A study of T19 in subjects with spinal muscular atrophy
| ISRCTN | ISRCTN21188633 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN21188633 |
| Secondary identifying numbers | CA-18-10 |
- Submission date
- 03/01/2023
- Registration date
- 08/01/2023
- Last edited
- 15/07/2025
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Nervous System Diseases
Plain English summary of protocol
Background and study aims
Spinal muscular atrophy (SMA) is the most common genetic cause of death for children below two years old. Previous studies by us and others have suggested that the fibrinolysis system is involved in nerve degeneration and regeneration and in respiratory failure. In this study, the clinical effects of T19, the key substrate of the fibrinolysis system, were investigated in SMA patients.
Who can participate?
Type I and 1 non-5q (IGHMBP2 gene deficiency) SMA patients
What does the study involve?
Patients were given an intravenous injection of T19 1 time per 1-3 days, for two weeks as one treatment course, with 2-week intervals between courses. Trained clinical evaluators will assess the patients’ motor function according to the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND). Respiratory function is assessed by the value of blood oxygen saturation.
What are the possible benefits and risks of participating?
The possible benefits of participating in the trial are improvement of the patient's motor function, respiration function and survival. Patients also get free medication. Considering the properties of T19, there may be a risk of bleeding, hypersensitivity reactions and infection after receiving a T19 injection.
Where is the study run from?
Beijing Chang’an Chinese and Western Integrated Medicine Hospital (China)
When is the study starting and how long is it expected to run for?
September 2018 to September 2021
Who is funding the study?
Talengen Institute of Life Sciences (China)
Who is the main contact?
Dr Jinan Li, jnl@talengen-pharma.com
Contact information
Principal Investigator
Room C602G
289 Digital Peninsula
Shunfeng Industrial Park
No.2 Red Willow Road
Futian District
Shenzhen
518000
China
 ORCID ID |
0000-0001-6746-967X |
|---|---|
| Phone | +86 15919440001 |
| jnl@talengen-pharma.com |
Public
Department of Applied Research
Talengen Institute of Life Sciences
Room C602G
289 Digital Peninsula
Shunfeng Industrial Park
No.2 Red Willow Road
Futian District
Shenzhen
518000
China
| ORCID ID |
0000-0001-5679-4389 |
|---|---|
| Phone | +86 15167735556 |
| guocy@talengen-pharma.com |
Scientific
Department of Applied Research
Talengen Institute of Life Sciences
Room C602G
289 Digital Peninsula
Shunfeng Industrial Park
No.2 Red Willow Road
Futian District
Shenzhen
518000
China
| Phone | +86 15167735556 |
|---|---|
| guocy@talengen-pharma.com |
Study information
| Study design | Open-label one-arm non-randomized study |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Non randomised study |
| Study setting(s) | Hospital |
| Study type | Treatment |
| Participant information sheet | Not available in web format, please use the contact details to request a participant information sheet |
| Scientific title | T19 shows rapid efficacy in treating patients with type I spinal muscular atrophy |
| Study objectives | Spinal muscular atrophy (SMA) is the most common genetic cause of death for children aged below two years old. Previous studies by us and others have suggested that the fibrinolysis system is involved in nerve degeneration and regeneration and in respiratory failure. In the present study, we investigated the clinical effects of T19. |
| Ethics approval(s) | Approved 05/09/2018, Ethics Committee of Beijing Chang’an Chinese and Western Integrated Medicine Hospital (19 Zaolinqian St, Xicheng District, Beijing, China; +86-13522667371; 421337949@qq.com), ref: CA-18-10 |
| Health condition(s) or problem(s) studied | Spinal muscular atrophy |
| Intervention | Spinal muscular atrophy (SMA) is the most common genetic cause of death for children below two years old. Previous studies by us and others have suggested that the fibrinolysis system is involved in nerve degeneration and regeneration and in respiratory failure. Clinical doctors or nursing staff with more than 5 years of clinical work experience will administer the intervention face-to-face. Based on the condition of the patients, the intervention is performed at the home of patients or at Beijing Chang’an Chinese and Western Integrated Medicine Hospital. The clinical study is an open-label, one-arm, and non-randomized study with a treatment duration of 72 weeks. Freeze-dried T19 (5 or 50 mg per vial), purified from human plasma fraction III at GMP-compliant facilities, was provided by the Talengen Institute of Life Sciences. T19 was dissolved in sterile water to produce 5 mg/ml solutions for use in the study. Patients were given an intravenous injection, at doses of 50-200 mg each time, 1 time per 1-3 days, with two weeks as one treatment course, and 2-week intervals between courses. Trained clinical evaluators assessed the patients’ motor function according to the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND). Respiratory function is assessed by the value of blood oxygen saturation. |
