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Trial record 1 of 1 for:    NCT01734850
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Safety Study of a Dual Anti-HIV Gene Transfer Construct to Treat HIV-1 Infection

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ClinicalTrials.gov Identifier: NCT01734850
Recruitment Status : Completed
First Posted : November 28, 2012
Results First Posted : August 6, 2020
Last Update Posted : August 6, 2020
Sponsor:
Information provided by (Responsible Party):
Calimmune, Inc.

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Human Immunodeficiency Virus
Interventions Drug: Busulfan
Biological: Cal-1 modified HSPC
Biological: Cal-1 modified CD4+ T lymphocytes
Enrollment 13
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Hide Arm/Group Description Cal-1 modified Hematopoietic stem/progenitor cells (HSPC) and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC) administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Period Title: Overall Study
Started 4 4 5
Completed 1 2 1
Not Completed 3 2 4
Reason Not Completed
Continued to Week 48 but recommenced ART             3             2             3
Not infused due to manufacturing failure             0             0             1
Arm/Group Title Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses) Total
Hide Arm/Group Description Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Total of all reporting groups
Overall Number of Baseline Participants 4 4 5 13
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 4 participants 5 participants 13 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
4
 100.0%
4
 100.0%
5
 100.0%
13
 100.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 4 participants 4 participants 5 participants 13 participants
43.0  (11.7) 48.5  (12.8) 46.8  (3.1) 46.2  (9.2)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 4 participants 5 participants 13 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Male
4
 100.0%
4
 100.0%
5
 100.0%
13
 100.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 4 participants 5 participants 13 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
1
  25.0%
0
   0.0%
1
   7.7%
White
4
 100.0%
3
  75.0%
5
 100.0%
12
  92.3%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 4 participants 4 participants 5 participants 13 participants
4 4 5 13
1.Primary Outcome
Title Number of Participants With Severe and Life-threatening Adverse Events (AEs)
Hide Description [Not Specified]
Time Frame Up to 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population (SP): The safety population includes all enrolled subjects signing informed consent who started the interventional phase of the trial (i.e. CD4+ Apheresis and discontinued subjects who re-commenced ART)
Arm/Group Title Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Hide Arm/Group Description:
Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC) administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Overall Number of Participants Analyzed 4 4 5
Measure Type: Count of Participants
Unit of Measure: Participants
Severe
0
   0.0%
4
 100.0%
4
  80.0%
Life-threatening
0
   0.0%
3
  75.0%
4
  80.0%
2.Primary Outcome
Title Number of Participants With Severe or Life-threatening AEs Related to CSL202
Hide Description [Not Specified]
Time Frame Up to 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
SP
Arm/Group Title Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Hide Arm/Group Description:
Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Overall Number of Participants Analyzed 4 4 5
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
1
  20.0%
3.Primary Outcome
Title Number of Participants With the Presence of Replication-competent Retrovirus
Hide Description [Not Specified]
Time Frame Up to 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
SP
Arm/Group Title Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Hide Arm/Group Description:
Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Overall Number of Participants Analyzed 4 4 5
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
4.Primary Outcome
Title Number of Participants With Predominant Integration Site Analysis
Hide Description Vector Integration Site Analysis performed only when Cal-1 Marking is >= 1%.
