Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Authorised, recruitment pending
Participants planned
300
Countries
1
Sites
6
Schizophrenia and Autism Spectrum Disorder (ASD)
To assess the long-term safety and tolerability of KarXT in the treatment of teenagers (13-17 years old) with schizophrenia and of KarXT+KarX-EC in the treatment of children and teenagers (5-17 years old) with irritability associated with ASD.
Key facts
- Sponsor
- Bristol-Myers Squibb Services Unlimited Company
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10], Psychiatry and Psychology [F] - Mental Disorders [F03]
- Decision date (initial)
- 2026-05-29
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2025-524062-16-00
- WHO UTN
- U1111-1325-9994
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy
To assess the long-term safety and tolerability of KarXT in the treatment of teenagers (13-17 years old) with schizophrenia and of KarXT+KarX-EC in the treatment of children and teenagers (5-17 years old) with irritability associated with ASD.
Secondary objectives 3
- To assess the effects of KarXT and of KarXT+KarX-EC on procholinergic symptoms in teenagers with schizophrenia and in children and teenagers (5-17 years old) with irritability associated with ASD, respectively.
- To assess the effects of KarXT and of KarXT+KarX-EC on the frequency and severity of suicidal ideation and behavior in teenagers with schizophrenia and in children and teenagers (5-17 years old) with irritability associated with ASD, respectively.
- To assess the effects of KarXT and of KarXT+KarX-EC on extrapyramidal symptoms (EPS; movement and coordination problems) in teenagers with schizophrenia and in children and teenagers (5-17 years old) with irritability associated with ASD, respectively.
Conditions and MedDRA coding
Schizophrenia and Autism Spectrum Disorder (ASD)
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10039626 | Schizophrenia | 100000004873 |
| 20.0 | LLT | 10076922 | Early onset schizophrenia | 10037175 |
| 20.0 | LLT | 10008520 | Childhood autism | 10037175 |
| 21.1 | PT | 10063844 | Autism spectrum disorder | 100000004873 |
Regulatory references
- EMA paediatric investigation plan (PIP)
- EMEA-000027-PIP00-79, EMEA-000029-PIP10-41
- Plan to share IPD
- Yes
- IPD plan description
- BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria. Additional information regarding Bristol Myers Squibb's data sharing policy and process can be found at: https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosure-commitment.html.
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 2
- Teenagers with schizophrenia must be between 13-17 years old at the time of consent in study CN0120020, have a diagnosis of schizophrenia, and have completed the double-blind treatment period (i.e., Visit 8) of study CN0120020 without an adverse event that the study doctor believes is an unacceptable safety risk.
- Children and teenagers with irritability associated with ASD must be between 5-17 years old at the time of consent in studies CN0120044/CN0120045, have a diagnosis of ASD, and have completed the double-blind treatment period (i.e., Week 8) of study CN0120044 or CN0120045 without an adverse event that the study doctor believes is an unacceptable safety risk.
Exclusion criteria 5
- Participants from study CN0120020 who have a primary diagnosis other than schizophrenia.
- Participants from study CN0120020 who are experiencing moderate to severe substance use disorder within the last year.
- Participants who, at the final end‑of‑treatment visit of Study CN0120020, Study CN0120044, or Study CN0120045 have any clinically significant abnormalities in physical examination, electrocardiogram, or vital signs (including heart rate or blood pressure) that, in the investigator’s judgment, could compromise their safety.
- Pregnant or breastfeeding women.
- Participants from Study CN0120044 and Study CN0120045 who have any primary psychiatric diagnosis other than ASD.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Occurrence of treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), and serious adverse events (SAEs) during the treatment and safety follow-up periods.
Secondary endpoints 3
- Occurrence of procholinergic and anticholinergic symptoms during the treatment period.
- Suicidal ideation and behavior assessed using the Columbia Suicide Severity Rating Scale (C-SSRS) during the treatment and safety follow-up periods.
