Ignite Creation Date:
2024-05-06 @ 7:41 PM
Last Modification Date:
2024-10-26 @ 3:11 PM
Study NCT ID:
NCT06097780
Status:
NOT_YET_RECRUITING
Last Update Posted:
2023-10-24
First Post:
2023-10-05
Brief Title:
Efficacy and Safety of NestaCell in Huntingtons Disease
Sponsor:
Azidus Brasil
Organization:
Azidus Brasil
Study Overview
Official Title:
Phase III Efficacy and Safety of NestaCell in Moderated Huntingtons Disease
Status:
NOT_YET_RECRUITING
Status Verified Date:
2023-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
STAR
Brief Summary:
Huntingtons disease HD is a rare neurodegenerative condition caused by increased CAG trinucleotide repeats in the HTT gene on chromosome 4 The estimated global prevalence is 271 cases per 100000 inhabitants In Brazil it is estimated that 13000 to 19000 people carry the gene and 65000 to 95000 are descendants at risk
HD usually manifests itself in the fourth decade of life with motor cognitive and behavioral symptoms such as chorea This condition profoundly affects quality of life and there is no treatment that can modify its course Tetrabenazine is the only medication approved to control chorea
A partnership between the Butantan Institute and Cellavita investigates the use of Human Dental Pulp Stem Cells hDPSCs to treat HD NestaCell was developed a product based on these cells which express high levels of BDNF an important neurotrophic factor for neuronal survival
Preclinical tests showed that NestaCell is distributed to several organs including the central nervous system being well tolerated in toxicological tests in rats
In phase I SAVE and phase II ADORE clinical trials NestaCell was administered to patients with HD The results indicated a significant improvement in motor scores and functional capacity compared to placebo demonstrating a clinically significant benefit
NestaCell also presented a good safety and tolerability profile with few adverse events related to the product The results support the conclusion that NestaCell is safe and well tolerated in HD patients within the doses tested
HD usually manifests itself in the fourth decade of life with motor cognitive and behavioral symptoms such as chorea This condition profoundly affects quality of life and there is no treatment that can modify its course Tetrabenazine is the only medication approved to control chorea
A partnership between the Butantan Institute and Cellavita investigates the use of Human Dental Pulp Stem Cells hDPSCs to treat HD NestaCell was developed a product based on these cells which express high levels of BDNF an important neurotrophic factor for neuronal survival
Preclinical tests showed that NestaCell is distributed to several organs including the central nervous system being well tolerated in toxicological tests in rats
In phase I SAVE and phase II ADORE clinical trials NestaCell was administered to patients with HD The results indicated a significant improvement in motor scores and functional capacity compared to placebo demonstrating a clinically significant benefit
NestaCell also presented a good safety and tolerability profile with few adverse events related to the product The results support the conclusion that NestaCell is safe and well tolerated in HD patients within the doses tested
Detailed Description:
This is a Phase III multicenter prospective randomized double-blind placebo-controlled clinical trial to evaluate the efficacy and safety of Nestacell in Huntingtons Disease
After signing the Informed Consent Form ICF the patients will perform the V-2 and V-1 screening procedures In the V-2 the site collects personal data and a physician clinically confirms the HD diagnosis The patient collects blood for CAG repeats and safety exams Females with childbearing potential perform a urine pregnancy test In the V-1 the investigator remotely reviews the results of the V-2 exams and authorizes the performance of the V-1 radiological exams MRI and Total-Body PET Scan in the radiology center
In the V0 the PI and his team perform the baseline UHDRS and body weight assessments The patient will be randomized to Nestacell or placebo Those weighing 50 to 679 kg will receive 100 million cells or placebo and those weighing 68 kg will receive 136 million cellsor placebo per administration The doses are based on the V0 weight assessment and will not change throughout the study unless the PI asks for safety reasons for example a relevant weight loss
The V1 marks the first investigational product administration It will happen in sites unrelated to those performing the outcomes evaluation by personnel specially trained to prepare and administer the investigational product After the females with childbearing potential performe the urine pregnancy test NestaCell or placebo will be administered intravenously in three cycles of three-monthly administrations with a monthly interval between cycles total of 9 administrations The other administrations happen at visits V2 V3 V5 V6 V7 V9 V10 and V11 To assure blindness UHDRS and other clinical evaluations will be carried out in the research center by the principal investigator PI while the administrations will be made in the Center for the Investigational Product Administration CIPA The outcome evaluations by the PI and hisher team happen at V4 V8 and V12 one month after the end of each cycle In the V13 the patients will also be directed to the radiology center to repeat the MRI exam
Each patient participates in the trial for approximately 14 months two months for screening and twelve months for investigational product administration and follow-up
After signing the Informed Consent Form ICF the patients will perform the V-2 and V-1 screening procedures In the V-2 the site collects personal data and a physician clinically confirms the HD diagnosis The patient collects blood for CAG repeats and safety exams Females with childbearing potential perform a urine pregnancy test In the V-1 the investigator remotely reviews the results of the V-2 exams and authorizes the performance of the V-1 radiological exams MRI and Total-Body PET Scan in the radiology center
In the V0 the PI and his team perform the baseline UHDRS and body weight assessments The patient will be randomized to Nestacell or placebo Those weighing 50 to 679 kg will receive 100 million cells or placebo and those weighing 68 kg will receive 136 million cellsor placebo per administration The doses are based on the V0 weight assessment and will not change throughout the study unless the PI asks for safety reasons for example a relevant weight loss
The V1 marks the first investigational product administration It will happen in sites unrelated to those performing the outcomes evaluation by personnel specially trained to prepare and administer the investigational product After the females with childbearing potential performe the urine pregnancy test NestaCell or placebo will be administered intravenously in three cycles of three-monthly administrations with a monthly interval between cycles total of 9 administrations The other administrations happen at visits V2 V3 V5 V6 V7 V9 V10 and V11 To assure blindness UHDRS and other clinical evaluations will be carried out in the research center by the principal investigator PI while the administrations will be made in the Center for the Investigational Product Administration CIPA The outcome evaluations by the PI and hisher team happen at V4 V8 and V12 one month after the end of each cycle In the V13 the patients will also be directed to the radiology center to repeat the MRI exam
Each patient participates in the trial for approximately 14 months two months for screening and twelve months for investigational product administration and follow-up
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| U1111-1298-5627 | OTHER | WHO | None |