Severe Haemophilia APhase 1/2NCT02576795

Gene Therapy Study in Severe Haemophilia A Patients (270-201)

A Phase 1/2 study testing valoctocogene roxaparvovec in people with Severe Haemophilia A, run by BioMarin Pharmaceutical at 5 sites in United Kingdom.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, dose escalation study in order to determine the safety and efficacy of valoctocogene roxaparvovec (an Adenovirus-Associated Virus based gene therapy vector in participants with severe haemophilia A. Read more about this study

Who is it for?: Ages 18 years and older, men only
Conditions the study is for: Severe Haemophilia A
What is being tested?: valoctocogene roxaparvovec
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 15 participants
Where: 5 sitesUnited Kingdom
Study dates: 28 Sep 2015to 14 Feb 2024
Phase: Phase 1/2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 18 years and older
Men only
No healthy volunteers
Adult, Older adult

Who may be able to join

Males 18 years or older with established severe Haemophilia A (endogenous FVIII level ≤1 IU/dL) as evidenced by their medical history.
Treated/exposed to FVIII concentrates or cryoprecipitate for a minimum of 150 exposure days (EDs)
Greater than or equal to 12 bleeding episodes for patients on on-demand FVIII replacement therapy over the previous 12 months. Does not apply to patients on prophylaxis
No history of inhibitor, and results from a modified Nijmegen Bethesda assay of less than 0.6 Bethesda Units (BU) 2 consecutive occasions at least one week apart within the past 12 months
Sexually active patients must be willing to use an acceptable method of contraception.

Who cannot join

Detectable pre-existing immunity to the AAV5 capsid as measured by adeno-associated virus 5 (AAV5) transduction inhibition (TI) or AAV5 total antibodies
Any evidence of immunosuppressive disorder or active chronic infection including hepatis B, hepatitis C, HIV
Significant liver dysfunction as defined by abnormal elevation ofliver function tests, or for patients who have undergone liver imaging or biopsy and found to have evidence of grade 3 or higher fibrosis
Evidence of any bleeding disorder not related to haemophilia A
12. Treatment with any investigational product within 30 days prior to the end of the screening period, or any previous exposure to any gene transfer therapy
Any disease or condition that per the physician's discretion would prevent the patient from fully complying with the requirements of the study including possible corticosteroid treatment outlined in the protocol. The physician may exclude patients unwilling or unable to agree on not using alcohol for the 16-week period following the viral infusion.

Trial locations and contacts

5 sites in 1 country.

City, postcode or hospital

5 sites in 1 country

United Kingdom5 sites

Queen Elizabeth Hospital Birmingham
Birmingham
Addenbrooke's Hospital
Cambridge
St. Thomas' Hospital
London
The Royal London Hospital
London
University Hospital Southampton NHS Foundation Trust
Southampton

Other trials in United Kingdom

About this study

Official title: A Phase 1/2, Dose-Escalation, Safety, Tolerability and Efficacy Study of Valoctocogene Roxaparvovec, an Adenovirus-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Patients With Severe Haemophilia A

Conditions: Severe Haemophilia A
Being tested: valoctocogene roxaparvovec
Keywords: Haemophilia A, Gene Therapy, Clotting Disorders, Blood Disorder, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorders, Hematologic Diseases, Coagulation Protein Disorders, Hemorrhagic Disorders, Genetic Diseases, Inborn, Factor VIII, Coagulants, AAV5 vector
Medical subject headings: Hemophilia A, Hemostatic Disorders, Hematologic Diseases, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorders, Coagulation Protein Disorders, Hemorrhagic Disorders, Genetic Diseases, Inborn

What participants receive

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

valoctocogene roxaparvovecBiological
Also called BMN 270
Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Severe Hemophilia A

Study groups in NCT02576795
Study group	Role	What they receive
valoctocogene roxaparvovec	Experimental	Single administration of valoctocogene roxaparvovec at escalating doses. Receives: Biological: valoctocogene roxaparvovec

