CDKL5 Deficiency DisorderPhase 3NCT05249556
Open-label, Single-arm Trial of Ganaxolone in CDD Patients 6 Months to Less Than 2 Years Old
A Phase 3 study testing Ganaxolone in people with CDKL5 Deficiency Disorder, run by Immedica Pharma AB.
Trial summary
The goal of this clinical trial is to learn how patients with CDKL-5 deficiency disorder (CDD) aged 6 months to 2 years of age respond to treatment with ganaxolone. The main questions the trial aims to answer are: The amount of the medicine that was in participants' bodies and the safety of the medicine. Read more about this study
- Who is it for?
- Ages 6 months to 2 years
- Conditions the study is for
- CDKL5 Deficiency Disorder
- What is being tested?
- Ganaxolone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- No sites listed yet
- Study dates
- Mar 2027to Dec 2029
- Phase
- Phase 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 6 months to 2 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- A diagnosis of CDD, including molecular confirmation of a pathogenic or likely pathogenic CDKL5 variant.
- Male or female participants aged 6 months to less than 2 years at the Baseline Visit.
- Failure to control seizures despite appropriate use of 1 or more anti-seizure medications at therapeutic doses. At least 3 participants will be included who failed to control seizures despite appropriate use of at least 2 anti-seizure medications.
- Participants will have refractory seizures with a history of at least 8 countable seizures of primary seizure types during the 28 days prospective Baseline Period prior to the Baseline Visit. Primary seizure types are seizures with or without impairment of consciousness with a clear motor component, including generalized tonic-clonic, focal to bilateral tonic-clonic, atonic/drop, bilateral clonic, bilateral tonic (sustained motor activity ≥ 3 seconds). Clusters of uncountable seizures will be counted as a single seizure.
- Primary seizures exclude epileptic spasms. Patients with epileptic spasms are eligible for enrolment but epileptic spasms will not be counted as qualifying countable seizures for eligibility. Epileptic spasms should not constitute the dominant seizure type during the Baseline Period.
- Primary seizures exclude myoclonic, absence, generalized non-motor seizures, and focal motor or non-motor seizures with or without impairment of awareness.
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- Participants should be receiving a stable regimen of anti-seizure medications at Screening through to the end of the 4-week prospective Baseline Period and have no foreseeable change for the duration of Part A.
- Concurrent use of adrenocorticotropic hormone, prednisone, other glucocorticoids, or vigabatrin are permitted.
- Vagus nerve stimulator (VNS) settings, ketogenic diet, or a modified Atkins diet should be unchanged for 1 month prior to the Screening Visit, throughout the Screening Period and the 4-week prospective Baseline Period and without foreseeable change for the duration of Part A at the time of the Baseline Visit.
- Parent/caregiver is able and willing to maintain an accurate and complete daily seizure diary for the duration of the trial.
Who cannot join
- Have an active CNS infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive as evaluated by brain imaging (magnetic resonance imaging).
- Have any disease or condition (medical or surgical; other than CDKL5 deficiency) that might compromise the hematologic, cardiovascular, pulmonary, renal, gastrointestinal, or hepatic systems; or other conditions that might interfere with the absorption, distribution, metabolism, or excretion of the investigational product or would place the participant at increased risk.
- History of or present AST (aspartate aminotransferase/serum glutamic-oxaloacetic transaminase) or ALT (alanine aminotransferase/serum glutamic-pyruvic transaminase) >3 times the upper limit of normal (ULN) at Baseline and confirmed by a repeat testing.
- History of or present total bilirubin levels greater than the ULN at trial Baseline and confirmed by a repeat testing. In cases of a documented, stable medical condition (i.e., Gilbert's Syndrome) resulting in levels of total bilirubin greater than the ULN, the Medical Monitor will determine if an exception can be made.
- Participants with significant renal insufficiency, with an estimated glomerular filtration rate (eGFR) <30 mL/min (calculated using the Cockcroft-Gault formula or Pediatric eGFR calculator or Bedside Schwartz).
- Regular use of St. John's Wort or strong CYP3A4 inducers, e.g., phenytoin, carbamazepine or phenobarbital (N.B. phenytoin and phenobarbital are permitted as rescue medication on as-need basis but are not permitted as continuous maintenance medication).
