A Phase 1/2 interventional study of rAAV9.CMV.hNAGLU in Mucopolysaccharidosis Type 3 B, sponsored by Abeona Therapeutics, Inc. Terminated at 4 sites in 4 countries. Per ClinicalTrials.gov, last updated 2022-05-05.
Sponsored by Abeona Therapeutics, Inc · Phase 1/2, Interventional, and Treatment
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Among 43 studies in Mucopolysaccharidosis III with a site in United States — 12 open to participants now, 15 under way, 4 of them phase 3. Mucopolysaccharidosis III · United States.
Among the 43 studies in Mucopolysaccharidosis III with a site in United States, this one is the 14th largest by enrollment of the 23 interventional ones that give a count, is one of 2 that started in 2017, is one of 10 in phase 1/2, and was the 21st registered.
58 studies on the registry are indexed under Mucopolysaccharidosis III; 16 are open to participants now.
Browse Mucopolysaccharidosis III studies →12 of the 43 studies in Mucopolysaccharidosis III with a site in United States are open now. Recruiting first, the newest first:
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center · Sites in United States
Ages 4+
Ozlem Goker-Alpan · Sites in United States
Ages 18–99
Phoenix Nest · Sites in United States, France
Ages 12 mo+
Denali Therapeutics Inc. · Sites in United States
Ages 0–18
Lysosomal and Rare Disorders Research and Treatment Center, Inc. · Sites in United States
Ages 5–99
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 8 Mucopolysaccharidosis III studies recruiting now, 5 set no upper age limit and 1 stops at 75 or younger; 7 take children under 18; none take healthy volunteers.
This study: all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
43 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 11 · Phase 2 18 · Phase 3 6 · Phase 4 1
First in 2006; this study’s year is marked. 2 started in 2017.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 11 is close to the median of 12 across 33 interventional studies indexed under Mucopolysaccharidosis III.
Median enrollment 17 across the 23 interventional studies, 30 across the 19 observational. Of the 25 that completed or were terminated, 10 (40%) have results posted.
This tab as a report to print or save as PDF → With an account.
30 of 43 have an industry lead sponsor. The 10 most frequent:
Abeona Therapeutics, Inc is the lead sponsor of 11 studies on the registry; 3 are open to participants now.
Abeona Therapeutics, Inc has 2 studies filed under Mucopolysaccharidosis III — fewer than ten, so there is no comparison to draw.
5 of the studies in Mucopolysaccharidosis III with a site in United States that are under way reach primary completion within a year, 2 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06333041Phase 3Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical CenterStudy of Cannabidiol in Sanfilippo Syndrome | 2027-04 |
| NCT04360265Phase 3Ultragenyx Pharmaceutical IncFollow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA | 2027-08 |
| NCT03655223RTI InternationalEarly Check: Expanded Screening in Newborns | 2026-11-30 |
| NCT06567769GC Biopharma CorpPhase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA) | 2027-06 |
| NCT05492799Allievex CorporationSafety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE | 2027-10 |
Add all 5 to your calendar (.ics) → With an account.
Among the 43 studies in Mucopolysaccharidosis III with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
Open-label, dose-escalation clinical trial of rAAV9.CMV.hNAGLU injected intravenously through a peripheral limb vein
Adeno-associated virus serotype 9 carrying the human NAGLU gene under the control of a CMV enhancer/promoter (rAAV9.CMV.hNAGLU) will be delivered one-time through a venous catheter inserted into a peripheral limb vein. A tapering course of prophylactic enteral prednisone or prednisolone will be administered for a period of at least two months.
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.
Confirmed diagnosis of MPSIIIB by both of the following two methods:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CTCAE grade
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
INR, PT and aPTT (clotting tests)
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Subjects will receive a single infusion: • Cohort 1 (Low Dose): 2 X 10E13 vg/kg (n=2 participants)
Biological: rAAV9.CMV.hNAGLU
Subjects will receive a single infusion: • Cohort 2 (Med Dose): 5 X 10E13 vg/kg (n=4-5 participants)
Biological: rAAV9.CMV.hNAGLU
Subjects will receive a single infusion: • Cohort 3 (High Dose): 1 X 10E14 vg/kg (n=4-8 participants)
Biological: rAAV9.CMV.hNAGLU
Adeno-associated virus serotype 9 carrying the human NAGLU gene under the control of a CMV enhancer/promoter (rAAV9.CMV.hNAGLU) will be delivered one-time through a venous catheter inserted into a peripheral limb vein.
Change from baseline in the Age Equivalent Developmental score (calculated by the Mullen Scales of Early Learning or the Kaufman Assessment Battery for Children Second Edition, based on developmental age) compared with Natural History Study data
Time frame: 24 months
Product safety as defined by the incidence, type and severity of treatment-related adverse events and serious adverse events
Time frame: 24 Months
Change from baseline of central spinal fluid heparan sulfate after treatment
Time frame: 24 months
Change from baseline of plasma or urine glycosaminoglycans or heparan sulfate after treatment
Time frame: 24 Months
Change from baseline in CSF or plasma NAGLU enzyme activity levels after treatment
Time frame: 24 Months
Change from baseline in liver and/or spleen volumes after treatment, as measured by magnetic resonance imaging
Time frame: 24 Months
Change from baseline in brain volumes after treatment, as measured by magnetic resonance imaging
Time frame: 24 Months
Change from baseline in the Cognitive Age Equivalent (Developmental Age) compared to Natural History Study, calculated using the Bayley Scales of Infant and Toddler Development or the Kaufman Assessment Battery for Children
Time frame: 24 Months
Change from baseline in the Adaptive Age Equivalent score after treatment compared to Natural History Study data, as assessed by parent report using the Vineland Adaptive Behavior Scale II Survey form
Time frame: 24 Months
Change from baseline Developmental Quotient after treatment compared to Natural History Study data assessed by the Mullen Scales of Early Learning or the Kaufman Assessment Battery for Children.
Time frame: 24 Months
Change from baseline in Pediatric Quality of Life Inventory (PedsQL™) Generic Core Scales total score
Time frame: 24 Months
Change from baseline in parent quality of life, using the Parenting Stress Index, 4th Edition (PSI-4) short form
Time frame: 24 Months
Determination of vector shedding analysis in plasma, saliva, urine and feces will provide preliminary data for the Environmental Risk Assessment
Time frame: 24 Months
Plan to share: No — There is no plan to share data
Ran from Oct 2017 to primary completion in Apr 2022, 53 months.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Apr 2022. You cannot join it, but the record below documents what was studied.
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Abeona Therapeutics, Inc