Trial Outcomes & Findings for Treatment With Acalabrutinib Post Blood or Marrow Transplantation in Subjects With Mantle Cell Lymphoma (NCT NCT04402138)

NCT ID: NCT04402138

Last Updated: 2025-05-13

Results Overview

Progression Free Survival Rate is defined as the Kaplan-Meier estimate of the percentage of participants who are alive and free of disease progression according to Response Evaluation Criteria in Lymphoma (RECIL 2017) for up to 2 years post-BMT. According to RECIL, disease progression is defined as following criteria: \>20% increase in sum of longest diameters of target lesions, for small lymph nodes measuring \<15 mm post therapy, a minimum absolute increase of 5 mm and the longest diameter should exceed 15mm, or appearance of new lesions.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 15 participants
• Primary outcome timeframe: 2 years after date of BMT procedure for each patient
• Results posted on: 2025-05-13

Participant Flow

Participant milestones

Measure	Acalabrutinib Acalabrutinib will be self-administered orally for up to approximately 2 years post-BMT. Acalabrutinib: Acalabrutinib 129 mg BID will be self-administered orally starting from 100 day (+/- 7 days) Post-BMT on a 28-day schedule, with or without food, until the patient has reached approximately 2 years post-BMT.
Overall Study STARTED	15
Overall Study COMPLETED	12
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Measure	Acalabrutinib Acalabrutinib will be self-administered orally for up to approximately 2 years post-BMT. Acalabrutinib: Acalabrutinib 129 mg BID will be self-administered orally starting from 100 day (+/- 7 days) Post-BMT on a 28-day schedule, with or without food, until the patient has reached approximately 2 years post-BMT.
Overall Study Progressive Disease	2
Overall Study Adverse Event	1

Baseline Characteristics

Treatment With Acalabrutinib Post Blood or Marrow Transplantation in Subjects With Mantle Cell Lymphoma

Baseline characteristics by cohort

Measure	Acalabrutinib n=15 Participants Acalabrutinib will be self-administered orally for up to approximately 2 years post-BMT. Acalabrutinib: Acalabrutinib 129 mg BID will be self-administered orally starting from 100 day (+/- 7 days) Post-BMT on a 28-day cycle schedule, with or without food, until the patient has reached 2 years post-BMT.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	6 Participants n=5 Participants
Age, Categorical >=65 years	9 Participants n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	11 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	12 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants
Region of Enrollment United States	15 participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years after date of BMT procedure for each patient

Population: Participants who receive at least one dose of acalabrutinib, who have an adequate baseline disease assessment and an adequate post-baseline assessment, or those who discontinue due to death or PD prior to their first assessment.

Progression Free Survival Rate is defined as the Kaplan-Meier estimate of the percentage of participants who are alive and free of disease progression according to Response Evaluation Criteria in Lymphoma (RECIL 2017) for up to 2 years post-BMT.

According to RECIL, disease progression is defined as following criteria: >20% increase in sum of longest diameters of target lesions, for small lymph nodes measuring <15 mm post therapy, a minimum absolute increase of 5 mm and the longest diameter should exceed 15mm, or appearance of new lesions.

Outcome measures

Measure	Acalabrutinib n=15 Participants Acalabrutinib will be self-administered orally for up to approximately 2 years post-BMT. Acalabrutinib: Acalabrutinib 129 mg BID will be self-administered orally starting from 100 day (+/- 7 days) Post-BMT on a 28-day schedule, with or without food, until the patient has reached approximately 2 years post-BMT.
Progression Free Survival Rate (PFS) at 2 Years Post-Blood or Marrow Transplant (BMT)	73.5 percentage of participants Interval 35.9 to 91.1

SECONDARY outcome

Timeframe: Assessed at day 100 post-BMT procedure (prior to beginning maintenance treatment with study drug) and 2 years post-BMT

Population: Participants who received one dose of study drug and had post-BMT MRD tested

Percentage of participants whose whole blood or bone marrow results transition from MRD positive before study treatment to MRD negative at 2 years post-BMT. An MRD negative status will be defined as all results from whole blood and bone marrow that are negative for the presence of residual clonal cells (with assay sensitivity of 10^-6) per Adaptive Biotechnologies' reporting methods.

Outcome measures

Measure	Acalabrutinib n=15 Participants Acalabrutinib will be self-administered orally for up to approximately 2 years post-BMT. Acalabrutinib: Acalabrutinib 129 mg BID will be self-administered orally starting from 100 day (+/- 7 days) Post-BMT on a 28-day schedule, with or without food, until the patient has reached approximately 2 years post-BMT.
Conversion Rate From Minimal Residual Disease Positive (MRD+) to Minimal Residual Disease Negative (MRD-)	13.3 percentage of participants

SECONDARY outcome

Timeframe: 2 years after date of BMT procedure for each patient

Population: Participants who receive at least one dose of acalabrutinib, who have an adequate baseline disease assessment and an adequate post-baseline assessment, or those who discontinue due to death or PD prior to their first assessment.

