Trial Outcomes & Findings for MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID (NCT NCT00794508)

Last Updated: 2021-04-23

Results Overview

Examine the safety of the procedure: harvesting bone marrow, isolating CD34+ hematopoietic stem/progenitor cells, performing ex vivo gene transduction with the MND-ADA gamma-retroviral vector, giving 90 mg/m2 busulfan to "make space" in the bone marrow to aid engraftment, and re-infusing the autologous gene-modified cells.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

10 participants

Primary outcome timeframe

2 years

Results posted on

2021-04-23

Participant Flow

Participant milestones

Participant milestones
Measure	Gamma-retroviral-mediated ADA Gene Transfer Transfer of the human ADA gene to isolated CD34+ cells from the bone marrow. ADA gene transfer: Autologous CD34+ cells transduced with the gamma-retroviral vector, MND-ADA, carrying the human ADA gene.
Overall Study STARTED	10
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Experimental Retroviral-mediated ADA Gene Transfer n=10 Participants Transfer of the human ADA gene to isolated CD34+ cells from the bone marrow
Age, Categorical <=18 years	10 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
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	4 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	1 Participants n=5 Participants
Race (NIH/OMB) White	7 Participants n=5 Participants
Race (NIH/OMB) More than one race	2 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	10 participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years

Outcome measures

Outcome measures
Measure	Gamma-retroviral Mediated ADA Gene Transfer n=10 Participants Transfer of the human ADA gene to isolated CD34+ cells from the bone marrow
Number of Participants With Adverse Events Expected Grade 3 or 4 AE related to procedure	10 participants
Number of Participants With Adverse Events Unexpected Grade 3 or 4 AE related to procedure	9 participants
Number of Participants With Adverse Events Grade 3 or 4 AE related to study product	0 participants
Number of Participants With Adverse Events Alive	10 participants
Number of Participants With Adverse Events Deaths	0 participants

SECONDARY outcome

Timeframe: 2 years

As measured by quantitative polymerase chain reaction in peripheral blood cells separated into mononuclear and granulocyte fractions.

Outcome measures

Outcome measures
Measure	Gamma-retroviral Mediated ADA Gene Transfer n=10 Participants Transfer of the human ADA gene to isolated CD34+ cells from the bone marrow
Number of Participants With Greater Than 1% of Gene-Modified Cells in the Peripheral Blood For peripheral blood mononuclear cells	10 participants
Number of Participants With Greater Than 1% of Gene-Modified Cells in the Peripheral Blood for peripheral blood granulocytes	4 participants

SECONDARY outcome

Timeframe: 2 years

As measured by ADA enzyme activity in peripheral blood mononuclear cells

Outcome measures

Outcome measures
Measure	Gamma-retroviral Mediated ADA Gene Transfer n=10 Participants Transfer of the human ADA gene to isolated CD34+ cells from the bone marrow
Number of Participants Reaching the Normal Range of ADA Enzyme Activity	9 participants

Adverse Events

Retroviral-mediated ADA Gene Transfer

Serious events: 5 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Retroviral-mediated ADA Gene Transfer n=10 participants at risk Transfer of the human ADA gene to isolated CD34+ cells from the bone marrow. ADA gene transfer: Autologous CD34+ cells transduced with the retroviral vector MND-ADA, carrying the human ADA gene.
Immune system disorders Infection requiring hospitalization	50.0% 5/10 • 1, 2, 3, 4, 5, 6, 8, 10, 12, 15, 18, 21, 24 months

Other adverse events

Other adverse events
Measure	Retroviral-mediated ADA Gene Transfer n=10 participants at risk Transfer of the human ADA gene to isolated CD34+ cells from the bone marrow. ADA gene transfer: Autologous CD34+ cells transduced with the retroviral vector MND-ADA, carrying the human ADA gene.
Blood and lymphatic system disorders neutropenia	90.0% 9/10 • 1, 2, 3, 4, 5, 6, 8, 10, 12, 15, 18, 21, 24 months

Additional Information

Dr. Donald B. Kohn

University of California Los Angeles

Phone: 310-794-1964

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place