Trial Outcomes & Findings for Safety Study of Multipotent Progenitor Cells for Immunomodulation Therapy After Liver Transplantation (NCT NCT01841632)

Last Updated: 2018-08-17

Results Overview

* For the description of intraportal toxicity a doppler ultrasound examination will be performed to assess various parameters that describe velocity of flow and flow pattern. * For pulmonary toxicity the assessment begins with an arterial blood gas. If this reveals pathological findings, a chest X-ray is required for clinical reasons independent of the study enrolment. In addition, clinical data describing the need for postoperative re-intubation will be recorded and the patient is assessed for the occurrence of a pulmonary embolism according to clinical guidelines. * For systemic toxicity, the occurrence of anaphylactic shock due to standard clinical guidelines is recorded.

Recruitment status

TERMINATED

Study phase

PHASE1

Target enrollment

3 participants

Primary outcome timeframe

up to day 30 (+10)

Results posted on

2018-08-17

Participant Flow

Participant milestones

Participant milestones
Measure	MultiStem - Cohort 1 Cohort 1 Drug: MultiStem, Dose 1 of MultiStem; Route and time: Two infusions; First: intra portal at liver transplantation (day 1), second: intra venous (day 3)
Overall Study STARTED	3
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Safety Study of Multipotent Progenitor Cells for Immunomodulation Therapy After Liver Transplantation

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	MultiStem - Cohort 1 n=3 Participants Cohort 1 Drug: MultiStem, Dose 1 of MultiStem; Route and time: Two infusions; First: intra portal at liver transplantation (day 1), second: intra venous (day 3)
Age, Categorical <=18 years	1 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=93 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants
Sex: Female, Male Female	1 Participants n=93 Participants
Sex: Female, Male Male	2 Participants n=93 Participants
Region of Enrollment Germany	3 participants n=93 Participants

PRIMARY outcome

Timeframe: up to day 30 (+10)

Population: A Per-protocol (PP) population consisting of all patients of the ITT population who showed no major protocol violations.

Outcome measures

Outcome measures
Measure	MultiStem - Cohort 1 n=3 Participants Cohort 1 Drug: MultiStem, Dose 1 of MultiStem; Route and time: Two infusions; First: intra portal at liver transplantation (day 1), second: intra venous (day 3)
Infusional and Acute Toxicity, Using Toxicity Scoring Mechanism	0 Participants

SECONDARY outcome

Timeframe: up to day 90 (+/-30)

Per protocol biopsies will be performed on days 1, 4, 10. Additional biopsies will be taken whenever clinically necessary.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to day 365 (+/-30)

Population: A Per-protocol (PP) population consisting of all patients of the ITT population who showed no major protocol violations. Protocol violations that may have an impact on the study outcome were considered as major protocol violations.

Four additional outpatient visits are planned to further evaluate the study patients (including screening for malignancies).

Outcome measures

Outcome measures
Measure	MultiStem - Cohort 1 n=3 Participants Cohort 1 Drug: MultiStem, Dose 1 of MultiStem; Route and time: Two infusions; First: intra portal at liver transplantation (day 1), second: intra venous (day 3)
Evidence Confirming That MultiStem Does Not Promote Malignant Transformation or Tumor Growth	0 Participants

SECONDARY outcome

Timeframe: up to six years

The results of routine examinations, which are necessary for all transplant patients, will be used once a year and analyzed retrospectively.

Outcome measures

Outcome data not reported

Adverse Events

MultiStem - Cohort 1

Serious events: 3 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	MultiStem - Cohort 1 n=3 participants at risk Cohort 1 Drug: MultiStem, Dose 1 of MultiStem; Route and time: Two infusions; First: intra portal at liver transplantation (day 1), second: intra venous (day 3)
Cardiac disorders Atrial fibrillation	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Injury, poisoning and procedural complications Biliary anastomosis complication	66.7% 2/3 • Number of events 3 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Investigations Liver function test abnormal	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Immune system disorders Liver transplant rejection	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Gastrointestinal disorders Papilla of Vater stenosis	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Injury, poisoning and procedural complications Wound dehiscence	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Injury, poisoning and procedural complications Post procedural bile leak	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Infections and infestations Subcutaneous abscess	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Infections and infestations Gastroenteritis	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Nervous system disorders Myoclonus	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Injury, poisoning and procedural complications Subcutaneous haematoma	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.

