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CompletedNCT02999984Updated Aug 3, 2022Results posted

Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

A Phase 1/2 interventional study of Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101) and busulfan in Severe Combined Immunodeficiency Due to ADA Deficiency, sponsored by University of California, Los Angeles. Completed at 1 site in United States. Open to participants aged 30 Days to 17 Years. Per ClinicalTrials.gov, last updated 2022-08-03.

Sponsored by University of California, Los Angeles · Phase 1/2, Interventional, and Treatment

Phase
Phase 1/2
Study type
Interventional
Enrollment
10
Allocation
Not applicable
Ages
30 Days to 17 Years
Sex
All
01

Study summary

This is a prospective, non-randomized, single-cohort, longitudinal, single-center, clinical study designed to assess the efficacy and safety of a cryopreserved formulation of OTL-101 (autologous CD34+ hematopoietic stem/progenitor cells transduced ex vivo with EFS (Elongation Factor 1α Short form) Lentiviral Vector (LV) encoding for the human ADA gene) administered to ADA-SCID subjects between the ages of 30 days and 17 years of age, who are not eligible for an Human Leukocyte Antigen (HLA) matched sibling/family donor and meeting the inclusion/exclusion criteria. The OTL-101 product is infused after a minimal interval of at least 24 hours following the completion of reduced intensity conditioning. For subjects who successfully receive the OTL-101 product, pegademase bovine (PEG-ADA) Enzyme Replacement Therapy (ERT) is discontinued at Day+30 (-3/+15) after the transplant. After their discharge from hospital, the subjects will be seen at regular intervals to review their history, perform examinations and draw blood samples to assess immunity and safety.

02

Conditions studied

  • Severe Combined Immunodeficiency Due to ADA Deficiency

Keywords

  • gene therapy
  • hematopoietic and progenitor cells
  • lentiviral vector
  • ADA-SCID
03

In context

Lead sponsor

University of California, Los Angeles is the lead sponsor of 1,167 studies on the registry; 192 are open to participants now.

Of its 94 completed or terminated interventional studies of FDA-regulated products, 69 (73%) have results posted.

Browse University of California, Los Angeles studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
30 Days to 17 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Provision of written informed consent prior to any study related procedures. In this study consent must be provided by the parents/legal guardians and, where applicable according to local laws, a signed assent from the child,
  2. Subjects ≥30 days and \<18 years of age,

  3. With a diagnosis of ADA-SCID based on:

    Evidence of ADA deficiency, defined as:

    i. Decreased ADA enzymatic activity in erythrocytes, leukocytes, skin fibroblasts, or in cultured fetal cells to levels consistent with ADA-SCID as determined by the reference laboratory, or ii. Identified mutations in ADA alleles consistent with a severe reduction in ADA activity,

    Evidence of ADA-SCID based on either:

    i. Family history of a first order relative with ADA deficiency and clinical and laboratory evidence of severe immunologic deficiency, or ii. Evidence of severe immunologic deficiency in subjects prior to the institution of immune restorative therapy, based on

    • Lymphopenia (absolute lymphocyte count (ALC) \<400 cells/µL) OR absence or low number of T cells (absolute CD3+ count \< 300 cells/µL), or
    • Severely decreased T lymphocyte blastogenic responses to phytohemagglutinin (either \<10% of lower limit of normal controls for the diagnostic laboratory, or \<10% of the response of the normal control of the day, or stimulation index \<10), or
    • Identification of SCID by neonatal screening revealing low T cell Receptor Excision Circle (TREC) levels.
  4. Ineligible for matched family allogeneic Bone Marrow (BM) transplantation, defined as the absence of a medically eligible HLA-identical sibling or family donor, with normal immune function, who could serve as an allogeneic bone marrow donor.
  5. Females of child-bearing age will be required to provide a negative pregnancy test 30 days prior to Visit 2.
  6. Subjects and their parents/legal guardians must be willing and able to comply with study restrictions and to remain at the clinic for the required duration during the study period and willing to return to the clinic for the follow up evaluation as specified in the protocol.

Exclusion criteria

Exclusion Criteria:

  1. Ineligible for autologous Hematopoietic Stem Cell Transplantation (HSCT) as per clinical site criteria.
  2. Other conditions which in the opinion of the Principal Investigator and/or Co Investigators, contraindicate the harvest of bone marrow, the administration of Busulfan and the infusion of transduced cells, or which indicate an inability of the subject or subject's parent/legal guardian to comply with the protocol.

