GRIN-related DisordersPhase 1NCT05818943
Honeycomb: Evaluation of Radiprodil in Children with GRIN-related Disorder
A Phase 1 study testing Radiprodil in people with GRIN-related Disorders, run by GRIN Therapeutics, Inc. at 15 sites in 8 countries.
Trial summary
Study RAD-GRIN-101 is a phase 1B trial to assess safety, tolerability, PK, and potential efficacy of radiprodil for the treatment of GRIN-related disorder in children with a Gain-of-Function (GoF) genetic variant. The study is open-label, so all participants will be treated with radiprodil. Read more about this study
- Who is it for?
- Ages 6 months to 12 years
- Conditions the study is for
- GRIN-related Disorders
- What is being tested?
- Radiprodil
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 15 sitesin 8 countries
- Study dates
- 7 Mar 2023to Nov 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 6 months to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age: ≥6 months to ≤12 years, with GRIN gene variants known to result in GoF of the NMDA receptor.
- Cohort 1 must have at least 1 observable motor seizure per week and ≥4 observable motor seizures (generalized or focal) during the prospective 4-week Observation Period and must have failed to obtain adequate seizure control with at least 2 antiseizure medications (ASMs) used at appropriate dose and duration.
- Cohort 2 must have significant behavioral and/or motor symptoms based on caregiver report with a CGI-S score ≥4.
- Stable antiseizure therapies and nonpharmacological treatments such as ketogenic diet throughout screening and study participation.
Who cannot join
- Any other clinically relevant medical, neurologic, or psychiatric condition and/or behavioral disorder unrelated to GRIN-related disorder that would preclude or jeopardize participant's safe participation or the conduct of the study according to the judgement of the investigator.
- Clinically significant laboratory or ECG abnormalities.
- Severe hepatic dysfunction (Child-Pugh grade C).
- History of brain surgery for epilepsy or any other reason.
- Receiving treatment with contraindicated concomitant drugs such as agonists or antagonists of the glutamate receptor, including but not limited to felbamate, memantine, and perampanel.
- Receiving treatment with hormonal therapy such as adrenocorticotrophic hormone or prednisolone.
Trial locations and contacts
15 sites in 8 countries.
15 sites in 8 countries
United States2 sites
Mid-Atlantic Epilepsy and Sleep Center
Bethesda, Maryland, 20817
Columbia University Irving Medical Center, Dept of Neurology
New York, New York, 10032
Canada2 sites
The Hospital for Sick Children (Sick Kids)
Toronto, Ontario, M5G 1X8
BC Children's Hospital
Vancouver, BC V6H 3N1
Germany2 sites
Abteilung für Neuropädiatrie, Klinik und Poliklinik für Kinder - und Jugendmedizin, Universitätsklinikum Leipzig
Leipzig, 04103
KBO-Kinderzentrum München gemeinnützige GmbH
München, 81377
Italy2 sites
Azienda Ospedaliero Universitaria Careggi (AOUC) Firenze - Azienda Ospedaliera Universitaria Meyer
Florence, Tuscany, 50139
Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale Pediatrico Bambino Gesu
Rome, Lazio, 00165
Netherlands2 sites
ERASMUS Medisch Centrum, Developmental & Genetic pediatrics
Rotterdam, 3015
UMC Utrecht - Wilhelmina Kinderziekenhuis, Polikliniek Kinderneurologie
Utrecht, 3508
Spain2 sites
Universitat de Barcelona - Hospital Sant Joan de Deu Barcelona (HSJDB)
Barcelona, 08950
Hospital Ruber Internacional
Madrid, 28034
United Kingdom2 sites
Royal Hospital for Children Glasgow
Glasgow, G51 4TF
Great Ormond Street Hospital For Children NHS Foundation Trust
London, WC1N 3JH
Australia1 site
Queensland Children's Hospital
South Brisbane, Queensland, 4101
About this study
Official title: A Multicenter Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms of Multiple Individually Titrated Doses of Radiprodil in Children with GRIN-related Disorder
Study RAD-GRIN-101 is a phase 1B trial to assess safety, tolerability, PK, and potential efficacy of radiprodil for the treatment of GRIN-related disorder in children with a Gain-of-Function (GoF) genetic variant. The study is open-label, so all participants will be treated with radiprodil.
Subjects' participation in the study is expected to last up to six months in Part A.
After the end of part A, all participants who are still eligible can choose to continue to receive radiprodil as part of an open-label long-term treatment period (Part B).
