Trial Outcomes & Findings for A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy (NCT NCT04718779)
NCT ID: NCT04718779
Last Updated: 2023-12-21
Results Overview
Hemoglobin concentration level was assessed from the blood sample. Clinical stability from baseline was calculated using prespecified threshold of hemoglobin concentration level less than (\<) 1.5 gram per deciliter (g/dl) decrease level. Number of participants with clinically stable hemoglobin from baseline up to Month 12 were reported.
COMPLETED
PHASE4
4 participants
From Baseline up to Month 12
2023-12-21
Participant Flow
The study was conducted at 3 sites in United States from 22 April 2021 to 16 February 2023.
A total of 4 participants were enrolled in the study. This is an interventional, retrospective and prospective study to describe the effect of the treatment change on the clinical parameters and patient reported outcomes (PROs) with the use of a digital engagement application.
Participant milestones
| Measure |
Arm A: Prospective
Participants with Gaucher's Disease 1 (GD1) who were treated with substrate reduction therapy (SRT) for at least 3 months before being switched to enzyme replacement therapy (ERT) velaglucerase alfa (VPRIV) were followed-up prospectively for 12 months from the switch from SRT to ERT (baseline). All participants were treated in accordance with physician treatment plan (standard clinical practice).
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Arm B: Retrospective
Participants with GD1 transitioning from SRT to ERT (VPRIV) or ERT to SRT and then to ERT (VPRIV) previously (within the past 5 years at the time of enrollment) were followed-up retrospectively for up to 12 months before being switched from SRT to ERT (VPRIV). All participants were treated in accordance with physician treatment plan (standard clinical practice).
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|---|---|---|
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Overall Study
STARTED
|
2
|
2
|
|
Overall Study
COMPLETED
|
2
|
2
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy
Baseline characteristics by cohort
| Measure |
All GD1 Participants
n=4 Participants
All participants with GD1 who were treated with SRT for at least 3 months before being switched to ERT (VPRIV) were followed-up prospectively for 12 months from the switch from SRT to ERT (baseline); and participants transitioning from SRT to ERT (VPRIV) or ERT to SRT and then to ERT (VPRIV) previously (within the past 5 years at the time of enrollment) were followed-up retrospectively for up to 12 months before being switched from SRT to ERT (VPRIV). All participants were treated in accordance with physician treatment plan (standard clinical practice).
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|---|---|
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Age, Continuous
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45.8 Years
STANDARD_DEVIATION 22.5 • n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From Baseline up to Month 12Population: Analysis population included all eligible participants whose medical charts were retrieved and observed in this study.
Hemoglobin concentration level was assessed from the blood sample. Clinical stability from baseline was calculated using prespecified threshold of hemoglobin concentration level less than (\<) 1.5 gram per deciliter (g/dl) decrease level. Number of participants with clinically stable hemoglobin from baseline up to Month 12 were reported.
Outcome measures
| Measure |
Arm A: Prospective
n=2 Participants
Participants with GD1 who were treated with SRT for at least 3 months before being switched to ERT (VPRIV) were followed-up prospectively for 12 months from the switch from SRT to ERT (baseline). All participants were treated in accordance with physician treatment plan (standard clinical practice).
|
Arm B: Retrospective
n=2 Participants
Participants with GD1 transitioning from SRT to ERT (VPRIV) or ERT to SRT and then to ERT (VPRIV) previously (within the past 5 years at the time of enrollment) were followed-up retrospectively for up to 12 months before being switched from SRT to ERT (VPRIV). All participants were treated in accordance with physician treatment plan (standard clinical practice).
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|---|---|---|
|
Number of Participants With Clinically Stable Hemoglobin (Hb) Concentration From Baseline up to Month 12
|
2 Participants
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2 Participants
|
PRIMARY outcome
Timeframe: From Baseline up to Month 12Population: Analysis population included all eligible participants whose medical charts were retrieved and observed in this study.
Platelet count concentration level was assessed from the blood sample. Clinical stability from baseline was calculated using prespecified threshold of platelet count \< 25 percent (%) decrease levels. Number of participants with clinically stable platelet count from baseline up to Month 12 were reported.
Outcome measures
| Measure |
Arm A: Prospective
n=2 Participants
Participants with GD1 who were treated with SRT for at least 3 months before being switched to ERT (VPRIV) were followed-up prospectively for 12 months from the switch from SRT to ERT (baseline). All participants were treated in accordance with physician treatment plan (standard clinical practice).
|
Arm B: Retrospective
n=2 Participants
Participants with GD1 transitioning from SRT to ERT (VPRIV) or ERT to SRT and then to ERT (VPRIV) previously (within the past 5 years at the time of enrollment) were followed-up retrospectively for up to 12 months before being switched from SRT to ERT (VPRIV). All participants were treated in accordance with physician treatment plan (standard clinical practice).
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|---|---|---|
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Number of Participants With Clinically Stable Platelet Count From Baseline in Platelet Count up to Month 12
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2 Participants
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2 Participants
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PRIMARY outcome
Timeframe: From Baseline up to Month 12Population: Data for this outcome measure could not be collected and analyzed as no participants were evaluable for the specified parameter.
Clinical stability from baseline was calculated using prespecified threshold of liver volume \< 20 % increase, was assessed using ultrasound or Magnetic Resonance Image (MRI). Number of participants with clinically stable liver volume from baseline up to Month 12 were reported.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: From Baseline up to Month 12Population: Data for this outcome measure could not be collected and analyzed as no participants were evaluable for the specified parameter.
Clinical stability from baseline was calculated using prespecified threshold of spleen volume \< 25 % increase was assessed using ultrasound or MRI. Number of participants with clinically stable spleen volume from baseline up to Month 12 were reported.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From start of the study up to Month 12Population: Analysis population included all eligible participants whose medical charts were retrieved and observed in this study.
An AE was defined as any untoward medical occurrence in a clinical investigation participant administered with a drug; it does not necessarily have to have a causal relationship with the treatment. An SAE was any event that resulted in: death; was life-threatening; required inpatient hospitalization or resulted in prolongation of existing hospitalization; persistent or significant disability/incapacity; was a congenital anomaly/birth defect or a medically important event. AEs included both SAEs, and non-serious AEs.
Outcome measures
| Measure |
Arm A: Prospective
n=2 Participants
Participants with GD1 who were treated with SRT for at least 3 months before being switched to ERT (VPRIV) were followed-up prospectively for 12 months from the switch from SRT to ERT (baseline). All participants were treated in accordance with physician treatment plan (standard clinical practice).
|
Arm B: Retrospective
n=2 Participants
Participants with GD1 transitioning from SRT to ERT (VPRIV) or ERT to SRT and then to ERT (VPRIV) previously (within the past 5 years at the time of enrollment) were followed-up retrospectively for up to 12 months before being switched from SRT to ERT (VPRIV). All participants were treated in accordance with physician treatment plan (standard clinical practice).
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|---|---|---|
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Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants with AEs
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0 Participants
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0 Participants
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|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants with Serious AEs
|
0 Participants
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0 Participants
|
Adverse Events
Arm A: Prospective
Arm B: Retrospective
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
- Publication restrictions are in place
Restriction type: OTHER