Hutchinson-Gilford Progeria SyndromePhase 1/2NCT03871972
Umbilical Cord Blood Transfusion in Progeria Syndrome
A Phase 1/2 study testing Umbilical Cord Blood Unit in people with Hutchinson-Gilford Progeria Syndrome, run by Bundang CHA Hospital at 1 site in South Korea.
Trial summary
This is a pilot study on safety and efficacy of umbilical cord blood therapy for patients with Hutchinson Gilford Progeria syndrome (HGPS). This is an 1 year trial with 3 IV infusions (4 months apart from each infusion) of umbilical cord blood units with oral Sirolimus to see the safety and efficacy. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hutchinson-Gilford Progeria Syndrome
- What is being tested?
- Umbilical Cord Blood Unit
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteSouth Korea
- Study dates
- 5 Mar 2019to 1 Jun 2020
- Phase
- Phase 1/2
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- those who were clinically and genetically diagnosed as Hutchinson-Gilford progeria syndrome
Who cannot join
- those who show definite hemorrhage or ischemia on brain MRI
- those who are affected with systemic infection during study enrolling period
- those who are not able to able to make consents to the study; those who are not accompanying any guardians
- those who were enrolled in other clinical trials within last 30 days
- those who are not appropriate according to laboratory criteria
- whose ALT/AST > 2 fold of normal limit
Show all 12Show fewer
- whose serum creatinine > 1.5 fold of normal limit
- whose total bilirubin > 2 fold of normal limit
- whose total WBC count < 3000/mm3
- whose platelet count < normal lower limit
- those who are diagnosed with other malignancies
- those who are affected by other serious medical (cardiopulmonary, gastrointestinal, endocrinologic, etc.) conditions
Trial locations and contacts
1 site in 1 country.
South Korea1 site
Bundang CHA Medical Center
Seongnam-si, Gyeonggi-do, 13496
About this study
Official title: Safety and Efficacy of Umbilical Cord Blood Transfusion in Patients With Hutchinson-Gilford Progeria Syndrome
This is a pilot study on safety and efficacy of umbilical cord blood therapy for patients with Hutchinson Gilford Progeria syndrome (HGPS). This is an 1 year trial with 3 IV infusions (4 months apart from each infusion) of umbilical cord blood units with oral Sirolimus to see the safety and efficacy.
Read the detailed description
This is a pilot study on safety and efficacy of umbilical cord blood therapy for patients with Hutchinson Gilford Progeria syndrome (HGPS). HGPS is a rare genetic disease where affected LMNA gene coding lamin A protein leads to premature aging and early death.
Teenagers with HGPS are in high risk of atherosclerosis and ischemic stroke, and these are major reason of mortality in HGPS.Currently, there are no definite cure for this rare genetic disease. Among the potential drugs under investigation, Lornafarnib (farnesyltransferase inhibitor) lowered the carotid-femoral pulse wave velocity (cfPWV) and also lowered mortality.
Stem cell therapy has proven its efficacy in progeria mouse model. We are trying to study safety and efficacy of umbilical cord blood therapy in human HGPS patients.
- Conditions
- Hutchinson-Gilford Progeria Syndrome
- Being tested
- Umbilical Cord Blood Unit
- Medical subject headings
- Progeria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Umbilical Cord Blood UnitDrug
Also called Oral Sirolimus
3 infusions of umbilical cord blood (UCB) unit (TNC > 2.0ⅹ107cells/kg) each 4 months apart and take oral Sirolimus (1 mg/m2/day) for 7 days (from 3 days before UCB infusion until 3 days after UCB infusion)
| Study group | Role | What they receive |
|---|---|---|
| UCB injection group | Experimental | This pilot study includes only 2 subjects who are enrolled by invitation. Both subjects are included in this single arm. Receives: Drug: Umbilical Cord Blood Unit |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline Carotid-femoral pulse wave velocity at 48 weeks
measured by carotid doppler ultrasonography
Measured: 48 weeks after UCB infusion
Change from baseline serum HDL cholesterol at 48 weeks
taken on routine lab
Measured: 48 weeks after UCB infusion
Change from baseline weight at 48 weeks
measured by bioimpedance analysis
Measured: 48 weeks after UCB infusion
Secondary outcomes (4)
Ankle-brachial index
measured by automatic blood pressure gauge
Measured: baseline, 48 weeks after UCB infusion
Body fat proportion
taken by bioimpedance analysis
Measured: baseline, 48 weeks after UCB infusion
Range of motion
measured manually
Measured: baseline, 48 weeks after UCB infusion
height
measured by bioimpedance analysis
Measured: baseline, 48 weeks after UCB infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Mar 2019
- Study start5 Mar 2019
- Primary completion1 Jun 2020
- Study completion1 Jun 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hutchinson-Gilford Progeria SyndromeStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bundang CHA Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, MinYoung Kim, MD, PhD, Bundang CHA Hospital
- Investigators
- Min Young Kim, MD, PhD, Principal investigator, CHA University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03871972
- Sponsor's protocol ID
- 2018-12-031
- Status last verified
- Nov 2020
- First submitted
- 7 Mar 2019
- Last updated
- 4 Nov 2020
- 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 4 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Umbilical Cord Blood Transfusion in Patients With Hutchinson-Gilford Progeria Syndrome
- ClinicalTrials.gov ID
- NCT03871972
- Acronym / sponsor ID
- 2018-12-031
- Conditions
- Hutchinson-Gilford Progeria Syndrome
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Bundang CHA HospitalUniversity, hospital or other
- Study start
- 5 Mar 2019 (actual)
- Primary completion
- 1 Jun 2020 (actual)
- Study completion
- 1 Jun 2020 (actual)
- First posted
- 12 Mar 2019 (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.
