Trial Outcomes & Findings for CC-4047 (Pomalidomide) for Graft vs. Host Disease (NCT NCT00770757)
NCT ID: NCT00770757
Last Updated: 2016-02-25
Results Overview
* CR is defined as complete resolution in all of signs and symptoms at all affected organs and tissues * PR is defined as improvement in greater than or equal to 1 organ/tissue with no progression in any other affected organ/tissue * Improvement in chronic GvHD symptoms less than what meets the definition of a PR is defined as other * Progressive disease is defined as failure of therapy to control chronic GvHD despite increasing the dose of primary therapy or adding second line treatments * No response is defined as no change in disease.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
13 participants
Primary outcome timeframe
1 year after last dose of CC-4047
Results posted on
2016-02-25
Participant Flow
The study opened to participant enrollment on 02/02/2009 and closed to participant enrollment on 03/29/2011.
Participant milestones
| Measure |
CC-4047 Arm
CC-4047 2 mg orally every day for 1 course (12 weeks or 84 days). Every 28 days of treatment are considered as 1 cycle and every 3 cycles are are considered as 1 course of treatment. A total of 4 courses of treatment are planned (12 months).
|
|---|---|
|
Overall Study
STARTED
|
13
|
|
Overall Study
COMPLETED
|
13
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
CC-4047 (Pomalidomide) for Graft vs. Host Disease
Baseline characteristics by cohort
| Measure |
CC-4047 Arm
n=13 Participants
CC-4047 2 mg orally every day for 1 course (12 weeks or 84 days). Every 28 days of treatment are considered as 1 cycle and every 3 cycles are are considered as 1 course of treatment. A total of 4 courses of treatment are planned (12 months).
|
|---|---|
|
Age, Continuous
|
46 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
13 participants
n=5 Participants
|
|
Donor type
Matched related donor
|
7 participants
n=5 Participants
|
|
Donor type
Matched unrelated donor
|
6 participants
n=5 Participants
|
|
Karnofsky performance status
|
80 scores on a scale
n=5 Participants
|
|
Chronic graft-versus-host disease onset
De novo
|
5 participants
n=5 Participants
|
|
Chronic graft-versus-host disease onset
Progressive
|
1 participants
n=5 Participants
|
|
Chronic graft-versus-host disease onset
Quiescent
|
7 participants
n=5 Participants
|
|
Global chronic graft-versus-host disease score
Severe
|
11 participants
n=5 Participants
|
|
Global chronic graft-versus-host disease score
Moderate
|
2 participants
n=5 Participants
|
|
Number of prior therapies
|
4 number of prior therapies
n=5 Participants
|
|
Years post-transplant
|
2.5 years
n=5 Participants
|
|
Years since chronic graft-versus-host disease
|
1.5 years
n=5 Participants
|
|
Major organ-systems involved
Skin
|
13 participants
n=5 Participants
|
|
Major organ-systems involved
Eyes
|
10 participants
n=5 Participants
|
|
Major organ-systems involved
Joint and Fascia
|
10 participants
n=5 Participants
|
|
Major organ-systems involved
Mouth
|
9 participants
n=5 Participants
|
|
Major organ-systems involved
Lungs
|
8 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 1 year after last dose of CC-4047Population: 4 participants discontinued treatment before the third cycle because of adverse-effects and were not evaluable
* CR is defined as complete resolution in all of signs and symptoms at all affected organs and tissues * PR is defined as improvement in greater than or equal to 1 organ/tissue with no progression in any other affected organ/tissue * Improvement in chronic GvHD symptoms less than what meets the definition of a PR is defined as other * Progressive disease is defined as failure of therapy to control chronic GvHD despite increasing the dose of primary therapy or adding second line treatments * No response is defined as no change in disease.
Outcome measures
| Measure |
CC-4047 Arm
n=9 Participants
CC-4047 2 mg orally every day for 1 course (12 weeks or 84 days). Every 28 days of treatment are considered as 1 cycle and every 3 cycles are are considered as 1 course of treatment. A total of 4 courses of treatment are planned (12 months).
|
|---|---|
|
Overall Response (Complete Response + Partial Response + Other)
Partial overall response
|
7 participants
|
|
Overall Response (Complete Response + Partial Response + Other)
No response
|
2 participants
|
|
Overall Response (Complete Response + Partial Response + Other)
Complete overall response
|
0 participants
|
|
Overall Response (Complete Response + Partial Response + Other)
Progressive disease
|
0 participants
|
SECONDARY outcome
Timeframe: 30 days after last dose of CC-4047 or until resolution of event-Toxicities will be graded according to the NCI CTCAE v3.0.