What is being measured

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

Number of Participants With Treatment Emergent Adverse Events
Adverse events (AEs) with onset or worsening after the investigational product were included. Participants with more than one AE of the same category were counted only once for that category.
Serious adverse event (SAE)
Measured: Approximately up to 7 years after dosing
Number of Participant With Median FVIII Activity Levels >= 5 IU/dL Using Chromogenic Substrate Assay (CSA)
Responder/Non responder status, where a responder was defined as a participant with median FVIII activity of >= 5 IU/dL during Week 13-16 post-BMN 270 infusion
Measured: Week 13-16 post-BMN 270 infusion
Median FVIII Activity as Measured by Chromogenic Substrate Assay During Week 13-16 Post-BMN 270 Infusion
Values for FVIII activity were excluded from analysis if obtained within 72 hours since the last infusion of exogenous FVIII replacement therapy
FVIII activity levels below the Lower limit of quantitation (LLOQ) will be imputed with 0 IU/dL
Q1: 25% Percentile; Q3: 75% Percentile
Measured: Week 13-16 post-BMN 270 infusion

Secondary outcomes (3)

Annualized Bleeding Rate Requiring Exogenous Factor VIII Replacement Treatment During Week 5 and Beyond
ABR= [Number of bleeding episodes during calculation period] / [Total number of days during the calculation period] ×365.25
A bleeding episode (treated) was defined as a bleed or symptoms associated with the development of a bleed (or multiple bleeds occurring in the same day) requiring FVIII replacement treatment within 72 hours of the start of the bleed.
The baseline values for the secondary efficacy endpoints were based on the historical data prior to study enrollment.
Annualized bleeding rate (ABR)
Measured: Week 5 and Beyond (Approximately 7 years post Infusion)
Annualized Factor VIII Utilization During Week 5 and Beyond
Annualized FVIII use (IU/kg/yr.) =[Sum of FVIII use (IU/kg) during calculation period] / [Total number of days during the calculation period] ×365.25
Measured: Week 5 and Beyond (Approximately 7 years post Infusion)
Annualized Factor VIII Infusion Rate During Week 5 and Beyond
Annualized FVIII infusion rate (count/yr.) = [Number of FVIII replacement infusions during calculation period] / [Total number of days during the calculation period] ×365.25
Measured: Week 5 and Beyond (Approximately 7 years post Infusion)

Timeline

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

28 Sep 201514 Feb 2024

First posted15 Oct 2015 (estimated)
Study start28 Sep 2015
Primary completion14 Feb 2024
Study completion14 Feb 2024

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (8)Papers the sponsor has linked to this study.
Other trials for Severe Haemophilia AStudies of the same condition, open and under way.
More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
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: 15 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02576795
Sponsor's protocol ID: BMN 270-201
Other IDs: 2014-003880-38, 2014
Status last verified: Mar 2025
First submitted: 5 Oct 2015
Last updated: 10 Apr 2025
Results posted: Yes, on ClinicalTrials.gov
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