To compare the Kaplan-Meier estimated PFS rate for MRD negative population with MRD positive population. Progression-free survival is defined as the time from date of BMT procedure (day 0) until the date of objective radiological PD according to Response Evaluation Criteria in Lymphoma (RECIL 2017) or death. According to RECIL, disease progression is defined as following criteria: >20% increase in sum of longest diameters of target lesions, for small lymph nodes measuring <15 mm post therapy, a minimum absolute increase of 5 mm and the longest diameter should exceed 15mm, or appearance of new lesions. An MRD negative status will be defined as all results from whole blood and bone marrow that are negative for the presence of residual clonal cells (with assay sensitivity of 10^-6) per Adaptive Biotechnologies' reporting methods.

Outcome measures

Measure	Acalabrutinib n=15 Participants Acalabrutinib will be self-administered orally for up to approximately 2 years post-BMT. Acalabrutinib: Acalabrutinib 129 mg BID will be self-administered orally starting from 100 day (+/- 7 days) Post-BMT on a 28-day schedule, with or without food, until the patient has reached approximately 2 years post-BMT.
Minimal Residual Disease (MRD) Correlation With Progression Free Survival (PFS) PFS Rate Estimate of MRD-Negative Participants	88.9 percentage of participants Interval 43.3 to 98.4
Minimal Residual Disease (MRD) Correlation With Progression Free Survival (PFS) PFS Rate Estimate of MRD-Positive Participants	37.5 percentage of participants Interval 1.1 to 80.8

SECONDARY outcome

Timeframe: Safety assessment will be done every cycle up to 2 years post-BMT

Population: All participants who have received at least one dose of study drug.

Number of participants with grade 3 or higher CTCAE Version 5.0 Adverse Events that are considered related to study drug

Outcome measures

Measure	Acalabrutinib n=15 Participants Acalabrutinib will be self-administered orally for up to approximately 2 years post-BMT. Acalabrutinib: Acalabrutinib 129 mg BID will be self-administered orally starting from 100 day (+/- 7 days) Post-BMT on a 28-day schedule, with or without food, until the patient has reached approximately 2 years post-BMT.
Incidence of Grade 3 or Greater Treatment-Related Adverse Events	6 Participants

Adverse Events

Acalabrutinib

Serious events: 3 serious events

Other events: 15 other events

Deaths: 0 deaths

Serious adverse events

Measure	Acalabrutinib n=15 participants at risk Acalabrutinib will be self-administered orally for up to approximately 2 years post-BMT. Acalabrutinib: Acalabrutinib 129 mg BID will be self-administered orally starting from 100 day (+/- 7 days) Post-BMT on a 28-day schedule, until the patient has reached approximately 2 years post-BMT.
Infections and infestations Coronavirus infection	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Infections and infestations Clostridium difficile infection	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Infections and infestations Sepsis	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.

