Trial Outcomes & Findings for Expanded Access Study With Peginterferon Alfa-2a (Pegasys) in Participants With Chronic Myelogenous Leukemia (CML) (NCT NCT02736721)
NCT ID: NCT02736721
Last Updated: 2016-09-20
Results Overview
Hematologic response was considered to be achieved if participants met all of the following criteria: normalization of white blood cells count to less than (\<) 10\*10\^9 per liter (/L) with normal differentiation, normalization of platelet count at \<450\*10\^9/L, and disappearance of all signs and symptoms of the disease. This response had to be confirmed at least 4 weeks after the first measure and beyond that.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
41 participants
Primary outcome timeframe
Up to approximately 7 years
Results posted on
2016-09-20
Participant Flow
Participant milestones
| Measure |
Peginterferon Alfa-2a
Participants who have previously participated in study ML16544 (NCT number not available), NO16006 (NCT number not available) or ML17228 (NCT number not available) and deemed to be a responder, received peginterferon alfa-2a subcutaneously in doses between 90 and 450 microgram (mcg) once weekly until medically indicated as judged by the treating investigator. Maximum treatment duration was approximately up to 7 years.
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|---|---|
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Overall Study
STARTED
|
41
|
|
Overall Study
COMPLETED
|
25
|
|
Overall Study
NOT COMPLETED
|
16
|
Reasons for withdrawal
| Measure |
Peginterferon Alfa-2a
Participants who have previously participated in study ML16544 (NCT number not available), NO16006 (NCT number not available) or ML17228 (NCT number not available) and deemed to be a responder, received peginterferon alfa-2a subcutaneously in doses between 90 and 450 microgram (mcg) once weekly until medically indicated as judged by the treating investigator. Maximum treatment duration was approximately up to 7 years.
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|---|---|
|
Overall Study
Adverse Event
|
8
|
|
Overall Study
Lost to Follow-up
|
3
|
|
Overall Study
Other
|
5
|
Baseline Characteristics
Expanded Access Study With Peginterferon Alfa-2a (Pegasys) in Participants With Chronic Myelogenous Leukemia (CML)
Baseline characteristics by cohort
| Measure |
Peginterferon Alfa-2a
n=41 Participants
Participants who have previously participated in study ML16544, NO16006, or ML17228 and deemed to be a responder, received peginterferon alfa-2a subcutaneously in doses between 90 and 450 mcg once weekly until medically indicated as judged by the treating investigator. Maximum treatment duration was approximately up to 7 years.
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|---|---|
|
Age, Continuous
|
48.5 years
STANDARD_DEVIATION 14.1 • n=5 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
27 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to approximately 7 yearsPopulation: Analysis population included all enrolled participants.
Hematologic response was considered to be achieved if participants met all of the following criteria: normalization of white blood cells count to less than (\<) 10\*10\^9 per liter (/L) with normal differentiation, normalization of platelet count at \<450\*10\^9/L, and disappearance of all signs and symptoms of the disease. This response had to be confirmed at least 4 weeks after the first measure and beyond that.
Outcome measures
| Measure |
Peginterferon Alfa-2a
n=41 Participants
Participants who have previously participated in study ML16544, NO16006, or ML17228 and deemed to be a responder, received peginterferon alfa-2a subcutaneously in doses between 90 and 450 mcg once weekly until medically indicated as judged by the treating investigator. Maximum treatment duration was approximately up to 7 years.
|
|---|---|
|
Number of Participants With Complete Hematologic Response
|
40 participants
|
PRIMARY outcome
Timeframe: Up to approximately 7 yearsPopulation: Analysis population included all enrolled participants.
Time to loss of previous hematologic response was defined as the interval between the first day of treatment in the study and the first day of loss of previous hematologic response during elapsed time of study. Hematologic response was considered to be achieved if participants met all of the following criteria: normalization of white blood cells count to \<10\*10\^9/L with normal differentiation, normalization of platelet count at \<450\*10\^9/L, and disappearance of all signs and symptoms of the disease. This response had to be confirmed at least 4 weeks after the first measure and beyond that.