  3. Hematologic abnormality, defined as:

    • Anemia (Hb \<8.0 g/dl).
    • Neutropenia (ANC \<500/mm3). Note: ANC \<500 with absence of myelodysplastic syndrome on bone marrow aspirate and biopsy and normal marrow cytogenetics are acceptable for eligibility.
    • Thrombocytopenia (platelet count \<50,000/mm3, at any age).
    • Prothrombin time or international normalized ratio (INR) and partial thromboplastin time (PTT) >2 x upper limit of normal (ULN) (subjects with a correctable deficiency controlled on medication will not be excluded).
    • Cytogenetic abnormalities on peripheral blood or bone marrow or amniotic fluid (if available).
    • Prior allogeneic HSCT with cytoreductive conditioning.

  4. Pulmonary abnormality, defined as:

    • Resting O2 saturation by pulse oximetry \<90% on room air.
    • Chest X-ray indicating active or progressive pulmonary disease. Note: Chest X ray indicating residual signs of treated pneumonitis is acceptable for eligibility.

  5. Cardiac abnormality, defined as:

    • Abnormal ECG indicating cardiac pathology.
    • Uncorrected congenital cardiac malformation with clinical symptoms.
    • Active cardiac disease, including clinical evidence of congestive heart failure, cyanosis, hypotension.
    • Poor cardiac function as evidenced by left ventricular ejection fraction \<40% on echocardiogram.

  6. Neurologic abnormality, defined as:

    • Significant neurologic abnormality revealed by examination.
    • Uncontrolled seizure disorder.

  7. Renal abnormality, defined as:

    • Renal insufficiency: serum creatinine ≥1.2 mg/dl (106 µmol/L), or ≥3+ proteinuria.
    • Abnormal serum sodium, potassium, calcium, magnesium or phosphate levels at >2 x ULN.

  8. Hepatic/gastrointestinal abnormality, defined as:

    • Serum transaminases >5 x ULN.
    • Serum bilirubin >2 x ULN.
    • Serum glucose >1.5 x ULN.

  9. Oncologic disease, defined as:

    • Evidence of active malignant disease other than dermatofibrosarcoma protuberans (DFSP).
    • Evidence of DFSP expected to require anti-neoplastic therapy within the 5 years following the infusion of genetically corrected cells (if anti-neoplastic therapy has been completed, a subject with a history of DFSP can be included).
    • Evidence of DFSP expected to be life limiting within the 5 years following the infusion of genetically corrected cells.
  10. Known sensitivity to Busulfan.

  11. Confirmation of an infectious disease by deoxyribonucleic acid (DNA) Polymerase chain reaction (PCR) positive at time of screening assessment for the following:

    • HIV-1,
    • Hepatitis B,
    • Parvovirus B19.
  12. The subject is pregnant or has a major congenital anomaly.
  13. Is likely to require treatment during the study with drugs that are not permitted by the study protocol.
  14. The subject has previously received another form of gene therapy.
05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
10 participants (actual)

Study arms

  • Experimental
    Gene Therapy

    Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells

    Genetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101) · Drug: busulfan · Drug: PEG-ADA ERT

Interventions

  • GeneticInfusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101)

    autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101) are infused intravenously

    Also known as: OTL-101

  • Drugbusulfan

    Busulfan is used for non-myeloablative conditioning

  • DrugPEG-ADA ERT

    PEG-ADA ERT is discontinued at Day +30 (-3/+15 days) after successful engraftment

06

What researchers measure

Primary outcomes

  1. Percentage of Participants With Treatment Efficacy After Treatment With OTL 101 (6 Months)

    Efficacy of OTL-101 treatment at 6 months post OTL-101 infusion based on the following parameters and thresholds: 1. ADA enzyme activity in erythrocytes above baseline/pretreatment level (i.e., \>0 units). ADA enzyme activity is measured to assess the amount of functional gene product produced from the normal ADA transgene delivered by EFS-ADA LV. 2. Absolute CD3+ T cell counts ≥200 cells/μL. Increase in CD3+ T cell counts is a marker of immune reconstitution. 3. Granulocyte samples positive for vector sequences by quantitative Polymerase Chain Reaction (PCR) (≥1/10,000 cells). Vector copy number (VCN) in the Peripheral Blood (PB) Granulocytes fraction that was T cell depleted, is a surrogate for amount of engrafted genetically modified Hematopoietic stem cell (HSC) that are producing granulocytes every 3-5 days.

    Time frame: 6 months

  2. Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)

    Overall survival is defined as the percentage of subjects alive at 12 months post- treatment with cryopreserved OTL-101.

