Type I Spinal Muscular AtrophyEarly Phase 1NCT06576388
Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I
A Early Phase 1 study testing EXG001-307 injection in people with Type I Spinal Muscular Atrophy, run by Guangzhou Jiayin Biotech Ltd at 1 site in China.
Trial summary
The purpose of this study was to evaluate the safety and preliminary efficacy of a single intravenous injection of exg001-307 in patients with type I spinal muscular atrophy. The research process includes the screening period (the screening period is from the time the subject signs the informed consent to the time… Read more about this study
- Who is it for?
- Ages Up to 180 days
- Conditions the study is for
- Type I Spinal Muscular Atrophy
- What is being tested?
- EXG001-307 injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteChina
- Study dates
- 21 Jan 2022to 21 Jan 2024
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
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 Up to 180 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- SMA was diagnosed by bilateral allele SMN1 mutation (deletion or point mutation) gene, and there were 2 copies of SMN2 gene.
- On the day of administration, the age of the subjects did not exceed the 180th day after birth.
- The clinical history and signs are consistent with the manifestations of type I SMA, that is, hypotonia, lagging development of motor function, poor head control, round shoulder posture and excessive joint activity.
- The legal guardian of the subject understands the purpose, possible risks and rights of the test, agrees the subject to participate in the test, completes all research steps, tests and visits, and voluntarily signs the informed consent.
- During the study period, according to the changes of the subject's condition, the subject's legal guardian was willing to carry out standard treatment requirements such as nasal feeding, noninvasive mechanical ventilation and expectoration machine according to the researcher's suggestions.
Who cannot join
- The gestational age at birth was less than 35 weeks (245 days).
- During the screening period, when the subjects were awake or asleep and did not receive any auxiliary oxygen supply or respiratory support, the blood oxygen saturation was less than 96%.
- Invasive ventilation or tracheotomy is required, or the current use of noninvasive ventilation support is ≥ 16 hours / day on average.
- According to the WHO child growth standard (who 2009), the weight is lower than the 3rd percentile by age.
- Before administration, if the subjects have not been vaccinated or delayed vaccination according to the national vaccination plan of the current month, it will significantly affect their safety according to the evaluation of the researcher and the medical manager of the project team;
- Active viral infections (including HIV, covid-19, seropositive for hepatitis B or C, torch virus, EBV virus and syphilis).
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- Severe non respiratory diseases within 2 weeks before screening.
- Upper respiratory tract infection or lower respiratory tract infection within 4 weeks before screening.
- There are other severe infections or diseases.
- There are known heart diseases or ECG abnormalities with clinical significance.
- Known allergy to prednisolone, other glucocorticoids or their excipients.
- Receive immunosuppressive therapy (e.g. cyclosporin, tacrolimus, methotrexate, cyclophosphamide, intravenous immunoglobulin, rituximab) requiring preventive administration within 3 months before administration.
- Immunomodulatory drugs (such as thymosin, interferon, etc.) are being used to treat myopathy, neuritis and diabetes (such as immunosuppressive agents, glucocorticoids, insulin).
- Anti AAV9 antibody titer > 1:50 (determined by ELISA). If the potential subject's anti AAV9 antibody titer > 1:50, it can be retested during the screening period. When the anti AAV9 antibody titer is ≤ 1:50, it can continue to participate in the screening.
- Abnormal laboratory test values with clinical significance (GGT, ALT and AST > 2.5) × ULN, bilirubin ≥ 3.0 mg / dl, creatinine ≥ 1.0 mg / dl, hemoglobin < 8 or > 18 g / dl; Leukocyte count > 20000 / cm3; Platelet count < 100000 / cm3).
- Previously participated in clinical research on the treatment of other SMA drugs.
- Major surgery was expected during study treatment.
- Other situations that the researcher judges are not suitable to participate in this study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Clinical Trial Evaluating the Safety and Efficacy of EXG001-307 in Patients With Type I Spinal Muscular Atrophy
The purpose of this study was to evaluate the safety and preliminary efficacy of a single intravenous injection of exg001-307 in patients with type I spinal muscular atrophy.
