Trial Outcomes & Findings for Novel Cardiac Magnetic Resonance Imaging to Define a Unique Restrictive Cardiomyopathy in Sickle Cell Disease (NCT NCT02410811)
NCT ID: NCT02410811
Last Updated: 2020-11-02
Results Overview
The occurrence of an abnormally increased extracellular volume (ECV) measurement \[i.e., the presence of the diffuse myocardial fibrosis phenotype\] as assessed by cardiac magnetic resonance imaging (CMR) using T1 mapping before and after administration of gadolinium. Expressed as number of participants with the diffuse myocardial fibrosis phenotype in each stratum.
Recruitment status
COMPLETED
Target enrollment
33 participants
Primary outcome timeframe
Assessed annually over a 2-year period (3 assessments over 2 years)
Results posted on
2020-11-02
Participant Flow
Participant milestones
| Measure |
Stratum A (6-13.99 Years)
* Age 6 to 13.99 years.
* Sickle cell anemia or sickle-β0-thalassemia.
|
Stratum B (14-20.99 Years)
* Age 14 to 20.99 years.
* Sickle cell anemia or sickle-β0-thalassemia.
* Detectible and quantifiable tricuspid regurgitant jet velocity (TRJV).
|
Stratum C (≥21 Years)
* Age ≥21 years.
* Sickle cell anemia or sickle-β0-thalassemia.
* Detectible and quantifiable tricuspid regurgitant jet velocity (TRJV).
|
Stratum D (Early Treatment)
* Age ≥6 years.
* Sickle cell anemia or sickle-β0-thalassemia.
* Current use of disease-modifying therapy \[hydroxyurea, chronic transfusions, or both (given concurrently, sequentially, or both)\] that was initiated at \<3 years of age, and for which there has been no interruption of therapy for \>6 consecutive months since the initiation of disease-modifying therapy.
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
5
|
10
|
11
|
7
|
|
Overall Study
COMPLETED
|
5
|
10
|
10
|
7
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
1
|
0
|
Reasons for withdrawal
| Measure |
Stratum A (6-13.99 Years)
* Age 6 to 13.99 years.
* Sickle cell anemia or sickle-β0-thalassemia.
|
Stratum B (14-20.99 Years)
* Age 14 to 20.99 years.
* Sickle cell anemia or sickle-β0-thalassemia.
* Detectible and quantifiable tricuspid regurgitant jet velocity (TRJV).
|
Stratum C (≥21 Years)
* Age ≥21 years.
* Sickle cell anemia or sickle-β0-thalassemia.
* Detectible and quantifiable tricuspid regurgitant jet velocity (TRJV).
|
Stratum D (Early Treatment)
* Age ≥6 years.
* Sickle cell anemia or sickle-β0-thalassemia.
* Current use of disease-modifying therapy \[hydroxyurea, chronic transfusions, or both (given concurrently, sequentially, or both)\] that was initiated at \<3 years of age, and for which there has been no interruption of therapy for \>6 consecutive months since the initiation of disease-modifying therapy.
|
|---|---|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
0
|
1
|
0
|
Baseline Characteristics
Novel Cardiac Magnetic Resonance Imaging to Define a Unique Restrictive Cardiomyopathy in Sickle Cell Disease
Baseline characteristics by cohort
| Measure |
Stratum A (6-13.99 Years)
n=5 Participants
* Age 6 to 13.99 years.
* Sickle cell anemia or sickle-β0-thalassemia.
|
Stratum B (14-20.99 Years)
n=10 Participants
* Age 14 to 20.99 years.
* Sickle cell anemia or sickle-β0-thalassemia.
* Detectible and quantifiable tricuspid regurgitant jet velocity (TRJV).
|
Stratum C (≥21 Years)
n=11 Participants
* Age ≥21 years.
* Sickle cell anemia or sickle-β0-thalassemia.
* Detectible and quantifiable tricuspid regurgitant jet velocity (TRJV).
|
Stratum D (Early Treatment)
n=7 Participants
* Age ≥6 years.
* Sickle cell anemia or sickle-β0-thalassemia.