Read the detailed description
The effect of radiprodil is assessed in two (2) cohorts of pediatric participants: one (1) cohort of participants with treatment-resistant seizures (with or without behavioral symptoms) (Cohort 1) and one (1) cohort of participants with behavioral symptoms but no qualifying seizures (Cohort 2) caused by Gain-of-Function (GoF) variants in the GRIN gene. As the daily doses of radiprodil will be individually titrated for every participant and all the participants will receive the study drug, this is in effect a "single group" study.
This study is divided into the following periods:
PART A:
- Screening/Observation Period (35 days): Investigators assess eligibility followed by a four(4)- week Observation Period to evaluate seizure frequency and/or behavioral symptoms.
- Titration Period (approx. 51 days): Overnight stay to administer radiprodil twice daily to assigned dose level, assessing plasma concentrations, safety, and tolerability during the titration period. Once a safe and potentially effective dose has been established, the participant will immediately enter the Maintenance Period.
- Maintenance Period (up to 53 days): During the Maintenance Period, the participant will continue to take the highest safe and potentially effective dose, as identified during the Titration Period. At the end of the Maintenance Period, there will be an additional overnight stay when the participant will either be invited to take part in Part B or enter the Tapering and Safety Follow-up Period.
- Tapering (15 days) and Safety Follow-up Period (14 days): the participant who doesn't take part in the long-term treatment period (Part B) will need to taper off (ie gradually decrease) the study medicine for 15 days and enter a safety Follow-up Period (14 days). In this case, the participant will make one (1) last visit to the study site 14 days after his/her last dose of radiprodil.
PART B:
- Long-Term Treatment Period (not specified): Participation in Part B of the study, at the dose established during part A, will be continued until such time as either the participant withdraws/is withdrawn from the study or sponsor terminates the study. During this period there will be four (4) visits per year, two(2) of which will require overnight stays. At the end of the Long-Term Treatment Period, the participant will enter the Tapering and Safety Follow-up Period.
- Tapering (15 days) and Safety Follow-up Period (14 days): the participant will need to taper off the study medicine for 15 days and enter a safety Follow-up Period of 14 days. In this case, the participant will make one (1) last visit to the study site 14 days after his/her last dose of radiprodil.
- Conditions
- GRIN-related Disorders
- Being tested
- Radiprodil
- Keywords
- GRIN1, GRIN2A, GRIN2B, GRIN2D
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RadiprodilDrug
Radiprodil is an orally active, negative allosteric modulator of the NR2B subunit of the NMDA receptor.
| Study group | Role | What they receive |
|---|---|---|
| Radiprodil | Experimental | Liquid suspension of radiprodil, at concentrations 0.25 mg/mL or 2.50 mg/mL for 1% and 10% formulation respectively. It will be administered twice a day (bid) either orally or via gastric or nasogastric tube, slowly up-titrated to the highest tolerated dose. Receives: Drug: Radiprodil |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs (SAEs), TEAEs Leading to Discontinuation and Severity of TEAEs
Frequency, type, severity and duration of adverse events, serious adverse events and adverse drug reactions.
Measured: through study completion (average of 2 years).
Maximum Plasma concentration of radiprodil (Cmax)
Measured: Titration Visit 1 (week 5): 0,1,2,4,6,8,10,12 hours post-dose. Titration Visits 2,3,4 (week 6 to 11) and Maintenance Visit 4 (week 20): 0,1,2,5 hours post-dose.
Pharmacokinetic plasma concentration of radiprodil: half-life (T1/2)
Measured: Titration Visit 1 (week 5): 0,1,2,4,6,8,10,12 hours post-dose. Titration Visits 2,3,4 (week 6 to 11) and Maintenance Visit 4 (week 20): 0,1,2,5 hours post-dose.
Plasma concentration of radiprodil versus time, area under the curve (AUCt)
Measured: Titration Visit 1 (week 5): 0,1,2,4,6,8,10,12 hours post-dose. Titration Visits 2,3,4 (week 6 to 11) and Maintenance Visit 4 (week 20): 0,1,2,5 hours post-dose.
Pharmacokinetic plasma concentration of radiprodil, clearance (Cl)
Measured: Titration Visit 1 (week 5): 0,1,2,4,6,8,10,12 hours post-dose. Titration Visits 2,3,4 (week 6 to 11) and Maintenance Visit 4 (week 20): 0,1,2,5 hours post-dose.
