Pediatric ALLPhase 2/3NCT04851717
Investigation of Remimazolam in Children Undergoing Sedation for Medical Procedures
A Phase 2/3 study testing Remimazolam in people with Pediatric ALL, run by Acacia Pharma Ltd at 8 sites in 2 countries.
Trial summary
To assess the efficacy of intravenous remimazolam in inducing and maintaining suitable sedation levels for paediatric patients undergoing diagnostic and/or therapeutic procedures Read more about this study
- Who is it for?
- Ages 0 to 17 years
- Conditions the study is for
- Pediatric ALL
- What is being tested?
- Remimazolam
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 8 sitesin 2 countries
- Study dates
- 15 Nov 2021to Oct 2027
- Phase
- Phase 2/3
- 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 0 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Signed informed consent form and/or assent and willingness of patient and parent(s) to participate in the trial.
- In US sites: Paediatric male or female patients, aged ≥3 and <18 years scheduled to undergo a diagnostic or therapeutic procedure, which is medically indicated and independent from the trial.
- In European sites: Paediatric male or female patients, aged full term birth to <18 years scheduled to undergo a diagnostic or therapeutic procedure, which is medically indicated and independent from the trial.
- Maximum planned duration of procedure: 2 hours
- ASA Physical Status I-III
- Planned spontaneous breathing during sedation
- A female who is of child bearing potential (i.e. after menarche) and sexually active must use a highly effective method of birth control during the trial period (from the time of consent until all specified observations are completed)
- Negative pregnancy test at screening and on treatment day -
Who cannot join
- Emergency procedures
- Condition/procedure that requires planned airway control via endotracheal tube or LMA/IGEL insertion
- Cranio-facial malformation, which would severely limit the possibilities for emergency airway rescue
- Other abnormalities relating to the airway (including large tonsils and anatomical abnormalities of upper airway or lower airway) which may compromise emergency airway rescue
- Known hypersensitivity to benzodiazepines, flumazenil, dextran or any of the ingredients of the drug product
- Known paradoxical reactions to benzodiazepines
Show all 14Show fewer
- History of sleep apnoea
- Active respiratory failure
- Active neuromuscular disease
- Active cardiac failure
- Active hepatic failure
- Breast feeding females
- Prohibited medication
- Any patient judged by the Principal Investigator (PI) or Sub-Investigator to be inappropriate for the trial for any other reason
Trial locations and contacts
8 sites in 2 countries.
8 sites in 2 countries
United States6 sites
Boston Children's Hospital
Boston, Massachusetts, 02115
Texas Children's Hospital
Houston, Texas, 77030
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, 55455
Stanford University
Palo Alto, California, 94306
University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
University of California Davis Children's Hospital
Sacramento, California, 95817
About this study
Official title: A Phase 2/3, Prospective, Open-label Trial Evaluating the Efficacy, Safety, and Pharmacokinetics of Remimazolam for Intravenous Sedation in Paediatric Patients Undergoing Diagnostic and/or Therapeutic Procedures
To assess the efficacy of intravenous remimazolam in inducing and maintaining suitable sedation levels for paediatric patients undergoing diagnostic and/or therapeutic procedures
Read the detailed description
This trial is part of the European Paediatric Investigational Plan and the US Pediatric Study Plan and has been developed in line with guidance from the EMA Paediatric Committee and the US FDA.
The trial will commence with cohort 1 (aged ≥6 and <18 years) and proceed to lower age groups: cohort 2 (≥3 and <6 years); and, in European sites only, cohort 3 (full-term birth to <3 years). The Data Monitoring committee (DMC) may convene any time after at least half of the subjects in cohort 1 or 2 have completed the study, to review PK,safety and efficacy data. If there are no concerns, the DMC may recommend to initiate concurrent dosing in the next younger cohort (cohort 2 or 3) in parallel to dosing the remaining subjects in cohort 1 or 2 Dosing for cohort 3 will be predicted based on PK modelling as well as efficacy and safety outcomes of the older age groups. Enrolment of patients aged <2 years will not be permitted until supported by adequate juvenile toxicity data.
The trial will consist of three visits: Screening (Day -21 to day 1),Treatment (Day 1), and Follow-up (Day 4 [+3/-1 days]).
- Conditions
- Pediatric ALL
- Being tested
- Remimazolam
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RemimazolamDrug
Remimazolam for intravenous sedation
| Study group | Role | What they receive |
|---|---|---|
| All Patients | Experimental | All paediatric patients undergoing diagnostic and/or therapeutic procedures Receives: Drug: Remimazolam |
What is being measured
The primary outcome is the main question the study is designed to answer.
Success of the procedure
Success of the procedure defined as: Completion of the procedure AND no requirement for rescue sedative medication AND no requirement for more than the permitted bolus or infusion regimen
Measured: 2 hours
Secondary outcomes (14)
Target depth of sedation achieved
Proportion of patients achieving predefined target depth of sedation (assessed using University of Michigan Sedation Score [UMSS]) during procedure
Measured: 2 hours
Target range of sedation achieved during 80% of procedure duration
Proportion of patients in whom predefined target range of sedation (assessed using University of Michigan Sedation Score [UMSS]) was achieved during at least 80% of procedure duration
Measured: 2 hours
Percentage of time within target range of sedation
Percentage of time spent by patients within predefined target range of sedation (assessed using University of Michigan Sedation Score [UMSS]) during procedure
Measured: 2 hours
Adequacy of sedation
Depth of sedation (assessed using Nurse Interpretation of Sedation Scale [NISS]) of patients over time
Measured: 2 hours
Time to start of procedure
Time between initial administration of study drug and start of procedure
Measured: 2 hours
Time to fully alert
Time between last dose of study drug, end of procedure and full alertness, defined as the first of three consecutive sedation scores showing no sedation
Measured: 2 hours
Time to ready for discharge
Time between last dose of study drug, end of procedure and discharge readiness
Measured: 2 hours
Signs of re-sedation
Occurrence, after reaching a University of Michigan Sedation Score (UMSS) of 0 after end of procedure, of a UMSS greater than zero
Measured: 2 hours
Procedure success excluding cases where the procedure could not be completed for non sedative reasons
Success of the procedure defined as: Completion of the procedure AND no requirement for rescue sedative medication AND no requirement for more than the permitted bolus or infusion regimen; excluding any patients where procedure was not completed for reasons other than failure of sedation
Measured: 2 hours
Safety: AEs
Incidence of treatment-emergent adverse events
Measured: 4 days
Safety: emergence of delirium
Incidence of paediatric anaesthesia emergence delirium between end of procedure until fit for discharge
Measured: 2 hours
Safety: need for ventilation
Incidence of use of any manual or mechanical ventilation
Measured: 2 hours
Safety: need for reversal
Incidence of use of flumazenil for reversal of benzodiazepine effect
Measured: 2 hours
PK: assessment of plasma concentration-time relationship
Graphical description of plasma concentration over time and comparison to predicted concentration-time relationship as calculated from existing pharmacokinetic/pharmacodynamic model
Measured: 3.5 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Apr 2021
- Study start15 Nov 2021
- Primary completionOct 2027 (estimated)
- Study completionOct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pediatric ALLStudies 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 2/3
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- A Phase 2/3 prospective, open-label trial
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Acacia Pharma Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Paion UK Ltd.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04851717
- Sponsor's protocol ID
- DA10030
- Other IDs
- CNS7056-026
- Status last verified
- Jun 2025
- First submitted
- 14 Apr 2021
- Last updated
- 1 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2/3, Prospective, Open-label Trial Evaluating the Efficacy, Safety, and Pharmacokinetics of Remimazolam for Intravenous Sedation in Paediatric Patients Undergoing Diagnostic and/or Therapeutic Procedures
- ClinicalTrials.gov ID
- NCT04851717
- Acronym / sponsor ID
- DA10030
- Conditions
- Pediatric ALL
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Acacia Pharma LtdIndustry (pharma, biotech, devices)
- Collaborators
- Paion UK Ltd.
- Study start
- 15 Nov 2021 (actual)
- Primary completion
- Oct 2027 (estimated)
- Study completion
- Oct 2027 (estimated)
- First posted
- 20 Apr 2021 (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.