    Time frame: 12 Months

  3. Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)

    Event-free survival is defined as the percentage of subjects alive with no "event", an "event" being the resumption of PEG-ADA ERT or the need for a rescue allogeneic Hematopoietic Stem Cell Transplant (HSCT), or death.

    Time frame: 12 Months

Secondary outcomes

  1. OS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years)

    OS is defined as the percentage of subjects alive at 24 months post- treatment with cryopreserved OTL-101.

    Time frame: 24 months

  2. EvFS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years)

    Event-free survival is defined as the percentage of subjects alive with no "event", an "event" being the resumption of PEG-ADA ERT or the need for a rescue allogenic Hematopoietic Stem Cell Transplant (HSCT), or death.

    Time frame: 24 months

  3. Change From Baseline in CD3+ T Cell Counts (2 Years)

    Immune reconstitution was assessed by change in CD3+ T Cell counts over time.

    Time frame: 24 months

  4. Severe Infections Excluding First 3 Months After Treatment

    The infections of interest in this study were severe infections or opportunistic infectious episodes, defined as infections requiring hospitalization or prolonging hospitalization and/or documented infections by opportunistic pathogens. Infections that took place in the first 3 months of follow-up post treatment were excluded from calculations to avoid possible bias introduced in the data by the effects of conditioning.

    Time frame: 24 months

  5. Change From Baseline in Quality of Life Measures (2 Years)

    Assessment of quality of life was measured by the Lansky Performance Status Scale. The maximum score on the Lansky Performance Scale is 100 - the child is fully active and able to carry on normal activity with no special care needed. The minimum score is 10 - the child is completely disabled, not even passive play. The scores at baseline (pre-treatment with OTL-101) and scores at Month 24 post-treatment with OTL-101 were compared to establish if there were any changes in the child's score in this timeframe.

    Time frame: 24 months

  6. Percentage of Patients Who Stopped Immunoglobulin Replacement Therapy (IgRT) (2 Years)

    Use of immunoglobulin replacement therapies prior to and after gene therapy were monitored. Indications for considering the discontinuation of immunoglobulin replacement therapy included: absolute CD4+ \>200, absolute B cell \>100/μl, IgA or IgM \> lower limit of normal for age or gene marking \>1% detectable in B cells.

    Time frame: 24 months

  7. Time to Cessation of IgRT for Those Who Stopped (2 Years)

    Use of immunoglobulin replacement therapies prior to and after gene therapy were monitored. For subjects who stopped IgRT during the study, the time of cessation was recorded.

    Time frame: 24 months

07

Results

Posted Jul 22, 2022

Participant flow

Participant flow — Overall Study
MilestoneGene Therapy
Started10
Completed9
Not completed1
Withdrew: Treatment failure1

Outcome measures

PrimaryPercentage of Participants With Treatment Efficacy After Treatment With OTL 101 (6 Months)

Efficacy of OTL-101 treatment at 6 months post OTL-101 infusion based on the following parameters and thresholds: 1. ADA enzyme activity in erythrocytes above baseline/pretreatment level (i.e., \>0 units). ADA enzyme activity is measured to assess the amount of functional gene product produced from the normal ADA transgene delivered by EFS-ADA LV. 2. Absolute CD3+ T cell counts ≥200 cells/μL. Increase in CD3+ T cell counts is a marker of immune reconstitution. 3. Granulocyte samples positive for vector sequences by quantitative Polymerase Chain Reaction (PCR) (≥1/10,000 cells). Vector copy number (VCN) in the Peripheral Blood (PB) Granulocytes fraction that was T cell depleted, is a surrogate for amount of engrafted genetically modified Hematopoietic stem cell (HSC) that are producing granulocytes every 3-5 days.

Time frame:
6 months
Reported as:
Number · percentage of participants
Percentage of Participants With Treatment Efficacy After Treatment With OTL 101 (6 Months)
percentage of participantsGene Therapy
% of subjects with an increase from baseline in RBC ADA activity100
% of subjects with CD3+ T-cell count >=200 cells/μL100
% of subjects with detectable gene-marked granulocytes by quantitative PCR >=1/10,000 cells90
PrimaryOverall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)

Overall survival is defined as the percentage of subjects alive at 12 months post- treatment with cryopreserved OTL-101.

Time frame:
12 Months
Reported as:
Number · percentage of participants
Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)
percentage of participantsGene Therapy
Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)100 (66.37 to 100)
PrimaryEvent-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)

Event-free survival is defined as the percentage of subjects alive with no "event", an "event" being the resumption of PEG-ADA ERT or the need for a rescue allogeneic Hematopoietic Stem Cell Transplant (HSCT), or death.