Other adverse events

Measure	Acalabrutinib n=15 participants at risk Acalabrutinib will be self-administered orally for up to approximately 2 years post-BMT. Acalabrutinib: Acalabrutinib 129 mg BID will be self-administered orally starting from 100 day (+/- 7 days) Post-BMT on a 28-day schedule, until the patient has reached approximately 2 years post-BMT.
Metabolism and nutrition disorders Decreased Appetite	13.3% 2/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Metabolism and nutrition disorders Dehydration	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Metabolism and nutrition disorders Hyperuricaemia	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Metabolism and nutrition disorders Hypokalaemia	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Blood and lymphatic system disorders Anaemia	13.3% 2/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Blood and lymphatic system disorders Neutropenia	13.3% 2/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Blood and lymphatic system disorders Thrombocytopenia	13.3% 2/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Blood and lymphatic system disorders Lymphadenopathy	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Cardiac disorders Palpitations	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Cardiac disorders Heart Rate Increased	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Ear and labyrinth disorders Ear Discomfort	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Ear and labyrinth disorders Ear Pruritus	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Ear and labyrinth disorders Hypoacusis	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Ear and labyrinth disorders Vertigo	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Endocrine disorders Hypothyroidism	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Endocrine disorders Androgen Deficiency	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Eye disorders Photophobia	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Gastrointestinal disorders Diarrhoea	53.3% 8/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Gastrointestinal disorders Nausea	26.7% 4/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Gastrointestinal disorders Abdominal Distension	13.3% 2/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Gastrointestinal disorders Abdominal Pain	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Gastrointestinal disorders Abdominal Pain Upper	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Gastrointestinal disorders Anal Incontinence	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Gastrointestinal disorders Colitis	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Gastrointestinal disorders Constipation	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Gastrointestinal disorders Gastrooesophageal Reflux Disease	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Gastrointestinal disorders Oesophagitis	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Gastrointestinal disorders Vomiting	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
General disorders Fatigue	60.0% 9/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
General disorders Pyrexia	13.3% 2/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
General disorders Chills	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
General disorders Malaise	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
General disorders Pain	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
General disorders Oedema	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Infections and infestations Covid-19	26.7% 4/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Infections and infestations Upper Respiratory Tract Infection	13.3% 2/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Infections and infestations Eye Infection	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Infections and infestations Pneumonia	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Infections and infestations Sars-Cov-2 Test Positive	13.3% 2/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Infections and infestations Bronchitis	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Infections and infestations Clostridium Difficile Infection	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Infections and infestations Coronavirus Infection	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Infections and infestations Exposure To Sars-Cov-2	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Infections and infestations Gastroenteritis Viral	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Infections and infestations Influenza	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Infections and infestations Pharyngitis	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Infections and infestations Sepsis	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Injury, poisoning and procedural complications Contusion	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Injury, poisoning and procedural complications Fall	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Injury, poisoning and procedural complications Spinal Compression Fracture	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Investigations Neutrophil Count Decreased	20.0% 3/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Investigations Blood Creatinine Increased	13.3% 2/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Investigations Weight Decreased	13.3% 2/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Investigations Aspartate Aminotransferase Increased	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Investigations Blood Alkaline Phosphatase Increased	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Investigations Lymphocyte Count Decreased	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Investigations White Blood Cell Count Decreased	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Investigations Blood Uric Acid Increased	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Investigations Weight Increased	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Metabolism and nutrition disorders Vitamin D Deficiency	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Metabolism and nutrition disorders Vitamin B12 Deficiency	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Musculoskeletal and connective tissue disorders Arthralgia	26.7% 4/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Musculoskeletal and connective tissue disorders Myalgia	26.7% 4/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Musculoskeletal and connective tissue disorders Bone Pain	13.3% 2/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Musculoskeletal and connective tissue disorders Muscle Spasms	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Musculoskeletal and connective tissue disorders Musculoskeletal Pain	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Musculoskeletal and connective tissue disorders Neck Pain	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Musculoskeletal and connective tissue disorders Pain In Extremity	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Musculoskeletal and connective tissue disorders Limb Injury	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Nervous system disorders Headache	40.0% 6/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Nervous system disorders Dizziness	13.3% 2/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Nervous system disorders Peripheral Sensory Neuropathy	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Nervous system disorders Syncope	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Nervous system disorders Aphasia	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Psychiatric disorders Insomnia	13.3% 2/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Renal and urinary disorders Dysuria	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Renal and urinary disorders Acute Kidney Injury	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Respiratory, thoracic and mediastinal disorders Dyspnoea	20.0% 3/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Respiratory, thoracic and mediastinal disorders Cough	13.3% 2/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Respiratory, thoracic and mediastinal disorders Sinus Congestion	13.3% 2/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Respiratory, thoracic and mediastinal disorders Nasal Congestion	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Respiratory, thoracic and mediastinal disorders Oropharyngeal Pain	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Respiratory, thoracic and mediastinal disorders Dyspnoea Exertional	13.3% 2/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Respiratory, thoracic and mediastinal disorders Hypoxia	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Respiratory, thoracic and mediastinal disorders Lower Respiratory Tract Congestion	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Respiratory, thoracic and mediastinal disorders Respiratory Tract Congestion	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Respiratory, thoracic and mediastinal disorders Upper-Airway Cough Syndrome	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Skin and subcutaneous tissue disorders Petechiae	13.3% 2/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Skin and subcutaneous tissue disorders Rash	13.3% 2/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Skin and subcutaneous tissue disorders Rash Maculo-Papular	13.3% 2/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Skin and subcutaneous tissue disorders Dry Skin	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Skin and subcutaneous tissue disorders Nail Disorder	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Skin and subcutaneous tissue disorders Pruritus	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Skin and subcutaneous tissue disorders Basal Cell Carcinoma	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Skin and subcutaneous tissue disorders Dermatitis Acneiform	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Skin and subcutaneous tissue disorders Increased Tendency To Bruise	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Skin and subcutaneous tissue disorders Rash Pruritic	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Skin and subcutaneous tissue disorders Skin Lesion	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.
Skin and subcutaneous tissue disorders Squamous Cell Carcinoma	6.7% 1/15 • Serious and/or other adverse events were assessed from the date of first dose to 30 days after last dose of study treatment, up to 2 years post-BMT procedure. All-Cause Mortality was monitored from date of consent up to 5 years post-BMT procedure. This study closed early so full follow-up not collected for all patients so All-Cause Mortality was collected for a maximum of 3.7 years. All patients who received at least one dose of protocol treatment were followed for safety. Adverse events and serious adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.

Additional Information

Sarah Cannon Development Innovations, LLC

Sarah Cannon Development Innovations, LLC

Phone: 844-710-6157

Email: SCRI.InnovationsMedical@scri.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place