| Intervention type | Drug |
| Pharmaceutical study type(s) | Not Applicable |
| Phase | Not Applicable |
| Drug / device / biological / vaccine name(s) | Plasminogen |
| Primary outcome measure | Motor function scores measured using the CHOP INTEND scoring system at baseline and weeks 2, 6, 10, 22 and 46 |
| Secondary outcome measures | 1. Respiratory function measured by the value of blood oxygen saturation without Oxygen inhalation in pulse oximetry at baseline and weeks 2, 6, 10, 22 and 46 2. Anthropometric nutritional status measured using the proportion of high body weight, body fat, and growth parameters at baseline and weeks 10, 22 and 46 3. Adverse events assessed by routine blood test, blood biochemistry, coagulation function, hemolysis function, urine routine test, 12 lead ECG, physical examination, vital signs, etc measured using standard procedures at baseline, and weeks 22 and 46 |
| Overall study start date | 05/09/2018 |
| Completion date | 05/09/2021 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Child |
| Sex | Both |
| Target number of participants | 30 |
| Total final enrolment | 20 |
| Key inclusion criteria | The subjects were diagnosed with type I SMA with SMN gene mutation or non-5q SMA with mutation in the gene encoding immunoglobulin-binding protein 2 (IGHMBP2), according to genetic tests and clinical symptoms |
| Key exclusion criteria | Patients receiving more than 16 hours of invasive ventilation per day |
| Date of first enrolment | 05/10/2018 |
| Date of final enrolment | 05/08/2020 |
Locations
Countries of recruitment
- China
Study participating centre
Xicheng District
Beijing
100010
China
Sponsor information
Industry
Room C602G
289 Digital Peninsula
Shunfeng Industrial Park
No.2 Red Willow Road
Futian District
Shenzhen
518000
China
| Phone | +86-15919440001 |
|---|---|
| guocy@talengen-pharma.com |
Funders
Funder type
Industry
No information available
Results and Publications
| Intention to publish date | 10/05/2023 |
|---|---|
| Individual participant data (IPD) Intention to share | Yes |
| IPD sharing plan summary | Available on request |
| Publication and dissemination plan | Planned publication in a high-impact peer reviewed publication |
| IPD sharing plan | The datasets generated during and/or analysed during the current study are/will be available upon request from Dr Jinan Li, jnl@talengen-pharma.com. The type of data that will be shared is the table of the scoring record, clinical observations in record form, images, videotape, and detection data. Data will be available from 05/10/2023 to 05/10/2033. Consent from participants was required and obtained. Except for the initial record, all patients use unique numbers in favour of anonymity in the experiment. The patient's data, pictures and other relevant information must be approved by the patient before being published. |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Protocol file | 06/01/2023 | No | No |
Additional files
Editorial Notes
15/07/2025: The word 'plasminogen' to the word 'T19' in the public and scientific titles, study objectives, interventions, and the Plain English summary of protocol.
14/11/2023: The following changes were made to the trial record:
1. The recruitment start date was changed from 05/09/2019 to 05/10/2018.
2. The recruitment end date was changed from 10/08/2022 to 05/08/2020.
3. The overall end date was changed from 05/12/2022 to 05/09/2021.
4. The plain English summary was updated to reflect these changes.
14/02/2023: The following changes were made to the trial record:
1. The overall trial end date has been changed from 05/10/2023 to 05/12/2022 and the plain English summary was updated accordingly.
2. The recruitment end date has been changed from 10/10/2022 to 10/08/2022.
10/02/2023: A contact was updated.
02/02/2023: The following changes were made to the trial record:
1. The ethics details were updated.
2. The target number of participants was changed from 15 to 30.
3. The total final enrolment was changed from 10 to 20.
06/01/2023: Trial's existence confirmed by the Ethics Committee of Beijing Chang’an Chinese and Western Integrated Medicine Hospital.
