BridgeMD
Tests treatment safety and results for SMA - Spinal Muscular Atrophy
Official title Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III
ClinicalTrials.gov ID: NCT06971094
What this study is testing
What is GC101 adeno-associated virus injection?
GC101 adeno-associated virus injection is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for sma - spinal muscular atrophy.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This trial employs a multicenter, randomized, open-label, standard-of-care-controlled design and plans to enroll 50 patients with Type 2 SMA aged 2 to 12 years who have previously received nusinersen. The primary objective of the trial is to evaluate the efficacy of GC101 in treating Type 2 SMA.
- Phase 3: a large, late-stage study
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 12
You may be able to join if
- Patients with a confirmed diagnosis of Type 2 5q-SMA through clinical phenotype and genetic testing.
- Patients who have been receiving regular treatment with nusinersen for more than one year prior to screening.
- Patients who have not received treatment with risdiplam within 2 months prior to screening and have no plans to receive risdiplam treatment within 12...
- Patients who can sit independently but cannot walk independently at the time of screening (according to the definitions of independent sitting and...
- Patients and/or their legal guardians are able to understand and are willing to comply with the requirements and procedures of the trial protocol...
You likely can't join if
- Patients with serum anti-AAV9 neutralizing antibody titers \> 1:50 at the time of screening.
- Patients who have received nusinersen treatment within 2 months prior to enrollment.
- Patients with any medical conditions that may affect the interpretation of study results or pose a risk to the safety of the participants, including...
- Patients with severe liver injury/hepatic insufficiency of any cause, including but not limited to alanine aminotransferase (ALT), aspartate...
- Patients deemed by the investigator to have contraindications to glucocorticoid use, such as severe hypertension, diabetes, systemic infectious...
- Patients with contraindications to lumbar puncture or intrathecal injection therapy.
See the full eligibility criteria
- Patients with a confirmed diagnosis of Type 2 5q-SMA through clinical phenotype and genetic testing.
- Patients who have been receiving regular treatment with nusinersen for more than one year prior to screening.
- Patients who have not received treatment with risdiplam within 2 months prior to screening and have no plans to receive risdiplam treatment within 12 months after enrollment.
- Patients who can sit independently but cannot walk independently at the time of screening (according to the definitions of independent sitting and walking in the WHO-MGRS motor milestones scale), and have an HFMSE score...
- Patients and/or their legal guardians are able to understand and are willing to comply with the requirements and procedures of the trial protocol, and voluntarily participate and sign the informed consent form
- Patients with serum anti-AAV9 neutralizing antibody titers \> 1:50 at the time of screening.
- Patients who have received nusinersen treatment within 2 months prior to enrollment.
- Patients with any medical conditions that may affect the interpretation of study results or pose a risk to the safety of the participants, including but not limited to organ dysfunction of any cause, acute infectious...
- Patients with severe liver injury/hepatic insufficiency of any cause, including but not limited to alanine aminotransferase (ALT), aspartate aminotransferase (AST) ≥3 times the upper limit of normal (ULN); total...
- Patients deemed by the investigator to have contraindications to glucocorticoid use, such as severe hypertension, diabetes, systemic infectious diseases, fungal infections, glaucoma, osteoporosis, peptic ulcer disease...
- Patients with contraindications to lumbar puncture or intrathecal injection therapy.
- Patients with any medical conditions that may affect the assessment of motor function, such as severe scoliosis, severe joint contracture deformities, planned spinal correction surgery during the trial period, severe...
- Patients positive for hepatitis B surface antigen (HBsAg), human immunodeficiency virus (HIV) antibodies, hepatitis C virus (HCV) antibodies, or syphilis antibodies.
- Patients who have received vaccinations within 2 weeks prior to dosing.
- Patients who have previously received gene therapy or participated in any clinical trial within 3 months prior to screening.
- Patients deemed by the investigator to be unsuitable for participation in this study.
The study team makes the final eligibility decision.
Where it's taking place
- Beijing, China
- Shanghai, China
- Shenzhen, China
- Suzhou, China
- Wuhan, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 12 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Beijing, China; Shanghai, China; Shenzhen, China; Suzhou, China; Wuhan, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.