Outcome measures
| Measure |
CC-4047 Arm
n=13 Participants
CC-4047 2 mg orally every day for 1 course (12 weeks or 84 days). Every 28 days of treatment are considered as 1 cycle and every 3 cycles are are considered as 1 course of treatment. A total of 4 courses of treatment are planned (12 months).
|
|---|---|
|
Safety as Measured by the Most Common Adverse Effects and Reasons for Dose Reductions or Study-discontinuation
Sepsis
|
1 participants
|
|
Safety as Measured by the Most Common Adverse Effects and Reasons for Dose Reductions or Study-discontinuation
Aseptic meningitis
|
1 participants
|
|
Safety as Measured by the Most Common Adverse Effects and Reasons for Dose Reductions or Study-discontinuation
Deep vein thrombosis/pulmonary embolism
|
1 participants
|
|
Safety as Measured by the Most Common Adverse Effects and Reasons for Dose Reductions or Study-discontinuation
Erythematous skin rash
|
1 participants
|
|
Safety as Measured by the Most Common Adverse Effects and Reasons for Dose Reductions or Study-discontinuation
Tremors/shakiness
|
4 participants
|
|
Safety as Measured by the Most Common Adverse Effects and Reasons for Dose Reductions or Study-discontinuation
Muscle cramps/musculoskeletal pain
|
12 participants
|
|
Safety as Measured by the Most Common Adverse Effects and Reasons for Dose Reductions or Study-discontinuation
Fatigue/anxiety
|
6 participants
|
|
Safety as Measured by the Most Common Adverse Effects and Reasons for Dose Reductions or Study-discontinuation
Sensory neuropathy
|
4 participants
|
|
Safety as Measured by the Most Common Adverse Effects and Reasons for Dose Reductions or Study-discontinuation
Respiratory syncytial virus (RSV) pneumonia
|
2 participants
|
|
Safety as Measured by the Most Common Adverse Effects and Reasons for Dose Reductions or Study-discontinuation
Community-acquired pneumonia
|
1 participants
|
POST_HOC outcome
Timeframe: 1 year after last dose of CC-4047Population: 4 participants discontinued treatment before the third cycle because of adverse-effects and were not evaluable
Outcome measures
| Measure |
CC-4047 Arm
n=9 Participants
CC-4047 2 mg orally every day for 1 course (12 weeks or 84 days). Every 28 days of treatment are considered as 1 cycle and every 3 cycles are are considered as 1 course of treatment. A total of 4 courses of treatment are planned (12 months).
|
|---|---|
|
Organ System Response
Eyes - complete organ response
|
1 participants
|
|
Organ System Response
Eyes - partial organ response
|
1 participants
|
|
Organ System Response
Gastrointestinal - complete organ response
|
2 participants
|
|
Organ System Response
Skin erythema - complete organ response
|
2 participants
|
|
Organ System Response
Skin erythema - partial organ response
|
3 participants
|
|
Organ System Response
Skin movable sclerosis - complete organ response
|
0 participants
|
|
Organ System Response
Skin movable sclerosis - partial organ response
|
1 participants
|
|
Organ System Response
Mouth - complete organ response
|
2 participants
|
|
Organ System Response
Mouth - partial organ response
|
0 participants
|
|
Organ System Response
Gastrointestinal - partial organ response
|
0 participants
|
POST_HOC outcome
Timeframe: 1 year after last dose of CC-4047* Global scoring of chronic GvHD consists of questions about various organs including skin, genital tract, lungs, liver, and multiple others. The physician scores each organ from 0 to 3. Score 0 means the patient has no symptoms. Score 1 means the patient has mild symptoms. Score 2 means the patient has moderate symptoms. Score 3 means the patient has severe syptoms. * Mild scoring of chronic GvHD is only 1 or 2 organs or site (except the lung). No clinically significant functional impairment (maximum of score 1 in all affected organs or sites) * Moderate scoring of chronic GvHD is at least 1 organ or site with clinically significant but no major disability (maximum score of 2 in any affected organ or site) or 3 or more organs or sites with no clinically significant functional impairment (maximum of 1 in all affected orgnas or sites) * Severe scoring of chronic GvHD is a major disability caused by chronic GvHD (score of 3 in any organ or site) and lung function score \>=2.