To assess the efficacy of intravenous remimazolam in inducing and maintaining suitable sedation levels for paediatric patients undergoing diagnostic and/or therapeutic procedures
Detailed description
This trial is part of the European Paediatric Investigational Plan and the US Pediatric Study Plan and has been developed in line with guidance from the EMA Paediatric Committee and the US FDA.
The trial will commence with cohort 1 (aged ≥6 and <18 years) and proceed to lower age groups: cohort 2 (≥3 and <6 years); and, in European sites only, cohort 3 (full-term birth to <3 years). The Data Monitoring committee (DMC) may convene any time after at least half of the subjects in cohort 1 or 2 have completed the study, to review PK,safety and efficacy data. If there are no concerns, the DMC may recommend to initiate concurrent dosing in the next younger cohort (cohort 2 or 3) in parallel to dosing the remaining subjects in cohort 1 or 2 Dosing for cohort 3 will be predicted based on PK modelling as well as efficacy and safety outcomes of the older age groups. Enrolment of patients aged <2 years will not be permitted until supported by adequate juvenile toxicity data.
The trial will consist of three visits: Screening (Day -21 to day 1),Treatment (Day 1), and Follow-up (Day 4 [+3/-1 days]).
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- A Phase 2/3 prospective, open-label trial
| Arm | Type | Receives |
|---|---|---|
| All Patients | Experimental | Drug: RemimazolamAll paediatric patients undergoing diagnostic and/or therapeutic procedures |
Who may qualify
- Ages
- 0 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- Signed informed consent form and/or assent and willingness of patient and parent(s) to participate in the trial.
- In US sites: Paediatric male or female patients, aged ≥3 and <18 years scheduled to undergo a diagnostic or therapeutic procedure, which is medically indicated and independent from the trial.
- In European sites: Paediatric male or female patients, aged full term birth to <18 years scheduled to undergo a diagnostic or therapeutic procedure, which is medically indicated and independent from the trial.
- Maximum planned duration of procedure: 2 hours
- ASA Physical Status I-III
Key exclusion criteria (14 listed)
- Emergency procedures
- Condition/procedure that requires planned airway control via endotracheal tube or LMA/IGEL insertion
- Cranio-facial malformation, which would severely limit the possibilities for emergency airway rescue
- Other abnormalities relating to the airway (including large tonsils and anatomical abnormalities of upper airway or lower airway) which may compromise emergency airway rescue
- Known hypersensitivity to benzodiazepines, flumazenil, dextran or any of the ingredients of the drug product
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 |
|---|---|---|
| RemimazolamRemimazolam for intravenous sedation | Drug | All Patients |
Primary outcome
Success of the procedure
Time frame: 2 hours
Success of the procedure defined as: Completion of the procedure AND no requirement for rescue sedative medication AND no requirement for more than the permitted bolus or infusion regimen
14 secondary outcomes
Target depth of sedation achieved
Time frame: 2 hours
Target range of sedation achieved during 80% of procedure duration
Time frame: 2 hours
Percentage of time within target range of sedation
Time frame: 2 hours
Adequacy of sedation
Time frame: 2 hours
Time to start of procedure
Time frame: 2 hours
Time to fully alert
Time frame: 2 hours
Time to ready for discharge
Time frame: 2 hours
Signs of re-sedation
Time frame: 2 hours
Procedure success excluding cases where the procedure could not be completed for non sedative reasons
Time frame: 2 hours
Safety: AEs
Time frame: 4 days
Safety: emergence of delirium
Time frame: 2 hours
Safety: need for ventilation
Time frame: 2 hours
Safety: need for reversal
Time frame: 2 hours
PK: assessment of plasma concentration-time relationship
Time frame: 3.5 hours
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 1 Jul 2025.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
8 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Remimazolam in people with Pediatric ALL, run by Acacia Pharma Ltd at 8 sites in 2 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 8 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: under way, not looking for participants, as of the sponsor's last update on 1 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States, Denmark. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Success of the procedure (time frame: 2 hours). 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 1 Jul 2025.