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| ID | Type | Description | Link |
|---|---|---|---|
| NCT05407805 | Registry Identifier | ClinicalTrials.gov |
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The purpose of this clinical trial is to evaluate the performance of the sickle cell disease (SCD) electronic diary in people with SCD who are on treatment that will change SCD and those not on such a treatment.
SCD is a type of condition when there are fewer red blood cells to carry oxygen around the body.
This disease can be passed on from parent to child and may cause pain, infections and damage to organs.
This study is seeking participants who:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Control Group | SCD participants not on disease modifying treatment. |
| |
| SCD Disease Modifying Treatment Group | SCD participants on a stable dose of a SCD disease modifying treatment regimen. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Electronic Diary | Other | Participants will be asked to complete a daily electronic patient reported outcome diary entry to report on their experience in the past 24 hours. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Physician-reported Medical Utilization (MU) Vaso-occlusive Crisis (VOC) Rate | Physician-reported MU VOC: defined as an acute episode of pain with no other cause other than a VOC event that required a medical facility visit or contact with a health care professional and treatment with oral or parenteral narcotics, or non-steroidal anti-inflammatory drugs. Acute chest syndrome, hepatic sequestration, splenic sequestration, and priapism (requiring a visit to a medical facility) were also considered MU VOC. Contact with healthcare professional included: called healthcare provider (or telemedicine visit) and received treatment, went to clinic and received treatment, went to emergency department and received treatment, admitted to the hospital and received treatment. VOC rate was derived as annualized rate. Annualized MU VOC rate = (Number of MU VOC events * 365)/ (number of days in the observation period). | Baseline up to Day 180 |
| VOC Day Rate | A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. VOC day was a self-report by the participant of experiencing a VOC during the past 24 hours. This was assessed through a dichotomous (Yes/No) item on the SCD ePRO system, "Did you have a pain crisis in the past 24 hours?" A response of "Yes" indicated a VOC day. VOC day rate was derived as annualized rate. VOC day rate = (Number of VOC days * 365)/ (number of days in the observation period). | Baseline up to Day 180 |
| Patient-reported VOC Event Rate | A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. Patient reported VOC Event is used to define a sequence of VOC days that could also include single intervening days with no pain crisis. The subsequent occurrence of two consecutive days with no pain crisis operationally defines the end of the respective VOC event. Rate were derived as annualized rate. Annualized VOC event rate = (Number of VOC events * 365)/ (number of days in the observation period). | Baseline up to Day 180 |
| Sickle Cell Disease (SCD) Electronic Patient Reported Outcome (ePRO) Daily Worst Pain Scores by VOC Status |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change in Physician-reported MU VOC Rate Per Unit of Change in VOC Day Rate | MU VOC: acute episode of pain with no other cause other than VOC event that required medical facility visit/contact with health care professional and treatment with oral/parenteral narcotics/NSAIDs. Acute chest syndrome,hepatic/splenic sequestration, priapism also considered MU VOC. MU VOC derived as annualized rate by:(Number of MU VOC events*365)/(number of days in observation period). VOC day rate: VOC day was self-report by participant experiencing VOC during past 24 hours. It was assessed by dichotomous (Yes/No) item on SCD ePRO system, "Did you have pain crisis in past 24 hours?" Response "Yes" indicated VOC day. VOC day rate derived as annualized rate by: (Number of VOC days*365)/(number of days in observation period). Parameter of interest was % change of MU VOC rate per unit of change in VOC Day rate. It was estimated via Negative Binomial model to evaluate association between physician-reported MU VOC rate and SCD ePRO VOC Day rate and was reported with corresponding 95% CI. |
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Inclusion Criteria (All Groups):
- Confirmed diagnosis of stable SCD (HbS/S or HbS/beta-zero-thalassemia).