The research process includes the screening period (the screening period is from the time the subject signs the informed consent to the time before hormone pretreatment, with a maximum of 28 days), the treatment period (the subject receives hospitalization and observation including hormone pretreatment and single infusion of study drugs), and the follow-up period (the end of the treatment period until the subject reaches the age of 18 months, loss of follow-up, active withdrawal from the study or death). The qualified subjects in the screening period enter the treatment period, receive exg001-307 treatment, and enter the follow-up period after hospitalization observation. At the end of the study visit (subjects 18 months old), eligible subjects will be asked to transfer to the long-term follow-up study.
- Conditions
- Type I Spinal Muscular Atrophy
- Being tested
- EXG001-307 injection
- Medical subject headings
- Spinal Muscular Atrophies of Childhood
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EXG001-307 injectionDrug
Research process includes filter (subjects signed informed consent for screening period before the pretreatment to the hormone, the longest 28 days), treatment period (the subjects accept including single hormone pretreatment, the study drug infusion) hospitalization and observation and follow-up treatment period (subjects to the participants to 18 months, lost to follow-up, active exit research subjects, or death). Subjects qualified in the screening period entered the treatment period and received EXG001-307, and entered the follow-up period after hospitalization. At the end of the study visit (subject at 18 months of age), eligible subjects will be asked to transfer to the long-term follow-up study.
| Study group | Role | What they receive |
|---|---|---|
| single Arm-experimental group | Experimental | The dose escalation study included two cohorts with three dose levels: 0.67×1014 vg/kg, 1.1×1014 vg/kg, and 1.5×1014 vg/kg. Among them, the first queue contains two levels, the first dose levels (0.67 x 1014 vg/kg) using the accelerated titration method (i.e. after the first dose levels in group 1 case subjects, when the subjects to 28 days after complete treatment of safety assessment, if not present dose limiting toxicity (DLT), increasing the dose to the next dose levels; Otherwise the study will be terminated .
Study treatment name:EXG001-307 injection; Dosage form: injection; frequency and duration: single dose Receives: Drug: EXG001-307 injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the safety and tolerability of EXG001-307 after a single intravenous infusion
including type and incidence of AE, SAE, AESI, vital signs, physical/neurological examination, immunogenicity, virology, injection/infusion site reactions, 12-lead electrocardiogram, and safety laboratory results recorded
Measured: up to 18months
Secondary outcomes (1)
Explore a safe and effective dose range；Assess initial effectiveness；
Explore dose limiting toxicity By the time of the study visit at the age of 18 months, the proportion of subjects who could sit alone for ≥ 30 seconds without support was reached according to the Bailey infant development scale version 3 (bsid-iii) Survival at 14 months. Changes in motor function assessment data relative to baseline after 6 months of treatment at the children's Hospital of Philadelphia neuromuscular disease infant test (chop-intend) Proportion of subjects with chop-int score above 40 after 6 months of treatment After 6 months of treatment, the proportion of subjects whose exercise intensity and functional improvement reached development milestones were evaluated according to bsid-iii scale (head control ≥ 3 seconds; sitting alone ≥ 5 seconds without support; sitting alone ≥ 30 seconds without support)
Measured: up to 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2024
- Study start21 Jan 2022
- Primary completion21 Sep 2023
- Study completion21 Jan 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Type I Spinal Muscular AtrophyStudies of the same condition, open and under way.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Guangzhou Jiayin Biotech Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06576388
- Sponsor's protocol ID
- EXG001-307-101
- Status last verified
- Aug 2024
- First submitted
- 26 Aug 2024
- Last updated
- 28 Aug 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Trial Evaluating the Safety and Efficacy of EXG001-307 in Patients With Type I Spinal Muscular Atrophy
- ClinicalTrials.gov ID
- NCT06576388
- Acronym / sponsor ID
- EXG001-307-101
- Conditions
- Type I Spinal Muscular Atrophy
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Guangzhou Jiayin Biotech LtdIndustry (pharma, biotech, devices)
- Study start
- 21 Jan 2022 (actual)
- Primary completion
- 21 Sep 2023 (actual)
- Study completion
- 21 Jan 2024 (actual)
- First posted
- 28 Aug 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study was to evaluate the safety and preliminary efficacy of a single intravenous injection of exg001-307 in patients with type I spinal muscular atrophy.