* Current use of disease-modifying therapy \[hydroxyurea, chronic transfusions, or both (given concurrently, sequentially, or both)\] that was initiated at \<3 years of age, and for which there has been no interruption of therapy for \>6 consecutive months since the initiation of disease-modifying therapy.
|
Total
n=33 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
5 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
16 Participants
n=21 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
17 Participants
n=21 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Age, Continuous
|
12.0 years
STANDARD_DEVIATION 9.2 • n=5 Participants
|
18.0 years
STANDARD_DEVIATION 9.1 • n=7 Participants
|
35.8 years
STANDARD_DEVIATION 13.1 • n=5 Participants
|
13.4 years
STANDARD_DEVIATION 6.9 • n=4 Participants
|
22.1 years
STANDARD_DEVIATION 12.7 • n=21 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
20 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
13 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
5 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
33 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Black or African American
|
5 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
33 Participants
n=21 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Region of Enrollment
United States
|
5 participants
n=5 Participants
|
10 participants
n=7 Participants
|
11 participants
n=5 Participants
|
7 participants
n=4 Participants
|
33 participants
n=21 Participants
|
|
Sickle cell disease genotype
Homozygous sickle cell anemia (HbSS)
|
4 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
32 Participants
n=21 Participants
|
|
Sickle cell disease genotype
Sickle-β0-thalassemia (HbSβ0)
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
PRIMARY outcome
Timeframe: Assessed annually over a 2-year period (3 assessments over 2 years)The occurrence of an abnormally increased extracellular volume (ECV) measurement \[i.e., the presence of the diffuse myocardial fibrosis phenotype\] as assessed by cardiac magnetic resonance imaging (CMR) using T1 mapping before and after administration of gadolinium. Expressed as number of participants with the diffuse myocardial fibrosis phenotype in each stratum.
Outcome measures
| Measure |
Stratum A (6-13.99 Years)
n=5 Participants
* Age 6 to 13.99 years.
* Sickle cell anemia or sickle-β0-thalassemia.
|
Stratum B (14-20.99 Years)
n=10 Participants
* Age 14 to 20.99 years.
* Sickle cell anemia or sickle-β0-thalassemia.
* Detectible and quantifiable tricuspid regurgitant jet velocity (TRJV).
|
Stratum C (≥21 Years)
n=10 Participants
* Age ≥21 years.
* Sickle cell anemia or sickle-β0-thalassemia.
* Detectible and quantifiable tricuspid regurgitant jet velocity (TRJV).
|
Stratum D (Early Treatment)
n=7 Participants
* Age ≥6 years.
* Sickle cell anemia or sickle-β0-thalassemia.
* Current use of disease-modifying therapy \[hydroxyurea, chronic transfusions, or both (given concurrently, sequentially, or both)\] that was initiated at \<3 years of age, and for which there has been no interruption of therapy for \>6 consecutive months since the initiation of disease-modifying therapy.
|
|---|---|---|---|---|
|
Frequency of the Diffuse Myocardial Fibrosis Phenotype
|
5 Participants
|
10 Participants
|
10 Participants
|
1 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Assessed annually over a 2-year period (3 assessments over 2 years)Population: Stratum D participants had a single CMR assessment only; no longitudinal data were collected.
The occurrence of a change \[from the baseline assessment\] in the classification \[presence or absence\] of the diffuse myocardial fibrosis phenotype, which is defined as an abnormally increased extracellular volume (ECV) measurement as assessed by cardiac magnetic resonance imaging (CMR) using T1 mapping before and after administration of gadolinium. Expressed as number of participants who had a change in classification of the diffuse myocardial fibrosis phenotype \[e.g., presence to absence, or absence to presence\] during the 2-year study in each stratum.
Outcome measures
| Measure |
Stratum A (6-13.99 Years)
n=5 Participants
* Age 6 to 13.99 years.
* Sickle cell anemia or sickle-β0-thalassemia.
|
Stratum B (14-20.99 Years)
n=10 Participants
* Age 14 to 20.99 years.
* Sickle cell anemia or sickle-β0-thalassemia.
* Detectible and quantifiable tricuspid regurgitant jet velocity (TRJV).
|
Stratum C (≥21 Years)
n=10 Participants
* Age ≥21 years.
* Sickle cell anemia or sickle-β0-thalassemia.
* Detectible and quantifiable tricuspid regurgitant jet velocity (TRJV).
|
Stratum D (Early Treatment)
* Age ≥6 years.
* Sickle cell anemia or sickle-β0-thalassemia.
* Current use of disease-modifying therapy \[hydroxyurea, chronic transfusions, or both (given concurrently, sequentially, or both)\] that was initiated at \<3 years of age, and for which there has been no interruption of therapy for \>6 consecutive months since the initiation of disease-modifying therapy.
|
|---|---|---|---|---|
|
Stability of the Diffuse Myocardial Fibrosis Phenotype Over Time
|
0 Participants
|
0 Participants
|
0 Participants
|
—
|
Adverse Events
Stratum A (6-13.99 Years)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Stratum B (14-20.99 Years)
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Stratum C (≥21 Years)
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Stratum D (Early Treatment)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Stratum A (6-13.99 Years)
n=5 participants at risk
* Age 6 to 13.99 years.
