Trial Outcomes & Findings for A Multi-center, Open-label Study of Oral ENMD-2076 for the Treatment of Patients With Advanced Fibrolamellar Carcinoma (NCT NCT02234986)
NCT ID: NCT02234986
Last Updated: 2025-04-18
Results Overview
6-month overall response rate (ORR rate) using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) 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; ORR = proportion of patients evaluated as CR + PR
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
35 participants
Primary outcome timeframe
6 months
Results posted on
2025-04-18
Participant Flow
43 patients screened; 35 patients enrolled
Participant milestones
| Measure |
ENMD-2076
ENMD-2076, oral capsule Once daily dose 250 mg/day
ENMD-2076: 250 mg oral dose, once daily (QD) for 28 day cycles
|
|---|---|
|
Overall Study
STARTED
|
35
|
|
Overall Study
COMPLETED
|
35
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
ENMD-2076
n=35 Participants
ENMD-2076, oral capsule Once daily dose 250 mg/day 28 day cycles
|
|---|---|
|
Age, Continuous
|
25 years
n=35 Participants
|
|
Sex: Female, Male
Female
|
19 Participants
n=35 Participants
|
|
Sex: Female, Male
Male
|
16 Participants
n=35 Participants
|
|
Eastern Cooperative Oncology Group (ECOG) status
ECOG 0: Fully active, able to carry on all pre-disease performance without restriction
|
12 participants
n=33 Participants • 33 of the 35 patients in this study was \>= 16 years of age.
|
|
Eastern Cooperative Oncology Group (ECOG) status
ECOG 1: Restricted in physically strenuous activity but ambulatory, able do work of a light nature
|
19 participants
n=33 Participants • 33 of the 35 patients in this study was \>= 16 years of age.
|
|
Eastern Cooperative Oncology Group (ECOG) status
ECOG 2: Ambulatory and capable of selfcare, unable to carry out any work; up >50% of waking hours
|
2 participants
n=33 Participants • 33 of the 35 patients in this study was \>= 16 years of age.
|
|
Lansky status
Lansky 100: Fully active
|
1 participants
n=2 Participants • Lansky status was applicable to patients \< 16 years of age. Two patients enrolled in the study were \< 16 years of age.
|
|
Lansky status
Lansky 90: Minor restriction in physically strenuous play
|
1 participants
n=2 Participants • Lansky status was applicable to patients \< 16 years of age. Two patients enrolled in the study were \< 16 years of age.
|
|
Number of prior systemic therapies
|
4 Number of prior systemic therapies
n=35 Participants
|
PRIMARY outcome
Timeframe: 6 monthsPopulation: Patients treated and evaluable for efficacy
6-month overall response rate (ORR rate) using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) 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; ORR = proportion of patients evaluated as CR + PR
Outcome measures
| Measure |
ENMD-2076
n=35 Participants
ENMD-2076, oral capsule Once daily dose 250 mg/day
ENMD-2076: 250 mg oral dose, QD for 28 day cycles
|
|---|---|
|
Overall Response Rate
Partial response
|
1 Participants
|
|
Overall Response Rate
Stable disease
|
20 Participants
|
|
Overall Response Rate
Progressive disease
|
10 Participants
|
|
Overall Response Rate
Other / unknown
|
4 Participants
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: patients treated and evaluable for efficacy
using RECIST v 1.1 criteria
Outcome measures
| Measure |
ENMD-2076
n=35 Participants
ENMD-2076, oral capsule Once daily dose 250 mg/day
ENMD-2076: 250 mg oral dose, QD for 28 day cycles
|
|---|---|
|
Progression Free Survival
|
3.9 months
Interval 2.3 to 5.5