Additional Inclusion Criteria (No Disease Modifying Treatment Control Group):
Additional Inclusion Criteria (SCD Disease Modifying Treatment Group):
Have experienced ≥1 episode(s) of MU VOC within 12 months prior to initiation of HU and/or crizanlizumab (whichever was initiated earlier).
Must be on a stable dose of their SCD treatment regimen ≥8 weeks prior to Day 1 with the intent of remaining on the same dose throughout the study, unless adjustments are medically necessary due to bone marrow suppression, in accordance with published guidelines and/or product specific guidance (eg, package label). Accepted SCD disease modifying treatment regimens include:
Data available for number of MU VOC(s) during the 12-month interval prior to initiation of any SCD disease modifying treatment, as described above, and a value for %HbF collected subsequent to 1 year of age, prior to initiation of any HU treatment, and in the absence of recent transfusion.
Exclusion Criteria (All Groups):
Additional Exclusion Criteria (No Disease Modifying Treatment Control Group):
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Participants with a confirmed diagnosis of stable Sickle Cell Disease (SCD) (hemoglobin S inherited from both parents [HbS/S] or hemoglobin S inherited from one parent and hemoglobin beta thalassemia inherited from the other parent [HbS/beta-zero-thalassemia] genotype) who are either not on disease modifying treatment or on a stable dose of a SCD disease modifying treatment regimen.
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Foundation for Sickle Cell Disease Research | Hollywood | Florida | 33024 | United States | ||
| Mid-Atlantic Permanente Medical Group Largo Medical Center |
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| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.
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Participants were asked to complete a daily SCD electronic patient reported outcome (ePRO) diary entry to report on their experience in the past 24 hours from Day 1 to Day 180.
Participants with stable sickle cell disease (SCD) (hemoglobin S inherited from both parents [HbS/S] or hemoglobin S inherited from one parent and hemoglobin beta thalassemia inherited from the other parent [HbS/beta-zero-thalassemia] genotype) were enrolled in two concurrent groups and were observed for 6 months in this study. This was an observational study, and under this study no therapeutic study interventions were administered.
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| ID | Title | Description |
|---|---|---|
| FG000 | Control Group | Eligible participants who were not on any disease modifying treatment for SCD were included in this group. |
| FG001 | Disease-Modifying Treatment Group | Eligible participants who were on a stable dose of disease modifying treatment for SCD under routine clinical practice in real world setting were included. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Evaluable Analysis population included all participants who signed the informed consent document and met the eligibility criteria.
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| ID | Title | Description |
|---|---|---|
| BG000 | Control Group | Eligible participants who were not on any disease modifying treatment for SCD were included in this group. |
| BG001 | Disease-Modifying Treatment Group | Eligible participants who were on a stable dose of disease modifying treatment for SCD under routine clinical practice in real world setting were included. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Physician-reported Medical Utilization (MU) Vaso-occlusive Crisis (VOC) Rate | Physician-reported MU VOC: defined as an acute episode of pain with no other cause other than a VOC event that required a medical facility visit or contact with a health care professional and treatment with oral or parenteral narcotics, or non-steroidal anti-inflammatory drugs. Acute chest syndrome, hepatic sequestration, splenic sequestration, and priapism (requiring a visit to a medical facility) were also considered MU VOC. Contact with healthcare professional included: called healthcare provider (or telemedicine visit) and received treatment, went to clinic and received treatment, went to emergency department and received treatment, admitted to the hospital and received treatment. VOC rate was derived as annualized rate. Annualized MU VOC rate = (Number of MU VOC events * 365)/ (number of days in the observation period). | Matched for efficacy population set consisted of all enrolled participants who had at least one SCD electronic patient reported outcome (ePRO) recording and was selected for analysis via matching procedure. | Posted | Median | Full Range | Events per year | Baseline up to Day 180 |
