Trial Outcomes & Findings for Stem Cell Transplantation for Hurler (NCT NCT00176917)

Results Overview

Donor-derived engraftment determined by restriction fragment length polymorphism (RFLP).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

41 participants

Primary outcome timeframe

at 21 days, 42 days, 60 days, 100 days, 6 months, and 1 year

Results posted on

2017-12-28

Participant Flow

Participant milestones

Participant milestones
Measure	Transplant Patients Patients that received hematopoietic stem cell transplant.
Overall Study STARTED	41
Overall Study COMPLETED	41
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Stem Cell Transplantation for Hurler

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Transplant Patients n=41 Participants Patients that received hematopoietic stem cell transplant.
Age, Categorical <=18 years	41 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	1.8 years STANDARD_DEVIATION 1.4 • n=5 Participants
Sex: Female, Male Female	23 Participants n=5 Participants
Sex: Female, Male Male	18 Participants n=5 Participants
Region of Enrollment United States	41 participants n=5 Participants

PRIMARY outcome

Timeframe: at 21 days, 42 days, 60 days, 100 days, 6 months, and 1 year

Population: Day 21 (24 patients included), Day 42 (15 pts), Day 60 (29 pts), Day 100 (25 pts), 6 Months (18 pts), 1 Year (16 pts).

Donor-derived engraftment determined by restriction fragment length polymorphism (RFLP).

Outcome measures

Outcome measures
Measure	Transplant Patients n=41 Participants Patients that received hematopoietic stem cell transplant.
Mean Percentage of Donor Cells in Study Population (Chimerism). 21 Days Post Transplant	85.8 Percentage Standard Deviation 28.4
Mean Percentage of Donor Cells in Study Population (Chimerism). 42 Days Post Transplant	73.2 Percentage Standard Deviation 31.6
Mean Percentage of Donor Cells in Study Population (Chimerism). 60 Days Post Transplant	84.6 Percentage Standard Deviation 27.9
Mean Percentage of Donor Cells in Study Population (Chimerism). 100 Days Post Transplant	81.1 Percentage Standard Deviation 26.8
Mean Percentage of Donor Cells in Study Population (Chimerism). 6 Months Post Transplant	81.6 Percentage Standard Deviation 29.6
Mean Percentage of Donor Cells in Study Population (Chimerism). 1 Year Post Transplant	91.5 Percentage Standard Deviation 18.8

SECONDARY outcome

Timeframe: at 100 days, 1 year, and 3 years post transplant

Population: Day 100 and 1 Year timepoints include all 41 patients. Year 3 includes 36 patients (5 pts not yet at followup timepoint.)

Number of patients surviving (alive) at specified timepoints.

Outcome measures

Outcome measures
Measure	Transplant Patients n=41 Participants Patients that received hematopoietic stem cell transplant.
Number of Patients Surviving on Study Day 100 Post Transplant	37 Participants
Number of Patients Surviving on Study 1 Year Post Transplant	28 Participants
Number of Patients Surviving on Study 3 Years Post Transplant	27 Participants

SECONDARY outcome

Timeframe: Day 42 Post Transplant

Population: 1 patient of 41 failed engraftment - per protocol.

Toxicity (undesireable effect) of hematologic donor cell engraftment is determined by failure to engraft at Day 42.

Outcome measures

Outcome measures
Measure	Transplant Patients n=41 Participants Patients that received hematopoietic stem cell transplant.
Number of Patients Who Failed Engraftment.	1 Participants

SECONDARY outcome

Timeframe: Day 100 Post Transplant

Toxicity (undesireable effect) of this stem cell transplant preparative regimen due to acute graft-versus-host disease.

Outcome measures

Outcome measures
Measure	Transplant Patients n=41 Participants Patients that received hematopoietic stem cell transplant.
Number of Patients With Grade III-IV Acute Graft-versus-host Disease (aGVHD).	2 Participants

Adverse Events

Transplant Patients

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Paul J. Orchard, M.D.

Masonic Cancer Center, University of Minnesota

Phone: 612-626-2313

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place