Outcome measures
| Measure |
CC-4047 Arm
n=13 Participants
CC-4047 2 mg orally every day for 1 course (12 weeks or 84 days). Every 28 days of treatment are considered as 1 cycle and every 3 cycles are are considered as 1 course of treatment. A total of 4 courses of treatment are planned (12 months).
|
|---|---|
|
Chronic Graft-versus-host Disease Global Score at the Start/End of the Study
Starting score Severe/Ending score Severe
|
11 participants
|
|
Chronic Graft-versus-host Disease Global Score at the Start/End of the Study
Starting score Moderate/Ending score Moderate
|
2 participants
|
POST_HOC outcome
Timeframe: Median follow-up 5.6 years (4.2-5.6 years)Population: Five participants have died and these were the participants who did not respond or were removed from study prior to cycle 3 due to adverse events.
Outcome measures
| Measure |
CC-4047 Arm
n=7 Participants
CC-4047 2 mg orally every day for 1 course (12 weeks or 84 days). Every 28 days of treatment are considered as 1 cycle and every 3 cycles are are considered as 1 course of treatment. A total of 4 courses of treatment are planned (12 months).
|
|---|---|
|
Survival Rate of CC-4047 Responders
|
100 percentage of participants
|
Adverse Events
CC-4047 Arm
Serious events: 5 serious events
Other events: 13 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
CC-4047 Arm
n=13 participants at risk
CC-4047 2 mg orally every day for 1 course (12 weeks or 84 days). Every 28 days of treatment are considered as 1 cycle and every 3 cycles are are considered as 1 course of treatment. A total of 4 courses of treatment are planned (12 months).
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
7.7%
1/13
|
|
Infections and infestations
Cellulitis
|
7.7%
1/13
|
|
Gastrointestinal disorders
Constipation
|
7.7%
1/13
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
15.4%
2/13
|
|
Gastrointestinal disorders
Diarrhea
|
7.7%
1/13
|
|
Nervous system disorders
Dizziness
|
7.7%
1/13
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
7.7%
1/13
|
|
General disorders
Fatigue
|
7.7%
1/13
|
|
General disorders
Fever
|
7.7%
1/13
|
|
Vascular disorders
Hypotension
|
7.7%
1/13
|
|
Musculoskeletal and connective tissue disorders
Muscle cramps
|
7.7%
1/13
|
|
Gastrointestinal disorders
Nausea
|
15.4%
2/13
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
7.7%
1/13
|
|
Infections and infestations
Pneumonia
|
7.7%
1/13
|
|
Infections and infestations
RSV infection
|
7.7%
1/13
|
|
Skin and subcutaneous tissue disorders
Rash
|
7.7%
1/13
|
|
General disorders
Rigors/chills
|
7.7%
1/13
|
|
Nervous system disorders
Tremors
|
7.7%
1/13
|
|
Gastrointestinal disorders
Vomiting
|
7.7%
1/13
|
Other adverse events
| Measure |
CC-4047 Arm
n=13 participants at risk
CC-4047 2 mg orally every day for 1 course (12 weeks or 84 days). Every 28 days of treatment are considered as 1 cycle and every 3 cycles are are considered as 1 course of treatment. A total of 4 courses of treatment are planned (12 months).
|
|---|---|
|
Metabolism and nutrition disorders
Albumin - low
|
53.8%
7/13
|
|
Investigations
Alkaline phosphatase
|
46.2%
6/13
|
|
Immune system disorders
Allergic reaction/hypersensitivity
|
15.4%
2/13
|
|
Respiratory, thoracic and mediastinal disorders
Allergic rhinitis
|
15.4%
2/13
|
|
Gastrointestinal disorders
Anal incontinence (intermittent)
|
7.7%
1/13
|
|
Metabolism and nutrition disorders
Anorexia
|
15.4%
2/13
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
23.1%
3/13
|
|
Skin and subcutaneous tissue disorders
Blistering/tenderness/erythema/desquamating rash
|
7.7%
1/13
|
|
Skin and subcutaneous tissue disorders
Bruising
|
7.7%
1/13
|
|
Metabolism and nutrition disorders
Calcium - low
|
46.2%
6/13
|
|
Gastrointestinal disorders
Constipation
|
15.4%
2/13
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
7.7%
1/13
|
|
Renal and urinary disorders
Creatinine
|
15.4%
2/13
|
|
