Amyotrophic Lateral SclerosisPhase 1NCT03214146
Safety/Efficacy Study of 2nd Cycle Treatment After 6 Months of 1st Cycle HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell Treatment in ALS
A Phase 1 study testing HYNRCS-Allo inj in people with Amyotrophic Lateral Sclerosis, run by Hanyang University Seoul Hospital at 1 site in South Korea.
Trial summary
The purpose of this study is to evaluate the safety and efficacy of HLA-haplo matched Allogenic Bone Marrow Derived stem cells("HYNRCS-Allo-ALS-02 inj"), through intrathecal delivery for the repeated treatment after 6 months of first treatment in patients with amyotrophic lateral sclerosis(ALS). Read more about this study
- Who is it for?
- Ages 25 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- HYNRCS-Allo inj
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteSouth Korea
- Study dates
- 1 Feb 2017to Oct 2021
- Phase
- 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 25 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients between 25 and 80 years old
- Patients diagnosed as 'Possible with lab-supported' or 'Possible' or 'Probable' or 'Definite' ALS according to the World Federation of Neurology El Escorial criteria
- Patients whose duration of disease is within 5 years from the first diagnosis
- Patients with ALSFRS-R score within 21 to 46 at screening
- Patients who can visit to a hospital by walk personally or by protector's help
- Patients who provide the written consent by oneself or his/her legal representative
- Patients who has HLA-haplo matched Bone marrow donor
Who cannot join
- Patients who doesn't appropriate to the diagnostic criteria of ALS
- Patients who doesn't have HLA-haplo-matched bone marrow donor
- Patients suspected of adverse effect after stem cell injection(patients suspected of malignant tumor, risk group of psychogenic shock, patients with serious hypertension)
- Patients with ALSFRS-R score below 21 at screening
- Patients performed Tracheostomy at screening
- Patients with suspected 20% or less of Forced vital capacity(FVC) at screening
Show all 14Show fewer
- Patients who doesn't agree with written consent form by oneself of his/her legal representative
- Patients who have taken any other drug for clinical trial within the past 3 months at screening entry
- Patients with epilepsy
- Patients with severe medical disease
- Pregnant woman, lactating woman, female patients who has a pregnancy planning or who doesn't agree with adoption of contraception methods proper medically, male patients who doesn't agree with adoption of contraception methods proper to his partner during participating this study
- Patients with hemorrhagic tendency at screening
- Patients with a known history of hypersensitivity/allergy to penicillin and streptomycin
- Patients with severe psychotic diseases (such as alzheimer, schizophrenia excepts slight cognitive dysfunction and secondary emotional disorder)
Trial locations and contacts
1 site in 1 country.
South Korea1 site
Hanyang University Seoul Hospital, Cell Therapy Center for Neurologic Disorders
Seoul, Haengdang-dong, Seongdong-gu, 133-792
About this study
Official title: An Open-label, Phase 1 Trial for Safety and Efficacy Study of 2nd Cycle Treatment After 6 Months of 1st Cycle HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell("HYNR-CS-Allo Inj") Treatment in Amyotrophic Lateral Sclerosis(ALS)
The purpose of this study is to evaluate the safety and efficacy of HLA-haplo matched Allogenic Bone Marrow Derived stem cells("HYNRCS-Allo-ALS-02 inj"), through intrathecal delivery for the repeated treatment after 6 months of first treatment in patients with amyotrophic lateral sclerosis(ALS).
This study is an open label, single-dose study to assess the safety and efficacy of HLA-haplo matched Allogenic Bone Marrow Derived stem cells("HYNRCS-Allo-ALS-02 inj")
Read the detailed description
Amyotrophic lateral sclerosis is a progressive neurodegenerative disease characterized by motor neuron loss. Despite of many trials for disease-modifying, no treatment has so far changed natural course of disease.
The investigators had performed the pre-clinical and clinical studies using autologous bone marrow-derived stem cells in ALS. In the investigators' results of clinical trial, intrathecal injection of autologous bone marrow-derived stem cells is safe and could slow down disease progression and might be used as a disease modifying strategy in patients with ALS.
In the new field, like cell therapy, it is an important issue whether a bone marrow derived mesenchymal stem cells can be used as an allograft. Many investigators had showed that the immunoprivileged and immunosuppressive properties of mesenchymal stem cells result from the absence of major histocompatibility class II antigens and the secretion of T helper type 2 cytokines.