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- Have been exposed to any other investigational drug within 30 days or less than its 5 half-lives prior to the Screening Visit.
- Previous treatment with ganaxolone.
- Participation in any other trial involving administration of an investigational medication or device.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
Clinical Project Manager contact
About this study
Official title: An Open-label, Single-arm, 52-week, Multicenter Phase 3 Trial to Assess Pharmacokinetics, Safety, Tolerability, and Activity of Adjunctive Ganaxolone for the Treatment of Seizures Associated With Genetically Confirmed Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) in Pediatric Patients From 6 Months to Less Than 2 Years of Age
The goal of this clinical trial is to learn how patients with CDKL-5 deficiency disorder (CDD) aged 6 months to 2 years of age respond to treatment with ganaxolone. The main questions the trial aims to answer are:
- The amount of the medicine that was in participants' bodies and the safety of the medicine.
- Does the medicine ganaxolone lower the number of seizures when used in addition to the participant's usual anti-seizure treatment.
Read more
Participants will:
Take ganaxolone 3 times daily for up to 52 weeks. Visit the clinic every 3 to 5 weeks for the initial 17 weeks of the trial and thereafter every 11 to 12 weeks.
The participant's caregiver will:
Keep a daily diary of the participants seizures and use of anti-seizure medications, including the trial drug ganaxolone.
Read the detailed description
This is an open-label, single-arm, 52-week, multicenter trial of adjunctive ganaxolone treatment in participants 6 months to less than 2 years of age with genetically confirmed CDD. The trial consists of an up to 2-week Screening Period, 4-week prospective Baseline Period, followed by 2 parts: Part A; a 17-week ganaxolone treatment phase (including 4 to 5 weeks of ganaxolone titration followed by 12 to 13 weeks of dose maintenance) and Part B; a 35-week ganaxolone maintenance phase, and a 2-week taper period at the end of the trial. Tapering will not apply for participants continuing ganaxolone treatment after the trial.
The Screening Period will follow the Screening Visit to ensure caregivers can classify seizures and comply with completion instructions of an electronic seizure and medication diary. This will be followed by a 4-week prospective Baseline Period to assess seizure frequency for trial eligibility.
Up to 15 participants will be enrolled to ensure at least 12 participants are evaluable for the primary PK endpoints at the end of Part A of the trial, including at least 3 participants experiencing uncontrolled seizures despite treatment with at least 2 anti-seizure medications.
Participants will be monitored at Baseline and at specified visits to assess the exposure, safety and tolerability and activity of ganaxolone. PK samples will be collected at specified timepoints up until completion of Part A.
Clinical laboratory testing will be performed at local laboratories at participating sites.
- Conditions
- CDKL5 Deficiency Disorder
- Being tested
- Ganaxolone
- Keywords
- CDKL5 deficiency disorder (CDD), Developmental and epileptic encephalopathy (DEE)
- Medical subject headings
- CDKL5 deficiency disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GanaxoloneDrug
Also called Ztalmy, Active Drug
Ganaxolone
| Study group | Role | What they receive |
|---|---|---|
| Ganaxolone | Experimental | Ganaxolone sodium benzoate-free oral suspension 50 mg/ml Receives: Drug: Ganaxolone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Ganaxolone exposure in participants receiving ganaxolone doses up to 63 mg/kg/day
Pharmacokinetic (PK) exposure parameter derived from population PK modelling and simulation: steady-state maximum concentration (Cmax,ss)
Measured: Baseline through 17 weeks
Ganaxolone exposure in participants receiving ganaxolone doses up to 63 mg/kg/day
PK exposure parameter derived from population PK modelling and simulation: trough concentration (Ctrough)
Measured: Baseline through 17 weeks
Ganaxolone exposure in participants receiving ganaxolone doses up to 63 mg/kg/day
PK exposure parameter derived from population PK modelling and simulation: systemic exposure (Caverage,ss derived as AUC over a dosing interval / tau)
Measured: Baseline through 17 weeks
Safety and tolerability of ganaxolone as adjunctive therapy for the treatment of seizures in participants receiving ganaxolone doses up to 63 mg/kg/day
Frequency, type, and severity of adverse events (AEs)
Measured: Baseline through to 52 weeks
Secondary outcomes (15)
Ganaxolone PK parameters at steady-state in participants receiving ganaxolone doses up to 63 mg/kg/day.
