A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
CompletedPhase 2, PHASE3Results postedLast updated 25 April 2022
What this trial tests
Phase 2, PHASE3 trial testing Lenti-D Drug Product (eli-cel) in Cerebral Adrenoleukodystrophy (CALD) in 32 participants. Completed in 26 March 2021.
Under 17, male only, with Cerebral Adrenoleukodystrophy (CALD). Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Percentage of Participants Who Were Alive and Have None of the 6 Major Functional Disabilities (MFDs) at Month 24 and Without Allo-HSCT or Rescue Cell AdministrationPrimary· At Month 24
The 6 MFDs consisted of loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, complete loss of voluntary movement. Month 24 MFD-Free survival criteria was defined as: alive at 24 months post-infusion; had not developed any of the MFDs by 24 months post-infusion; had not received rescue cell administration or allo-HSCT by 24 months post-infusion; and had not withdrawn from the study or had not been lost to follow-up by 24 months post-infusion. Percentage of participants who were alive and have none of the 6 major functional disabilities (MFDs) at Mo
Group
Value
95% CI
Lenti-D Drug Product
90.6
75.0 – 98.0
Proportion of Participants Who Had Experienced Either Acute ([>or=] Grade II) or Chronic Graft Versus Host Disease (GVHD) by Month 24Primary· By Month 24
Acute GVHD graded on the Acute GVHD Grading Scale (I-IV): Grade I is characterized as mild disease, Grade II as moderate, Grade III as severe (involvement of any organ system), and Grade IV as life-threatening; chronic GVHD was determined by the Investigator. Percentage of participants who experienced with either acute (\>= Grade II) or chronic GVHD at Month 24 were reported.
Group
Value
95% CI
Lenti-D Drug Product
0.0
0.0 – 10.9
Percentage of Participants Who Demonstrated Resolution of Gadolinium Positivity on Magnetic Resonance Imaging (MRI) at Month 24Secondary· At Month 24
Percentage of participants who demonstrated resolution of gadolinium positivity (i.e., GdE-) on MRI at Month 24 were reported.
Group
Value
95% CI
Lenti-D Drug Product
86.7
69.3 – 96.2
Time to Sustained Resolution of Gadolinium Positivity on MRISecondary· Up to Month 24
Sustained resolution of gadolinium positivity was defined as having at least two consecutive GdE- results by MRI without a subsequent evaluation indicating GdE+.
Group
Value
95% CI
Lenti-D Drug Product
77
25 – 551
Number of Participants With Change in Total Neurologic Function Score (NFS) From Baseline up to Month 24Secondary· Baseline up to Month 24
NFS was a 25-point score used to evaluate the severity of gross neurologic dysfunction in CALD by scoring 15 symptoms (functional domains) across 6 categories. Listed here are the 15 symptoms followed by their maximal score out of 25 points: a) Hearing / auditory processing problems-1, b) Aphasia/apraxia-1, c) Loss of communication-3, d) Vision impairment/field cut-1, e) Cortical blindness-2, f) Swallowing/other CNS dysfunctions-2, g) Tube feeding-2, h) Running difficulties/hyperreflexia-1, i) Walking difficulties/spasticity/spastic gait (no assistance)-1, j) Spastic gait (needs assistance)-2,
Group
Value
95% CI
Lenti-D Drug Product
4
Major Functional Disability (MFD)-Free Survival RateSecondary· At 24 months after Lenti-D drug infusion
MFD-free survival rate was defined as percentage of participants from drug product infusion to either second transplant, MFD, or death due to any cause, whichever occurs first. MFD-free survival rate was analyzed using Kaplan-Meier Analysis. Kaplan-Meier estimated MFD-free survival rate at 24 months after Lenti-D drug infusion was reported.
Group
Value
95% CI
Lenti-D Drug Product
90.6
73.7 – 96.9
Overall Survival RateSecondary· At 24 months after Lenti-D drug infusion
Overall survival rate was defined as percentage of participants alive from date of Lenti-D drug product infusion (Day 0) to date of death of all causes. Overall survival rate was censored at the date of last visit if the participant were alive. Participants who are alive were censored at the date of last contact. Overall survival rate was analyzed using Kaplan-Meier Analysis. Kaplan-Meier estimated overall survival rate at 24 months after Lenti-D drug infusion was reported.