One potential advantage of allogenic bone marrow derived cells could be avoiding the need for procedural delay before treatment. And it is also hypothesized that the function of autologous bone marrow derived cells could be impaired in patients with co-morbidities or advanced age.
This study is to evaluate safety and efficacy of repeated treatment after 6 months of first HYNRCS-Allo-ALS-02 inj(HLA-haplo matched Allogenic bone marrow-derived stem cells) treatment in patients with ALS.
The patients enrolled in the trial will be successively allocated into single cohort for HYNRCS-Allo-ALS-02 inj., 1.0 X 10^6 cells/kg, according to the protocol design. Only a maximum of six patients will be given a particular dosage.
The scheduled assessments and visits will be carried out over three periods: run-in period, treatment period, and follow-up period.
The run-in period includes the screening visit where a written informed consent is obtained and the screening period where patients are assessed for eligibility. It will be completed within 56 days prior to enrollment. The patients meeting inclusion criteria will start the treatment period.
During the treatment period, subjects will be administered HYNRCS-Allo-ALS-02 inj. 2 times(first treatment) by intrathecal administration with 28 days interval and there will be repeated treatment after 6 months of the first treatment.
The Follow-up period starts once subjects complete the treatment period and will continue until the final follow-up visit.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- HYNRCS-Allo inj
- Keywords
- ALS, Motor Neuron Disease, Neuromuscular Disease, Neurodegenerative Disease, Central Nervous System Disease, HLA-haplo matched allogenic bone marrow derived stem cells
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Neuromuscular Diseases, Neurodegenerative Diseases, Central Nervous System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HYNRCS-Allo injBiological
Also called HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell
The patients enrolled in the trial will be successively allocated into single cohort for HYNRCS-Allo inj., 1.0 X 10^6 cells/kg according to the protocol design.
| Study group | Role | What they receive |
|---|---|---|
| HYNRCS-Allo inj. | Experimental | 2 cycles of HYNRCS-Allo inj. with 6 months interval through intrathecal injection.
*1 cycle of HYNRCS-Allo inj. is 2 times administration with 28 days interval by intrathecal. Receives: Biological: HYNRCS-Allo inj |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Incidence of any treatment related serious adverse events(SAE)
Measured: 12 months
Secondary outcomes (3)
ALS-Functional rating scales(ALS-FRS)
Measured: 12 months
Incidence & Degree of Adverse Events(AE)
Measured: 12 months
PRA test to identify generation status of HLA antibody
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jul 2017
- Study start1 Feb 2017
- Primary completion25 Mar 2019
- Study completionOct 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies 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
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hanyang University Seoul Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Corestemchemon, Inc.
- Responsible party
- Principal investigator, Seung Hyun Kim, Hanyang University Seoul Hospital
- Investigators
- Seung Hyun Kim, M.D.,Ph.D., Principal investigator, Hanyang University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03214146
- Sponsor's protocol ID
- HYNR-CS-Allo-02
- Status last verified
- May 2019
- First submitted
- 9 Jul 2017
- Last updated
- 16 May 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Phase 1 Trial for Safety and Efficacy Study of 2nd Cycle Treatment After 6 Months of 1st Cycle HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell("HYNR-CS-Allo Inj") Treatment in Amyotrophic Lateral Sclerosis(ALS)
- ClinicalTrials.gov ID
- NCT03214146
- Acronym / sponsor ID
- HYNR-CS-Allo-02
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS, Motor Neuron Disease, Neuromuscular Disease, Neurodegenerative Disease, Central Nervous System Disease, HLA-haplo matched allogenic bone marrow derived stem cells
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Hanyang University Seoul HospitalUniversity, hospital or other
- Collaborators
- Corestemchemon, Inc.
- Study start
- 1 Feb 2017 (actual)
- Primary completion
- 25 Mar 2019 (actual)
- Study completion
- Oct 2021 (estimated)
- First posted
- 11 Jul 2017 (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 is to evaluate the safety and efficacy of HLA-haplo matched Allogenic Bone Marrow Derived stem cells("HYNRCS-Allo-ALS-02 inj"), through intrathecal delivery for the repeated treatment after 6 months of first treatment in patients with amyotrophic lateral sclerosis(ALS).
This study is an open label, single-dose study to assess the safety and efficacy of HLA-haplo matched Allogenic Bone Marrow Derived stem cells("HYNRCS-Allo-ALS-02 inj")
Detailed description
Amyotrophic lateral sclerosis is a progressive neurodegenerative disease characterized by motor neuron loss. Despite of many trials for disease-modifying, no treatment has so far changed natural course of disease.