Santos S, Robinson TM, Trueman D. Estimated Long-Term Durability of Valoctocogene Roxaparvovec Treatment in Male patients with Severe Hemophilia A: An Extrapolation of Clinical Data. Adv Ther. 2025 Nov;42(11):5781-5793. doi: 10.1007/s12325-025-03368-4. Epub 2025 Sep 23.
PubMed 40986187
Symington E, Rangarajan S, Lester W, Madan B, Pierce GF, Raheja P, Millar C, Osmond D, Li M, Robinson TM. Valoctocogene roxaparvovec gene therapy provides durable haemostatic control for up to 7 years for haemophilia A. Haemophilia. 2024 Sep;30(5):1138-1147. doi: 10.1111/hae.15071. Epub 2024 Jul 8.
PubMed 38975624
Quinn J, Delaney KA, Wong WY, Miesbach W, Bullinger M. Psychometric Validation of the Haemo-QOL-A in Participants with Hemophilia A Treated with Gene Therapy. Patient Relat Outcome Meas. 2022 Jul 18;13:169-180. doi: 10.2147/PROM.S357555. eCollection 2022.
PubMed 35879931
Fong S, Yates B, Sihn CR, Mattis AN, Mitchell N, Liu S, Russell CB, Kim B, Lawal A, Rangarajan S, Lester W, Bunting S, Pierce GF, Pasi KJ, Wong WY. Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A. Nat Med. 2022 Apr;28(4):789-797. doi: 10.1038/s41591-022-01751-0. Epub 2022 Apr 11.
PubMed 35411075
Pasi KJ, Laffan M, Rangarajan S, Robinson TM, Mitchell N, Lester W, Symington E, Madan B, Yang X, Kim B, Pierce GF, Wong WY. Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A. Haemophilia. 2021 Nov;27(6):947-956. doi: 10.1111/hae.14391. Epub 2021 Aug 11.
PubMed 34378280
Rosen S, Tiefenbacher S, Robinson M, Huang M, Srimani J, Mackenzie D, Christianson T, Pasi KJ, Rangarajan S, Symington E, Giermasz A, Pierce GF, Kim B, Zoog SJ, Vettermann C. Activity of transgene-produced B-domain-deleted factor VIII in human plasma following AAV5 gene therapy. Blood. 2020 Nov 26;136(22):2524-2534. doi: 10.1182/blood.2020005683.
PubMed 32915950
Pasi KJ, Rangarajan S, Mitchell N, Lester W, Symington E, Madan B, Laffan M, Russell CB, Li M, Pierce GF, Wong WY. Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A. N Engl J Med. 2020 Jan 2;382(1):29-40. doi: 10.1056/NEJMoa1908490.
PubMed 31893514
Rangarajan S, Walsh L, Lester W, Perry D, Madan B, Laffan M, Yu H, Vettermann C, Pierce GF, Wong WY, Pasi KJ. AAV5-Factor VIII Gene Transfer in Severe Hemophilia A. N Engl J Med. 2017 Dec 28;377(26):2519-2530. doi: 10.1056/NEJMoa1708483. Epub 2017 Dec 9.
PubMed 29224506

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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Active, not recruitingPhase 4
A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis
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All Severe Haemophilia A trials

Study at a glance

From the registry record, last updated by the sponsor on 10 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 1/2, Dose-Escalation, Safety, Tolerability and Efficacy Study of Valoctocogene Roxaparvovec, an Adenovirus-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Patients With Severe Haemophilia A
ClinicalTrials.gov ID: NCT02576795
Acronym / sponsor ID: BMN 270-201
Conditions: Severe Haemophilia A
Keywords: Haemophilia A, Gene Therapy, Clotting Disorders, Blood Disorder, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorders, Hematologic Diseases, Coagulation Protein Disorders
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 15 participants (actual)
Lead sponsor: BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
Study start: 28 Sep 2015 (actual)
Primary completion: 14 Feb 2024 (actual)
Study completion: 14 Feb 2024 (actual)
First posted: 15 Oct 2015 (estimated)
Results: Posted 10 Apr 2025

Clinical summary

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

Study design

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

Arm	Type	Receives
valoctocogene roxaparvovec	Experimental	Biological: valoctocogene roxaparvovecSingle administration of valoctocogene roxaparvovec at escalating doses.

Who may qualify

Ages: 18 years and older
Sex: Male only
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (5 listed)

Males 18 years or older with established severe Haemophilia A (endogenous FVIII level ≤1 IU/dL) as evidenced by their medical history.
Treated/exposed to FVIII concentrates or cryoprecipitate for a minimum of 150 exposure days (EDs)
Greater than or equal to 12 bleeding episodes for patients on on-demand FVIII replacement therapy over the previous 12 months. Does not apply to patients on prophylaxis
No history of inhibitor, and results from a modified Nijmegen Bethesda assay of less than 0.6 Bethesda Units (BU) 2 consecutive occasions at least one week apart within the past 12 months
Sexually active patients must be willing to use an acceptable method of contraception.

Key exclusion criteria (6 listed)

Detectable pre-existing immunity to the AAV5 capsid as measured by adeno-associated virus 5 (AAV5) transduction inhibition (TI) or AAV5 total antibodies
Any evidence of immunosuppressive disorder or active chronic infection including hepatis B, hepatitis C, HIV
Significant liver dysfunction as defined by abnormal elevation ofliver function tests, or for patients who have undergone liver imaging or biopsy and found to have evidence of grade 3 or higher fibrosis
Evidence of any bleeding disorder not related to haemophilia A
12. Treatment with any investigational product within 30 days prior to the end of the screening period, or any previous exposure to any gene transfer therapy

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
valoctocogene roxaparvovecAlso: BMN 270Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Severe Hemophilia A	Biological	valoctocogene roxaparvovec