Day 1 up to Day 180
Evaluable analysis population included all participants who signed the informed consent document and met the eligibility criteria.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Control Group | Eligible participants who were not on any disease modifying treatment for SCD were included in this group. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 27.0 | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Aug 25, 2022 | Jun 2, 2025 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | May 29, 2024 | Jun 2, 2025 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D000098644 | Vaso-Occlusive Crises |
| D012805 | Sickle Cell Trait |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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|
A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. VOC day was defined as the day on which a SCD participant self-reports sickle pain crisis that was recorded in the SCD ePRO. A non-VOC day was defined as the day on which a SCD participant does not self-reports sickle pain crisis. Participants rated their pain by selecting the one number that best described their pain at its worst in the past 24 hours from 0-10, where 0= no pain and 10= as bad as you can imagine. Higher scores indicated worse pain. Data in this outcome measure was presented separately for VOC state and non-VOC state. |
| Baseline up to Day 180 |
| Sickle Cell Disease Electronic Patient Reported Outcome Daily Worst Tiredness Scores by VOC Status | A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. VOC day was defined as the day on which a SCD participant self-reports sickle pain crisis that was recorded in the SCD ePRO. A non-VOC day was defined as the day on which a SCD participant does not self-reports sickle pain crisis. Participants rated their tiredness by selecting the one number that best described their tiredness at its worst in the past 24 hours from 0-10, where 0= no tiredness and 10= as bad as you can imagine. Higher scores indicated worse tiredness. Data in this outcome measure was presented separately for VOC state and non-VOC state. | Baseline up to Day 180 |
| Sickle Cell Disease Electronic Patient Reported Outcome Daily Rating for Ability to Perform Usual Physical Activity (UPA) by VOC Status | A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. VOC day was defined as the day on which a SCD participant self-reports sickle pain crisis that was recorded in the SCD ePRO. A non-VOC day was defined as the day on which a SCD participant does not self-reports sickle pain crisis. A measure of participant's ability to perform their UPA (e.g. walking, climbing stairs, or household chores) during a VOC event was assessed on SCD ePRO recorded by participants using a scale ranging from 1-4 scale, where 1= able to perform with no difficulty and 4= unable to perform usual physical activities, where higher score indicated worse status. Data in this outcome measure was presented separately for VOC state and non-VOC state. | Baseline up to Day 180 |
| Baseline up to Day 180 |
| Percent Change in Physician-reported MU VOC Rate Per Unit of Change in Patient-reported VOC Event Rate | MU VOC: acute episode of pain with no other cause other than VOC event that required medical facility visit/contact with health care professional and treatment with oral/parenteral narcotics/NSAIDs. Acute chest syndrome, hepatic/splenic sequestration, priapism also considered MU VOC. MU VOC derived as annualized rate by:(Number of MU VOC events*365)/(number of days in observation period). VOC Event rate: Patient-reported VOC Event is used to define a sequence of VOC days. The subsequent occurrence of two consecutive days with no pain crisis operationally defines the end of the respective VOC event. Rate were derived as annualized rate by: (Number of VOC events * 365)/ (number of days in the observation period). Parameter of interest was % change of MU VOC rate per unit of change in VOC Event rate. It was estimated via Negative Binomial model to evaluate association between physician-reported MU VOC rate and SCD ePRO VOC Event rate and was reported with corresponding 95% CI. | Baseline up to Day 180 |
| Upper Marlboro |
| Maryland |
| 20774 |
| United States |
| Sanguine Biosciences, Inc. | Waltham | Massachusetts | 02451 | United States |
| Cohen Children's Medical Center | New Hyde Park | New York | 11040 | United States |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| ID |
|---|
| Title |
|---|
| Description |
|---|
| OG000 | Control Group | Eligible participants who were not on any disease modifying treatment for SCD were included in this group. |
| OG001 | Disease-Modifying Treatment Group | Eligible participants who were on a stable dose of disease modifying treatment for SCD under routine clinical practice in real world setting were included. |
|
|
|
| Primary | VOC Day Rate | A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. VOC day was a self-report by the participant of experiencing a VOC during the past 24 hours. This was assessed through a dichotomous (Yes/No) item on the SCD ePRO system, "Did you have a pain crisis in the past 24 hours?" A response of "Yes" indicated a VOC day. VOC day rate was derived as annualized rate. VOC day rate = (Number of VOC days * 365)/ (number of days in the observation period). | Matched for efficacy population set consisted of all enrolled participants who had at least one SCD ePRO recording and was selected for analysis via matching procedure. | Posted | Mean | 95% Confidence Interval | Days per year | Baseline up to Day 180 |
|
|
|
|
| Primary | Patient-reported VOC Event Rate | A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. Patient reported VOC Event is used to define a sequence of VOC days that could also include single intervening days with no pain crisis. The subsequent occurrence of two consecutive days with no pain crisis operationally defines the end of the respective VOC event. Rate were derived as annualized rate. Annualized VOC event rate = (Number of VOC events * 365)/ (number of days in the observation period). | Matched for efficacy population set consisted of all enrolled participants who had at least one SCD ePRO recording and was selected for analysis via matching procedure. | Posted | Mean | 95% Confidence Interval | Events per year | Baseline up to Day 180 |
|
|
|
|
| Primary | Sickle Cell Disease (SCD) Electronic Patient Reported Outcome (ePRO) Daily Worst Pain Scores by VOC Status | A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. VOC day was defined as the day on which a SCD participant self-reports sickle pain crisis that was recorded in the SCD ePRO. A non-VOC day was defined as the day on which a SCD participant does not self-reports sickle pain crisis. Participants rated their pain by selecting the one number that best described their pain at its worst in the past 24 hours from 0-10, where 0= no pain and 10= as bad as you can imagine. Higher scores indicated worse pain. Data in this outcome measure was presented separately for VOC state and non-VOC state. | Matched for efficacy population set consisted of all enrolled participants who had at least one SCD ePRO recording and was selected for analysis via matching procedure. Here, "Overall Number of Participants Analyzed" signifies participants evaluable for this outcome measure. "Number Analyzed" signifies number of participants evaluable for specified rows. | Posted | Least Squares Mean | 95% Confidence Interval | Score on a scale | Baseline up to Day 180 |
|
|
|
|
| Primary | Sickle Cell Disease Electronic Patient Reported Outcome Daily Worst Tiredness Scores by VOC Status | A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. VOC day was defined as the day on which a SCD participant self-reports sickle pain crisis that was recorded in the SCD ePRO. A non-VOC day was defined as the day on which a SCD participant does not self-reports sickle pain crisis. Participants rated their tiredness by selecting the one number that best described their tiredness at its worst in the past 24 hours from 0-10, where 0= no tiredness and 10= as bad as you can imagine. Higher scores indicated worse tiredness. Data in this outcome measure was presented separately for VOC state and non-VOC state. | Matched for efficacy population set consisted of all enrolled participants who had at least one SCD ePRO recording and was selected for analysis via matching procedure. Here, "Overall Number of Participants" signifies participants evaluable for this outcome measure. "Number Analyzed" signifies number of participants evaluable for specified rows. | Posted | Least Squares Mean | 95% Confidence Interval | Score on a scale | Baseline up to Day 180 |
|
|
|
|