This is a pilot study on safety and efficacy of umbilical cord blood therapy for patients with Hutchinson Gilford Progeria syndrome (HGPS). This is an 1 year trial with 3 IV infusions (4 months apart from each infusion) of umbilical cord blood units with oral Sirolimus to see the safety and efficacy.
Detailed description
This is a pilot study on safety and efficacy of umbilical cord blood therapy for patients with Hutchinson Gilford Progeria syndrome (HGPS). HGPS is a rare genetic disease where affected LMNA gene coding lamin A protein leads to premature aging and early death.
Teenagers with HGPS are in high risk of atherosclerosis and ischemic stroke, and these are major reason of mortality in HGPS.Currently, there are no definite cure for this rare genetic disease. Among the potential drugs under investigation, Lornafarnib (farnesyltransferase inhibitor) lowered the carotid-femoral pulse wave velocity (cfPWV) and also lowered mortality.
Stem cell therapy has proven its efficacy in progeria mouse model. We are trying to study safety and efficacy of umbilical cord blood therapy in human HGPS patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| UCB injection group | Experimental | Drug: Umbilical Cord Blood UnitThis pilot study includes only 2 subjects who are enrolled by invitation. Both subjects are included in this single arm. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- those who were clinically and genetically diagnosed as Hutchinson-Gilford progeria syndrome
Key exclusion criteria (12 listed)
- those who show definite hemorrhage or ischemia on brain MRI
- those who are affected with systemic infection during study enrolling period
- those who are not able to able to make consents to the study; those who are not accompanying any guardians
- those who were enrolled in other clinical trials within last 30 days
- those who are not appropriate according to laboratory criteria
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 |
|---|---|---|
| Umbilical Cord Blood UnitAlso: Oral Sirolimus3 infusions of umbilical cord blood (UCB) unit (TNC > 2.0ⅹ107cells/kg) each 4 months apart and take oral Sirolimus (1 mg/m2/day) for 7 days (from 3 days before UCB infusion until 3 days after UCB infusion) | Drug | UCB injection group |
Primary outcome
Change from baseline Carotid-femoral pulse wave velocity at 48 weeks
Time frame: 48 weeks after UCB infusion
measured by carotid doppler ultrasonography
Change from baseline serum HDL cholesterol at 48 weeks
Time frame: 48 weeks after UCB infusion
taken on routine lab
Change from baseline weight at 48 weeks
Time frame: 48 weeks after UCB infusion
measured by bioimpedance analysis
4 secondary outcomes
Ankle-brachial index
Time frame: baseline, 48 weeks after UCB infusion
Body fat proportion
Time frame: baseline, 48 weeks after UCB infusion
Range of motion
Time frame: baseline, 48 weeks after UCB infusion
height
Time frame: baseline, 48 weeks after UCB infusion
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 4 Nov 2020.
- Individual participant dataSharing planned: No
Related trials for Hutchinson-Gilford Progeria Syndrome
Not yet recruitingObservational study
A Study Observing Disease Progression in a Patient With Hutchinson-Gilford Progeria Syndrome (HGPS)
Hutchinson-Gilford Progeria Syndrome
Not yet recruitingObservational study
Creating Stem Cells From a Progeria Patient's Blood to Test Candidate Treatments in the Lab
Hutchinson-Gilford Progeria Syndrome
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.
Min Young Kim, MD, PhD principal investigator
CHA University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Umbilical Cord Blood Unit in people with Hutchinson-Gilford Progeria Syndrome, run by Bundang CHA 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 1 inclusion and 12 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 4 Nov 2020. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Change from baseline Carotid-femoral pulse wave velocity at 48 weeks (time frame: 48 weeks after UCB infusion). The study lists 3 primary outcomes in all. 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 4 Nov 2020.