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Also known as Adjuvant Immunomodulatory (IM) Therapy
Adjuvant Immunomodulatory Therapy · 1 trial · 4 indications
| Arm | Type | Description |
|---|---|---|
| Cohort A | OTHER | Participants who have participated in a prior clinical trial involving the administration of UX111 (rebisufligene etisparvovec) and are able to comply with onsite scheduled visits and assessments. Select participants may receive adjuvant IM therapy. |
| Cohort B | OTHER | Participants who have participated in a prior clinical trial involving the administration of UX111 (rebisufligene etisparvovec) and who cannot participate in Cohort A. Participants will partake in a reduced number of assessments, performed either onsite or at home via a combination of telehealth and home healthcare visits. |
| Name | Type | Description |
|---|---|---|
| No Investigational Product | OTHER | No investigational product will be administered in this follow-up trial. |
| Adjuvant Immunomodulatory (IM) Therapy | DRUG | The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IM therapy. Not all participants may receive IM therapy. |
Inclusion Criteria: * Participants that have participated in a prior clinical trial in which they received UX111 * Parent(s)/legal guardian(s) of participant willing and able to complete the informed consent process and comply with study procedures and visit schedule Exclusion Criteria: * Planned...
Adjuvant Immunomodulatory Therapy is an investigational small molecule being studied for use in Mucopolysaccharidosis IIIA, also known as MPS IIIA or Sanfilippo syndrome type A. It is being evaluated as part of a Phase 3 clinical trial in combination with gene transfer therapy for this rare genetic condition.
Adjuvant Immunomodulatory Therapy is being developed by Ultragenyx Pharmaceutical Inc., a biopharmaceutical company traded on NASDAQ under the ticker RARE. The company is investigating this therapy in the context of a Phase 3 clinical trial for Mucopolysaccharidosis IIIA.
Adjuvant Immunomodulatory Therapy is currently in Phase 3 clinical development. It is an investigational therapy, meaning it has not been approved by regulatory authorities and is still being studied in clinical trials for Mucopolysaccharidosis IIIA.
Adjuvant Immunomodulatory Therapy is being studied in one Phase 3 clinical trial with the identifier NCT04360265. This trial is a follow-up study of AAV-mediated gene transfer (UX111, previously known as ABO-102) for MPS type IIIA, enrolling 41 participants in the United States, Australia, and Spain.
Yes, Adjuvant Immunomodulatory Therapy is also known as Adjuvant Immunomodulatory (IM) Therapy. These names refer to the same investigational therapy being developed by Ultragenyx Pharmaceutical Inc. for use in Mucopolysaccharidosis IIIA.
Adjuvant Immunomodulatory Therapy is a small molecule designed to modulate the immune system. It is being studied as an adjuvant to gene transfer therapy, potentially helping to manage immune responses during treatment for Mucopolysaccharidosis IIIA. The specific molecular target has not been disclosed.