Non-compartmental-like steady-state PK parameters derived from population PK modelling and simulation: steady-state time to maximum concentration (tmax,ss)
Measured: Baseline through to 17 weeks
Ganaxolone PK parameters at steady-state in participants receiving ganaxolone doses up to 63 mg/kg/day.
Non-compartmental-like steady-state PK parameters derived from population PK modelling and simulation: exposure accumulation ratios
Measured: Baseline through to 17 weeks
Ganaxolone PK parameters at steady-state in participants receiving ganaxolone doses up to 63 mg/kg/day.
Non-compartmental-like steady-state PK parameters derived from population PK modelling and simulation: swing [(Cmax-Cmin)/Cmin])
Measured: Baseline through to 17 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 17-week treatment period (Part A)
Percent change from baseline in 28-day primary seizure frequency during the treatment period relative to the 4-week prospective Baseline Phase
Measured: Baseline through to 17 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 17-week treatment period (Part A)
Percentage of participants experiencing ≥50% reduction in the 28-day primary seizure frequency compared to the 4-week baseline
Measured: Baseline through to 17 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 17-week treatment period (Part A)
Percent change in seizures by primary seizure type
Measured: Baseline through to 17 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 17-week treatment period (Part A)
Percent change in primary seizure-free days
Measured: Baseline through to 17 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 17-week treatment period (Part A)
Clinical Global Impression of Improvement (CGI-I); 7-point Likert scale, higher scores mean a worse outcome
Measured: Baseline through to 17 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 17-week treatment period (Part A)
Caregiver Global Impression of Change in Seizure Intensity/Duration (CGI-CSID); 7-point Likert scale, higher scores mean a worse outcome
Measured: Baseline through to 17 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 52-week treatment period (Part B)
Percent change from baseline in 28-day primary seizure frequency during the treatment period relative to the 4-week prospective Baseline Phase
Measured: Baseline through to 52 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 52-week treatment period (Part B)
Percentage of participants experiencing ≥50% reduction in the 28-day primary seizure frequency compared to the 4-week baseline
Measured: Baseline through to 52 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 52-week treatment period (Part B)
Percent change in seizures by primary seizure type
Measured: Baseline through to 52 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 52-week treatment period (Part B)
Percent change in primary seizure-free days
Measured: Baseline through to 52 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 52-week treatment period (Part B)
Clinical Global Impression of Improvement (CGI-I); 7-point Likert scale, higher scores mean a worse outcome
Measured: Baseline through to 52 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 52-week treatment period (Part B)
Caregiver Global Impression of Change in Seizure Intensity/Duration (CGI-CSID); 7-point Likert scale, higher scores mean a worse outcome
Measured: Baseline through to 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2022
- Study startMar 2027 (estimated)
- Primary completionDec 2029 (estimated)
- Study completionDec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for CDKL5 Deficiency DisorderStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This is an open-label, single-arm trial
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Immedica Pharma AB
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05249556
- Sponsor's protocol ID
- 1042-CDD-3002
- Other IDs
- 2026-528611-26-00
- Status last verified
- Oct 2026
- First submitted
- 27 Jan 2022
- Last updated
- 4 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Yasmen N, Sluter MN, Yu Y, Jiang J. Ganaxolone for management of seizures associated with CDKL5 deficiency disorder. Trends Pharmacol Sci. 2023 Feb;44(2):128-129. doi: 10.1016/j.tips.2022.11.007. Epub 2022 Dec 12. No abstract available.