| Primary | Sickle Cell Disease Electronic Patient Reported Outcome Daily Rating for Ability to Perform Usual Physical Activity (UPA) by VOC Status | A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. VOC day was defined as the day on which a SCD participant self-reports sickle pain crisis that was recorded in the SCD ePRO. A non-VOC day was defined as the day on which a SCD participant does not self-reports sickle pain crisis. A measure of participant's ability to perform their UPA (e.g. walking, climbing stairs, or household chores) during a VOC event was assessed on SCD ePRO recorded by participants using a scale ranging from 1-4 scale, where 1= able to perform with no difficulty and 4= unable to perform usual physical activities, where higher score indicated worse status. Data in this outcome measure was presented separately for VOC state and non-VOC state. | Matched for efficacy population set consisted of all enrolled participants who had at least one SCD ePRO recording and was selected for analysis via matching procedure. Here, "Overall Number of Participants" signifies participants evaluable for this outcome measure. "Number Analyzed" signifies number of participants evaluable for specified rows. | Posted | Least Squares Mean | 95% Confidence Interval | Score on a scale | Baseline up to Day 180 |
|
|
|
|
| Secondary | Percent Change in Physician-reported MU VOC Rate Per Unit of Change in VOC Day Rate | MU VOC: acute episode of pain with no other cause other than VOC event that required medical facility visit/contact with health care professional and treatment with oral/parenteral narcotics/NSAIDs. Acute chest syndrome,hepatic/splenic sequestration, priapism also considered MU VOC. MU VOC derived as annualized rate by:(Number of MU VOC events*365)/(number of days in observation period). VOC day rate: VOC day was self-report by participant experiencing VOC during past 24 hours. It was assessed by dichotomous (Yes/No) item on SCD ePRO system, "Did you have pain crisis in past 24 hours?" Response "Yes" indicated VOC day. VOC day rate derived as annualized rate by: (Number of VOC days*365)/(number of days in observation period). Parameter of interest was % change of MU VOC rate per unit of change in VOC Day rate. It was estimated via Negative Binomial model to evaluate association between physician-reported MU VOC rate and SCD ePRO VOC Day rate and was reported with corresponding 95% CI. | Analysis population included all participants who signed the informed consent document and met the eligibility criteria. One participant was originally included into control group and later was found to receive disease modifying treatment during 18 months before enrollment and was excluded from analysis. | Posted | Number | Percent change | Baseline up to Day 180 |
|
|
|
| Secondary | Percent Change in Physician-reported MU VOC Rate Per Unit of Change in Patient-reported VOC Event Rate | MU VOC: acute episode of pain with no other cause other than VOC event that required medical facility visit/contact with health care professional and treatment with oral/parenteral narcotics/NSAIDs. Acute chest syndrome, hepatic/splenic sequestration, priapism also considered MU VOC. MU VOC derived as annualized rate by:(Number of MU VOC events*365)/(number of days in observation period). VOC Event rate: Patient-reported VOC Event is used to define a sequence of VOC days. The subsequent occurrence of two consecutive days with no pain crisis operationally defines the end of the respective VOC event. Rate were derived as annualized rate by: (Number of VOC events * 365)/ (number of days in the observation period). Parameter of interest was % change of MU VOC rate per unit of change in VOC Event rate. It was estimated via Negative Binomial model to evaluate association between physician-reported MU VOC rate and SCD ePRO VOC Event rate and was reported with corresponding 95% CI. | Analysis population included all participants who signed the informed consent document and met the eligibility criteria. One participant was originally included into control group and later was found to receive disease modifying treatment during 18 months before enrollment and was excluded from analysis. | Posted | Number | Percent change | Baseline up to Day 180 |
|
|
|
| 0 |
| 32 |
| 0 |
| 32 |
| 0 |
| 32 |
| EG001 | Disease-Modifying Treatment Group | Eligible participants who were on a stable dose of disease modifying treatment for SCD under routine clinical practice in real world setting were included. | 0 | 65 | 0 | 65 | 1 | 65 |
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publication until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| Non-VOC State |
|
|
| =0.4314 |
| LS Mean Difference |
| 0.5 |
| 2-Sided |
| 95 |
| -0.7 |
| 1.7 |
| Superiority |
| Non-VOC State |
|
|
| =0.8262 |
| LS Mean Difference |
| 0.1 |
| 2-Sided |
| 95 |
| -1.1 |
| 1.4 |
| Superiority |
| Non-VOC State |
|
|
| =0.3335 |
| LS Mean Difference |
| 0.1 |
| 2-Sided |
| 95 |
| -0.1 |
| 0.3 |
| Superiority |