Pharmacokinetic plasma concentration of radiprodil, Time corresponding to occurrence of Cmax (Tmax)
Measured: Titration Visit 1 (week 5): 0,1,2,4,6,8,10,12 hours post-dose. Titration Visits 2,3,4 (week 6 to 11) and Maintenance Visit 4 (week 20): 0,1,2,5 hours post-dose.
Secondary outcomes (10)
Percent change from baseline in V-EEG seizure burden
Assessed by 8- to 24- hour video electroencephalogram
Measured: Baseline (week 5) to study completion (average of 2 years).
Change from baseline in seizure frequency
Assessed by seizure diaries
Measured: Baseline (week 5) to study completion (average of 2 years).
Aberrant Behavior Checklist-Community (ABC-C)
The ABCC is a standardized 58-item caregiver-reported problem-behavior rating scale, originally designed to assess treatment effects in people with intellectual disabilities. Each item is scored from 0 (never a problem) to 3 (severe problem). Items load onto one of five empirically derived subscales: Irritability, Agitation, \& Crying (15 items); Lethargy/Social Withdrawal (16 items); Stereotypic Behavior (7 items); Hyperactivity/Noncompliance (16 items); and Inappropriate Speech (4 items). A total score would range from 0 to 174.
Measured: Baseline (week 5) to study completion (average of 2 years).
Caregiver Global Impression of Change (CaGI-C)
The CaGI-C is a 7-point caregiver-rated scale ranging from 1 (very much improved) to 7 (very much worse).
Measured: Baseline (week 5) to study completion (average of 2 years).
Change from Baseline in Clinical Global Impression - Severity [CGI-S]
The CGI-S scale is a clinician-rated measures of severity of a symptom or condition, using a single item, 6- or 7-point scale. The CGI-S scale ranges from 1 ("None") to 6 ("Very Severe").
Measured: Baseline (week 5) to study completion (average of 2 years).
Clinical Global Impression of Change [CGI-C]
The CGI scale is a clinician-rated measures of change of a symptom or condition, using a single item, 6- or 7-point scale. The CGI-C scale ranges from 1 ("Very much worse") to 7 ("Very much improved").
Measured: Baseline (week 5) to study completion (average of 2 years).
Gross Motor Function Measure (GMFM)
The GMFM is a standardized observational instrument designed and validated to measure change in gross motor function over time in children with cerebral palsy. There is a 4-point (0 to 3) scoring system for each of the 5 dimensions of gross motor function, with higher scores denoting better performance.
Measured: Baseline (week 5) to study completion (average of 2 years).
Sleep Disturbance Scale for Children (SDSC)
The SDSC is a parent-report measure to screen for sleep disturbances within the preceding period. It is a 27-item questionnaire rated on a 5-point Likert scale, with higher scores being indicative of more acute sleep disturbances.
Measured: Baseline (week 5) to study completion (average of 2 years).
Pediatric Quality of Life Inventory [PedsQL]
The PedsQL is a 23-item generic health status instrument assessing 5 domains of health in children. It's a 0-100 scale, and higher scores are indicative of better health-related quality of life.
Measured: Baseline (week 5) to study completion (average of 2 years).
Caregiver Burden Inventory (CBI)
The CBI is a validated scale providing information regarding the impact of caregiving on the lives of caregivers. It comprises 24 closed questions divided into 5 dimensions. Each dimension includes 4 or 5 items. Each item is given a score between 0 and 4, where higher scores indicate greater caregiver burden.
Measured: Baseline (week 5) to study completion (average of 2 years).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2023
- Study start7 Mar 2023
- Primary completionNov 2026 (estimated)
- Study completionNov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for GRIN-related DisordersStudies 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
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- GRIN Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Vijay Rai, PhD, Study director, Associate Director of Clinical OperationsMichael Panzara, MD, MPH, Study chair, Chief Medical Officer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05818943
- Sponsor's protocol ID
- RAD-GRIN-101
- Other IDs
- 2022-000317-14
- Status last verified
- Nov 2024
- First submitted
- 22 Feb 2023
- Last updated
- 15 Nov 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 15 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms of Multiple Individually Titrated Doses of Radiprodil in Children with GRIN-related Disorder
- ClinicalTrials.gov ID
- NCT05818943
- Acronym / sponsor ID
- RAD-GRIN-101
- Conditions
- GRIN-related Disorders
- Keywords
- GRIN1, GRIN2A, GRIN2B, GRIN2D
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- GRIN Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 7 Mar 2023 (actual)
- Primary completion
- Nov 2026 (estimated)
- Study completion
- Nov 2026 (estimated)
- First posted
- 19 Apr 2023 (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.
