NCT02396342 is a Phase 1, PHASE2 clinical trial of AAV5-hFIX in Hemophilia B, sponsored by CSL Behring.

Who is sponsoring NCT02396342?

NCT02396342 is sponsored by CSL Behring.

What drugs are being tested in NCT02396342?

Interventions in NCT02396342: AAV5-hFIX.

What condition does NCT02396342 study?

NCT02396342 studies Hemophilia B.

Is NCT02396342 still recruiting?

NCT02396342 is currently completed. Last updated 27 June 2022.

Are results published for NCT02396342?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2022-06-27 · How we verify

NCT02396342

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B

Completed Phase 1, PHASE2 Results posted Last updated 27 June 2022

What this trial tests

Phase 1, PHASE2 trial testing AAV5-hFIX in Hemophilia B in 10 participants. Completed in 15 April 2021.

Timeline

10 June 2015

Primary endpoint
15 April 2021

15 April 2021

Quick facts

Lead sponsor	CSL Behring
Phase	Phase 1, PHASE2
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	10
Start date	10 June 2015
Primary completion	15 April 2021
Estimated completion	15 April 2021
Sites	7 locations across Denmark, Netherlands, Germany

Drugs / interventions tested

AAV5-hFIX

Conditions studied

Hemophilia B — all drugs for Hemophilia B →

Sponsor

CSL Behring — full company profile →

Who can join

18 and older, male only, with Hemophilia B. 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.

Number of Participants With Adverse Events Primary · From AMT-060 infusion through end of study (5 years post-dose)

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	5
AAV5-hFIX High Dose (Cohort 2)	5

FIX-replacement-therapy-free FIX Activity Secondary · From AMT-060 infusion through end of study (5 years post-dose)

FIX activity measured any time from 72 hours after latest FIX replacement therapy administration and until next administration of FIX replacement therapy. Only assessments performed more than 10 days after most recent FIX-replacement therapy administration included.

one-stage aPTT assay

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	7.43	± 1.28
AAV5-hFIX High Dose (Cohort 2)	6.60	± 1.96

amidolytic/chromogenic assay

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	4.58	± 2.88
AAV5-hFIX High Dose (Cohort 2)	4.74	± 1.43

Total Annualized Bleeding Rate (ABR) Secondary · From AMT-060 infusion through end of study (5 years post-dose)

Annualized: Sum of post-treatment bleeding episodes divided by subjects' average number of years (365.25 days) from treatment start to until the data cutoff date.

One Year Prior to Screening

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	14.40	± 5.73
AAV5-hFIX High Dose (Cohort 2)	4.00	± 3.16

Post-tapering Period

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	5.39	± 5.94
AAV5-hFIX High Dose (Cohort 2)	0.71	± 0.58

Total Consumption of FIX Replacement Therapy Secondary · From AMT-060 infusion through end of study (5 years post dose).

One year prior to screening

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	326532	± 234900
AAV5-hFIX High Dose (Cohort 2)	233778	± 156873

Post-tapering period

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	252950	± 222790
AAV5-hFIX High Dose (Cohort 2)	85800	± 84482

Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores Secondary · From AMT-060 infusion through the end of study (5 years post dose)

Scores range from 0 to 100, with a higher score defining a more favorable health state.

Physical Functioning

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	0.00	± 10.00
AAV5-hFIX High Dose (Cohort 2)	-7.00	± 9.75

Role-Physical

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	-15.00	± 8.39
AAV5-hFIX High Dose (Cohort 2)	-10.00	± 22.79

Bodily Pain

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	-9.00	± 9.00
AAV5-hFIX High Dose (Cohort 2)	1.20	± 14.81

General Health

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	-0.80	± 20.22
AAV5-hFIX High Dose (Cohort 2)	-2.40	± 8.99

Vitality

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	-11.25	± 19.96
AAV5-hFIX High Dose (Cohort 2)	-6.25	± 12.50

Social Functioning

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	-20.00	± 25.92
AAV5-hFIX High Dose (Cohort 2)	-5.00	± 14.25

Role-Emotional

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	-13.33	± 27.39
AAV5-hFIX High Dose (Cohort 2)	-10.00	± 13.69

Mental Health

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	-13.00	± 22.80
AAV5-hFIX High Dose (Cohort 2)	-9.00	± 12.94

Time to Vector DNA Stopped Shedding From Blood, Nasal Secretions, Saliva, Urine, Feces, and Semen Secondary · From AMT-060 infusion through end of study (5 years post dose).

