Expanded Access Study With Peginterferon Alfa-2a (Pegasys) in Participants With Chronic Myelogenous Leukemia (CML)

Multicenter Open Label Expanded Access-Program of Pegylated Interferon Alfa-2a (Pegasys) in Patients With Chronic Myelogenous Leukemia (CML)

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02736721

Enrollment

Registered

2016-04-13

Start date

2003-09-30

Completion date

2010-11-30

Last updated

2016-09-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Myelogenous Leukemia, Chronic

Brief summary

This study will evaluate the efficacy, safety and tolerability of long-term use of peginterferon alfa-2a in participants with CML who have previously participated in peginterferon alfa-2a study ML16544 (NCT number not available), NO16006 (NCT number not available) or ML17228 (NCT number not available) and treating physician has decided to continue treatment with peginterferon alfa-2a within the frame of another clinical study.

Interventions

DRUGPeginterferon alfa-2a

Peginterferon alfa-2a in doses between 90 and 450 mcg will be administered subcutaneously once weekly until medically indicated as judged by the treating investigator.

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

* Participants with CML * Previous participation in studies: ML16544, NO16006 or ML17228; for which the treating physician recommends a continuation of the study drug according to defined minimal criteria

Exclusion criteria

* Major protocol violator in the participated study prior to participation in this study

Design outcomes

Primary

Measure	Time frame	Description
Number of Participants With Complete Hematologic Response	Up to approximately 7 years	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.
Time to Loss of Previous Hematologic Response	Up to approximately 7 years	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.
Number of Participants With Major Cytogenetic Response (CyR)	Up to approximately 7 years	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+.
Time to Loss of Previous CyR	Up to approximately 7 years	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+.
Number of Participants With Molecular Response (MR)	Up to approximately 7 years	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.
Time to Loss of Previous MR	Up to approximately 7 years	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.

Countries

Germany

Participant flow

Participants by arm

Arm	Count
Peginterferon Alfa-2a 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.	41
Total	41

Withdrawals & dropouts

Period	Reason	FG000
Overall Study	Adverse Event	8
Overall Study	Lost to Follow-up	3
Overall Study	Other	5

Baseline characteristics

Characteristic	Peginterferon Alfa-2a
Age, Continuous	48.5 years STANDARD_DEVIATION 14.1
Sex: Female, Male Female	14 Participants
Sex: Female, Male Male	27 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	— / —
other Total, other adverse events	35 / 41
serious Total, serious adverse events	13 / 41

Outcome results

Primary

Number of Participants With Complete Hematologic Response

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.