Musculoskeletal and connective tissue disorders
Decreased range of motion (joint function)
|
23.1%
3/13
|
|
General disorders
Diaphoresis
|
7.7%
1/13
|
|
Gastrointestinal disorders
Diarrhea
|
7.7%
1/13
|
|
Gastrointestinal disorders
Distension/bloating
|
7.7%
1/13
|
|
Gastrointestinal disorders
Dysgeusia
|
7.7%
1/13
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
23.1%
3/13
|
|
Renal and urinary disorders
Dysuria
|
7.7%
1/13
|
|
General disorders
Edema
|
7.7%
1/13
|
|
General disorders
Edema (generalized)
|
7.7%
1/13
|
|
General disorders
Edema (limb)
|
15.4%
2/13
|
|
General disorders
Fatigue
|
30.8%
4/13
|
|
Injury, poisoning and procedural complications
Fracture
|
7.7%
1/13
|
|
Injury, poisoning and procedural complications
Fracture of vertebra (compression)
|
7.7%
1/13
|
|
Metabolism and nutrition disorders
Glucose - high
|
61.5%
8/13
|
|
Metabolism and nutrition disorders
Glucose - low
|
7.7%
1/13
|
|
Nervous system disorders
Headache
|
7.7%
1/13
|
|
Blood and lymphatic system disorders
Hemoglobin
|
69.2%
9/13
|
|
Musculoskeletal and connective tissue disorders
Hip pain
|
7.7%
1/13
|
|
Skin and subcutaneous tissue disorders
Hyperpigmentation
|
7.7%
1/13
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
61.5%
8/13
|
|
Skin and subcutaneous tissue disorders
Hypopigmentation
|
7.7%
1/13
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
7.7%
1/13
|
|
Infections and infestations
Infection without neutropenia
|
46.2%
6/13
|
|
Investigations
Lymphopenia
|
69.2%
9/13
|
|
Metabolism and nutrition disorders
Magnesium - high
|
7.7%
1/13
|
|
Metabolism and nutrition disorders
Magnesium - low
|
15.4%
2/13
|
|
Psychiatric disorders
Mood alteration - anxiety
|
23.1%
3/13
|
|
Psychiatric disorders
Mood alteration - depression
|
7.7%
1/13
|
|
Gastrointestinal disorders
Mucositis - mouth sensitivity
|
7.7%
1/13
|
|
Gastrointestinal disorders
Mucositis - oral ulcers
|
23.1%
3/13
|
|
Musculoskeletal and connective tissue disorders
Muscle pain
|
30.8%
4/13
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness
|
7.7%
1/13
|
|
Gastrointestinal disorders
Nausea
|
23.1%
3/13
|
|
Renal and urinary disorders
Nephrolithiasis
|
7.7%
1/13
|
|
Renal and urinary disorders
Nephrolithiasis - passing stones
|
7.7%
1/13
|
|
Nervous system disorders
Neuropathy - sensory (pain)
|
7.7%
1/13
|
|
Nervous system disorders
Neuropathy - motor (weakness)
|
7.7%
1/13
|
|
Nervous system disorders
Neuropathy - sensory (numbness)
|
15.4%
2/13
|
|
Investigations
Neutrophils (ANC)
|
7.7%
1/13
|
|
Musculoskeletal and connective tissue disorders
Osteonecrosis (avascular)
|
7.7%
1/13
|
|
Musculoskeletal and connective tissue disorders
Osteoporosis
|
7.7%
1/13
|
|
General disorders
Pain NOS
|
7.7%
1/13
|
|
Investigations
Platelets
|
15.4%
2/13
|
|
Metabolism and nutrition disorders
Potassium - low
|
30.8%
4/13
|
|
Infections and infestations
RSV infection
|
7.7%
1/13
|
|
Skin and subcutaneous tissue disorders
Rash
|
23.1%
3/13
|
|
Investigations
SGOT (AST)
|
69.2%
9/13
|
|
Investigations
SGPT (ALT)
|
84.6%
11/13
|
|
Skin and subcutaneous tissue disorders
Sclerodermatous fibrosis
|
7.7%
1/13
|
|
Musculoskeletal and connective tissue disorders
Shoulder pain
|
15.4%
2/13
|
|
Skin and subcutaneous tissue disorders
Skin wound/break
|
7.7%
1/13
|
|
Metabolism and nutrition disorders
Sodium - high
|
23.1%
3/13
|
|
Metabolism and nutrition disorders
Sodium - low
|
15.4%
2/13
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
7.7%
1/13
|
|
Vascular disorders
Thrombosis/embolism
|
7.7%
1/13
|
|
Nervous system disorders
Tremor
|
23.1%
3/13
|
|
Renal and urinary disorders
Urinary frequency/urgency
|
7.7%
1/13
|
|
Gastrointestinal disorders
Vomiting
|
15.4%
2/13
|
|
Musculoskeletal and connective tissue disorders
Wrist pain
|
7.7%
1/13
|
Additional Information
John F. DiPersio, M.D., Ph.D.
Washington University School of Medicine
Phone: 314-454-8603
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place