* Sickle cell anemia or sickle-β0-thalassemia.
|
Stratum B (14-20.99 Years)
n=10 participants at risk
* Age 14 to 20.99 years.
* Sickle cell anemia or sickle-β0-thalassemia.
* Detectible and quantifiable tricuspid regurgitant jet velocity (TRJV).
|
Stratum C (≥21 Years)
n=10 participants at risk
* Age ≥21 years.
* Sickle cell anemia or sickle-β0-thalassemia.
* Detectible and quantifiable tricuspid regurgitant jet velocity (TRJV).
|
Stratum D (Early Treatment)
n=7 participants at risk
* Age ≥6 years.
* Sickle cell anemia or sickle-β0-thalassemia.
* Current use of disease-modifying therapy \[hydroxyurea, chronic transfusions, or both (given concurrently, sequentially, or both)\] that was initiated at \<3 years of age, and for which there has been no interruption of therapy for \>6 consecutive months since the initiation of disease-modifying therapy.
|
|---|---|---|---|---|
|
Gastrointestinal disorders
Nausea and vomiting
|
0.00%
0/5 • AEs were collected for three, separate 30-day intervals over the entire study period of 2 years. Each 30-day interval was defined as starting with the study visit during which annual study activities were performed (e.g., cardiac magnetic resonance imaging [CMR]) and ending 30 days after that study visit. AEs were assessed as they occurred during each study visit, and AEs that occurred in the 30 days after each study visit were collected by scripted phone call from the study team.
|
0.00%
0/10 • AEs were collected for three, separate 30-day intervals over the entire study period of 2 years. Each 30-day interval was defined as starting with the study visit during which annual study activities were performed (e.g., cardiac magnetic resonance imaging [CMR]) and ending 30 days after that study visit. AEs were assessed as they occurred during each study visit, and AEs that occurred in the 30 days after each study visit were collected by scripted phone call from the study team.
|
10.0%
1/10 • Number of events 1 • AEs were collected for three, separate 30-day intervals over the entire study period of 2 years. Each 30-day interval was defined as starting with the study visit during which annual study activities were performed (e.g., cardiac magnetic resonance imaging [CMR]) and ending 30 days after that study visit. AEs were assessed as they occurred during each study visit, and AEs that occurred in the 30 days after each study visit were collected by scripted phone call from the study team.
|
0.00%
0/7 • AEs were collected for three, separate 30-day intervals over the entire study period of 2 years. Each 30-day interval was defined as starting with the study visit during which annual study activities were performed (e.g., cardiac magnetic resonance imaging [CMR]) and ending 30 days after that study visit. AEs were assessed as they occurred during each study visit, and AEs that occurred in the 30 days after each study visit were collected by scripted phone call from the study team.
|
|
Blood and lymphatic system disorders
Vaso-occlusive pain episode
|
0.00%
0/5 • AEs were collected for three, separate 30-day intervals over the entire study period of 2 years. Each 30-day interval was defined as starting with the study visit during which annual study activities were performed (e.g., cardiac magnetic resonance imaging [CMR]) and ending 30 days after that study visit. AEs were assessed as they occurred during each study visit, and AEs that occurred in the 30 days after each study visit were collected by scripted phone call from the study team.
|
30.0%
3/10 • Number of events 3 • AEs were collected for three, separate 30-day intervals over the entire study period of 2 years. Each 30-day interval was defined as starting with the study visit during which annual study activities were performed (e.g., cardiac magnetic resonance imaging [CMR]) and ending 30 days after that study visit. AEs were assessed as they occurred during each study visit, and AEs that occurred in the 30 days after each study visit were collected by scripted phone call from the study team.
|
0.00%
0/10 • AEs were collected for three, separate 30-day intervals over the entire study period of 2 years. Each 30-day interval was defined as starting with the study visit during which annual study activities were performed (e.g., cardiac magnetic resonance imaging [CMR]) and ending 30 days after that study visit. AEs were assessed as they occurred during each study visit, and AEs that occurred in the 30 days after each study visit were collected by scripted phone call from the study team.
|
0.00%
0/7 • AEs were collected for three, separate 30-day intervals over the entire study period of 2 years. Each 30-day interval was defined as starting with the study visit during which annual study activities were performed (e.g., cardiac magnetic resonance imaging [CMR]) and ending 30 days after that study visit. AEs were assessed as they occurred during each study visit, and AEs that occurred in the 30 days after each study visit were collected by scripted phone call from the study team.
|
Additional Information
Dr. Charles T. Quinn
Cincinnati Children's Hospital Medical Center
Phone: 5138033086
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place