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 6 monthsPopulation: patients treated and evaluable for toxicity assessment; regardless of relationship to study medication according to CTCAE 5.0
Frequency and severity of adverse events in patients evaluable for toxicity
Outcome measures
| Measure |
ENMD-2076
n=35 Participants
ENMD-2076, oral capsule Once daily dose 250 mg/day
ENMD-2076: 250 mg oral dose, QD for 28 day cycles
|
|---|---|
|
Frequency and Severity of Adverse Events
Fatigue
|
27 Participants
|
|
Frequency and Severity of Adverse Events
ALT increased
|
26 Participants
|
|
Frequency and Severity of Adverse Events
AST increased
|
24 Participants
|
|
Frequency and Severity of Adverse Events
Abdominal pain
|
23 Participants
|
|
Frequency and Severity of Adverse Events
Diarrhea
|
23 Participants
|
Adverse Events
ENMD-2076
Serious events: 4 serious events
Other events: 35 other events
Deaths: 3 deaths
Serious adverse events
| Measure |
ENMD-2076
n=35 participants at risk
ENMD-2076, oral capsule Once daily dose 250 mg/day
ENMD-2076: 250 mg oral dose, QD for 28 day cycles
|
|---|---|
|
Infections and infestations
Sepsis
|
2.9%
1/35 • Number of events 1 • 2 years
|
|
Hepatobiliary disorders
Liver failure
|
2.9%
1/35 • Number of events 1 • 2 years
|
|
Cardiac disorders
Respiratory failure
|
2.9%
1/35 • Number of events 1 • 2 years
|
|
Nervous system disorders
Seizure
|
2.9%
1/35 • Number of events 1 • 2 years
|
Other adverse events
| Measure |
ENMD-2076
n=35 participants at risk
ENMD-2076, oral capsule Once daily dose 250 mg/day
ENMD-2076: 250 mg oral dose, QD for 28 day cycles
|
|---|---|
|
General disorders
Fatigue
|
77.1%
27/35 • Number of events 27 • 2 years
|
|
General disorders
Fever
|
8.6%
3/35 • Number of events 3 • 2 years
|
|
General disorders
Chills
|
5.7%
2/35 • Number of events 2 • 2 years
|
|
Gastrointestinal disorders
Nausea
|
57.1%
20/35 • Number of events 20 • 2 years
|
|
Gastrointestinal disorders
Vomiting
|
25.7%
9/35 • Number of events 9 • 2 years
|
|
Gastrointestinal disorders
Diarrhea
|
65.7%
23/35 • Number of events 23 • 2 years
|
|
Gastrointestinal disorders
Constipation
|
22.9%
8/35 • Number of events 8 • 2 years
|
|
Blood and lymphatic system disorders
Anemia
|
25.7%
9/35 • Number of events 9 • 2 years
|
|
Blood and lymphatic system disorders
Decreased neutrophil count
|
5.7%
2/35 • Number of events 2 • 2 years
|
|
Investigations
Decreased platelet count
|
14.3%
5/35 • Number of events 5 • 2 years
|
|
Investigations
Increased hemoglobin
|
11.4%
4/35 • Number of events 4 • 2 years
|
|
Investigations
INR increased
|
8.6%
3/35 • Number of events 3 • 2 years
|
|
Investigations
Prolonged aPTT
|
17.1%
6/35 • Number of events 6 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
25.7%
9/35 • Number of events 9 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngolaryngeal pain
|
8.6%
3/35 • Number of events 3 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Sinus disorder
|
5.7%
2/35 • Number of events 2 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
28.6%
10/35 • Number of events 10 • 2 years
|
|
Investigations
Weight loss
|
20.0%
7/35 • Number of events 7 • 2 years
|
|
Metabolism and nutrition disorders
Decreased appetite
|
28.6%
10/35 • Number of events 10 • 2 years
|
|
Gastrointestinal disorders
Dry mouth
|
5.7%
2/35 • Number of events 2 • 2 years
|
|
Gastrointestinal disorders
Abdominal distension
|
5.7%
2/35 • Number of events 2 • 2 years
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
8.6%
3/35 • Number of events 3 • 2 years
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
28.6%
10/35 • Number of events 10 • 2 years
|
|
Vascular disorders
Hypertension
|
45.7%