Study RAD-GRIN-101 is a phase 1B trial to assess safety, tolerability, PK, and potential efficacy of radiprodil for the treatment of GRIN-related disorder in children with a Gain-of-Function (GoF) genetic variant. The study is open-label, so all participants will be treated with radiprodil.
Subjects' participation in the study is expected to last up to six months in Part A.
After the end of part A, all participants who are still eligible can choose to continue to receive radiprodil as part of an open-label long-term treatment period (Part B).
Detailed description
The effect of radiprodil is assessed in two (2) cohorts of pediatric participants: one (1) cohort of participants with treatment-resistant seizures (with or without behavioral symptoms) (Cohort 1) and one (1) cohort of participants with behavioral symptoms but no qualifying seizures (Cohort 2) caused by Gain-of-Function (GoF) variants in the GRIN gene. As the daily doses of radiprodil will be individually titrated for every participant and all the participants will receive the study drug, this is in effect a "single group" study.
This study is divided into the following periods:
PART A:
- Screening/Observation Period (35 days): Investigators assess eligibility followed by a four(4)- week Observation Period to evaluate seizure frequency and/or behavioral symptoms.
- Titration Period (approx. 51 days): Overnight stay to administer radiprodil twice daily to assigned dose level, assessing plasma concentrations, safety, and tolerability during the titration period. Once a safe and potentially effective dose has been established, the participant will immediately enter the Maintenance Period.
- Maintenance Period (up to 53 days): During the Maintenance Period, the participant will continue to take the highest safe and potentially effective dose, as identified during the Titration Period. At the end of the Maintenance Period, there will be an additional overnight stay when the participant will either be invited to take part in Part B or enter the Tapering and Safety Follow-up Period.
- Tapering (15 days) and Safety Follow-up Period (14 days): the participant who doesn't take part in the long-term treatment period (Part B) will need to taper off (ie gradually decrease) the study medicine for 15 days and enter a safety Follow-up Period (14 days). In this case, the participant will make one (1) last visit to the study site 14 days after his/her last dose of radiprodil.
PART B:
- Long-Term Treatment Period (not specified): Participation in Part B of the study, at the dose established during part A, will be continued until such time as either the participant withdraws/is withdrawn from the study or sponsor terminates the study. During this period there will be four (4) visits per year, two(2) of which will require overnight stays. At the end of the Long-Term Treatment Period, the participant will enter the Tapering and Safety Follow-up Period.
- Tapering (15 days) and Safety Follow-up Period (14 days): the participant will need to taper off the study medicine for 15 days and enter a safety Follow-up Period of 14 days. In this case, the participant will make one (1) last visit to the study site 14 days after his/her last dose of radiprodil.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Radiprodil | Experimental | Drug: RadiprodilLiquid suspension of radiprodil, at concentrations 0.25 mg/mL or 2.50 mg/mL for 1% and 10% formulation respectively. It will be administered twice a day (bid) either orally or via gastric or nasogastric tube, slowly up-titrated to the highest tolerated dose. |
Who may qualify
- Ages
- 6 months to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Age: ≥6 months to ≤12 years, with GRIN gene variants known to result in GoF of the NMDA receptor.
- Cohort 1 must have at least 1 observable motor seizure per week and ≥4 observable motor seizures (generalized or focal) during the prospective 4-week Observation Period and must have failed to obtain adequate seizure control with at least 2 antiseizure medications (ASMs) used at appropriate dose and duration.
- Cohort 2 must have significant behavioral and/or motor symptoms based on caregiver report with a CGI-S score ≥4.
- Stable antiseizure therapies and nonpharmacological treatments such as ketogenic diet throughout screening and study participation.
Key exclusion criteria (6 listed)
- Any other clinically relevant medical, neurologic, or psychiatric condition and/or behavioral disorder unrelated to GRIN-related disorder that would preclude or jeopardize participant's safe participation or the conduct of the study according to the judgement of the investigator.
- Clinically significant laboratory or ECG abnormalities.
- Severe hepatic dysfunction (Child-Pugh grade C).
- History of brain surgery for epilepsy or any other reason.
- Receiving treatment with contraindicated concomitant drugs such as agonists or antagonists of the glutamate receptor, including but not limited to felbamate, memantine, and perampanel.
