Mucopolysaccharidosis IHPhase 1/2TigetT10_MPSIHNCT03488394

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant

A Phase 1/2 study testing Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LVV, encoding for the α-L-iduronidase cDNA, in their final formulation medium. in people with Mucopolysaccharidosis IH, run by Orchard Therapeutics at 1 site in Italy.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record

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Trial summary

This is a phase I/II study evaluating safety and efficacy of autologous hematopoietic stem and progenitor cells genetically modified with IDUA lentiviral vector encoding for the human α-L-iduronidase gene for the treatment of patients affected by Mucopolysaccharidosis Type I, Hurler variant Read more about this study

Who is it for?: Ages 28 days to 11 years
Conditions the study is for: Mucopolysaccharidosis IH
What is being tested?: Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LVV, encoding for the α-L-iduronidase cDNA, in their final formulation medium.
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 8 participants
Where: 1 siteItaly
Study dates: 11 May 2018to Mar 2035
Phase: Phase 1/2
Are there results?: Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 28 days to 11 years
All sexes
No healthy volunteers
Child

Who may be able to join

Written informed consent by parent/legal guardian
Sex: Males and Females
Age: ≥ 28 days and ≤ 11 years old
Biochemically and molecularly proven MPS IH
Lansky index >80%
Indication to hematopoietic stem cell transplant
Lack of a non-heterozygous (for mutated IDUA) HLA-matched sibling donor or a ≥7/8 (4 digits high-resolution typing) HLA-matched cord blood donor with a cellularity ≥5 x 10^7 Total Nucleated Cells (TNC)/Kg after 1-month search.(This criterion will not apply to patients whose country of origin does not offer unrelated donor cord blood transplantation).
Adequate cardiac, renal, hepatic and pulmonary functions

Who cannot join

Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long-acting agents)
Severe, active viral, bacterial or fungal infection at eligibility evaluation
Patients affected by neoplasia or family history of familial cancer syndromes
Cytogenetic alterations associated with high risk of developing hematological malignancies
History of uncontrolled seizures
Patients with end-organ damage or any other severe disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study

Show all 10Show fewer

Positivity for HIV (serology or RNA), and/or HbsAg and/or HBV DNA and/or HCV RNA and/or Treponema Pallidum or Mycoplasma active infection
Patients with DQ/IQ <70
Previous allogeneic hematopoietic stem cells transplantation or gene therapy with a different product
Contraindications to PeIMP (G-CSF, Plerixafor, Busulfan, Fludarabine, Rituximab)

Trial locations and contacts

1 site in 1 country.

Italy1 site

Ospedale San Raffaele
Milan, 20132

Other trials in Italy

About this study

Official title: Phase I/II Study Evaluating Safety and Efficacy of Autologous Hematopoietic Stem and Progenitor Cells Genetically Modified With IDUA Lentiviral Vector Encoding for the Human α-L-iduronidase Gene for the Treatment of Patients Affected by Mucopolysaccharidosis Type I, Hurler Variant

Read the detailed description

Pediatric patients with mucopolysaccharidosis type I will be treated with genetically modified autologous hematopoietic stem cells collected from mobilized peripheral blood (or bone marrow if mobilization is not feasible) and transduced with IDUA lentiviral vector encoding for the human α-L-iduronidase gene.

Participants will be followed for up to 15 years post treatment (per regulatory guidelines for follow up of patients treated with ATMPs, under this study (TigetT10_MPSIH) or via enrollment into a separate long term follow-up (LTFU) study for the overall OTL-203 Clinical Development Program, if one is set up prior to their Year 15 visit.

Conditions: Mucopolysaccharidosis IH
Being tested: Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LVV, encoding for the α-L-iduronidase cDNA, in their final formulation medium.
Keywords: Mucopolysaccharidosis IH, Gene Therapy, Transplantation, Autologous, Lentiviral vector
Medical subject headings: Mucopolysaccharidosis I

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LVV, encoding for the α-L-iduronidase cDNA, in their final formulation medium.Genetic
The drug product target dose is more or equal to 8x10^6 CD34+ cells/Kg, with a minimum dose of 4x10^6 CD34+ cells/Kg and a maximum dose of 35x10^6 CD34+ cells/Kg.
The product will be injected intravenously.

