Trial Outcomes & Findings for Study of Efficacy and Safety of Reinfusion of Tisagenlecleucel in Pediatric and Young Adult Patients With Acute Lymphoblastic Leukemia (ALL) (NCT NCT04225676)
NCT ID: NCT04225676
Last Updated: 2023-01-30
Results Overview
Percentage of patients who establish B-cell aplasia at any visit following re-infusion with tisagenlecleucel. B-cell aplasia is defined as peripheral blood (PB) absolute B lymphocyte count \<50/μL. Planned timeframe was 12 months but actual timeframe was approximately 9 months due to early termination of the trial. Day 1 is post lymphodepleting chemotherapy and pre-reinfusion of tisagenlecleucel.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
5 participants
Primary outcome timeframe
Post-reinfusion up to 9 months (Day 1 is excluded)
Results posted on
2023-01-30
Participant Flow
There were 7 participants screened.
Participant milestones
| Measure |
Tisagenlecleucel
Tisagenlecleucel Cell Dispersion for Infusion given once during the study.
The approved dose range for tisagenlecleucel is: 0.2 to 5.0×10\^6 CAR positive viable T cells / kg for patients' ≤ 50 kg body weight or 0.1 to 2.5×10\^8 CAR-positive viable T cells for patients \> 50 kg body weight.
|
|---|---|
|
Overall Study
STARTED
|
5
|
|
Overall Study
Full Analysis, Safety and Enrolled Sets
|
5
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
5
|
Reasons for withdrawal
| Measure |
Tisagenlecleucel
Tisagenlecleucel Cell Dispersion for Infusion given once during the study.
The approved dose range for tisagenlecleucel is: 0.2 to 5.0×10\^6 CAR positive viable T cells / kg for patients' ≤ 50 kg body weight or 0.1 to 2.5×10\^8 CAR-positive viable T cells for patients \> 50 kg body weight.
|
|---|---|
|
Overall Study
Physician Decision
|
1
|
|
Overall Study
Lack of Efficacy
|
1
|
|
Overall Study
Study terminated by sponsor
|
3
|
Baseline Characteristics
Study of Efficacy and Safety of Reinfusion of Tisagenlecleucel in Pediatric and Young Adult Patients With Acute Lymphoblastic Leukemia (ALL)
Baseline characteristics by cohort
| Measure |
Tisagenlecleucel
n=5 Participants
Tisagenlecleucel Cell Dispersion for Infusion given once during the study.
The approved dose range for tisagenlecleucel is: 0.2 to 5.0×10\^6 CAR positive viable T cells / kg for patients' ≤ 50 kg body weight or 0.1 to 2.5×10\^8 CAR-positive viable T cells for patients \> 50 kg body weight.
|
|---|---|
|
Age, Customized
< 10 years
|
2 Participants
n=99 Participants
|
|
Age, Customized
>=10 to <18 years
|
2 Participants
n=99 Participants
|
|
Age, Customized
>=18 years
|
1 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=99 Participants
|
|
Race/Ethnicity, Customized
White
|
5 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: Post-reinfusion up to 9 months (Day 1 is excluded)Population: Full analysis set
Percentage of patients who establish B-cell aplasia at any visit following re-infusion with tisagenlecleucel. B-cell aplasia is defined as peripheral blood (PB) absolute B lymphocyte count \<50/μL. Planned timeframe was 12 months but actual timeframe was approximately 9 months due to early termination of the trial. Day 1 is post lymphodepleting chemotherapy and pre-reinfusion of tisagenlecleucel.
Outcome measures
| Measure |
Tisagenlecleucel
n=5 Participants
Tisagenlecleucel Cell Dispersion for Infusion given once during the study.
The approved dose range for tisagenlecleucel is: 0.2 to 5.0×10\^6 CAR positive viable T cells / kg for patients' ≤ 50 kg body weight or 0.1 to 2.5×10\^8 CAR-positive viable T cells for patients \> 50 kg body weight.
|
Complete Remission Without Complete Blood Count Recovery
Complete remission as determined by the investigator without complete blood count recovery
|
|---|---|---|
|
Percentage of Patients Who Establish B Cell Aplasia Within 9 Months of Reinfusion
|
2 Participants
|
—
|
SECONDARY outcome
Timeframe: Post-reinfusion up to 9 monthsPopulation: Full analysis set - Data was not adequate to perform overall remission rate analysis considering evaluable population size
Overall remission rate (ORR) was defined as the percentage of participants with a best overall disease response of complete remission (CR) or CR with incomplete blood count recovery (CRi). However, the rate was not calculated due to low enrollment. Participants' best responses have been listed by day and participants may be counted more than once.
Outcome measures
| Measure |
Tisagenlecleucel
n=5 Participants
Tisagenlecleucel Cell Dispersion for Infusion given once during the study.
The approved dose range for tisagenlecleucel is: 0.2 to 5.0×10\^6 CAR positive viable T cells / kg for patients' ≤ 50 kg body weight or 0.1 to 2.5×10\^8 CAR-positive viable T cells for patients \> 50 kg body weight.