Time frame:
12 Months
Reported as:
Number · percentage of participants
Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)
percentage of participantsGene Therapy
Percentage event-free at 12 months90.00 (55.50 to 99.75)
Percentage without reinstitution of PEG-ADA by 12 months90.00 (55.50 to 99.75)
Percentage without rescue HSCT by 12 months100 (66.37 to 100)
SecondaryOS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years)

OS is defined as the percentage of subjects alive at 24 months post- treatment with cryopreserved OTL-101.

Time frame:
24 months
Reported as:
Number · percentage of participants
OS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years)
percentage of participantsGene Therapy
OS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years)100 (66.37 to 100)
SecondaryEvFS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years)

Event-free survival is defined as the percentage of subjects alive with no "event", an "event" being the resumption of PEG-ADA ERT or the need for a rescue allogenic Hematopoietic Stem Cell Transplant (HSCT), or death.

Time frame:
24 months
Reported as:
Number · percentage of participants
EvFS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years)
percentage of participantsGene Therapy
Percentage event-free at 24 months90.00 (55.50 to 99.75)
Percentage without reinstitution of PEG-ADA by 24 months90.00 (55.50 to 99.75)
Percentage without rescue HSCT by 24 months100 (66.37 to 100)
SecondaryChange From Baseline in CD3+ T Cell Counts (2 Years)

Immune reconstitution was assessed by change in CD3+ T Cell counts over time.

Time frame:
24 months
Reported as:
Median · cells/μL
Change From Baseline in CD3+ T Cell Counts (2 Years)
cells/μLGene Therapy
Change From Baseline in CD3+ T Cell Counts (2 Years)418.5 (-332 to 1976)
SecondarySevere Infections Excluding First 3 Months After Treatment

The infections of interest in this study were severe infections or opportunistic infectious episodes, defined as infections requiring hospitalization or prolonging hospitalization and/or documented infections by opportunistic pathogens. Infections that took place in the first 3 months of follow-up post treatment were excluded from calculations to avoid possible bias introduced in the data by the effects of conditioning.

Time frame:
24 months
Reported as:
Number · Infections per person per year
Severe Infections Excluding First 3 Months After Treatment
Infections per person per yearGene Therapy
Severe Infections Excluding First 3 Months After Treatment0.12
SecondaryChange From Baseline in Quality of Life Measures (2 Years)

Assessment of quality of life was measured by the Lansky Performance Status Scale. The maximum score on the Lansky Performance Scale is 100 - the child is fully active and able to carry on normal activity with no special care needed. The minimum score is 10 - the child is completely disabled, not even passive play. The scores at baseline (pre-treatment with OTL-101) and scores at Month 24 post-treatment with OTL-101 were compared to establish if there were any changes in the child's score in this timeframe.

Time frame:
24 months
Reported as:
Median · Lansky Performance Score
Change From Baseline in Quality of Life Measures (2 Years)
Lansky Performance ScoreGene Therapy
Change From Baseline in Quality of Life Measures (2 Years)0 (0 to 0)
SecondaryPercentage of Patients Who Stopped Immunoglobulin Replacement Therapy (IgRT) (2 Years)

Use of immunoglobulin replacement therapies prior to and after gene therapy were monitored. Indications for considering the discontinuation of immunoglobulin replacement therapy included: absolute CD4+ \>200, absolute B cell \>100/μl, IgA or IgM \> lower limit of normal for age or gene marking \>1% detectable in B cells.

Time frame:
24 months
Reported as:
Number · percentage of participants
Percentage of Patients Who Stopped Immunoglobulin Replacement Therapy (IgRT) (2 Years)
percentage of participantsGene Therapy
Percentage of Patients Who Stopped Immunoglobulin Replacement Therapy (IgRT) (2 Years)78
SecondaryTime to Cessation of IgRT for Those Who Stopped (2 Years)

Use of immunoglobulin replacement therapies prior to and after gene therapy were monitored. For subjects who stopped IgRT during the study, the time of cessation was recorded.