Time Frame Up to 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)
Arm/Group Title Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Hide Arm/Group Description:
Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Overall Number of Participants Analyzed 4 4 4
Measure Type: Count of Participants
Unit of Measure: Participants
NA [1]  NA [1]  NA [1] 
[1]
Not done because Cal-1 Marking was <1% at the corresponding time point required
5.Primary Outcome
Title Mean Cell Dose for CD4+ Cells (Ttn)
Hide Description [Not Specified]
Time Frame Up to 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)
Arm/Group Title Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Hide Arm/Group Description:
Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Overall Number of Participants Analyzed 4 4 4
Mean (Full Range)
Unit of Measure: Number (10^8 cells)
16.940
(14.61 to 20.62)
81.855
(38.94 to 138.68)
49.553
(20.70 to 79.29)
6.Primary Outcome
Title Mean Cell Dose for CD34+ Cells (HSPCtn)
Hide Description [Not Specified]
Time Frame Up to 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)
Arm/Group Title Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Hide Arm/Group Description:
Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Overall Number of Participants Analyzed 4 4 4
Mean (Full Range)
Unit of Measure: Number (10^6 cells/kg body weight)
2.1
(0.35 to 5.0)
2.4
(1.0 to 4.0)
10.6
(7.4 to 12.5)
7.Primary Outcome
Title Percent Transduction Efficiency of CD4+ Cells (Ttn) and CD34+ Cells (HSPCtn) of Final Cell Product
Hide Description [Not Specified]
Time Frame Up to 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)
Arm/Group Title Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Hide Arm/Group Description:
Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Overall Number of Participants Analyzed 4 4 4
Mean (Full Range)
Unit of Measure: Percent transduction efficiency
CD4+ Cells (Ttn)
41.0
(21.0 to 60.0)
29.2
(13.0 to 39.0)
66.8
(39.0 to 84.0)
CD34+ Cells (HSPCtn)
58.6
(51.0 to 77.0)
26.3
(12.0 to 53.0)
31.3
(21.0 to 49.0)
8.Primary Outcome
Title Total Area Under the Curve (AUC) for Busulfan
Hide Description Cohort 3: Total AUC = first dose AUC value + second dose AUC value
Time Frame Up to 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)
Arm/Group Title Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Hide Arm/Group Description:

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC) administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Overall Number of Participants Analyzed 4 4
Mean (Full Range)
Unit of Measure: micromolar*min
6521.5
(5863 to 7259)
8296.8
(7893 to 8601)
9.Secondary Outcome
Title Percent Cal-1 Marking in Peripheral Blood
Hide Description [Not Specified]
Time Frame Up to 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)
Arm/Group Title Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Hide Arm/Group Description:
Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Overall Number of Participants Analyzed 4 4 4
Mean (Full Range)
Unit of Measure: percent Cal-1 marking
Peak marking
0.3500
(0.0000 to 1.1600)
0.7650
(0.6100 to 0.8900)
3.1200
(0.1800 to 7.4600)
Week 48
0.0000
(0.0000 to 0.0000)
0.1275
(0.0000 to 0.3800)
0.1275
(0.0000 to 0.4900)
10.Secondary Outcome
Title Cal-1 Marking in Gut-associated Lymphoid Tissue (GALT) (10-15 cm)
Hide Description Samples were collected via endoscopic biopsy from the sigmoid colon: 10-15 cm from the anal margin
Time Frame Up to 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)
Arm/Group Title Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Hide Arm/Group Description:
Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Overall Number of Participants Analyzed 4 4 4
Mean (Full Range)
Unit of Measure: copies/cell
Peak marking
0.8121
(0.0 to 2.7054)
0.0007
(0.0 to 0.0019)
0.0022
(0.0006 to 0.0060)
Week 48
0.8121
(0.0 to 2.7054)
0.0004
(0.0 to 0.0010)
0.0001
(0.0 to 0.0004)
11.Secondary Outcome
Title Cal-1 Marking in GALT (25-35 cm)
Hide Description Samples were collected via endoscopic biopsy from the sigmoid colon: 25-35 cm from the anal margin