PubMed 36517284
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Single-arm, 52-week, Multicenter Phase 3 Trial to Assess Pharmacokinetics, Safety, Tolerability, and Activity of Adjunctive Ganaxolone for the Treatment of Seizures Associated With Genetically Confirmed Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) in Pediatric Patients From 6 Months to Less Than 2 Years of Age
- ClinicalTrials.gov ID
- NCT05249556
- Acronym / sponsor ID
- 1042-CDD-3002
- Conditions
- CDKL5 Deficiency Disorder
- Keywords
- CDKL5 deficiency disorder (CDD), Developmental and epileptic encephalopathy (DEE)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Immedica Pharma ABIndustry (pharma, biotech, devices)
- Study start
- Mar 2027 (estimated)
- Primary completion
- Dec 2029 (estimated)
- Study completion
- Dec 2029 (estimated)
- First posted
- 21 Feb 2022 (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 goal of this clinical trial is to learn how patients with CDKL-5 deficiency disorder (CDD) aged 6 months to 2 years of age respond to treatment with ganaxolone. The main questions the trial aims to answer are:
- The amount of the medicine that was in participants' bodies and the safety of the medicine.
- Does the medicine ganaxolone lower the number of seizures when used in addition to the participant's usual anti-seizure treatment.
Participants will:
Take ganaxolone 3 times daily for up to 52 weeks. Visit the clinic every 3 to 5 weeks for the initial 17 weeks of the trial and thereafter every 11 to 12 weeks.
The participant's caregiver will:
Keep a daily diary of the participants seizures and use of anti-seizure medications, including the trial drug ganaxolone.
Detailed description
This is an open-label, single-arm, 52-week, multicenter trial of adjunctive ganaxolone treatment in participants 6 months to less than 2 years of age with genetically confirmed CDD. The trial consists of an up to 2-week Screening Period, 4-week prospective Baseline Period, followed by 2 parts: Part A; a 17-week ganaxolone treatment phase (including 4 to 5 weeks of ganaxolone titration followed by 12 to 13 weeks of dose maintenance) and Part B; a 35-week ganaxolone maintenance phase, and a 2-week taper period at the end of the trial. Tapering will not apply for participants continuing ganaxolone treatment after the trial.
The Screening Period will follow the Screening Visit to ensure caregivers can classify seizures and comply with completion instructions of an electronic seizure and medication diary. This will be followed by a 4-week prospective Baseline Period to assess seizure frequency for trial eligibility.
Up to 15 participants will be enrolled to ensure at least 12 participants are evaluable for the primary PK endpoints at the end of Part A of the trial, including at least 3 participants experiencing uncontrolled seizures despite treatment with at least 2 anti-seizure medications.
Participants will be monitored at Baseline and at specified visits to assess the exposure, safety and tolerability and activity of ganaxolone. PK samples will be collected at specified timepoints up until completion of Part A.
Clinical laboratory testing will be performed at local laboratories at participating sites.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is an open-label, single-arm trial
| Arm | Type | Receives |
|---|---|---|
| Ganaxolone | Experimental | Drug: GanaxoloneGanaxolone sodium benzoate-free oral suspension 50 mg/ml |
Who may qualify
- Ages
- 6 months to 2 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (10 listed)
- A diagnosis of CDD, including molecular confirmation of a pathogenic or likely pathogenic CDKL5 variant.
- Male or female participants aged 6 months to less than 2 years at the Baseline Visit.
- Failure to control seizures despite appropriate use of 1 or more anti-seizure medications at therapeutic doses. At least 3 participants will be included who failed to control seizures despite appropriate use of at least 2 anti-seizure medications.
- Participants will have refractory seizures with a history of at least 8 countable seizures of primary seizure types during the 28 days prospective Baseline Period prior to the Baseline Visit. Primary seizure types are seizures with or without impairment of consciousness with a clear motor component, including generalized tonic-clonic, focal to bilateral tonic-clonic, atonic/drop, bilateral clonic, bilateral tonic (sustained motor activity ≥ 3 seconds). Clusters of uncountable seizures will be counted as a single seizure.
- Participants should be receiving a stable regimen of anti-seizure medications at Screening through to the end of the 4-week prospective Baseline Period and have no foreseeable change for the duration of Part A.
Key exclusion criteria (9 listed)
- Have an active CNS infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive as evaluated by brain imaging (magnetic resonance imaging).
- Have any disease or condition (medical or surgical; other than CDKL5 deficiency) that might compromise the hematologic, cardiovascular, pulmonary, renal, gastrointestinal, or hepatic systems; or other conditions that might interfere with the absorption, distribution, metabolism, or excretion of the investigational product or would place the participant at increased risk.