Group
Value
95% CI
Lenti-D Drug Product
96.7
78.6 – 99.5
Proportion of Participants With Neutrophil Engraftment by 42 Days Post-drug Product InfusionSecondary· By 42 days post-drug infusion
Neutrophil engraftment (NE) was defined as achieving 3 consecutive absolute neutrophil count (ANC) laboratory values of \>= 0.5×10\^9 cells/Liter (L) (after initial post-infusion nadir) obtained on different days by 42 days post-infusion of Lenti-D Drug Product (Relative Day 43). Percentage of participants with neutrophil engraftment by 42 Days post-drug product infusion were reported.
Group
Value
95% CI
Lenti-D Drug Product
100.0
89.1 – 100
Time to Neutrophil Engraftment Post-drug Product InfusionSecondary· By 42 days post-drug infusion
Neutrophil Engraftment was defined as achieving 3 consecutive ANC laboratory values of \>= 0.5×10\^9 cells/L (after initial post-infusion nadir) obtained on different days by 42 days post-infusion of Lenti-D Drug Product (Relative Day 43). Time to neutrophil engraftment post-drug product infusion was reported.
Group
Value
95% CI
Lenti-D Drug Product
13.0
11 – 41
Proportion of Participants With Platelet Engraftment by Month 24Secondary· By Month 24
Platelet Engraftment was defined as achieving 3 consecutive unsupported platelet counts of \>=20 × 10\^9 cells/L (after initial post-infusion nadir) obtained on different days while no platelet transfusions were administered for 7 days immediately preceding and during the evaluation period. The first day of 3 consecutive platelet counts \>=20 × 10\^9 cells/L was the day of PE. Percentage of participants with Platelet Engraftment by Month 24 (Rel Day 730) were reported.
Group
Value
95% CI
Lenti-D Drug Product
100
89.1 – 100.0
Time to Platelet Engraftment Post-drug Product InfusionSecondary· By Month 24
Platelet Engraftment was defined as achieving 3 consecutive unsupported platelet counts of \> or =20 × 10\^9 cells/L (after initial post-infusion nadir) obtained on different days while no platelet transfusions were administered for 7 days immediately preceding and during the evaluation period. The first day of 3 consecutive platelet counts \>=20 × 10\^9 cells/L was the day of PE. Time to Platelet Engraftment post-drug product infusion up to Month 24 was reported.
Group
Value
95% CI
Lenti-D Drug Product
32
16 – 60
Proportion of Participants With Engraftment Failure By Month 24Secondary· By Month 24
Participants were considered to have primary engraftment failure if they did not achieve NE by Relative Day 43. A participant was considered to have secondary engraftment failure if they achieved and then subsequently lost NE by the Month 24, i.e., if they met both the conditions; Achieved NE by Relative Day 43 as defined above and had sustained decline in ANC to \< 0.5×10\^9 cells/L for 3 consecutive measurements on different days after Relative Day 43, without alternate etiology. First day of the 3 consecutive ANC decline to \< 0.5×10\^9 cells/L was considered the day of secondary engraftmen
Group
Value
95% CI
Lenti-D Drug Product
0.0
0.0 – 11.9
Adverse events — posted to ClinicalTrials.gov
Time frame: From date of informed consent up to Month 24.
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
This trial assessed the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector (also called elivaldogene autotemcel or eli-cel), for the treatment of cerebral adrenoleukodystrophy (CALD). A participant's blood stem cells were collected and modified (transduced) using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification (transduction) with the Lenti-D lentiviral vector, the cells were transplanted back into the participant following myeloablative conditioning. Participants in this study will be continuously followed in study LTF-304.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
Other recruiting trials for Cerebral Adrenoleukodystrophy (CALD)
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Other Genetix Biotherapeutics Inc. trials
Trials by the same sponsor.
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Genetix Biotherapeutics Inc.
Last refreshed: 25 April 2022
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01896102.