16/35 • Number of events 16 • 2 years
|
|
Metabolism and nutrition disorders
Hypernatremia
|
8.6%
3/35 • Number of events 3 • 2 years
|
|
Investigations
Aspartate aminotransferase increased
|
68.6%
24/35 • Number of events 24 • 2 years
|
|
Gastrointestinal disorders
Abdominal pain
|
65.7%
23/35 • Number of events 23 • 2 years
|
|
Investigations
Alanine aminotransferase increased
|
74.3%
26/35 • Number of events 26 • 2 years
|
|
Investigations
Alkaline phosphatase increased
|
42.9%
15/35 • Number of events 15 • 2 years
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
28.6%
10/35 • Number of events 10 • 2 years
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
8.6%
3/35 • Number of events 3 • 2 years
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
48.6%
17/35 • Number of events 17 • 2 years
|
|
Metabolism and nutrition disorders
Hyponatremia
|
25.7%
9/35 • Number of events 9 • 2 years
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
17.1%
6/35 • Number of events 6 • 2 years
|
|
Gastrointestinal disorders
Mucositis oral
|
11.4%
4/35 • Number of events 4 • 2 years
|
|
Nervous system disorders
Dizziness
|
8.6%
3/35 • Number of events 3 • 2 years
|
|
Cardiac disorders
Palpitations
|
5.7%
2/35 • Number of events 2 • 2 years
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
5.7%
2/35 • Number of events 2 • 2 years
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
17.1%
6/35 • Number of events 6 • 2 years
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
14.3%
5/35 • Number of events 5 • 2 years
|
|
Nervous system disorders
Headache
|
34.3%
12/35 • Number of events 12 • 2 years
|
|
Psychiatric disorders
Insomnia
|
14.3%
5/35 • Number of events 5 • 2 years
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
11.4%
4/35 • Number of events 4 • 2 years
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia syndrome
|
11.4%
4/35 • Number of events 4 • 2 years
|
|
Renal and urinary disorders
Proteinuria
|
20.0%
7/35 • Number of events 7 • 2 years
|
|
Eye disorders
Blurred vision
|
5.7%
2/35 • Number of events 2 • 2 years
|
|
Investigations
White blood cell decreased
|
5.7%
2/35 • Number of events 2 • 2 years
|
|
Nervous system disorders
Encephalopathy
|
5.7%
2/35 • Number of events 2 • 2 years
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
11.4%
4/35 • Number of events 4 • 2 years
|
|
Investigations
Blood bilirubin increased
|
17.1%
6/35 • Number of events 6 • 2 years
|
|
General disorders
Non-cardiac chest pain
|
5.7%
2/35 • Number of events 2 • 2 years
|
|
Gastrointestinal disorders
Dyspepsia
|
5.7%
2/35 • Number of events 2 • 2 years
|
|
Cardiac disorders
Left ventricular systolic dysfunction
|
8.6%
3/35 • Number of events 3 • 2 years
|
|
Infections and infestations
Enteritis
|
5.7%
2/35 • Number of events 2 • 2 years
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
14.3%
5/35 • Number of events 5 • 2 years
|
|
Reproductive system and breast disorders
Pelvic pain
|
5.7%
2/35 • Number of events 2 • 2 years
|
|
Gastrointestinal disorders
Colitis
|
8.6%
3/35 • Number of events 3 • 2 years
|
|
Metabolism and nutrition disorders
Dehydration
|
5.7%
2/35 • Number of events 2 • 2 years
|
|
Vascular disorders
Thromboembolic event
|
5.7%
2/35 • Number of events 2 • 2 years
|
|
Endocrine disorders
Hypothyroidism
|
5.7%
2/35 • Number of events 2 • 2 years
|
|
Investigations
Lymphocyte count decreased
|
5.7%
2/35 • Number of events 2 • 2 years
|
|
Cardiac disorders
Sinus tachycardia
|
8.6%
3/35 • Number of events 3 • 2 years
|
|
General disorders
Pain
|
17.1%
6/35 • Number of events 6 • 2 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place