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 |
|---|---|---|
| RadiprodilRadiprodil is an orally active, negative allosteric modulator of the NR2B subunit of the NMDA receptor. | Drug | Radiprodil |
Primary outcome
Incidence of Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs (SAEs), TEAEs Leading to Discontinuation and Severity of TEAEs
Time frame: through study completion (average of 2 years).
Frequency, type, severity and duration of adverse events, serious adverse events and adverse drug reactions.
Maximum Plasma concentration of radiprodil (Cmax)
Time frame: Titration Visit 1 (week 5): 0,1,2,4,6,8,10,12 hours post-dose. Titration Visits 2,3,4 (week 6 to 11) and Maintenance Visit 4 (week 20): 0,1,2,5 hours post-dose.
Pharmacokinetic plasma concentration of radiprodil: half-life (T1/2)
Time frame: Titration Visit 1 (week 5): 0,1,2,4,6,8,10,12 hours post-dose. Titration Visits 2,3,4 (week 6 to 11) and Maintenance Visit 4 (week 20): 0,1,2,5 hours post-dose.
Plasma concentration of radiprodil versus time, area under the curve (AUCt)
Time frame: Titration Visit 1 (week 5): 0,1,2,4,6,8,10,12 hours post-dose. Titration Visits 2,3,4 (week 6 to 11) and Maintenance Visit 4 (week 20): 0,1,2,5 hours post-dose.
Pharmacokinetic plasma concentration of radiprodil, clearance (Cl)
Time frame: Titration Visit 1 (week 5): 0,1,2,4,6,8,10,12 hours post-dose. Titration Visits 2,3,4 (week 6 to 11) and Maintenance Visit 4 (week 20): 0,1,2,5 hours post-dose.
Pharmacokinetic plasma concentration of radiprodil, Time corresponding to occurrence of Cmax (Tmax)
Time frame: Titration Visit 1 (week 5): 0,1,2,4,6,8,10,12 hours post-dose. Titration Visits 2,3,4 (week 6 to 11) and Maintenance Visit 4 (week 20): 0,1,2,5 hours post-dose.
10 secondary outcomes
Percent change from baseline in V-EEG seizure burden
Time frame: Baseline (week 5) to study completion (average of 2 years).
Change from baseline in seizure frequency
Time frame: Baseline (week 5) to study completion (average of 2 years).
Aberrant Behavior Checklist-Community (ABC-C)
Time frame: Baseline (week 5) to study completion (average of 2 years).
Caregiver Global Impression of Change (CaGI-C)
Time frame: Baseline (week 5) to study completion (average of 2 years).
Change from Baseline in Clinical Global Impression - Severity [CGI-S]
Time frame: Baseline (week 5) to study completion (average of 2 years).
Clinical Global Impression of Change [CGI-C]
Time frame: Baseline (week 5) to study completion (average of 2 years).
Gross Motor Function Measure (GMFM)
Time frame: Baseline (week 5) to study completion (average of 2 years).
Sleep Disturbance Scale for Children (SDSC)
Time frame: Baseline (week 5) to study completion (average of 2 years).
Pediatric Quality of Life Inventory [PedsQL]
Time frame: Baseline (week 5) to study completion (average of 2 years).
Caregiver Burden Inventory (CBI)
Time frame: Baseline (week 5) to study completion (average of 2 years).
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Nov 2024.
- Individual participant dataSharing planned: Undecided
Related trials for GRIN-related Disorders
RecruitingPhase 3
Beeline: A Phase 3 Study in GRIN-related Neurodevelopmental Disorder
GRIN-related Neurodevelopmental Disorder
RecruitingPhase 3
TAP-GRIN: Interventional Study on Patients With GRIN-related Neurodevelopmental Disorders
GRIN-related Disorders, GRIN1
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.
Vijay Rai, PhD study director
Associate Director of Clinical Operations
Michael Panzara, MD, MPH study chair
Chief Medical Officer
15 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Radiprodil in people with GRIN-related Disorders, run by GRIN Therapeutics, Inc. at 15 sites in 8 countries.
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 4 inclusion and 6 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: under way, not looking for participants, as of the sponsor's last update on 15 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, Canada, Germany, Italy, Netherlands and 3 more countries. 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?
This study is still under way. Results are not available in the source record yet.
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?
Incidence of Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs (SAEs), TEAEs Leading to Discontinuation and Severity of TEAEs (time frame: through study completion (average of 2 years).). The study lists 6 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 15 Nov 2024.