The investigators had performed the pre-clinical and clinical studies using autologous bone marrow-derived stem cells in ALS. In the investigators' results of clinical trial, intrathecal injection of autologous bone marrow-derived stem cells is safe and could slow down disease progression and might be used as a disease modifying strategy in patients with ALS.
In the new field, like cell therapy, it is an important issue whether a bone marrow derived mesenchymal stem cells can be used as an allograft. Many investigators had showed that the immunoprivileged and immunosuppressive properties of mesenchymal stem cells result from the absence of major histocompatibility class II antigens and the secretion of T helper type 2 cytokines.
One potential advantage of allogenic bone marrow derived cells could be avoiding the need for procedural delay before treatment. And it is also hypothesized that the function of autologous bone marrow derived cells could be impaired in patients with co-morbidities or advanced age.
This study is to evaluate safety and efficacy of repeated treatment after 6 months of first HYNRCS-Allo-ALS-02 inj(HLA-haplo matched Allogenic bone marrow-derived stem cells) treatment in patients with ALS.
The patients enrolled in the trial will be successively allocated into single cohort for HYNRCS-Allo-ALS-02 inj., 1.0 X 10^6 cells/kg, according to the protocol design. Only a maximum of six patients will be given a particular dosage.
The scheduled assessments and visits will be carried out over three periods: run-in period, treatment period, and follow-up period.
The run-in period includes the screening visit where a written informed consent is obtained and the screening period where patients are assessed for eligibility. It will be completed within 56 days prior to enrollment. The patients meeting inclusion criteria will start the treatment period.
During the treatment period, subjects will be administered HYNRCS-Allo-ALS-02 inj. 2 times(first treatment) by intrathecal administration with 28 days interval and there will be repeated treatment after 6 months of the first treatment.
The Follow-up period starts once subjects complete the treatment period and will continue until the final follow-up visit.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HYNRCS-Allo inj. | Experimental | Biological: HYNRCS-Allo inj2 cycles of HYNRCS-Allo inj. with 6 months interval through intrathecal injection. *1 cycle of HYNRCS-Allo inj. is 2 times administration with 28 days interval by intrathecal. |
Who may qualify
- Ages
- 25 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Patients between 25 and 80 years old
- Patients diagnosed as 'Possible with lab-supported' or 'Possible' or 'Probable' or 'Definite' ALS according to the World Federation of Neurology El Escorial criteria
- Patients whose duration of disease is within 5 years from the first diagnosis
- Patients with ALSFRS-R score within 21 to 46 at screening
- Patients who can visit to a hospital by walk personally or by protector's help
Key exclusion criteria (14 listed)
- Patients who doesn't appropriate to the diagnostic criteria of ALS
- Patients who doesn't have HLA-haplo-matched bone marrow donor
- Patients suspected of adverse effect after stem cell injection(patients suspected of malignant tumor, risk group of psychogenic shock, patients with serious hypertension)
- Patients with ALSFRS-R score below 21 at screening
- Patients performed Tracheostomy at screening
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 |
|---|---|---|
| HYNRCS-Allo injAlso: HLA-haplo Matched Allogenic Bone Marrow Derived Stem CellThe patients enrolled in the trial will be successively allocated into single cohort for HYNRCS-Allo inj., 1.0 X 10^6 cells/kg according to the protocol design. | Biological | HYNRCS-Allo inj. |
Primary outcome
The Incidence of any treatment related serious adverse events(SAE)
Time frame: 12 months
3 secondary outcomes
ALS-Functional rating scales(ALS-FRS)
Time frame: 12 months
Incidence & Degree of Adverse Events(AE)
Time frame: 12 months
PRA test to identify generation status of HLA antibody
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 May 2019.
- Individual participant dataSharing planned: No
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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.
Seung Hyun Kim, M.D.,Ph.D. principal investigator
Hanyang University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing HYNRCS-Allo inj in people with Amyotrophic Lateral Sclerosis, run by Hanyang University Seoul Hospital at 1 site in South Korea.
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 7 inclusion and 14 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: status not confirmed, as of the sponsor's last update on 16 May 2019. 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 South Korea. 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?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
The Incidence of any treatment related serious adverse events(SAE) (time frame: 12 months). 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 16 May 2019.