Study groups in NCT03488394
Study group	Role	What they receive
Treatment	Experimental	Gene therapy (autologous, CD34+ cell enriched cells fraction containing HSPCs, transduced with the IDUA LVV encoding for the human IDUA gene and cryopreserved in cryoformulation medium) Receives: Genetic: Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LVV, encoding for the α-L-iduronidase cDNA, in their final formulation medium.

What is being measured

The primary outcome is the main question the study is designed to answer.

Overall survival
Number and percentage of subjects alive at the end of the trial
Measured: Assessed at multiple timepoints up to 15 years post-treatment
Achievement of haematological engraftment
Percentage of subjects with both neutrophil count more than 500/mm3 and platelets more than 20,000/mm3 (in the absence of platelet transfusion for seven consecutive days) on 3 consecutive blood counts in the first 45 days from ATIMP injection.
Measured: within day +45 after gene therapy
Safety of the administration of autologous haematopoietic stem cells transduced with IDUA LVV - Short term tolerability
Percentage of subjects not experiencing short-term adverse events of any grade and systemic reactions
Measured: 0-24 hours from ATIMP injection
Safety of the administration of autologous haematopoietic stem cells transduced with IDUA LVV - Absence of Replication Competent Lentivirus
Percentage of subjects without Replication Competent Lentivirus
Measured: Assessed at multiple timepoints up to 8 years post-treatment, or if clinically indicated
Safety of the administration of autologous haematopoietic stem cells transduced with IDUA LVV - Absence of malignancy or abnormal clonal proliferation
Percentage of subjects without abnormal clonal proliferation
Measured: Assessed at multiple timepoints up to 15 years post-treatment
Overall safety and tolerability (AE)
The number of AEs (expected/unexpected and/or related/not related) and SAEs (expected/unexpected and/or related/not related) and the percentage of subjects experiencing AEs (expected/unexpected and/or related/not related) and SAEs (expected/unexpected and/or related/not related) will be summarized by severity and within body system involved. Narratives will also be presented. The rate of occurrence of these events will also be estimated.
Measured: Assessed at multiple timepoints up to 15 years post-treatment
IDUA activity in blood (up to supraphysiologic levels) at 1-year post-treatment
IDUA activity measured on peripheral dried blood spot
Measured: At 1 year post-treatment

Secondary outcomes (7)

Anti-IDUA antibody immune response
Presence or absence and titer of anti-IDUA antibody on plasma or serum
Measured: Assessed at multiple timepoints up to 8 years post-treatment, or if clinically indicated
Achievement of supraphysiologic IDUA activity in blood
IDUA activity measured on peripheral dried blood spots up to supraphysiologic levels as compared with healthy donors. A supraphysiologic IDUA level is defined as >24.31 μmol/L/h, which is the 97.5th percentile of the IDUA distribution in healthy children
Measured: Assessed at multiple timepoints up to 15 years post-treatment
IDUA activity in plasma
IDUA activity measured on plasma samples from peripheral blood.
Measured: Assessed at multiple timepoints up to 15 years post-treatment
Engraftment of transduced cells ≥ 0.30 VCN/genome
Percentage of subjects with engraftment of transduced cells ≥ 0.30 VCN/genome on peripheral blood mononuclear cells (PBMC) and/or bone marrow (BM) progenitor cells.
Measured: Assessed at multiple timepoints up to 15 years post-treatment
Normalization of urinary GAGs
Percentage of subjects achieving normalization of urinary GAG levels (heparan sulfate and dermatan sulfate) measured by HPLC
Measured: Assessed at multiple timepoints up to 15 years post-treatment
Normalization of spleen and liver
Percentage of subjects with normal spleen and liver assessed by clinical examination (palpation) and/or ultrasound
Measured: Assessed at multiple timepoints up to 15 years post-treatment
Growth velocity
Length/height for age (cm) per month vs. WHO percentiles
Measured: Assessed at multiple timepoints up to 15 years post-treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