Time frame:
24 months
Reported as:
Median · months
Time to Cessation of IgRT for Those Who Stopped (2 Years)
monthsGene Therapy
Time to Cessation of IgRT for Those Who Stopped (2 Years)11.6 (9 to 20)

Adverse events

Collected over Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101.. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Gene Therapy0/10 (0%)3/10 (30%)10/10 (100%)
Most frequent serious events
Most frequent serious events
EventGene Therapy
BacteraemiaInfections and infestations1/10
BronchiolitisInfections and infestations1/10
Respiratory tract infection viralInfections and infestations1/10
LeukopeniaBlood and lymphatic system disorders1/10
NeutropeniaBlood and lymphatic system disorders1/10
DehydrationMetabolism and nutrition disorders1/10
Most frequent other events
Showing 10 of 51
Most frequent other events
EventGene Therapy
LeukopeniaBlood and lymphatic system disorders10/10
NeutropeniaBlood and lymphatic system disorders10/10
AnaemiaBlood and lymphatic system disorders8/10
Dermatitis diaperSkin and subcutaneous tissue disorders6/10
Aspartate aminotransferase increasedInvestigations5/10
Alanine aminotransferase increasedInvestigations5/10
CoughRespiratory, thoracic and mediastinal disorders5/10
Viral upper respiratory tract infectionInfections and infestations4/10
ThrombocytopeniaBlood and lymphatic system disorders3/10
Upper respiratory tract infectionInfections and infestations3/10

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Gene Therapy
<=18 years10
Between 18 and 65 years0
>=65 years0
Sex: Female, Male
Sex: Female, Male(Participants)Gene Therapy
Female6
Male4
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)Gene Therapy
Caucasian6
Afro-American2
Other1
Unknown1
Region of Enrollment
Region of Enrollment(participants)Gene Therapy
United States10
08

Study locations

1 site
  • University of California, Los Angeles
    Los Angeles, California 90095, United States
09

References and documents

Publications

  • Carbonaro DA, Zhang L, Jin X, Montiel-Equihua C, Geiger S, Carmo M, Cooper A, Fairbanks L, Kaufman ML, Sebire NJ, Hollis RP, Blundell MP, Senadheera S, Fu PY, Sahaghian A, Chan RY, Wang X, Cornetta K, Thrasher AJ, Kohn DB, Gaspar HB. Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency. Mol Ther. 2014 Mar;22(3):607-622. doi: 10.1038/mt.2013.265. Epub 2013 Nov 20. PubMed 24256635 ↗
  • Kohn DB, Booth C, Shaw KL, Xu-Bayford J, Garabedian E, Trevisan V, Carbonaro-Sarracino DA, Soni K, Terrazas D, Snell K, Ikeda A, Leon-Rico D, Moore TB, Buckland KF, Shah AJ, Gilmour KC, De Oliveira S, Rivat C, Crooks GM, Izotova N, Tse J, Adams S, Shupien S, Ricketts H, Davila A, Uzowuru C, Icreverzi A, Barman P, Campo Fernandez B, Hollis RP, Coronel M, Yu A, Chun KM, Casas CE, Zhang R, Arduini S, Lynn F, Kudari M, Spezzi A, Zahn M, Heimke R, Labik I, Parrott R, Buckley RH, Reeves L, Cornetta K, Sokolic R, Hershfield M, Schmidt M, Candotti F, Malech HL, Thrasher AJ, Gaspar HB. Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency. N Engl J Med. 2021 May 27;384(21):2002-2013. doi: 10.1056/NEJMoa2027675. Epub 2021 May 11. PubMed 33974366 ↗

Study documents

  • Study protocol · Mar 28, 2018
  • Statistical analysis plan · Jan 25, 2018

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Ran from Dec 2016 to primary completion in Oct 2018, 21 months.

Results were first posted in Jul 2022, 3 years 9 months after primary completion. A results delay was requested in Jan 2020.

How these figures are worked out, and which studies are left out of them.

Registered
Dec 19, 2016
First posted
Dec 21, 2016
StartActual
Dec 16, 2016
Primary completionActual
Oct 11, 2018
CompletionActual
Sep 26, 2019
Results delay requested
Jan 24, 2020
Results first posted
Jul 22, 2022
Last update
Aug 3, 2022
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Aug 3, 2022, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02999984
Lead sponsor
University of California, Los Angeles
Collaborators
California Institute for Regenerative Medicine (CIRM), Orchard Therapeutics
Responsible party
Donald B. Kohn, M.D. (Principal Investigator, University of California, Los Angeles) — Principal investigator
First posted
Dec 21, 2016
Start date
Dec 16, 2016
Primary completion
Oct 11, 2018
Completion
Sep 26, 2019
Results posted
Jul 22, 2022
Last update
Aug 3, 2022

Study contacts

Donald B. Kohn, MD
study director · University of California, Los Angeles

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
View the source record on ClinicalTrials.gov ↗

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