Outcome measures
| Measure |
Peginterferon Alfa-2a
n=41 Participants
Participants who have previously participated in study ML16544, NO16006, or ML17228 and deemed to be a responder, received peginterferon alfa-2a subcutaneously in doses between 90 and 450 mcg once weekly until medically indicated as judged by the treating investigator. Maximum treatment duration was approximately up to 7 years.
|
|---|---|
|
Time to Loss of Previous Hematologic Response
|
NA months
The median and corresponding 95% confidence interval (CI) data was not estimable because only 1 participant, out of total 41 participants, lost previous hematological response.
|
PRIMARY outcome
Timeframe: Up to approximately 7 yearsPopulation: Analysis population included all enrolled participants.
CyR was based on the prevalence of Philadelphia chromosome-positive (Ph+) bone marrow cells in metaphase determined from reverse transcriptase polymerase chain reaction (RT-PCR). Major cytogenetic response was categorized as either complete cytogenetic response (CCyR) or partial cytogenetic response (PCyR). CCyR was achieved when there was absence of detectable Ph+ bone marrow cells and PCyR was achieved when 1 to 34 percent (%) of bone marrow cells were Ph+.
Outcome measures
| Measure |
Peginterferon Alfa-2a
n=41 Participants
Participants who have previously participated in study ML16544, NO16006, or ML17228 and deemed to be a responder, received peginterferon alfa-2a subcutaneously in doses between 90 and 450 mcg once weekly until medically indicated as judged by the treating investigator. Maximum treatment duration was approximately up to 7 years.
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|---|---|
|
Number of Participants With Major Cytogenetic Response (CyR)
|
40 participants
|
PRIMARY outcome
Timeframe: Up to approximately 7 yearsPopulation: Analysis population included all enrolled participants.
Time to loss of previous CyR was defined as the interval between the first day of treatment in the study and the first day of loss of previous CyR. CyR is based on the prevalence of Ph+ bone marrow cells in metaphase. Major CyR was categorized as either CCyR or PCyR. CCyR was achieved when there was absence of detectable Ph+ bone marrow cells and PCyR was achieved when 1 to 34% of bone marrow cells were Ph+.
Outcome measures
| Measure |
Peginterferon Alfa-2a
n=41 Participants
Participants who have previously participated in study ML16544, NO16006, or ML17228 and deemed to be a responder, received peginterferon alfa-2a subcutaneously in doses between 90 and 450 mcg once weekly until medically indicated as judged by the treating investigator. Maximum treatment duration was approximately up to 7 years.
|
|---|---|
|
Time to Loss of Previous CyR
|
NA years
The median and corresponding 95% CI data was not estimable because only 1 participant, out of total 41 participants, lost previous CyR.
|
PRIMARY outcome
Timeframe: Up to approximately 7 yearsPopulation: Analysis population included all enrolled participants.
MR was assessed using breakpoint cluster region - Abelson (BCR-ABL) proto-oncogene transcript levels measured by RT-PCR from peripheral blood. Number of participants with BCR-ABL/ABL ratio less than or equal to 10 (%) was reported.
Outcome measures
| Measure |
Peginterferon Alfa-2a
n=41 Participants
Participants who have previously participated in study ML16544, NO16006, or ML17228 and deemed to be a responder, received peginterferon alfa-2a subcutaneously in doses between 90 and 450 mcg once weekly until medically indicated as judged by the treating investigator. Maximum treatment duration was approximately up to 7 years.
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|---|---|
|
Number of Participants With Molecular Response (MR)
|
38 participants
|
PRIMARY outcome
Timeframe: Up to approximately 7 yearsPopulation: Analysis population included all enrolled participants.
Time to loss of previous MR was defined as the interval between the first day of treatment in the study and the first day of loss of previous MR. MR was assessed using BCR-ABL transcript levels measured by RT-PCR from peripheral blood.