The research process includes the screening period (the screening period is from the time the subject signs the informed consent to the time before hormone pretreatment, with a maximum of 28 days), the treatment period (the subject receives hospitalization and observation including hormone pretreatment and single infusion of study drugs), and the follow-up period (the end of the treatment period until the subject reaches the age of 18 months, loss of follow-up, active withdrawal from the study or death). The qualified subjects in the screening period enter the treatment period, receive exg001-307 treatment, and enter the follow-up period after hospitalization observation. At the end of the study visit (subjects 18 months old), eligible subjects will be asked to transfer to the long-term follow-up study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| single Arm-experimental group | Experimental | Drug: EXG001-307 injectionThe dose escalation study included two cohorts with three dose levels: 0.67×1014 vg/kg, 1.1×1014 vg/kg, and 1.5×1014 vg/kg. Among them, the first queue contains two levels, the first dose levels (0.67 x 1014 vg/kg) using the accelerated titration method (i.e. after the first dose levels in group 1 case subjects, when the subjects to 28 days after complete treatment of safety assessment, if not present dose limiting toxicity (DLT), increasing the dose to the next dose levels; Otherwise the study will be terminated . Study treatment name:EXG001-307 injection; Dosage form: injection; frequency and duration: single dose |
Who may qualify
- Ages
- Up to 180 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- SMA was diagnosed by bilateral allele SMN1 mutation (deletion or point mutation) gene, and there were 2 copies of SMN2 gene.
- On the day of administration, the age of the subjects did not exceed the 180th day after birth.
- The clinical history and signs are consistent with the manifestations of type I SMA, that is, hypotonia, lagging development of motor function, poor head control, round shoulder posture and excessive joint activity.
- The legal guardian of the subject understands the purpose, possible risks and rights of the test, agrees the subject to participate in the test, completes all research steps, tests and visits, and voluntarily signs the informed consent.
- During the study period, according to the changes of the subject's condition, the subject's legal guardian was willing to carry out standard treatment requirements such as nasal feeding, noninvasive mechanical ventilation and expectoration machine according to the researcher's suggestions.
Key exclusion criteria (18 listed)
- The gestational age at birth was less than 35 weeks (245 days).
- During the screening period, when the subjects were awake or asleep and did not receive any auxiliary oxygen supply or respiratory support, the blood oxygen saturation was less than 96%.
- Invasive ventilation or tracheotomy is required, or the current use of noninvasive ventilation support is ≥ 16 hours / day on average.
- According to the WHO child growth standard (who 2009), the weight is lower than the 3rd percentile by age.
- Before administration, if the subjects have not been vaccinated or delayed vaccination according to the national vaccination plan of the current month, it will significantly affect their safety according to the evaluation of the researcher and the medical manager of the project team;
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 |
|---|---|---|
| EXG001-307 injectionResearch process includes filter (subjects signed informed consent for screening period before the pretreatment to the hormone, the longest 28 days), treatment period (the subjects accept including single hormone pretreatment, the study drug infusion) hospitalization and observation and follow-up treatment period (subjects to the participants to 18 months, lost to follow-up, active exit research subjects, or death). Subjects qualified in the screening period entered the treatment period and received EXG001-307, and entered the follow-up period after hospitalization. At the end of the study visit (subject at 18 months of age), eligible subjects will be asked to transfer to the long-term follow-up study. | Drug | single Arm-experimental group |
Primary outcome
To evaluate the safety and tolerability of EXG001-307 after a single intravenous infusion
Time frame: up to 18months
including type and incidence of AE, SAE, AESI, vital signs, physical/neurological examination, immunogenicity, virology, injection/infusion site reactions, 12-lead electrocardiogram, and safety laboratory results recorded
1 secondary outcome
Explore a safe and effective dose range；Assess initial effectiveness；
Time frame: up to 18 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Aug 2024.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing EXG001-307 injection in people with Type I Spinal Muscular Atrophy, run by Guangzhou Jiayin Biotech Ltd at 1 site in China.
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 18 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 28 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. 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 phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
To evaluate the safety and tolerability of EXG001-307 after a single intravenous infusion (time frame: up to 18months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 28 Aug 2024.