11 May 2018Mar 2035

First posted5 Apr 2018
Study start11 May 2018
Primary completionJan 2035 (estimated)
Study completionMar 2035 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (3)Papers the sponsor has linked to this study.
Other trials for Mucopolysaccharidosis IHStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1/2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 8 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03488394
Sponsor's protocol ID: 2024-514870-29-00
Acronym: TigetT10_MPSIH
Status last verified: Jul 2026
First submitted: 22 Mar 2018
Last updated: 16 Jul 2026
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: Undecided

Publications

Consiglieri G, Tucci F, De Pellegrin M, Guerrini B, Cattoni A, Risca G, Scarparo S, Sarzana M, Pontesilli S, Mellone R, Gasperini S, Galimberti S, Silvani P, Filisetti C, Darin S, Forni G, Miglietta S, Santi L, Facchini M, Corti A, Fumagalli F, Cicalese MP, Calbi V, Migliavacca M, Barzaghi F, Ferrua F, Gallo V, Recupero S, Canarutto D, Doglio M, Tedesco L, Volpi N, Rovelli A, la Marca G, Valsecchi MG, Zancan S, Ciceri F, Naldini L, Baldoli C, Parini R, Gentner B, Aiuti A, Bernardo ME. Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome. Sci Transl Med. 2024 May;16(745):eadi8214. doi: 10.1126/scitranslmed.adi8214. Epub 2024 May 1.
PubMed 38691622
Gentner B, Tucci F, Galimberti S, Fumagalli F, De Pellegrin M, Silvani P, Camesasca C, Pontesilli S, Darin S, Ciotti F, Sarzana M, Consiglieri G, Filisetti C, Forni G, Passerini L, Tomasoni D, Cesana D, Calabria A, Spinozzi G, Cicalese MP, Calbi V, Migliavacca M, Barzaghi F, Ferrua F, Gallo V, Miglietta S, Zonari E, Cheruku PS, Forni C, Facchini M, Corti A, Gabaldo M, Zancan S, Gasperini S, Rovelli A, Boelens JJ, Jones SA, Wynn R, Baldoli C, Montini E, Gregori S, Ciceri F, Valsecchi MG, la Marca G, Parini R, Naldini L, Aiuti A, Bernardo ME; MPSI Study Group. Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome. N Engl J Med. 2021 Nov 18;385(21):1929-1940. doi: 10.1056/NEJMoa2106596.
PubMed 34788506
Tucci F, Uria Oficialdegui ML, Consiglieri G, Cossutta M, Filisetti C, Fumagalli F, Butera C, Santangelo R, Colombo M, Manitto MP, Stoppani M, Martina E, Dane G, Camesasca C, Risca G, De Pellegrin M, Scarparo S, Sarzana M, Puricelli C, Galimberti S, Darin S, Silvani P, Bonanomi S, Gasperini S, Naldini L, Gentner B, Parini R, Del Toro M, Diaz-de-Heredia C, Aiuti A, Bernardo ME. Non-neurological, non-skeletal outcomes after hematopoietic stem and progenitor cell-gene therapy (OTL-203) for Hurler syndrome. Mol Ther. 2026 Jan 7;34(1):443-454. doi: 10.1016/j.ymthe.2025.09.042. Epub 2025 Sep 27.
PubMed 41017152

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
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ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
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RecruitingObservational study
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All Mucopolysaccharidosis IH trials