Primary outcome

Number of Participants With Treatment Emergent Adverse Events
Time frame: Approximately up to 7 years after dosing
Adverse events (AEs) with onset or worsening after the investigational product were included. Participants with more than one AE of the same category were counted only once for that category. Serious adverse event (SAE)
Number of Participant With Median FVIII Activity Levels >= 5 IU/dL Using Chromogenic Substrate Assay (CSA)
Time frame: Week 13-16 post-BMN 270 infusion
Responder/Non responder status, where a responder was defined as a participant with median FVIII activity of >= 5 IU/dL during Week 13-16 post-BMN 270 infusion
Median FVIII Activity as Measured by Chromogenic Substrate Assay During Week 13-16 Post-BMN 270 Infusion
Time frame: Week 13-16 post-BMN 270 infusion
Values for FVIII activity were excluded from analysis if obtained within 72 hours since the last infusion of exogenous FVIII replacement therapy FVIII activity levels below the Lower limit of quantitation (LLOQ) will be imputed with 0 IU/dL Q1: 25% Percentile; Q3: 75% Percentile

3 secondary outcomes

Annualized Bleeding Rate Requiring Exogenous Factor VIII Replacement Treatment During Week 5 and Beyond
Time frame: Week 5 and Beyond (Approximately 7 years post Infusion)
Annualized Factor VIII Utilization During Week 5 and Beyond
Time frame: Week 5 and Beyond (Approximately 7 years post Infusion)
Annualized Factor VIII Infusion Rate During Week 5 and Beyond
Time frame: Week 5 and Beyond (Approximately 7 years post Infusion)

Results and safety

Results posted to the registry on 10 Apr 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
BMN 270 6E12 vg/kg	1	1
BMN 270 2E13 vg/kg	1	1
BMN 270 4E13 vg/kg	6	5
BMN 270 6E13 vg/kg	7	7

Primary outcome: Number of Participants With Treatment Emergent Adverse Events

Time frame: Approximately up to 7 years after dosing. Count of participants. Unit: Participants

Measure	BMN 270 6E12 vg/kg n = 1	BMN 270 2E13 vg/kg n = 1	BMN 270 4E13 vg/kg n = 6	BMN 270 6E13 vg/kg n = 7
Participants with any AE	1	1	6	7
Participants with any SAE	1	0	3	5
Participants with any treatment-related AE	1	0	6	7
Treatment-related SAEs	0	0	1	0
Participants with any AE of Grade >= 3	1	1	1	2
AEs leading to dose adjustment during infusion	0	0	0	0
AEs leading to dose interruption during infusion	0	0	0	0
AEs leading to study drug discontinuation	0	0	0	0

Population analysed: Safety analysis Population: The Safety analysis population was defined as all enrolled participants who received any amount of study drug.

Primary outcome: Number of Participant With Median FVIII Activity Levels >= 5 IU/dL Using Chromogenic Substrate Assay (CSA)

Time frame: Week 13-16 post-BMN 270 infusion. Count of participants. Unit: Participants

Measure	BMN 270 6E12 vg/kg n = 1	BMN 270 2E13 vg/kg n = 1	BMN 270 4E13 vg/kg n = 6	BMN 270 6E13 vg/kg n = 7
Value	0	0	5	7

Population analysed: Full Analysis Set (FAS) population: defined as all enrolled participants who receive study drug and have at least one Baseline and post-Baseline assessment.

Primary outcome: Median FVIII Activity as Measured by Chromogenic Substrate Assay During Week 13-16 Post-BMN 270 Infusion

Time frame: Week 13-16 post-BMN 270 infusion. Median (Inter-Quartile Range). Unit: IU/dL

Measure	BMN 270 6E12 vg/kg n = 1	BMN 270 2E13 vg/kg n = 1	BMN 270 4E13 vg/kg n = 6	BMN 270 6E13 vg/kg n = 7
Value	0.00 (0.00 to 0.00)	0.00 (0.00 to 0.00)	16.30 (11.05 to 19.90)	50.40 (31.30 to 64.95)

