CDKL5 DisorderCDDNCT04678479
Ganaxolone Expanded Access Program Compassionate Use
A study testing Ganaxolone in people with CDKL5 Disorder, run by Marinus Pharmaceuticals.
Trial summary
The primary objective is to provide GNX to patients ≥ 2 years with CDD-related seizures who are refractory to, or intolerant of, standard therapy. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- CDKL5 Disorder
- What is being tested?
- Ganaxolone
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 2 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Molecular confirmation of a pathogenic CDKL5 variant, early onset, difficult to control seizures, and neurodevelopmental impairment are required. Male or female patients aged ≥ 2 years. In the opinion of the investigator, the patient has inadequate seizure control on current anti-seizure medications at therapeutic doses.
Who cannot join
- -
About this study
Official title: Expanded Access With Adjunctive Ganaxolone Treatment in Children and Young Adults With Cyclin Dependent Kinase-like 5 Deficiency Disorder
The primary objective is to provide GNX to patients ≥ 2 years with CDD-related seizures who are refractory to, or intolerant of, standard therapy.
Read the detailed description
This is a multi-center, long-term, open-label, expanded access protocol of adjunctive GNX treatment in children, adolescents, and adults with CDD. Patients with inadequate seizure control on their current anti seizure medications at therapeutic doses will be eligible for enrollment.
- Conditions
- CDKL5 Disorder
- Being tested
- Ganaxolone
- 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
ganaxolone
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Dec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CDKL5 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Marinus Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Paula Bokesch, MD, FAAP, Study director, Marinus Pharmaceuticals, Inc.Igor Grachev, MD, PhD, Study director, Marinus Pharmaceuticals, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04678479
- Sponsor's protocol ID
- 1042-CDD-EAP-3005
- Acronym
- CDD
- Status last verified
- Jan 2024
- First submitted
- 16 Dec 2020
- Last updated
- 22 Jan 2024
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access With Adjunctive Ganaxolone Treatment in Children and Young Adults With Cyclin Dependent Kinase-like 5 Deficiency Disorder
- ClinicalTrials.gov ID
- NCT04678479
- Acronym / sponsor ID
- CDD / 1042-CDD-EAP-3005
- Conditions
- CDKL5 Disorder
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Marinus PharmaceuticalsIndustry (pharma, biotech, devices)
- First posted
- 22 Dec 2020 (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 primary objective is to provide GNX to patients ≥ 2 years with CDD-related seizures who are refractory to, or intolerant of, standard therapy.
Detailed description
This is a multi-center, long-term, open-label, expanded access protocol of adjunctive GNX treatment in children, adolescents, and adults with CDD. Patients with inadequate seizure control on their current anti seizure medications at therapeutic doses will be eligible for enrollment.
Study design
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Molecular confirmation of a pathogenic CDKL5 variant, early onset, difficult to control seizures, and neurodevelopmental impairment are required. Male or female patients aged ≥ 2 years. In the opinion of the investigator, the patient has inadequate seizure control on current anti-seizure medications at therapeutic doses.
Key exclusion criteria (1 listed)
- -
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 |
|---|---|---|
| Ganaxoloneganaxolone | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jan 2024.
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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.
Paula Bokesch, MD, FAAP study director
Marinus Pharmaceuticals, Inc.
Igor Grachev, MD, PhD study director
Marinus Pharmaceuticals, Inc.
Questions about this trial
What is this clinical trial studying?
A study testing Ganaxolone in people with CDKL5 Disorder, run by Marinus Pharmaceuticals.
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 1 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: status not confirmed, as of the sponsor's last update on 22 Jan 2024. 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.
Are results available for this study?
Results are not available in the source record.
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.
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 22 Jan 2024.