Jordan's SyndromePhase 2NCT06717438
Study of Zatolmilast (BPN14770) in Participants With PPP2R5D Neurodevelopmental Disorder (Jordan's Syndrome [JS])
A Phase 2 study testing BPN14770 in people with Jordan's Syndrome and PPP2R5D Neurodevelopmental Disorder, run by Shionogi at 3 sites in United States.
Trial summary
The primary objective of this study is to assess the efficacy, safety, and tolerability of BPN14770 in participants aged 9 to 45 years with JS. Read more about this study
- Who is it for?
- Ages 9 to 45 years
- Conditions the study is for
- Jordan's Syndrome, PPP2R5D Neurodevelopmental Disorder
- What is being tested?
- BPN14770
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 30 participants
- Where
- 3 sitesUnited States
- Study dates
- 13 May 2025to 31 Dec 2027
- Phase
- Phase 2
- 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 9 to 45 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Participant is aged 9 to 45 years, inclusive.
- Participant has confirmed/documented history of PPP2R5D Neurodevelopmental Disorder.
- Current treatment with no more than 3 prescribed psychotropic medications.
- Participant has a parent, legal authorized guardian or consistent caregiver.
Who cannot join
- Participant has body weight less than 25 kilograms (kg).
- Clinically significant abnormalities, in the investigator's judgment, in safety laboratory tests, vital signs, or electrocardiogram (ECG), as measured during Screening.
- Concurrent major psychiatric condition (e.g., Major Depressive Disorder, Schizophrenia or Bipolar Disorder) as diagnosed by the investigator. Participants with additional diagnosis of Autism Spectrum Disorder or Anxiety Disorder will be allowed.
- Participant is planning to commence psychotherapy or cognitive behavior therapy (CBT) during the period of the study or had begun psychotherapy or CBT within 4 weeks prior to Screening.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Boston Children's Hospital
Boston, Massachusetts, 02115
Rush University Medical Center
Chicago, Illinois, 60612
Seattle Children's Hospital
Seattle, Washington, 98101
About this study
Official title: A Phase 2 Randomized, Double-blind, Placebo-controlled, Study of Zatolmilast (BPN14770) in Subjects With PPP2R5D Neurodevelopmental Disorder (Jordan's Syndrome)
The primary objective of this study is to assess the efficacy, safety, and tolerability of BPN14770 in participants aged 9 to 45 years with JS.
- Being tested
- BPN14770, Placebo
- Keywords
- Jordan's Syndrome, JS, PPP2R5D Neurodevelopmental Disorder, Zatolmilast, BPN14770
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BPN14770Drug
Also called Zatolmilast
Capsules for oral administration
PlaceboDrug
Capsules for oral administration
| Study group | Role | What they receive |
|---|---|---|
| BPN14770 | Experimental | Participants will receive a weight adjusted dose of BPN14770 twice daily (BID) during the 24-week double-blind period.
Participants who complete the double-blind period will have the opportunity to continue in the open-label extension (OLE) period. Participants will receive a weight adjusted dose of BPN14770 BID during the 24-week OLE period.
Eligible participants who complete the double-blind period and OLE period may be offered the opportunity to participate in a 48-week extended open-label extension (Ext OLE) period. Adult participants (≥18 years of age at the start of Ext OLE) will receive an age-adjusted dose of BPN14770 BID during the Ext OLE period, while participants younger than 18 years at the start of Ext OLE will receive a weight-adjusted dose of BPN14770 BID. Receives: Drug: BPN14770 |
| Placebo | Placebo comparator | Participants will receive BPN14770-matching placebo BID during the 24-week double-blind period.
Participants who complete the double-blind period will have the opportunity to continue in the OLE period. Participants will receive a weight adjusted dose of BPN14770 BID during the 24-week OLE period.
Eligible participants who complete the double-blind period and OLE period may be offered the opportunity to participate in a 48-week Ext OLE period. Adult participants (≥18 years of age at the start of Ext OLE) will receive an age-adjusted dose of BPN14770 BID during the Ext OLE period, while participants younger than 18 years at the start of Ext OLE will receive a weight-adjusted dose of BPN14770 BID. Receives: Drug: BPN14770; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline at Week 24 in Numerical Rating Scale Within the Domain of Language/Communication
Measured: Baseline, Week 24
Number of Participants with Treatment-emergent Adverse Events (TEAEs)
Measured: Day 1 up to Week 96
Discontinuation-emergent Signs and Symptoms (DESS) Questionnaire Score
Measured: Week 98
Number of Participants with Seizures
Measured: Up to Week 96
Number of Participants at Suicidality Risk
Measured: Up to Week 96
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Dec 2024
- Study start13 May 2025
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Jordan's SyndromeStudies of the same condition, open and under way.