Study at a glance

From the registry record, last updated by the sponsor on 16 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Phase I/II Study Evaluating Safety and Efficacy of Autologous Hematopoietic Stem and Progenitor Cells Genetically Modified With IDUA Lentiviral Vector Encoding for the Human α-L-iduronidase Gene for the Treatment of Patients Affected by Mucopolysaccharidosis Type I, Hurler Variant
ClinicalTrials.gov ID: NCT03488394
Acronym / sponsor ID: TigetT10_MPSIH / 2024-514870-29-00
Conditions: Mucopolysaccharidosis IH
Keywords: Mucopolysaccharidosis IH, Gene Therapy, Transplantation, Autologous, Lentiviral vector
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Active, not recruiting
Enrolment: 8 participants (actual)
Lead sponsor: Orchard TherapeuticsIndustry (pharma, biotech, devices)
Collaborators: Fondazione Telethon
Study start: 11 May 2018 (actual)
Primary completion: Jan 2035 (estimated)
Study completion: Mar 2035 (estimated)
First posted: 5 Apr 2018 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Treatment	Experimental	Genetic: Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LVV, encoding for the α-L-iduronidase cDNA, in their final formulation medium.Gene therapy (autologous, CD34+ cell enriched cells fraction containing HSPCs, transduced with the IDUA LVV encoding for the human IDUA gene and cryopreserved in cryoformulation medium)

Who may qualify

Ages: 28 days to 11 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (8 listed)

Written informed consent by parent/legal guardian
Sex: Males and Females
Age: ≥ 28 days and ≤ 11 years old
Biochemically and molecularly proven MPS IH
Lansky index >80%

Key exclusion criteria (10 listed)

Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long-acting agents)
Severe, active viral, bacterial or fungal infection at eligibility evaluation
Patients affected by neoplasia or family history of familial cancer syndromes
Cytogenetic alterations associated with high risk of developing hematological malignancies
History of uncontrolled seizures

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LVV, encoding for the α-L-iduronidase cDNA, in their final formulation medium.The drug product target dose is more or equal to 8x10^6 CD34+ cells/Kg, with a minimum dose of 4x10^6 CD34+ cells/Kg and a maximum dose of 35x10^6 CD34+ cells/Kg. The product will be injected intravenously.	Genetic	Treatment

Primary outcome

Overall survival
Time frame: Assessed at multiple timepoints up to 15 years post-treatment
Number and percentage of subjects alive at the end of the trial
Achievement of haematological engraftment
Time frame: within day +45 after gene therapy
Percentage of subjects with both neutrophil count more than 500/mm3 and platelets more than 20,000/mm3 (in the absence of platelet transfusion for seven consecutive days) on 3 consecutive blood counts in the first 45 days from ATIMP injection.
Safety of the administration of autologous haematopoietic stem cells transduced with IDUA LVV - Short term tolerability
Time frame: 0-24 hours from ATIMP injection
Percentage of subjects not experiencing short-term adverse events of any grade and systemic reactions
Safety of the administration of autologous haematopoietic stem cells transduced with IDUA LVV - Absence of Replication Competent Lentivirus
Time frame: Assessed at multiple timepoints up to 8 years post-treatment, or if clinically indicated
Percentage of subjects without Replication Competent Lentivirus
Safety of the administration of autologous haematopoietic stem cells transduced with IDUA LVV - Absence of malignancy or abnormal clonal proliferation
Time frame: Assessed at multiple timepoints up to 15 years post-treatment
Percentage of subjects without abnormal clonal proliferation
Overall safety and tolerability (AE)
Time frame: Assessed at multiple timepoints up to 15 years post-treatment
The number of AEs (expected/unexpected and/or related/not related) and SAEs (expected/unexpected and/or related/not related) and the percentage of subjects experiencing AEs (expected/unexpected and/or related/not related) and SAEs (expected/unexpected and/or related/not related) will be summarized by severity and within body system involved. Narratives will also be presented. The rate of occurrence of these events will also be estimated.
IDUA activity in blood (up to supraphysiologic levels) at 1-year post-treatment
Time frame: At 1 year post-treatment
IDUA activity measured on peripheral dried blood spot