Time Frame Up to 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)
Arm/Group Title Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Hide Arm/Group Description:
Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Overall Number of Participants Analyzed 4 4 4
Mean (Full Range)
Unit of Measure: copies/cell
Peak marking
0.0255
(0.0 to 0.0677)
0.0003
(0.0 to 0.0011)
0.0068
(0.0020 to 0.0186)
Week 48
0.0255
(0.0 to 0.0677)
0.0003
(0.0 to 0.0011)
0.0055
(0.0 to 0.0186)
12.Secondary Outcome
Title Cal-1 Marking in Bone Marrow
Hide Description [Not Specified]
Time Frame Up to 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)
Arm/Group Title Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Hide Arm/Group Description:
Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Overall Number of Participants Analyzed 4 4 4
Mean (Full Range)
Unit of Measure: copies/cell
Peak marking
0.0007
(0.0 to 0.0026)
0.0539
(0.0005 to 0.2027)
0.0021
(0.0009 to 0.0030)
Week 48
0.0007
(0.0 to 0.0026)
0.0015
(0.0005 to 0.0044)
0.0009
(0.0003 to 0.0025)
13.Secondary Outcome
Title Cal-1 C46 Expression in Peripheral Blood
Hide Description C46 relative expression will be analyzed by reverse transcriptase-quantitative polymerase chain reaction (RT-qPCR) and normalized to the expression of β2-microglobulin (β2M) mRNA
Time Frame Up to 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)
Arm/Group Title Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Hide Arm/Group Description:
Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Overall Number of Participants Analyzed 4 4 4
Mean (Full Range)
Unit of Measure: units of relative expression
Peak marking
0.0000
(0.0000 to 0.0000)
1.0496
(0.0000 to 2.9977)
2.1701
(0.0000 to 6.4800)
Week 48
0.0000
(0.0000 to 0.0000)
0.0000
(0.0000 to 0.0000)
0.0000
(0.0000 to 0.0000)
14.Secondary Outcome
Title Cal-1 sh5 Expression in Peripheral Blood
Hide Description sh5 relative expression will be analyzed by RT-qPCR and normalized to the expression of RNU38B microRNA
Time Frame Up to 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)
Arm/Group Title Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Hide Arm/Group Description:
Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Overall Number of Participants Analyzed 4 4 4
Mean (Full Range)
Unit of Measure: units of relative expression
Peak marking
0.0000
(0.0000 to 0.0000)
0.2027
(0.0000 to 0.6489)
1.6933
(0.1930 to 4.6736)
Week 48
0.0000
(0.0000 to 0.0000)
0.0000
(0.0000 to 0.0000)
0.0000
(0.0000 to 0.0000)
15.Secondary Outcome
Title HIV Viral Load at Baseline Screening and Week 48 or at Anti-retroviral Therapy (ART) Re-commencement
Hide Description [Not Specified]
Time Frame Up to 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)
Arm/Group Title Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Hide Arm/Group Description:
Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Overall Number of Participants Analyzed 4 4 4
Mean (Full Range)
Unit of Measure: Log10 (copies/mL)
Baseline screening
4.59
(4.06 to 4.87)
4.51
(3.98 to 4.82)
4.28
(3.45 to 4.62)
Week 48 or at ART re-commencement
4.56
(4.00 to 4.93)
4.70
(3.88 to 4.96)
4.61
(4.20 to 4.95)
16.Secondary Outcome
Title CD4+ Count at Baseline Screening and Week 48 or at ART Re-commencement
Hide Description [Not Specified]
Time Frame Up to 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)
Arm/Group Title Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Hide Arm/Group Description:
Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Overall Number of Participants Analyzed 4 4 4
Mean (Full Range)
Unit of Measure: CD4+ cells/cubic mm
Baseline screening
680.75
(522.5 to 809.5)
575.13
(537.0 to 677.5)
668.63
(529.0 to 878.0)
Week 48 or at ART re-commencement
553.3
(450 to 759)
383.5
(274 to 549)
245.5
(152 to 385)
17.Secondary Outcome
Title Number of Participants With HIV-1 Tropism Shift
Hide Description Shift from R5 to X4 or dual/mixed tropism
Time Frame Up to 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
SP
Arm/Group Title Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Hide Arm/Group Description:
Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Overall Number of Participants Analyzed 4 4 5
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
Time Frame Up to 48 weeks per participant
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Hide Arm/Group Description Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC) administered as pre-conditioning for transplant

Busulfan: Intravenous busulfan

All-Cause Mortality
Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/4 (0.00%)      0/4 (0.00%)      0/5 (0.00%)    
Hide Serious Adverse Events
Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/4 (0.00%)      0/4 (0.00%)      1/5 (20.00%)    
Gastrointestinal disorders       
gastroenteritis   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Cohort 1 (CSL202 With No Busulfan) Cohort 2 (CSL202 With 1 Busulfan Dose) Cohort 3 (CSL202 With 2 Busulfan Doses)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   4/4 (100.00%)      4/4 (100.00%)      5/5 (100.00%)    
Blood and lymphatic system disorders       
Neutropenia   0/4 (0.00%)  0 4/4 (100.00%)  38 5/5 (100.00%)  21
Thrombocytopenia   0/4 (0.00%)  0 4/4 (100.00%)  19 4/5 (80.00%)  40
Leukopenia   0/4 (0.00%)  0 4/4 (100.00%)  18 3/5 (60.00%)  4
Leukocytosis   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Ear and labyrinth disorders       
ear congestion   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Eye disorders       
Eye irritation   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Eye pruritus   0/4 (0.00%)  0 1/4 (25.00%)  1 0/5 (0.00%)  0
Vision blurred   1/4 (25.00%)  1 0/4 (0.00%)  0 0/5 (0.00%)  0
Gastrointestinal disorders       
Nausea   0/4 (0.00%)  0 2/4 (50.00%)  3 5/5 (100.00%)  5
Diarrhoea   1/4 (25.00%)  3 0/4 (0.00%)  0 4/5 (80.00%)  5
Vomiting   0/4 (0.00%)  0 0/4 (0.00%)  0 2/5 (40.00%)  3
Constipation   0/4 (0.00%)  0 2/4 (50.00%)  2 0/5 (0.00%)  0
Retching   0/4 (0.00%)  0 0/4 (0.00%)  0 2/5 (40.00%)  2
Toothache   1/4 (25.00%)  1 1/4 (25.00%)  1 0/5 (0.00%)  0
Abdominal distension   0/4 (0.00%)  0 1/4 (25.00%)  1 0/5 (0.00%)  0
Dry mouth   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Faeces soft   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Food poisoning   0/4 (0.00%)  0 1/4 (25.00%)  1 0/5 (0.00%)  0
Haematochezia   0/4 (0.00%)  0 1/4 (25.00%)  1 0/5 (0.00%)  0
Stomatitis   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Tongue discolouration   0/4 (0.00%)  0 1/4 (25.00%)  1 0/5 (0.00%)  0
Gastroenteritis   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
General disorders       
Fatigue   2/4 (50.00%)  3 3/4 (75.00%)  5 3/5 (60.00%)  4
Chills   1/4 (25.00%)  1 1/4 (25.00%)  1 3/5 (60.00%)  3
Pain   1/4 (25.00%)  1 1/4 (25.00%)  1 2/5 (40.00%)  2
Pyrexia   1/4 (25.00%)  1 0/4 (0.00%)  0 3/5 (60.00%)  3
Asthenia   0/4 (0.00%)  0 1/4 (25.00%)  1 1/5 (20.00%)  1
Malaise   1/4 (25.00%)  1 1/4 (25.00%)  1 0/5 (0.00%)  0
Night Sweats   0/4 (0.00%)  0 0/4 (0.00%)  0 2/5 (40.00%)  2
Catheter site pain   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Influenza like illness   1/4 (25.00%)  1 0/4 (0.00%)  0 0/5 (0.00%)  0
Infections and infestations       
Upper respiratory tract infection   3/4 (75.00%)  5 1/4 (25.00%)  2 1/5 (20.00%)  1
Cellulitis   0/4 (0.00%)  0 0/4 (0.00%)  0 2/5 (40.00%)  2
Sinusitis   1/4 (25.00%)  1 0/4 (0.00%)  0 1/5 (20.00%)  1
Acarodermatitis   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  2
Bronchitis   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Diverticulitis   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Gingivitis   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Secondary syphilis   0/4 (0.00%)  0 1/4 (25.00%)  1 0/5 (0.00%)  0
Staphylococcal infection   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Tinea pedis   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Injury, poisoning and procedural complications       
Contusion   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Head injury   0/4 (0.00%)  0 1/4 (25.00%)  1 0/5 (0.00%)  0