|
Complete Remission Without Complete Blood Count Recovery
n=5 Participants
Complete remission as determined by the investigator without complete blood count recovery
|
|---|---|---|
|
Complete Response (CR) or Complete Response With Incomplete Blood Count Recovery (CRi) by Day
Day 24
|
0 responses
|
1 responses
|
|
Complete Response (CR) or Complete Response With Incomplete Blood Count Recovery (CRi) by Day
Day 28
|
1 responses
|
0 responses
|
|
Complete Response (CR) or Complete Response With Incomplete Blood Count Recovery (CRi) by Day
Day 29
|
1 responses
|
0 responses
|
|
Complete Response (CR) or Complete Response With Incomplete Blood Count Recovery (CRi) by Day
Day 32
|
1 responses
|
0 responses
|
|
Complete Response (CR) or Complete Response With Incomplete Blood Count Recovery (CRi) by Day
Day 34
|
1 responses
|
0 responses
|
|
Complete Response (CR) or Complete Response With Incomplete Blood Count Recovery (CRi) by Day
Day 67
|
1 responses
|
0 responses
|
|
Complete Response (CR) or Complete Response With Incomplete Blood Count Recovery (CRi) by Day
Day 84
|
0 responses
|
1 responses
|
|
Complete Response (CR) or Complete Response With Incomplete Blood Count Recovery (CRi) by Day
Day 91
|
0 responses
|
1 responses
|
|
Complete Response (CR) or Complete Response With Incomplete Blood Count Recovery (CRi) by Day
Day 177
|
0 responses
|
1 responses
|
|
Complete Response (CR) or Complete Response With Incomplete Blood Count Recovery (CRi) by Day
Day 182
|
1 responses
|
0 responses
|
|
Complete Response (CR) or Complete Response With Incomplete Blood Count Recovery (CRi) by Day
Day 211
|
0 responses
|
1 responses
|
|
Complete Response (CR) or Complete Response With Incomplete Blood Count Recovery (CRi) by Day
Day 274
|
0 responses
|
1 responses
|
SECONDARY outcome
Timeframe: Reinfusion up to 9 monthsPopulation: Full analysis set - Data was not adequate to perform time to event analysis considering evaluable population size.
An event was defined as one of the following: death from any cause after remission, relapse, treatment failure (defined as no response in the study or discontinuation from the study for death, adverse event, lack of efficacy or progressive disease or new cancer therapy).
Outcome measures
| Measure |
Tisagenlecleucel
n=5 Participants
Tisagenlecleucel Cell Dispersion for Infusion given once during the study.
The approved dose range for tisagenlecleucel is: 0.2 to 5.0×10\^6 CAR positive viable T cells / kg for patients' ≤ 50 kg body weight or 0.1 to 2.5×10\^8 CAR-positive viable T cells for patients \> 50 kg body weight.
|
Complete Remission Without Complete Blood Count Recovery
Complete remission as determined by the investigator without complete blood count recovery
|
|---|---|---|
|
Participants With an Event
|
1 participant
|
—
|
SECONDARY outcome
Timeframe: Reinfusion up to 9 monthsPopulation: Full analysis set - Data was not adequate to perform overall survival analysis considering evaluable population size
Deaths due to any reason.
Outcome measures
| Measure |
Tisagenlecleucel
n=5 Participants
Tisagenlecleucel Cell Dispersion for Infusion given once during the study.
The approved dose range for tisagenlecleucel is: 0.2 to 5.0×10\^6 CAR positive viable T cells / kg for patients' ≤ 50 kg body weight or 0.1 to 2.5×10\^8 CAR-positive viable T cells for patients \> 50 kg body weight.
|
Complete Remission Without Complete Blood Count Recovery
Complete remission as determined by the investigator without complete blood count recovery
|
|---|---|---|
|
Overall Survival (OS)
|
0 deaths
|
—
|
Adverse Events
Tisagenlecleucel
Serious events: 3 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Tisagenlecleucel
n=5 participants at risk
Tisagenlecleucel Cell Dispersion for Infusion given once during the study.
The approved dose range for tisagenlecleucel is: 0.2 to 5.0×10\^6 CAR positive viable T cells / kg for patients' ≤ 50 kg body weight or 0.1 to 2.5×10\^8 CAR-positive viable T cells for patients \> 50 kg body weight.
|
|---|---|
|
General disorders
Pyrexia
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Immune system disorders
Cytokine release syndrome
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Nervous system disorders
Headache
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Nervous system disorders
Paraesthesia
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
Other adverse events
| Measure |
Tisagenlecleucel
n=5 participants at risk
Tisagenlecleucel Cell Dispersion for Infusion given once during the study.
The approved dose range for tisagenlecleucel is: 0.2 to 5.0×10\^6 CAR positive viable T cells / kg for patients' ≤ 50 kg body weight or 0.1 to 2.5×10\^8 CAR-positive viable T cells for patients \> 50 kg body weight.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
40.0%
2/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Eye disorders
Vision blurred
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Gastrointestinal disorders
Abdominal pain
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Gastrointestinal disorders
Constipation
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Gastrointestinal disorders
Dental caries
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Gastrointestinal disorders
Nausea
|
40.0%
2/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Gastrointestinal disorders
Stomatitis
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Gastrointestinal disorders
Tooth development disorder
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Gastrointestinal disorders
Vomiting
|
40.0%
2/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
General disorders
Catheter site erythema
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
General disorders
Chills
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
General disorders
Fatigue
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
General disorders
Pain
|
40.0%
2/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
General disorders
Pyrexia
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Infections and infestations
Conjunctivitis
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Infections and infestations
HCoV-OC43 infection
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Infections and infestations
Paronychia
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Infections and infestations
Polyomavirus viraemia
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Investigations
Enterovirus test positive
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Investigations
Human rhinovirus test positive
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Investigations
Neutrophil count decreased
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Investigations
Platelet count decreased
|
40.0%
2/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Investigations
Respirovirus test positive
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Metabolism and nutrition disorders
Decreased appetite
|
60.0%
3/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Metabolism and nutrition disorders
Hypoalbuminaemia
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
60.0%
3/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Cancer fatigue
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Nervous system disorders
Headache
|
40.0%
2/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Skin and subcutaneous tissue disorders
Dermatitis acneiform
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Skin and subcutaneous tissue disorders
Rash
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
|
Vascular disorders
Hypertension
|
20.0%
1/5 • Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
- Publication restrictions are in place
Restriction type: OTHER