7 secondary outcomes

Anti-IDUA antibody immune response
Time frame: Assessed at multiple timepoints up to 8 years post-treatment, or if clinically indicated
Achievement of supraphysiologic IDUA activity in blood
Time frame: Assessed at multiple timepoints up to 15 years post-treatment
IDUA activity in plasma
Time frame: Assessed at multiple timepoints up to 15 years post-treatment
Engraftment of transduced cells ≥ 0.30 VCN/genome
Time frame: Assessed at multiple timepoints up to 15 years post-treatment
Normalization of urinary GAGs
Time frame: Assessed at multiple timepoints up to 15 years post-treatment
Normalization of spleen and liver
Time frame: Assessed at multiple timepoints up to 15 years post-treatment
Growth velocity
Time frame: Assessed at multiple timepoints up to 15 years post-treatment

Results and safety

This study is still under way. Results are not available in the source record yet.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2026.
Background: PubMed 38691622Consiglieri G, Tucci F, De Pellegrin M, Guerrini B, Cattoni A, Risca G, Scarparo S, Sarzana M, Pontesilli S, Mellone R, Gasperini S, Galimberti S, Silvani P, Filisetti C, Darin S, Forni G, Miglietta S, Santi L, Facchini M, Corti A, Fumagalli F, Cicalese MP, Calbi V, Migliavacca M, Barzaghi F, Ferrua F, Gallo V, Recupero S, Canarutto D, Doglio M, Tedesco L, Volpi N, Rovelli A, la Marca G, Valsecchi MG, Zancan S, Ciceri F, Naldini L, Baldoli C, Parini R, Gentner B, Aiuti A, Bernardo ME. Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome. Sci Transl Med. 2024 May;16(745):eadi8214. doi: 10.1126/scitranslmed.adi8214. Epub 2024 May 1.
Background: PubMed 34788506Gentner B, Tucci F, Galimberti S, Fumagalli F, De Pellegrin M, Silvani P, Camesasca C, Pontesilli S, Darin S, Ciotti F, Sarzana M, Consiglieri G, Filisetti C, Forni G, Passerini L, Tomasoni D, Cesana D, Calabria A, Spinozzi G, Cicalese MP, Calbi V, Migliavacca M, Barzaghi F, Ferrua F, Gallo V, Miglietta S, Zonari E, Cheruku PS, Forni C, Facchini M, Corti A, Gabaldo M, Zancan S, Gasperini S, Rovelli A, Boelens JJ, Jones SA, Wynn R, Baldoli C, Montini E, Gregori S, Ciceri F, Valsecchi MG, la Marca G, Parini R, Naldini L, Aiuti A, Bernardo ME; MPSI Study Group. Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome. N Engl J Med. 2021 Nov 18;385(21):1929-1940. doi: 10.1056/NEJMoa2106596.
Background: PubMed 41017152Tucci F, Uria Oficialdegui ML, Consiglieri G, Cossutta M, Filisetti C, Fumagalli F, Butera C, Santangelo R, Colombo M, Manitto MP, Stoppani M, Martina E, Dane G, Camesasca C, Risca G, De Pellegrin M, Scarparo S, Sarzana M, Puricelli C, Galimberti S, Darin S, Silvani P, Bonanomi S, Gasperini S, Naldini L, Gentner B, Parini R, Del Toro M, Diaz-de-Heredia C, Aiuti A, Bernardo ME. Non-neurological, non-skeletal outcomes after hematopoietic stem and progenitor cell-gene therapy (OTL-203) for Hurler syndrome. Mol Ther. 2026 Jan 7;34(1):443-454. doi: 10.1016/j.ymthe.2025.09.042. Epub 2025 Sep 27.
Individual participant dataSharing planned: Undecided

Related trials for Mucopolysaccharidosis IH

Active, not recruitingObservational study
Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I
Active, not recruitingPhase 2
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Mucopolysaccharidosis Disorders, Hurler Syndrome
RecruitingPhase 1
ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
Enrolling by invitationObservational study
Long Term Follow-Up for RGX-111
Mucopolysaccharidosis I
RecruitingObservational study
Registry of Patients Diagnosed With Lysosomal Storage Diseases
Mucopolysaccharidosis I, Mucopolysaccharidosis II

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 16 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall survival (time frame: Assessed at multiple timepoints up to 15 years post-treatment). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03488394, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2026.