- Change from baseline (CFB) on the Simpson-Angus Scale (SAS), Barnes-Akathisia Rating Scale (BARS), and Abnormal Involuntary Movement Scale (AIMS) by visit during the treatment period.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 11
PRD12408422 · Product
- Active substance
- Xanomeline Tartrate
- Substance synonyms
- LY246708 tartrate, 3-(4-(Hexyloxy)-1,2,5-thiadiazol-3-yl)-1,2,5,6-tetrahydro-1-methylpyridine (-)-(+)-tartrate (1:1)
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL
- Max daily dose
- 9999 mg milligram(s)
- Max total dose
- 9999 mg milligram(s)
- Max treatment duration
- 9999 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
PRD12408417 · Product
- Active substance
- Xanomeline Tartrate
- Substance synonyms
- LY246708 tartrate, 3-(4-(Hexyloxy)-1,2,5-thiadiazol-3-yl)-1,2,5,6-tetrahydro-1-methylpyridine (-)-(+)-tartrate (1:1)
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL
- Max daily dose
- 9999 mg milligram(s)
- Max total dose
- 9999 mg milligram(s)
- Max treatment duration
- 9999 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
PRD12408431 · Product
- Active substance
- Xanomeline Tartrate
- Substance synonyms
- LY246708 tartrate, 3-(4-(Hexyloxy)-1,2,5-thiadiazol-3-yl)-1,2,5,6-tetrahydro-1-methylpyridine (-)-(+)-tartrate (1:1)
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL
- Max daily dose
- 9999 mg milligram(s)
- Max total dose
- 9999 mg milligram(s)
- Max treatment duration
- 9999 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
PRD12408423 · Product
- Active substance
- Xanomeline Tartrate
- Substance synonyms
- LY246708 tartrate, 3-(4-(Hexyloxy)-1,2,5-thiadiazol-3-yl)-1,2,5,6-tetrahydro-1-methylpyridine (-)-(+)-tartrate (1:1)
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL
- Max daily dose
- 9999 mg milligram(s)
- Max total dose
- 9999 mg milligram(s)
- Max treatment duration
- 9999 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
PRD12404377 · Product
- Active substance
- Trospium Chloride
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL
- Max daily dose
- 9999 mg milligram(s)
- Max total dose
- 9999 mg milligram(s)
- Max treatment duration
- 9999 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
PRD12404386 · Product
- Active substance
- Trospium Chloride
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL
- Max daily dose
- 9999 mg milligram(s)
- Max total dose
- 9999 mg milligram(s)
- Max treatment duration
- 9999 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
PRD12404394 · Product
- Active substance
- Trospium Chloride
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL
- Max daily dose
- 9999 mg milligram(s)
- Max total dose
- 9999 mg milligram(s)
- Max treatment duration
- 9999 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
PRD12404368 · Product
- Active substance
- Trospium Chloride
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL
- Max daily dose
- 9999 mg milligram(s)
- Max total dose
- 9999 mg milligram(s)
- Max treatment duration
- 9999 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
PRD12327546 · Product
- Active substance
- Trospium Chloride
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL
- Max daily dose
- 9999 mg milligram(s)
- Max total dose
- 9999 mg milligram(s)
- Max treatment duration
- 9999 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
PRD12327577 · Product
- Active substance
- Trospium Chloride
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL
- Max daily dose
- 9999 mg milligram(s)
- Max total dose
- 9999 mg milligram(s)
- Max treatment duration
- 9999 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
PRD12327569 · Product
- Active substance
- Trospium Chloride
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL
- Max daily dose
- 9999 mg milligram(s)
- Max total dose
- 9999 mg milligram(s)
- Max treatment duration
- 9999 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Bristol-Myers Squibb Services Unlimited Company
- Sponsor organisation
- Bristol-Myers Squibb Services Unlimited Company
- Address
- Plaza 254, Blanchardstown Corporate Park 2 Blanchardstown Corporate Park 2
- City
- Dublin 15
- Postcode
- D15 T867
- Country
- Ireland
Scientific contact point
- Organisation
- Bristol-Myers Squibb Services Unlimited Company
- Contact name
- GSM-CT
Public contact point
- Organisation
- Bristol-Myers Squibb Services Unlimited Company
- Contact name
- GSM-CT
Third parties 6
| Organisation | City, country | Duties |
|---|---|---|
| Greenphire LLC ORG-100041621
|
King Of Prussia, United States | Other |
| Accenture Solutions Private Limited ORG-100032592
|
Chennai, India | Other |
| Iqvia Holdings Inc. ORG-100043905
|
Durham, United States | Other |
| Labcorp Central Laboratory Services LP ORG-100032236
|
Indianapolis, United States | Other |
| Signant Health Global LLC ORG-100040604
|
Blue Bell, United States | Other |
| Syneos Health Inc. ORG-100008382
|
Morrisville, United States | Other |
Locations
1 EU/EEA country · 6 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Romania | Authorised, recruitment pending | 40 | 6 |
| Rest of world
United States, Colombia, Argentina, Japan
|
— | 260 | — |
Investigational sites
Institutul De Psihiatrie Socola Iasi
Pediatric Psychiatry, Soseaua Bucium 36, 700282, Iasi
Cabinet Medical Individual Dr Gheorghita Karina Lidia
Pediatric Neurology, Strada Nita Elinescu nr 6C, 031871, Bucharest
Spitalul Clinic De Psihiatrie Prof.Dr.Alexandru Obregia
Pediatric Psychiatry, Soseaua Berceni 4, 041914, Bucharest
Spitalul Clinic de Urgenta pentru Copii Cluj Napoca
Pediatric Psychiatry, Str. Motilor nr.68, 400370, Cluj Napoca
Spitalul Clinic De Urgenta Pentru Copii Louis Turcanu Timisoara
Pediatric Psychiatry, Strada Doctor Iosif Nemoianu 2, 300011, Timisoara
Clinica Animedica
Pediatric Psychiatry, Strada Caimetei nr.20, 021056, Bucharest
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 10 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol 2025-524062-16-_redacted | 1 |
| Recruitment arrangements (for publication) | K1_ Recruitment arrangements | 2 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF Assent 13-17_Redacted | 1.2 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF Main_Redacted | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Assent 13-17 EN_Redacted | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Future Research EN_Redacted | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Future Research_Redacted | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Main EN_Redacted | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_2025-524062-16_EN | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_2025-524062-16_RO | 1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2026-02-02 | Romania | Acceptable with conditions 2026-05-25
|
2026-05-29 |