Outcome measures
| Measure |
Peginterferon Alfa-2a
n=41 Participants
Participants who have previously participated in study ML16544, NO16006, or ML17228 and deemed to be a responder, received peginterferon alfa-2a subcutaneously in doses between 90 and 450 mcg once weekly until medically indicated as judged by the treating investigator. Maximum treatment duration was approximately up to 7 years.
|
|---|---|
|
Time to Loss of Previous MR
|
NA years
The median and corresponding 95% CI data was not estimable because limited number of participants lost previous MR.
|
Adverse Events
Peginterferon Alfa-2a
Serious events: 13 serious events
Other events: 35 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Peginterferon Alfa-2a
n=41 participants at risk
Participants who have previously participated in study ML16544, NO16006, or ML17228 and deemed to be a responder, received peginterferon alfa-2a subcutaneously in doses between 90 and 450 mcg once weekly until medically indicated as judged by the treating investigator. Maximum treatment duration was approximately up to 7 years.
|
|---|---|
|
Cardiac disorders
Atrial fibrillation
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Gastrointestinal disorders
Intestinal haemorrhage
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
General disorders
General physical health deterioration
|
4.9%
2/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Hepatobiliary disorders
Cholelithiasis
|
4.9%
2/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Hepatobiliary disorders
Cholecystitis
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Infections and infestations
Appendicitis
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Infections and infestations
Cellulitis
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Infections and infestations
Gastroenteritis
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Infections and infestations
Infection
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Infections and infestations
Pneumonia
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Infections and infestations
Respiratory tract infection
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Infections and infestations
Urinary tract infection
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Musculoskeletal and connective tissue disorders
Osteoarthritis
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Musculoskeletal and connective tissue disorders
Osteonecrosis
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Musculoskeletal and connective tissue disorders
Rotator cuff syndrome
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Bladder neoplasm
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Bowen's disease
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Colon cancer
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Uterine leiomyoma
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Psychiatric disorders
Depression
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Surgical and medical procedures
Colostomy
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Surgical and medical procedures
Finger amputation
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Vascular disorders
Deep vein thrombosis
|
2.4%
1/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
Other adverse events
| Measure |
Peginterferon Alfa-2a
n=41 participants at risk
Participants who have previously participated in study ML16544, NO16006, or ML17228 and deemed to be a responder, received peginterferon alfa-2a subcutaneously in doses between 90 and 450 mcg once weekly until medically indicated as judged by the treating investigator. Maximum treatment duration was approximately up to 7 years.
|
|---|---|
|
Blood and lymphatic system disorders
Neutropenia
|
9.8%
4/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Blood and lymphatic system disorders
Leukopenia
|
7.3%
3/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
7.3%
3/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Gastrointestinal disorders
Nausea
|
9.8%
4/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Gastrointestinal disorders
Abdominal pain
|
7.3%
3/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
General disorders
Fatigue
|
12.2%
5/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
General disorders
Influenza like illness
|
12.2%
5/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
General disorders
Pyrexia
|
9.8%
4/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
General disorders
Oedema
|
7.3%
3/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Infections and infestations
Bronchitis
|
12.2%
5/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Infections and infestations
Nasopharyngitis
|
12.2%
5/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Infections and infestations
Upper respiratory tract infection
|
9.8%
4/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Infections and infestations
Cystitis
|
7.3%
3/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Infections and infestations
Respiratory tract infection
|
7.3%
3/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Infections and infestations
Sinusitis
|
7.3%
3/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
17.1%
7/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
12.2%
5/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
12.2%
5/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
9.8%
4/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
9.8%
4/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Psychiatric disorders
Depression
|
17.1%
7/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
12.2%
5/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Skin and subcutaneous tissue disorders
Night sweats
|
7.3%
3/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Skin and subcutaneous tissue disorders
Rash
|
7.3%
3/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
|
Vascular disorders
Hypertension
|
9.8%
4/41 • Up to approximately 7 years
Analysis population included all enrolled participants.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
- Publication restrictions are in place
Restriction type: OTHER