Blood

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	508.8	± 261.7
AAV5-hFIX High Dose (Cohort 2)	705.4	± 245.1

Nasal secretions

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	83.4	± 41.7
AAV5-hFIX High Dose (Cohort 2)	108.4	± 66.0

Saliva

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	75.8	± 38.4
AAV5-hFIX High Dose (Cohort 2)	129.2	± 48.9

Urine

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	46.4	± 20.9
AAV5-hFIX High Dose (Cohort 2)	82.0	± 41.1

Feces

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	74.0	± 25.7
AAV5-hFIX High Dose (Cohort 2)	165.0	± 68.9

Semen

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	227.8	± 147.7
AAV5-hFIX High Dose (Cohort 2)	157.2	± 78.4

Number of Subjects Developing Neutralizing Antibodies to AAV5 Secondary · From AMT-060 infusion through end of study (5 years post dose)

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	5
AAV5-hFIX High Dose (Cohort 2)	5

Total IgG and IgM Antibody Titers to AAV5 Secondary · AMT-060 infusion through end of study (5 years post dose)

For subjects with a titer of 109350 and 50, the actual titer is \>109350 and \<50.

IgG (subject 1)

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	79499
AAV5-hFIX High Dose (Cohort 2)	109350

IgG (subject 2)

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	109350
AAV5-hFIX High Dose (Cohort 2)	109350

IgG (subject 3)

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	109350
AAV5-hFIX High Dose (Cohort 2)	109350

IgG (subject 4)

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	109350
AAV5-hFIX High Dose (Cohort 2)	107344

IgG (subject 5)

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	109350
AAV5-hFIX High Dose (Cohort 2)	109350

IgM (subject 1)

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	56
AAV5-hFIX High Dose (Cohort 2)	30071

IgM (subject 2)

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	1321
AAV5-hFIX High Dose (Cohort 2)	20000

IgM (subject 3)

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	557
AAV5-hFIX High Dose (Cohort 2)	6649

Number of Subjects With a Positive AAV5 Capsid-specific T Cell Response Secondary · From AMT-060 infusion through 26 weeks post-dose

Specific AAV5 response (results \>17 SFC/million PBMCs) were regarded as positive.

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	1
AAV5-hFIX High Dose (Cohort 2)	0

Number of Subjects With Antibodies to FIX Secondary · From AMT-060 infusion through the end of study (5 years post dose)

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	1
AAV5-hFIX High Dose (Cohort 2)	0

Number of Subjects With FIX Inhibitors Secondary · From AMT-060 infusion through the end of study (5 years post dose)

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	0
AAV5-hFIX High Dose (Cohort 2)	0

Number of Subjects With Clinically Significant Inflammatory Markers: IL-1β, IL-2, IL-6, INFγ, MCP-1 Secondary · From AMT-060 infusion through 18 weeks post dose

Group	Value	95% CI
AAV5-hFIX Low Dose (Cohort 1)	0
AAV5-hFIX High Dose (Cohort 2)	0

Adverse events — posted to ClinicalTrials.gov

Time frame: 5 years post-dose. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

AAV5-hFIX Low Dose (Cohort 1)

Serious: 3/5 (60%)

Deaths: 0/5

AAV5-hFIX High Dose (Cohort 2)

Serious: 2/5 (40%)

Deaths: 0/5

Serious adverse events (6 terms)

Reaction	System	AAV5-hFIX Low Dose (Cohort…	AAV5-hFIX High Dose (Cohor…
Hepatic Enzyme Increased	Investigations	—	—
Alanine Aminotransferase Increased	Investigations	—	—
Myelopathy	Nervous system disorders	—	—
Pyrexia	General disorders	—	—
Renal Colic	Renal and urinary disorders	—	—
Calculus Ureteric	Renal and urinary disorders	—	—

Other adverse events (88 terms — click to expand)

