverse Events
Treatment (Cediranib Maleate)
Serious events: 12 serious events
Other events: 26 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Treatment (Cediranib Maleate)
n=26 participants at risk
Patients receive 30 mg oral AZD2171 once daily on days 1-28. Treatment repeats every 28 days for 26 courses in the absence of disease progression or unacceptable toxicity. Patients with responding or stable disease may continue treatment beyond 26 courses in the absence of disease progression or unacceptable toxicity.
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|---|---|
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Blood and lymphatic system disorders
Blood disorder
|
3.8%
1/26 • Number of events 1
|
|
Cardiac disorders
Left ventricular failure
|
7.7%
2/26 • Number of events 2
|
|
Cardiac disorders
Pericarditis
|
3.8%
1/26 • Number of events 1
|
|
Gastrointestinal disorders
Esophagitis
|
3.8%
1/26 • Number of events 1
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|
Investigations
Alanine aminotransferase increased
|
15.4%
4/26 • Number of events 5
|
|
Investigations
Aspartate aminotransferase increased
|
11.5%
3/26 • Number of events 3
|
|
Investigations
Blood bilirubin increased
|
11.5%
3/26 • Number of events 7
|
|
Investigations
Neutrophil count decreased
|
7.7%
2/26 • Number of events 2
|
|
Investigations
Weight loss
|
3.8%
1/26 • Number of events 4
|
|
Nervous system disorders
Depressed level of consciousness
|
3.8%
1/26 • Number of events 1
|
|
Psychiatric disorders
Insomnia
|
3.8%
1/26 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory disorder
|
3.8%
1/26 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
3.8%
1/26 • Number of events 1
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|
Skin and subcutaneous tissue disorders
Rash desquamating
|
3.8%
1/26 • Number of events 1
|
Other adverse events
| Measure |
Treatment (Cediranib Maleate)
n=26 participants at risk
Patients receive 30 mg oral AZD2171 once daily on days 1-28. Treatment repeats every 28 days for 26 courses in the absence of disease progression or unacceptable toxicity. Patients with responding or stable disease may continue treatment beyond 26 courses in the absence of disease progression or unacceptable toxicity.
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|---|---|
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Blood and lymphatic system disorders
Blood disorder
|
23.1%
6/26 • Number of events 51
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|
Blood and lymphatic system disorders
Hemoglobin decreased
|
15.4%
4/26 • Number of events 48
|
|
Blood and lymphatic system disorders
Hemolysis
|
3.8%
1/26 • Number of events 1
|
|
Cardiac disorders
Cardiac disorder
|
3.8%
1/26 • Number of events 1
|
|
Cardiac disorders
Left ventricular failure
|
7.7%
2/26 • Number of events 5
|
|
Cardiac disorders
Palpitations
|
3.8%
1/26 • Number of events 2
|
|
Ear and labyrinth disorders
Tinnitus
|
3.8%
1/26 • Number of events 1
|
|
Endocrine disorders
Hyperthyroidism
|
7.7%
2/26 • Number of events 2
|
|
Endocrine disorders
Hypothyroidism
|
30.8%
8/26 • Number of events 65
|
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Eye disorders
Vision blurred
|
3.8%
1/26 • Number of events 1
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|
Gastrointestinal disorders
Abdominal distension
|
3.8%
1/26 • Number of events 1
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|
Gastrointestinal disorders
Abdominal pain
|
23.1%
6/26 • Number of events 10
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|
Gastrointestinal disorders
Constipation
|
26.9%
7/26 • Number of events 16
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|
Gastrointestinal disorders
Diarrhea
|
88.5%
23/26 • Number of events 135
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|
Gastrointestinal disorders
Dry mouth
|
11.5%
3/26 • Number of events 4
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|
Gastrointestinal disorders
Dyspepsia
|
19.2%
5/26 • Number of events 8
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|
Gastrointestinal disorders
Dysphagia
|
3.8%
1/26 • Number of events 1
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|
Gastrointestinal disorders
Ear, nose and throat examination abnormal
|
11.5%
3/26 • Number of events 3
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|
Gastrointestinal disorders
Esophagitis
|
7.7%
2/26 • Number of events 2
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|
Gastrointestinal disorders
Flatulence
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3.8%
1/26 • Number of events 1
|
|
Gastrointestinal disorders
Hemorrhoidal hemorrhage
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3.8%
1/26 • Number of events 1
|
|
Gastrointestinal disorders
Lower gastrointestinal hemorrhage
|
7.7%
2/26 • Number of events 3
|
|
Gastrointestinal disorders
Mucositis oral
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26.9%
7/26 • Number of events 8
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|
Gastrointestinal disorders
Nausea
|
46.2%
12/26 • Number of events 37
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|
Gastrointestinal disorders
Vomiting
|
34.6%
9/26 • Number of events 10
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General disorders
Chills
|
3.8%
1/26 • Number of events 1
|
|
General disorders
Edema limbs
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3.8%
1/26 • Number of events 1
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|
General disorders
Fatigue
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61.5%
16/26 • Number of events 125
|
|
General disorders
Fever
|
7.7%
2/26 • Number of events 2
|
|
General disorders
Flu-like symptoms
|
7.7%
2/26 • Number of events 5
|
|
General disorders
General symptom
|
3.8%
1/26 • Number of events 3
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|
General disorders
Pain
|
11.5%
3/26 • Number of events 4
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|
Immune system disorders
Hypersensitivity
|
3.8%
1/26 • Number of events 1
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|
Infections and infestations
Bone infection
|
3.8%
1/26 • Number of events 1
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|
Infections and infestations
Infection
|
3.8%
1/26 • Number of events 2
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|
Infections and infestations
Skin infection
|
3.8%
1/26 • Number of events 1
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|
Injury, poisoning and procedural complications
Bruising
|
15.4%
4/26 • Number of events 5
|
|
Investigations
Alanine aminotransferase increased
|
30.8%
8/26 • Number of events 36
|
|
Investigations
Alkaline phosphatase increased
|
30.8%
8/26 • Number of events 27
|
|
Investigations
Aspartate aminotransferase increased
|
23.1%
6/26 • Number of events 19
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|
Investigations
Blood bilirubin increased
|
34.6%
9/26 • Number of events 69
|
|
Investigations
Creatinine increased
|
3.8%
1/26 • Number of events 1
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|
Investigations
Laboratory test abnormal
|
3.8%
1/26 • Number of events 1
|
|
Investigations
Leukocyte count decreased
|
11.5%
3/26 • Number of events 10
|
|
Investigations
Lymphocyte count decreased
|
3.8%
1/26 • Number of events 2
|
|
Investigations
Neutrophil count decreased
|
7.7%
2/26 • Number of events 2
|
|
Investigations
Platelet count decreased
|
19.2%
5/26 • Number of events 10
|
|
Investigations
Serum cholesterol increased
|
3.8%
1/26 • Number of events 2
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|
Investigations
Weight loss
|
42.3%
11/26 • Number of events 139
|
|
Metabolism and nutrition disorders
Anorexia
|
23.1%
6/26 • Number of events 13
|
|
Metabolism and nutrition disorders
Blood glucose increased
|
50.0%
13/26 • Number of events 39
|
|
Metabolism and nutrition disorders
Dehydration
|
7.7%
2/26 • Number of events 3
|
|
Metabolism and nutrition disorders
Serum calcium decreased
|
3.8%
1/26 • Number of events 1
|
|
Metabolism and nutrition disorders
Serum calcium increased
|
3.8%
1/26 • Number of events 1
|
|
Metabolism and nutrition disorders
Serum glucose decreased
|
19.2%
5/26 • Number of events 12
|
|
Metabolism and nutrition disorders
Serum potassium decreased
|
19.2%
5/26 • Number of events 13
|
|
Metabolism and nutrition disorders
Serum potassium increased
|
3.8%
1/26 • Number of events 2
|
|
Metabolism and nutrition disorders
Serum sodium decreased
|
7.7%
2/26 • Number of events 3
|
|
Metabolism and nutrition disorders
Serum sodium increased
|
11.5%
3/26 • Number of events 3
|
|
Metabolism and nutrition disorders
Serum triglycerides increased
|
7.7%
2/26 • Number of events 12
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
3.8%
1/26 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
15.4%
4/26 • Number of events 9
|
|
Musculoskeletal and connective tissue disorders
Chest wall pain
|
3.8%
1/26 • Number of events 21
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness
|
34.6%
9/26 • Number of events 25
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness lower limb
|
3.8%
1/26 • Number of events 4
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
11.5%
3/26 • Number of events 4
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
19.2%
5/26 • Number of events 6
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Tumor pain
|
3.8%
1/26 • Number of events 18
|
|
Nervous system disorders
Depressed level of consciousness
|
3.8%
1/26 • Number of events 1
|
|
Nervous system disorders
Dizziness
|
23.1%
6/26 • Number of events 14
|
|
Nervous system disorders
Headache
|
53.8%
14/26 • Number of events 62
|
|
Nervous system disorders
Hypoglossal nerve disorder
|
3.8%
1/26 • Number of events 1
|
|
Nervous system disorders
Memory impairment
|
3.8%
1/26 • Number of events 4
|
|
Nervous system disorders
Peripheral motor neuropathy
|
11.5%
3/26 • Number of events 10
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
34.6%
9/26 • Number of events 44
|
|
Nervous system disorders
Sinus pain
|
3.8%
1/26 • Number of events 1
|
|
Nervous system disorders
Tremor
|
11.5%
3/26 • Number of events 11
|
|
Psychiatric disorders
Insomnia
|
19.2%
5/26 • Number of events 20
|
|
Renal and urinary disorders
Cystitis
|
3.8%
1/26 • Number of events 1
|
|
Renal and urinary disorders
Proteinuria
|
38.5%
10/26 • Number of events 30
|
|
Reproductive system and breast disorders
Irregular menstruation
|
11.5%
3/26 • Number of events 25
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
15.4%
4/26 • Number of events 12
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
15.4%
4/26 • Number of events 11
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
3.8%
1/26 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
3.8%
1/26 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory disorder
|
3.8%
1/26 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Voice alteration
|
23.1%
6/26 • Number of events 18
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
3.8%
1/26 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
7.7%
2/26 • Number of events 5
|
|
Skin and subcutaneous tissue disorders
Hand-and-foot syndrome
|
38.5%
10/26 • Number of events 40
|
|
Skin and subcutaneous tissue disorders
Skin disorder
|
7.7%
2/26 • Number of events 2
|
|
Skin and subcutaneous tissue disorders
Sweating
|
3.8%
1/26 • Number of events 2
|
|
Vascular disorders
Flushing
|
3.8%
1/26 • Number of events 2
|
|
Vascular disorders
Hypertension
|
42.3%
11/26 • Number of events 74
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60