Other adverse events

Other adverse events
Measure	MultiStem - Cohort 1 n=3 participants at risk Cohort 1 Drug: MultiStem, Dose 1 of MultiStem; Route and time: Two infusions; First: intra portal at liver transplantation (day 1), second: intra venous (day 3)
Gastrointestinal disorders Abdominal distension	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Gastrointestinal disorders Abdominal pain upper	66.7% 2/3 • Number of events 2 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Gastrointestinal disorders Abnormal faeces	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Metabolism and nutrition disorders Acidosis	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Psychiatric disorders Agitation	66.7% 2/3 • Number of events 5 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Hepatobiliary disorders Bile duct stenosis	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Hepatobiliary disorders Cholangitis	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Hepatobiliary disorders Cholestasis	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Gastrointestinal disorders Constipation	100.0% 3/3 • Number of events 4 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Respiratory, thoracic and mediastinal disorders Cough	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Infections and infestations Cytomegalovirus infection	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Skin and subcutaneous tissue disorders Dermatitis contact	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Gastrointestinal disorders Diarrhoea	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Gastrointestinal disorders Dyspepsia	100.0% 3/3 • Number of events 3 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Psychiatric disorders Dysphemia	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
General disorders Fatigue	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Gastrointestinal disorders Flatulence	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Vascular disorders Haemorrhage	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Respiratory, thoracic and mediastinal disorders Hypercapnia	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Metabolism and nutrition disorders Hyperglycaemia	66.7% 2/3 • Number of events 3 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Metabolism and nutrition disorders Hyperkalaemia	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Metabolism and nutrition disorders Hyperlipidaemia	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Vascular disorders Hypertension	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Endocrine disorders Hyperthyroidism	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Skin and subcutaneous tissue disorders Hypertrichosis	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Metabolism and nutrition disorders Hypomagnesaemia	66.7% 2/3 • Number of events 2 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Gastrointestinal disorders Ileus	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
General disorders Impaired healing	66.7% 2/3 • Number of events 2 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Injury, poisoning and procedural complications Incisional hernia	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Respiratory, thoracic and mediastinal disorders Increased bronchial secretion	33.3% 1/3 • Number of events 2 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Infections and infestations Infection	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Blood and lymphatic system disorders Leukopenia	33.3% 1/3 • Number of events 2 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Investigations Liver function test abnormal	66.7% 2/3 • Number of events 2 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Eye disorders Myopia	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Infections and infestations Nasopharyngitis	66.7% 2/3 • Number of events 4 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Skin and subcutaneous tissue disorders Night sweats	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
General disorders Oedema	33.3% 1/3 • Number of events 2 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
General disorders Oedema peripheral	33.3% 1/3 • Number of events 2 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Gastrointestinal disorders Oesophagitis	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Renal and urinary disorders Oliguria	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Infections and infestations Oral candidiasis	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Infections and infestations Oral herpes	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Gastrointestinal disorders Oropharyngeal pain	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Musculoskeletal and connective tissue disorders Osteoporosis	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
General disorders Pain	100.0% 3/3 • Number of events 3 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Blood and lymphatic system disorders Pancytopenia	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Nervous system disorders Paraesthesia	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Infections and infestations Paronychia	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Respiratory, thoracic and mediastinal disorders Pleural effusion	100.0% 3/3 • Number of events 3 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Skin and subcutaneous tissue disorders Pruritus	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Renal and urinary disorders Renal failure	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Investigations Renal function test abnormal	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Infections and infestations Rhinitis	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Skin and subcutaneous tissue disorders Skin lesion	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Psychiatric disorders Sleep disorder	66.7% 2/3 • Number of events 2 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Nervous system disorders Somnolence	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Nervous system disorders Tremor	66.7% 2/3 • Number of events 2 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Infections and infestations Urinary tract infection	33.3% 1/3 • Number of events 2 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Gastrointestinal disorders Vomiting	66.7% 2/3 • Number of events 2 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.
Infections and infestations Wound infection	33.3% 1/3 • Number of events 1 • The period of (S)AE reported for each trial patient was from the time of first study-specific procedure until 30 days after the final trial visit (approximately 52 weeks) or until the date of patient withdrawal.

Additional Information

Prof. Dr. Marc-H. Dahlke, Ph. D.

University Hospital Regensburg

Phone: +49941944

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place