Trial Outcomes & Findings for Reduced Intensity AlloTransplant For Osteopetrosis (NCT NCT00638820)

NCT ID: NCT00638820

Last Updated: 2017-12-28

Results Overview

Number of patients with persistent presence of donor-derived cells at Day 100

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 3 participants
• Primary outcome timeframe: Day 100
• Results posted on: 2017-12-28

Participant Flow

This study was terminated early due to stopping rules. The first 3 patients enrolled died by Day 100 of study.

Participant milestones

Measure	Patients With Osteopetrosis Who Received Transplant All patients enrolled with osteopetrosis and received transplant.
Overall Study STARTED	3
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Reduced Intensity AlloTransplant For Osteopetrosis

Baseline characteristics by cohort

Measure	Patients With Osteopetrosis Who Received Transplant n=3 Participants All patients enrolled with osteopetrosis and received transplant.
Age, Categorical <=18 years	2 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Region of Enrollment United States	3 participants n=5 Participants

PRIMARY outcome

Timeframe: Day 100

Number of patients with persistent presence of donor-derived cells at Day 100

Outcome measures

Measure	Patients With Osteopetrosis Who Received Transplant n=3 Participants All patients enrolled with osteopetrosis and received transplant.
Number of Patients Achieving Donor Cell Engraftment	2 Participants

SECONDARY outcome

Timeframe: Day 100

Number of participants died during study by Day 100 and reason for death was related to transplant.

Outcome measures

Measure	Patients With Osteopetrosis Who Received Transplant n=3 Participants All patients enrolled with osteopetrosis and received transplant.
Number of Patients With Transplant Related Death	3 Participants

SECONDARY outcome

Timeframe: Day 100

Number of patients experiencing adverse effects due to transplant categorized by body system using Common Terminology Criteria for Adverse Events coding from the National Cancer Institute, Version 3.0.

Outcome measures

Measure	Patients With Osteopetrosis Who Received Transplant n=3 Participants All patients enrolled with osteopetrosis and received transplant.
Number of Patients With Transplant Related Toxicity Neurologic	1 Participants
Number of Patients With Transplant Related Toxicity Renal	1 Participants
Number of Patients With Transplant Related Toxicity Pulmonary	3 Participants
Number of Patients With Transplant Related Toxicity Peri-Transplant Mortality (Death by Day 100)	3 Participants
Number of Patients With Transplant Related Toxicity Vascular	1 Participants

SECONDARY outcome

Timeframe: Day 100, 6 months, 1, 2 and 5 years

These outcome measures were not assessed due to early study termination.

Outcome measures

Outcome data not reported

Adverse Events

Patients With Osteopetrosis Who Received Transplant

Serious events: 3 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Measure	Patients With Osteopetrosis Who Received Transplant n=3 participants at risk All patients enrolled with osteopetrosis and received transplant.
General disorders Death, peri-transplant	100.0% 3/3 • Number of events 3 • Adverse events were collected from Day 1 through Day 100 of the study.

Other adverse events

Measure	Patients With Osteopetrosis Who Received Transplant n=3 participants at risk All patients enrolled with osteopetrosis and received transplant.
Nervous system disorders Neurologic	33.3% 1/3 • Number of events 1 • Adverse events were collected from Day 1 through Day 100 of the study.
Renal and urinary disorders Renal	33.3% 1/3 • Number of events 1 • Adverse events were collected from Day 1 through Day 100 of the study.
Respiratory, thoracic and mediastinal disorders Pulmonary	100.0% 3/3 • Number of events 3 • Adverse events were collected from Day 1 through Day 100 of the study.
Vascular disorders Vascular	33.3% 1/3 • Number of events 1 • Adverse events were collected from Day 1 through Day 100 of the study.

Additional Information

Paul Orchard, M.D.

Masonic Cancer Center, University of Minnesota

Phone: 612-626-2313

Email: orcha001@umn.edu

Results disclosure agreements

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