Population analysed: FAS Population

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: Approximately up to 7 years after dosing. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
BMN 270 6E12 vg/kg	1 of 1 (100%)	1 of 1 (100%)	0 of 1
BMN 270 2E13 vg/kg	0 of 1 (0%)	1 of 1 (100%)	0 of 1
BMN 270 4E13 vg/kg	3 of 6 (50%)	6 of 6 (100%)	0 of 6
BMN 270 6E13 vg/kg	5 of 7 (71.4%)	7 of 7 (100%)	0 of 7
Overall	9 of 15 (60%)	15 of 15 (100%)	0 of 15

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 10 Apr 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 40986187Santos S, Robinson TM, Trueman D. Estimated Long-Term Durability of Valoctocogene Roxaparvovec Treatment in Male patients with Severe Hemophilia A: An Extrapolation of Clinical Data. Adv Ther. 2025 Nov;42(11):5781-5793. doi: 10.1007/s12325-025-03368-4. Epub 2025 Sep 23.
Derived from this study: PubMed 38975624Symington E, Rangarajan S, Lester W, Madan B, Pierce GF, Raheja P, Millar C, Osmond D, Li M, Robinson TM. Valoctocogene roxaparvovec gene therapy provides durable haemostatic control for up to 7 years for haemophilia A. Haemophilia. 2024 Sep;30(5):1138-1147. doi: 10.1111/hae.15071. Epub 2024 Jul 8.
Derived from this study: PubMed 35879931Quinn J, Delaney KA, Wong WY, Miesbach W, Bullinger M. Psychometric Validation of the Haemo-QOL-A in Participants with Hemophilia A Treated with Gene Therapy. Patient Relat Outcome Meas. 2022 Jul 18;13:169-180. doi: 10.2147/PROM.S357555. eCollection 2022.
Derived from this study: PubMed 35411075Fong S, Yates B, Sihn CR, Mattis AN, Mitchell N, Liu S, Russell CB, Kim B, Lawal A, Rangarajan S, Lester W, Bunting S, Pierce GF, Pasi KJ, Wong WY. Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A. Nat Med. 2022 Apr;28(4):789-797. doi: 10.1038/s41591-022-01751-0. Epub 2022 Apr 11.
Derived from this study: PubMed 34378280Pasi KJ, Laffan M, Rangarajan S, Robinson TM, Mitchell N, Lester W, Symington E, Madan B, Yang X, Kim B, Pierce GF, Wong WY. Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A. Haemophilia. 2021 Nov;27(6):947-956. doi: 10.1111/hae.14391. Epub 2021 Aug 11.
Derived from this study: PubMed 32915950Rosen S, Tiefenbacher S, Robinson M, Huang M, Srimani J, Mackenzie D, Christianson T, Pasi KJ, Rangarajan S, Symington E, Giermasz A, Pierce GF, Kim B, Zoog SJ, Vettermann C. Activity of transgene-produced B-domain-deleted factor VIII in human plasma following AAV5 gene therapy. Blood. 2020 Nov 26;136(22):2524-2534. doi: 10.1182/blood.2020005683.
Derived from this study: PubMed 31893514Pasi KJ, Rangarajan S, Mitchell N, Lester W, Symington E, Madan B, Laffan M, Russell CB, Li M, Pierce GF, Wong WY. Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A. N Engl J Med. 2020 Jan 2;382(1):29-40. doi: 10.1056/NEJMoa1908490.
Derived from this study: PubMed 29224506Rangarajan S, Walsh L, Lester W, Perry D, Madan B, Laffan M, Yu H, Vettermann C, Pierce GF, Wong WY, Pasi KJ. AAV5-Factor VIII Gene Transfer in Severe Hemophilia A. N Engl J Med. 2017 Dec 28;377(26):2519-2530. doi: 10.1056/NEJMoa1708483. Epub 2017 Dec 9.

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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.

Medical Director, MD study director

BioMarin Pharmaceutical

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

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing valoctocogene roxaparvovec in people with Severe Haemophilia A, run by BioMarin Pharmaceutical at 5 sites in United Kingdom.

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 5 inclusion and 6 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: finished, as of the sponsor's last update on 10 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in United Kingdom. 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?

Yes. Results are posted in the registry (first posted 10 Apr 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Number of Participants With Treatment Emergent Adverse Events (time frame: Approximately up to 7 years after dosing). The study lists 3 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 NCT02576795, which also lists 8 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 10 Apr 2025.