- More trials run by ShionogiThe sponsor's other registered studies.
- 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shionogi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06717438
- Sponsor's protocol ID
- BPN14770-CNS-205
- Status last verified
- Jun 2026
- First submitted
- 2 Dec 2024
- Last updated
- 9 Jun 2026
- Results posted
- No
- 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 9 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Randomized, Double-blind, Placebo-controlled, Study of Zatolmilast (BPN14770) in Subjects With PPP2R5D Neurodevelopmental Disorder (Jordan's Syndrome)
- ClinicalTrials.gov ID
- NCT06717438
- Acronym / sponsor ID
- BPN14770-CNS-205
- Keywords
- Jordan's Syndrome, JS, PPP2R5D Neurodevelopmental Disorder, Zatolmilast, BPN14770
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 30 participants (actual)
- Lead sponsor
- ShionogiIndustry (pharma, biotech, devices)
- Study start
- 13 May 2025 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 5 Dec 2024 (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 of this study is to assess the efficacy, safety, and tolerability of BPN14770 in participants aged 9 to 45 years with JS.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| BPN14770 | Experimental | Drug: BPN14770Participants will receive a weight adjusted dose of BPN14770 twice daily (BID) during the 24-week double-blind period. Participants who complete the double-blind period will have the opportunity to continue in the open-label extension (OLE) period. Participants will receive a weight adjusted dose of BPN14770 BID during the 24-week OLE period. Eligible participants who complete the double-blind period and OLE period may be offered the opportunity to participate in a 48-week extended open-label extension (Ext OLE) period. Adult participants (≥18 years of age at the start of Ext OLE) will receive an age-adjusted dose of BPN14770 BID during the Ext OLE period, while participants younger than 18 years at the start of Ext OLE will receive a weight-adjusted dose of BPN14770 BID. |
| Placebo | Placebo comparator | Drug: BPN14770, Drug: PlaceboParticipants will receive BPN14770-matching placebo BID during the 24-week double-blind period. Participants who complete the double-blind period will have the opportunity to continue in the OLE period. Participants will receive a weight adjusted dose of BPN14770 BID during the 24-week OLE period. Eligible participants who complete the double-blind period and OLE period may be offered the opportunity to participate in a 48-week Ext OLE period. Adult participants (≥18 years of age at the start of Ext OLE) will receive an age-adjusted dose of BPN14770 BID during the Ext OLE period, while participants younger than 18 years at the start of Ext OLE will receive a weight-adjusted dose of BPN14770 BID. |
Who may qualify
- Ages
- 9 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Participant is aged 9 to 45 years, inclusive.
- Participant has confirmed/documented history of PPP2R5D Neurodevelopmental Disorder.
- Current treatment with no more than 3 prescribed psychotropic medications.
- Participant has a parent, legal authorized guardian or consistent caregiver.
Key exclusion criteria (4 listed)
- Participant has body weight less than 25 kilograms (kg).
- Clinically significant abnormalities, in the investigator's judgment, in safety laboratory tests, vital signs, or electrocardiogram (ECG), as measured during Screening.
- Concurrent major psychiatric condition (e.g., Major Depressive Disorder, Schizophrenia or Bipolar Disorder) as diagnosed by the investigator. Participants with additional diagnosis of Autism Spectrum Disorder or Anxiety Disorder will be allowed.
- Participant is planning to commence psychotherapy or cognitive behavior therapy (CBT) during the period of the study or had begun psychotherapy or CBT within 4 weeks prior to Screening.
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 |
|---|---|---|
| BPN14770Also: ZatolmilastCapsules for oral administration | Drug | BPN14770, Placebo |
| PlaceboCapsules for oral administration | Drug | Placebo |
Primary outcome
Change From Baseline at Week 24 in Numerical Rating Scale Within the Domain of Language/Communication
Time frame: Baseline, Week 24
Number of Participants with Treatment-emergent Adverse Events (TEAEs)
Time frame: Day 1 up to Week 96
Discontinuation-emergent Signs and Symptoms (DESS) Questionnaire Score
Time frame: Week 98
Number of Participants with Seizures
Time frame: Up to Week 96
Number of Participants at Suicidality Risk
Time frame: Up to Week 96
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 9 Jun 2026.
- Individual participant dataSharing planned: No
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.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BPN14770 in people with Jordan's Syndrome and PPP2R5D Neurodevelopmental Disorder, run by Shionogi at 3 sites in United States.
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 4 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 9 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Change From Baseline at Week 24 in Numerical Rating Scale Within the Domain of Language/Communication (time frame: Baseline, Week 24). The study lists 5 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 9 Jun 2026.