Road traffic accident   1/4 (25.00%)  1 0/4 (0.00%)  0 0/5 (0.00%)  0
Investigations       
Alanine aminotransferase increased   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Aspartate aminotransferase increased   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
CD4 lymphocytes decreased   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Metabolism and nutrition disorders       
Decreased appetite   1/4 (25.00%)  1 0/4 (0.00%)  0 1/5 (20.00%)  1
Musculoskeletal and connective tissue disorders       
Back pain   3/4 (75.00%)  3 3/4 (75.00%)  5 2/5 (40.00%)  2
Arthralgia   2/4 (50.00%)  4 0/4 (0.00%)  0 0/5 (0.00%)  0
Bone pain   1/4 (25.00%)  1 0/4 (0.00%)  0 0/5 (0.00%)  0
Nervous system disorders       
Headache   1/4 (25.00%)  1 1/4 (25.00%)  1 1/5 (20.00%)  2
Insomnia   1/4 (25.00%)  1 0/4 (0.00%)  0 2/5 (40.00%)  3
Dizziness   1/4 (25.00%)  1 0/4 (0.00%)  0 1/5 (20.00%)  1
Confusional state   1/4 (25.00%)  1 0/4 (0.00%)  0 0/5 (0.00%)  0
Hypoaesthesia   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Migraine   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Paraesthesia   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Somnolence   1/4 (25.00%)  1 0/4 (0.00%)  0 0/5 (0.00%)  0
Psychiatric disorders       
Depression   0/4 (0.00%)  0 1/4 (25.00%)  1 0/5 (0.00%)  0
Irritability   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Somnambulism   1/4 (25.00%)  1 0/4 (0.00%)  0 0/5 (0.00%)  0
Reproductive system and breast disorders       
Haematospermia   0/4 (0.00%)  0 1/4 (25.00%)  2 0/5 (0.00%)  0
Respiratory, thoracic and mediastinal disorders       
Cough   1/4 (25.00%)  1 0/4 (0.00%)  0 3/5 (60.00%)  3
Dyspnoea   1/4 (25.00%)  1 1/4 (25.00%)  1 2/5 (40.00%)  2
Oropharyngeal pain   1/4 (25.00%)  2 0/4 (0.00%)  0 1/5 (20.00%)  1
Wheezing   0/4 (0.00%)  0 0/4 (0.00%)  0 2/5 (40.00%)  2
Epistaxis   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Paranasal sinus hypersecretion   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Sinus congestion   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Sneezing   1/4 (25.00%)  1 0/4 (0.00%)  0 0/5 (0.00%)  0
Throat irritation   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Tonsillar disorder   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Upper-airway cough syndrome   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Skin and subcutaneous tissue disorders       
Skin odour abnormal   3/4 (75.00%)  3 2/4 (50.00%)  2 0/5 (0.00%)  0
Alopecia   0/4 (0.00%)  0 3/4 (75.00%)  3 1/5 (20.00%)  1
Rash   0/4 (0.00%)  0 2/4 (50.00%)  3 1/5 (20.00%)  1
Dermatitis allergic   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  2
Rash vesicular   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  2
Acne   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Dry skin   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Eosinophilic pustular folliculitis   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Excoriation   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Photosensitivity reaction   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Pruritus   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Rash pruritic   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Seborrhoeic dermatitis   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Skin erosion   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Skin mass   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Urticaria   0/4 (0.00%)  0 0/4 (0.00%)  0 1/5 (20.00%)  1
Vascular disorders       
Hot flush   1/4 (25.00%)  1 0/4 (0.00%)  0 0/5 (0.00%)  0
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Trial Registration Coordinator
Organization: CSL Behring
Phone: 484-878-4000
EMail: clinicaltrials@cslbehring.com
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Responsible Party: Calimmune, Inc.
ClinicalTrials.gov Identifier: NCT01734850    
Other Study ID Numbers: CAL-USA-11
First Submitted: November 19, 2012
First Posted: November 28, 2012
Results First Submitted: June 25, 2020
Results First Posted: August 6, 2020
Last Update Posted: August 6, 2020