- History of or present AST (aspartate aminotransferase/serum glutamic-oxaloacetic transaminase) or ALT (alanine aminotransferase/serum glutamic-pyruvic transaminase) >3 times the upper limit of normal (ULN) at Baseline and confirmed by a repeat testing.
- History of or present total bilirubin levels greater than the ULN at trial Baseline and confirmed by a repeat testing. In cases of a documented, stable medical condition (i.e., Gilbert's Syndrome) resulting in levels of total bilirubin greater than the ULN, the Medical Monitor will determine if an exception can be made.
- Participants with significant renal insufficiency, with an estimated glomerular filtration rate (eGFR) <30 mL/min (calculated using the Cockcroft-Gault formula or Pediatric eGFR calculator or Bedside Schwartz).
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 |
|---|---|---|
| GanaxoloneAlso: Ztalmy, Active DrugGanaxolone | Drug | Ganaxolone |
Primary outcome
Ganaxolone exposure in participants receiving ganaxolone doses up to 63 mg/kg/day
Time frame: Baseline through 17 weeks
Pharmacokinetic (PK) exposure parameter derived from population PK modelling and simulation: steady-state maximum concentration (Cmax,ss)
Ganaxolone exposure in participants receiving ganaxolone doses up to 63 mg/kg/day
Time frame: Baseline through 17 weeks
PK exposure parameter derived from population PK modelling and simulation: trough concentration (Ctrough)
Ganaxolone exposure in participants receiving ganaxolone doses up to 63 mg/kg/day
Time frame: Baseline through 17 weeks
PK exposure parameter derived from population PK modelling and simulation: systemic exposure (Caverage,ss derived as AUC over a dosing interval / tau)
Safety and tolerability of ganaxolone as adjunctive therapy for the treatment of seizures in participants receiving ganaxolone doses up to 63 mg/kg/day
Time frame: Baseline through to 52 weeks
Frequency, type, and severity of adverse events (AEs)
15 secondary outcomes
Ganaxolone PK parameters at steady-state in participants receiving ganaxolone doses up to 63 mg/kg/day.
Time frame: Baseline through to 17 weeks
Ganaxolone PK parameters at steady-state in participants receiving ganaxolone doses up to 63 mg/kg/day.
Time frame: Baseline through to 17 weeks
Ganaxolone PK parameters at steady-state in participants receiving ganaxolone doses up to 63 mg/kg/day.
Time frame: Baseline through to 17 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 17-week treatment period (Part A)
Time frame: Baseline through to 17 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 17-week treatment period (Part A)
Time frame: Baseline through to 17 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 17-week treatment period (Part A)
Time frame: Baseline through to 17 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 17-week treatment period (Part A)
Time frame: Baseline through to 17 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 17-week treatment period (Part A)
Time frame: Baseline through to 17 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 17-week treatment period (Part A)
Time frame: Baseline through to 17 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 52-week treatment period (Part B)
Time frame: Baseline through to 52 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 52-week treatment period (Part B)
Time frame: Baseline through to 52 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 52-week treatment period (Part B)
Time frame: Baseline through to 52 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 52-week treatment period (Part B)
Time frame: Baseline through to 52 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 52-week treatment period (Part B)
Time frame: Baseline through to 52 weeks
Activity of ganaxolone as adjunctive therapy for the treatment of primary seizures at the end of the 52-week treatment period (Part B)
Time frame: Baseline through to 52 weeks
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 4 Nov 2026.
- Derived from this study: PubMed 36517284Yasmen N, Sluter MN, Yu Y, Jiang J. Ganaxolone for management of seizures associated with CDKL5 deficiency disorder. Trends Pharmacol Sci. 2023 Feb;44(2):128-129. doi: 10.1016/j.tips.2022.11.007. Epub 2022 Dec 12. No abstract available.
- 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.
Clinical Project Manager contact
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Ganaxolone in people with CDKL5 Deficiency Disorder, run by Immedica Pharma AB.
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 10 inclusion and 9 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: opening soon, as of the sponsor's last update on 4 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
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 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?
Contact Clinical Project Manager (+46 8 533 39 500, clinical@immedica.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Ganaxolone exposure in participants receiving ganaxolone doses up to 63 mg/kg/day (time frame: Baseline through 17 weeks). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05249556, which also lists 1 linked publication.
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 2026.