Reaction	System	AAV5-hFIX Low Dose (Cohort…	AAV5-hFIX High Dose (Cohor…
Influenza like illness	General disorders	—	—
Arthralgia	Musculoskeletal and connective tissue disorders	—	—
Nasopharyngitis	Infections and infestations	—	—
Pyrexia	General disorders	—	—
Dizziness	Nervous system disorders	—	—
Joint swelling	Musculoskeletal and connective tissue disorders	—	—
Musculoskeletal pain	Musculoskeletal and connective tissue disorders	—	—
Influenza	Infections and infestations	—	—
Haematoma	Vascular disorders	—	—
Lymphoedema	Vascular disorders	—	—
Hypersensitivity	Immune system disorders	—	—
Fatigue	General disorders	—	—
Peripheral swelling	General disorders	—	—
Malaise	General disorders	—	—
Pain	General disorders	—	—
Drug ineffective	General disorders	—	—
Sleep disorder	Psychiatric disorders	—	—
Anxiety	Psychiatric disorders	—	—
Prostatitis	Reproductive system and breast disorders	—	—
Fall	Injury, poisoning and procedural complications	—	—
Upper limb fracture	Injury, poisoning and procedural complications	—	—
Ulna fracture	Injury, poisoning and procedural complications	—	—
Tooth fracture	Injury, poisoning and procedural complications	—	—
Joint injury	Injury, poisoning and procedural complications	—	—
Hand fracture	Injury, poisoning and procedural complications	—	—
Bone contusion	Injury, poisoning and procedural complications	—	—
Injury	Injury, poisoning and procedural complications	—	—
Muscle strain	Injury, poisoning and procedural complications	—	—
Arthropod bite	Injury, poisoning and procedural complications	—	—
Hepatic enzyme increased	Investigations	—	—
Haemoglobin decreased	Investigations	—	—
Transaminases increased	Investigations	—	—
Platelet count decreased	Investigations	—	—
Blood urine present	Investigations	—	—
Tachycardia	Cardiac disorders	—	—
Palpitations	Cardiac disorders	—	—
Chronic obstructive pulmonary disease	Respiratory, thoracic and mediastinal disorders	—	—
Splenomegaly	Blood and lymphatic system disorders	—	—
Headache	Nervous system disorders	—	—
Nervous system disorder	Nervous system disorders	—	—

Most-reported serious reactions: Hepatic Enzyme Increased, Alanine Aminotransferase Increased, Myelopathy, Pyrexia, Renal Colic, Calculus Ureteric.

Data from ClinicalTrials.gov NCT02396342 adverse events section.

Sponsor's own description

This study evaluates how safe gene therapy treatment with AAV5-hFIX is in adult patients with severe or moderately severe hemophilia B and severe bleeding type.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Adeno-Associated Virus (AAV) as a Vector for Gene Therapy.
Naso MF, Tomkowicz B, Perry WL, Strohl WR. · · 2017 · cited 936× · PMID 28669112 · DOI 10.1007/s40259-017-0234-5
AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer.
Costa Verdera H, Kuranda K, Mingozzi F. · · 2020 · cited 493× · PMID 31972133 · DOI 10.1016/j.ymthe.2019.12.010
Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B.
Miesbach W, Meijer K, Coppens M, Kampmann P, et al · · 2018 · cited 239× · PMID 29246900 · DOI 10.1182/blood-2017-09-804419
Gene Therapy Leaves a Vicious Cycle.
Goswami R, Subramanian G, Silayeva L, Newkirk I, et al · · 2019 · cited 221× · PMID 31069169 · DOI 10.3389/fonc.2019.00297
Unraveling the Complex Story of Immune Responses to AAV Vectors Trial After Trial.
Vandamme C, Adjali O, Mingozzi F. · · 2017 · cited 185× · PMID 28835127 · DOI 10.1089/hum.2017.150
Viral vector-based gene therapies in the clinic.
Zhao Z, Anselmo AC, Mitragotri S. · · 2022 · cited 171× · PMID 35079633 · DOI 10.1002/btm2.10258
Update on clinical gene therapy for hemophilia.
Perrin GQ, Herzog RW, Markusic DM. · · 2019 · cited 128× · PMID 30559260 · DOI 10.1182/blood-2018-07-820720
Anti-AAV Antibodies in AAV Gene Therapy: Current Challenges and Possible Solutions.
Weber T. · · 2021 · cited 126× · PMID 33815421 · DOI 10.3389/fimmu.2021.658399

Verify or expand the search:

Other recruiting trials for Hemophilia B

Currently open trials in the same condition.

NCT07080905 — Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or · Phase 3 · recruiting
NCT06727669 — Longitudinal Cohort of Thrombosis and Hemostasis Diseases · recruiting
NCT06565481 — Measurement Properties in People with Hemophilia · NA · recruiting
NCT06379789 — A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) · Phase 1, PHASE2 · recruiting
NCT06399289 — Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Pre · Phase 3 · active not recruiting

Other CSL Behring trials

Trials by the same sponsor.

NCT07326592 — Phase 4, Double-blind Study Evaluating the Response on Computed Tomography (CT) Lung Density Decline Rates of Respreeza · Phase 4 · not yet recruiting
NCT07224360 — Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD) · Phase 2 · recruiting
NCT07332091 — Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary He · Phase 2 · recruiting
NCT07094087 — Efficacy and Safety of 4F-PCC (4-Factor Prothrombin Complex Concentrate) in Adult Patients Undergoing Complex Cardiovasc · Phase 3 · recruiting
NCT07048262 — Dose Range Finding, Efficacy, and Safety Study of Nebulized CSL787 in Adults With Non-cystic Fibrosis Bronchiectasis (NC · Phase 2 · recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT02396342 (US National Library of Medicine, public domain)
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 CSL Behring
Last refreshed: 27 June 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/NCT02396342.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing