Overview
Sponsor-declared trial summary
Phase
Phase II and Phase III (Integrated)
Status
Ongoing, recruiting
Participants planned
66
Countries
1
Sites
7
Generalized myasthenia gravis
1. Phase 2: To characterize the safety and tolerability of KYV-101. 2. Phase 2: …
Key facts
- Sponsor
- Kyverna Therapeutics Inc.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Immune System Diseases [C20]
- Trial duration
- 9 Jul 2024 → ongoing
- Decision date (initial)
- 2024-05-13
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Kyverna Therapeutics, Inc
External identifiers
- EU CT number
- 2023-509892-17-00
- ClinicalTrials.gov
- NCT06193889
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy, Others
1. Phase 2: To characterize the safety and tolerability of KYV-101.
2. Phase 2: To evaluate the efficacy of KYV-101.
3. Phase 3: To evaluate the efficacy of KYV-101.
Secondary objectives 2
- 1. Phase 3: To evaluate the efficacy of KYV-101.
- 2. Phase 3: To characterize the safety and tolerability of KYV-101.
Conditions and MedDRA coding
Generalized myasthenia gravis
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.1 | PT | 10028417 | Myasthenia gravis | 100000004852 |
Regulatory references
- Scientific advice from competent authorities
- Paul-Ehrlich-Institut, European Medicines Agency
- Plan to share IPD
- No
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 12
- 1. Patients must be ≥ 18 to ≤ 75 years of age with a diagnosis of generalized MG
- 10. No use of intravenous immune globulin (IVIG) or plasmapheresis (PLEX) within 4 weeks of screening or pre-dose baseline (unless this is part of their SOC treatment regimen).
- 2. Presence of autoantibodies to AChR or MuSK at screening (not just historical).
- 3. Myasthenia Gravis Foundation of America (MGFA) Class IIb-IV.
- 4. MG-ADL total score of ≥6 at screening and confirmed at pre-dose baseline.
- 5. QMG total score of ≥11 at screening and confirmed at pre-dose baseline.
- 6. Failed treatment with 2 or more immunosuppressive/immunomodulatory therapies, or failed at least 1 immunosuppressive therapy and required chronic plasmapheresis, or IVIG to control symptoms. a. Failed treatment may consist of either lack of sufficient efficacy with an adequate trial, intolerable adverse effects, or contraindications to therapy, as determined by the investigator.
- 7. In Germany only, failed standard-of-care immunosuppressive and immunomodulatory therapies including complement inhibitors and FcRn modulators. o Failed treatment may consist of either lack of sufficient efficacy with an adequate trial, intolerable adverse effects, or contraindications to therapy, as determined by the investigator. o Failure of chronic plasmapheresis or IVIG to control symptoms.
- 8. On a stable dose of glucocorticoids and/or other immunotherapies for ≥1 month prior to screening. For azathioprine, being on a stable dose for ≥2 months prior to screening is required.
- 9. No change in dose of acetylcholinesterase inhibitors for ≥2 weeks prior to screening
- 11. No use of rituximab (or any other anti-CD20 or CD19 monoclonal antibody) within 12 weeks prior to screening.
- 12. No use of FcRn inhibitors within 4 weeks prior to screening.
Exclusion criteria 21
- 1. Unable to washout or interrupt autoimmune disease therapy prior to apheresis as specificed in protocol
- 2. Co-occurring neurological autoimmune disease (ie, Lambert-Eaton Myasthenic Syndrome) or any disease affecting the neuromuscular junction or muscle causing weakness (eg, myositis, myopathy, motor neuropathy)
- 3. History of stroke (with residual sequalae and/or risk for recurrence), seizure (even if well controlled on antiepileptics), neurodegenerative disease, altered mental status (unexplained and/or recent/current), or uncontrolled/severe psychiatric disease
- 4. Any serious and/or uncontrolled medical condition that, in the investigator’s judgment, would cause unacceptable safety risk, interfere with study procedures or results, or compromise compliance with the protocol, including but not limited to, clinically significant cardiac or pulmonary disease.
- 5. History of primary immunodeficiency, organ or allogeneic bone marrow transplant, or splenectomy.
- 6. Active, uncontrolled, viral, bacterial, or systemic fungal infection or recent history of repeated infections.
- 7. Previous or concurrent malignancy with the following exceptions: a. Adequately treated basal cell or squamous cell carcinoma (adequate wound healing is required prior to screening). b. In situ carcinoma (eg, of the cervix or breast), treated curatively and without evidence of recurrence for at least 3 years prior to screening. c. A primary malignancy which has been completely resected, or treated, and is in complete remission for at least 5 years prior to screening.
- 8. Major surgery within 4 weeks prior to apheresis or planned within 4 weeks after KYV-101 administration. For surgery planned after 4 weeks post KYV-101 administration, discuss with the sponsor
- 9. Thymectomy ≤ 12 months of screening or planned during the study.
- 10. Patients requiring chronic anticoagulation therapy that cannot be discontinued for medical procedures (such as apheresis
- 11. Prior treatment with gene therapy product or cellular immunotherapy (eg, CAR T) requiring vector integration and directed at any target.
- 12. Plan to receive live, attenuated vaccine after signing ICF (inactive vaccines, such as the flu vaccine, are allowed).
- 13. Contraindications or life-threatening allergies, hypersensitivity, or intolerance to KYV-101 or its excipients, including dimethyl sulfoxide; or to CYC, FLU, or tocilizumab.
- 14. Have evidence of latent or active TB infection, as documented by a positive QuantiFERON-TB Gold test at screening.
- 15. Positive hepatitis B surface antigen (HBsAg) at screening. Patients who are HBsAg negative but hepatitis B core antibody (HBcAb) positive must have negative hepatitis B virus (HBV) DNA testing.
- 16. Positive hepatitis C serology confirmed by polymerase chain reaction (PCR).
- 17. Positive serology for human immunodeficiency virus (HIV).
- 18. Positive screening test for SARS-CoV-2, by PCR or rapid antigen testing (not antibody testing). Note: Patients with a positive SARS-CoV-2 at screening can be enrolled if the patient has all 3 of the following: no active signs or symptoms of SARS-Cov-2 infection after 10 days, a negative repeat PCR or rapid antigen test, and no sequelae or complications of SARS-CoV-2 infection.
- 19. Any laboratory values at screening exceeding the cutoff values
- 20. Any of the following vital sign parameters at rest: • Systolic Blood Pressure (mmHg) < 95 or > 150 • Diastolic Blood pressure (mmHg) < 55 or > 95 • Heart Rate < 50 or > 100 bpm • Oral Temperature > 37.7° C/afebrile • Respiratory rate < 12 or > 20 bpm (Note: Up to 3 assessments of blood pressure, heart rate, and respiratory rate may be made within a 24-hour period)
- 21. Pregnant or breastfeeding, plans to become pregnant/breastfeed or father a child, risk of unintentional pregnancy/fathering, or plans to donate eggs/sperm in the timeframe provided in the protocol
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 4
- 1. Phase 2: Incidence and severity of AEs and laboratory abnormalities.
- 2. Phase 2: MG-ADL change from baseline at 24 weeks.
- 3. Phase 3: MG-ADL change from baseline at 24 weeks for KYV-101 compared to SOC (co-primary)
- 4. Phase 3: QMG change from baseline at 24 weeks for KYV-101 compared to SOC (co-primary)
Secondary endpoints 6
- 1. Phase 3: MGC change from baseline at 24 weeks for KYV-101 compared to SOC.
- 2. Phase 3: Percent change from baseline in anti-AChR or anti-MuSK antibody levels at week 24 for KYV-101 compared to SOC.
- 3. Phase 3: Proportion of patients with a ≥3 point improvement from baseline in MG-ADL at 24 weeks for KYV-101 compared to SOC.
- 4. Phase 3: Proportion of patients with MSE at week 24 for KYV-101 compared to SOC.
- 5. Phase 3: MG-QoL15r change from baseline at 24 weeks for KYV-101 compared to SOC.
- 6. Phase 3: Incidence and severity of AEs and laboratory abnormalities.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD9974051 · Product
- Active substance
- KYV-101
- Pharmaceutical form
- SUSPENSION
- Route of administration
- INTRAVENOUS INFUSION
- Max daily dose
- 100000000 Other
- Max total dose
- 100000000 Other
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Not Authorised
- ATC code
- NOTASSIGN — -
- MA holder
- KYVERNA THERAPEUTICS INC.
- Paediatric formulation
- No
- Orphan designation
- No
Comparator 23
Privigen 100 mg/ml solution for infusion
PRD339234 · Product
- Active substance
- Human Normal Immunoglobulin
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS INFUSION
- Max daily dose
- 1000 mg/kg milligram(s)/kilogram
- Max total dose
- 280 g gram(s)
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- J06BA02 — IMMUNOGLOBULINS, NORMAL HUMAN, FOR INTRAVASCULAR ADM.
- Marketing authorisation
- EU/1/08/446/001
- MA holder
- CSL BEHRING GMBH
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
PRD2153965 · Product
- Active substance
- Mycophenolate Mofetil
- Pharmaceutical form
- CAPSULE, HARD
- Route of administration
- ORAL
- Max daily dose
- 2.5 g gram(s)
- Max total dose
- 700 g gram(s)
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AA06 — MYCOPHENOLIC ACID
- Marketing authorisation
- EU/1/96/005/001
- MA holder
- ROCHE REGISTRATION GMBH
- MA country
- Iceland
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
PRD11855448 · Product
- Active substance
- Tacrolimus
- Substance synonyms
- TACROLIMUS ANHYDROUS
- Pharmaceutical form
- CAPSULE, HARD
- Route of administration
- ORAL
- Max daily dose
- 4 mg milligram(s)
- Max total dose
- 1.1 g gram(s)
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AD02 — -
- Marketing authorisation
- 41954.01.00
- MA holder
- ASTELLAS PHARMA GMBH
- MA country
- Germany
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/17/1912
- Modified vs. Marketing Authorisation
- No
Ultomiris 300 mg/30 mL concentrate for solution for infusion
PRD7445250 · Product
- Active substance
- Ravulizumab
- Substance synonyms
- Fc- and CDR-modified humanised monoclonal antibody against C5, ALXN1210
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS INFUSION
- Max daily dose
- 3600 mg milligram(s)
- Max total dose
- 11.2 g gram(s)
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AJ02 — -
- Marketing authorisation
- EU/1/19/1371/001
- MA holder
- ALEXION EUROPE SAS
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Azathioprine Tablets 50mg Oprisine
PRD7713388 · Product
- Active substance
- Azathioprine
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 2.5 mg/kg milligram(s)/kilogram
- Max total dose
- 700 mg/kg milligram(s)/kilogram
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AX01 — AZATHIOPRINE
- Marketing authorisation
- PL 13606/0093
- MA holder
- STRIDES PHARMA UK LIMITED
- MA country
- XI
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
PRD11534406 · Product
- Active substance
- Methotrexate Sodium
- Substance synonyms
- SODIUM METHOTREXATE, MTX SODIUM
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 2 g gram(s)
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AX03 — -
- Marketing authorisation
- PL 12762/0231
- MA holder
- MERCURY PHARMACEUTICALS LTD.
- MA country
- XI
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/25/3135
- Modified vs. Marketing Authorisation
- No
PRD11534403 · Product
- Active substance
- Methotrexate Sodium
- Substance synonyms
- SODIUM METHOTREXATE, MTX SODIUM
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 2 g gram(s)
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AX03 — -
- Marketing authorisation
- PL 12762/0231
- MA holder
- MERCURY PHARMACEUTICALS LTD.
- MA country
- XI
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/25/3135
- Modified vs. Marketing Authorisation
- No
PRD11534405 · Product
- Active substance
- Methotrexate Sodium
- Substance synonyms
- SODIUM METHOTREXATE, MTX SODIUM
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 2 g gram(s)
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AX03 — -
- Marketing authorisation
- PL 12762/0231
- MA holder
- MERCURY PHARMACEUTICALS LTD.
- MA country
- XI
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/25/3135
- Modified vs. Marketing Authorisation
- No
PRD11534408 · Product
- Active substance
- Methotrexate Sodium
- Substance synonyms
- SODIUM METHOTREXATE, MTX SODIUM
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 2 g gram(s)
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AX03 — -
- Marketing authorisation
- PL 12762/0231
- MA holder
- MERCURY PHARMACEUTICALS LTD.
- MA country
- XI
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/25/3135
- Modified vs. Marketing Authorisation
- No
PRD11534407 · Product
- Active substance
- Methotrexate Sodium
- Substance synonyms
- SODIUM METHOTREXATE, MTX SODIUM
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 2 g gram(s)
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AX03 — -
- Marketing authorisation
- PL 12762/0231
- MA holder
- MERCURY PHARMACEUTICALS LTD.
- MA country
- XI
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/25/3135
- Modified vs. Marketing Authorisation
- No
Zilbrysq 23 mg solution for injection in pre-filled syringe
PRD10984301 · Product
- Active substance
- Zilucoplan
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 0.3 mg/kg milligram(s)/kilogram
- Max total dose
- 0.9 g gram(s)
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AJ06 — -
- Marketing authorisation
- EU/1/23/1764/003
- MA holder
- UCB PHARMA S.A. (ANDERL BE)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Zilbrysq 16.6 mg solution for injection in pre-filled syringe
PRD10982512 · Product
- Active substance
- Zilucoplan
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 0.3 mg/kg milligram(s)/kilogram
- Max total dose
- 0.9 g gram(s)
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AJ06 — -
- Marketing authorisation
- EU/1/23/1764/001
- MA holder
- UCB PHARMA S.A. (ANDERL BE)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
PRD992060 · Product
- Active substance
- Prednisolone
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 60 mg milligram(s)
- Max total dose
- 16.8 g gram(s)
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- A07EA01 — PREDNISOLONE
- Marketing authorisation
- PL 29831/0178
- MA holder
- WOCKHARDT UK LTD
- MA country
- XI
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Soliris 300 mg concentrate for solution for infusion
PRD640284 · Product
- Active substance
- Eculizumab
- Substance synonyms
- ABP-959, H5G1.1
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS INFUSION
- Max daily dose
- 1200 mg milligram(s)
- Max total dose
- 24 g gram(s)
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AJ01 — -
- Marketing authorisation
- EU/1/07/393/001
- MA holder
- ALEXION EUROPE SAS
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/14/1304
- Modified vs. Marketing Authorisation
- No
NEORAL® Soft Gelatin Capsules 10mg
PRD11355194 · Product
- Active substance
- Ciclosporin
- Pharmaceutical form
- CAPSULE, SOFT
- Route of administration
- ORAL
- Max daily dose
- 3.5 mg/kg milligram(s)/kilogram
- Max total dose
- 980 mg/kg milligram(s)/kilogram
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AD01 — -
- Marketing authorisation
- PL 00101/0483
- MA holder
- NOVARTIS PHARMACEUTICALS UK LIMITED
- MA country
- XI
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
NEORAL® Soft Gelatin Capsules 10mg
PRD11355196 · Product
- Active substance
- Ciclosporin
- Pharmaceutical form
- CAPSULE, SOFT
- Route of administration
- ORAL
- Max daily dose
- 3.5 mg/kg milligram(s)/kilogram
- Max total dose
- 980 mg/kg milligram(s)/kilogram
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AD01 — -
- Marketing authorisation
- PL 00101/0483
- MA holder
- NOVARTIS PHARMACEUTICALS UK LIMITED
- MA country
- XI
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
NEORAL® Soft Gelatin Capsules 50mg
PRD11355209 · Product
- Active substance
- Ciclosporin
- Substance synonyms
- CYCLOSPORIN A, CYCLOSPORINE, CICLOSPORINE, CYCLOSPORIN
- Pharmaceutical form
- CAPSULE, SOFT
- Route of administration
- ORAL
- Max daily dose
- 3.5 mg/kg milligram(s)/kilogram
- Max total dose
- 980 mg/kg milligram(s)/kilogram
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AD01 — -
- Marketing authorisation
- PL 00101/0388
- MA holder
- NOVARTIS PHARMACEUTICALS UK LIMITED
- MA country
- XI
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
NEORAL® Soft Gelatin Capsules 25mg
PRD11355208 · Product
- Active substance
- Ciclosporin
- Substance synonyms
- CYCLOSPORIN A, CYCLOSPORINE, CICLOSPORINE, CYCLOSPORIN
- Pharmaceutical form
- CAPSULE, SOFT
- Route of administration
- ORAL
- Max daily dose
- 3.5 mg/kg milligram(s)/kilogram
- Max total dose
- 980 mg/kg milligram(s)/kilogram
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AD01 — -
- Marketing authorisation
- PL 00101/0387
- MA holder
- NOVARTIS PHARMACEUTICALS UK LIMITED
- MA country
- XI
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
NEORAL® Soft Gelatin Capsules 25mg
PRD11355214 · Product
- Active substance
- Ciclosporin
- Substance synonyms
- CYCLOSPORIN A, CYCLOSPORINE, CICLOSPORINE, CYCLOSPORIN
- Pharmaceutical form
- CAPSULE, SOFT
- Route of administration
- ORAL
- Max daily dose
- 3.5 mg/kg milligram(s)/kilogram
- Max total dose
- 980 mg/kg milligram(s)/kilogram
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AD01 — -
- Marketing authorisation
- PL 00101/0387
- MA holder
- NOVARTIS PHARMACEUTICALS UK LIMITED
- MA country
- XI
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
NEORAL® Soft Gelatin Capsules 10mg
PRD11355195 · Product
- Active substance
- Ciclosporin
- Pharmaceutical form
- CAPSULE, SOFT
- Route of administration
- ORAL
- Max daily dose
- 3.5 mg/kg milligram(s)/kilogram
- Max total dose
- 980 mg/kg milligram(s)/kilogram
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AD01 — -
- Marketing authorisation
- PL 00101/0483
- MA holder
- NOVARTIS PHARMACEUTICALS UK LIMITED
- MA country
- XI
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
NEORAL® Soft Gelatin Capsules 100mg
PRD11355207 · Product
- Active substance
- Ciclosporin
- Substance synonyms
- CYCLOSPORIN A, CYCLOSPORINE, CICLOSPORINE, CYCLOSPORIN
- Pharmaceutical form
- CAPSULE, SOFT
- Route of administration
- ORAL
- Max daily dose
- 3.5 mg/kg milligram(s)/kilogram
- Max total dose
- 980 mg/kg milligram(s)/kilogram
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AD01 — -
- Marketing authorisation
- PL 00101/0389
- MA holder
- NOVARTIS PHARMACEUTICALS UK LIMITED
- MA country
- XI
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
NEORAL® Soft Gelatin Capsules 100mg
PRD11355213 · Product
- Active substance
- Ciclosporin
- Substance synonyms
- CYCLOSPORIN A, CYCLOSPORINE, CICLOSPORINE, CYCLOSPORIN
- Pharmaceutical form
- CAPSULE, SOFT
- Route of administration
- ORAL
- Max daily dose
- 3.5 mg/kg milligram(s)/kilogram
- Max total dose
- 980 mg/kg milligram(s)/kilogram
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AD01 — -
- Marketing authorisation
- PL 00101/0389
- MA holder
- NOVARTIS PHARMACEUTICALS UK LIMITED
- MA country
- XI
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
NEORAL® Soft Gelatin Capsules 50mg
PRD11355215 · Product
- Active substance
- Ciclosporin
- Substance synonyms
- CYCLOSPORIN A, CYCLOSPORINE, CICLOSPORINE, CYCLOSPORIN
- Pharmaceutical form
- CAPSULE, SOFT
- Route of administration
- ORAL
- Max daily dose
- 3.5 mg/kg milligram(s)/kilogram
- Max total dose
- 980 mg/kg milligram(s)/kilogram
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AD01 — -
- Marketing authorisation
- PL 00101/0388
- MA holder
- NOVARTIS PHARMACEUTICALS UK LIMITED
- MA country
- XI
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Kyverna Therapeutics Inc.
- Sponsor organisation
- Kyverna Therapeutics Inc.
- Address
- 5980 Horton Street Ste 550
- City
- Emeryville
- Postcode
- 94608-2045
- Country
- United States
Scientific contact point
- Organisation
- Kyverna Therapeutics Inc.
- Contact name
- Medical Kyverna
Public contact point
- Organisation
- Kyverna Therapeutics Inc.
- Contact name
- Medical Kyverna
Third parties 17
| Organisation | City, country | Duties |
|---|---|---|
| PPD Global Central Labs ORG-100046496
|
Zaventem, Belgium | Laboratory analysis |
| Precision For Medicine Inc. ORG-100041895
|
Houston, United States | Laboratory analysis |
| Icon (Lr) Limited ORG-100042612
|
Dublin 18, Ireland | Laboratory analysis |
| Pra International ORG-100032850
|
Lenexa, United States | Laboratory analysis |
| Molecularmd Corp. ORG-100047559
|
Portland, United States | Laboratory analysis |
| PPD Development LP ORG-100011560
|
Wilmington, United States | On site monitoring, Code 10, Code 11, Code 12, Laboratory analysis, Code 5, Data management, E-data capture |
| Precision for Medicine GmbH ORG-100044456
|
Berlin, Germany | Other |
| Precision For Medicine Inc. ORG-100041895
|
Frederick, United States | Code 5 |
| Definitive Media Corp. ORG-100044065
|
Tustin, United States | Other |
| Acm Global Central Laboratory Limited ORG-100042459
|
York, United Kingdom | Laboratory analysis |
| WCG Clinical Inc. ORG-100040730
|
Puyallup, United States | Other |
| Elevatebio Base Camp Inc. ORG-100053841
|
Waltham, United States | Other |
| Medidata Solutions Inc. ORG-100016256
|
New York, United States | Other |
| Wuxi Advanced Therapies Inc. ORG-100032133
|
Philadelphia, United States | Other |
| Pharma Bio-Research Group ORG-100006268
|
Assen, Netherlands | Laboratory analysis |
| SYNLAB International GmbH ORG-100013007
|
Munich, Germany | Laboratory analysis |
| Icon Laboratory Services Inc. ORG-100037135
|
Farmingdale, United States | Other |
Locations
1 EU/EEA country · 7 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Germany | Ongoing, recruiting | 17 | 7 |
| Rest of world
Brazil, Australia, United States, United Kingdom, Saudi Arabia
|
— | 49 | — |
Investigational sites
Charite Universitaetsmedizin Berlin KöR
Neurology, Chariteplatz 1, Mitte, Berlin
University Medical Center Hamburg-Eppendorf
Stem Cell Transplantation, Center of Oncology, Martinistrasse 52, Eppendorf, Hamburg
Universitaetsklinikum Magdeburg AöR
Hematology, Oncology and Cell Therapy, Leipziger Strasse 44, 39120, Magdeburg
St. Josef-Hospital
Neurology, Gudrunstrasse 56, Grumme, Bochum
Medizinische Hochschule Hannover
Neurology, Carl-Neuberg-Strasse 1, Gross Buchholz, Hanover
Universitaetsklinikum Jena KöR
Neurology, Am Klinikum 1, Lobeda, Jena
Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)
Neurology, Ismaninger Strasse 22, Au-Haidhausen, Munich
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Germany | 2024-07-09 | 2024-09-26 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 22 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Kyverna_KYV101-006_Protocol_2023-509892-17-00_EEA_Public | 8.2.1 EEA |
| Protocol (for publication) | D1_Kyverna_KYV101-006_Risk-Benefit-Assessment_2023-509892-17-00_Public | 8.2 |
| Protocol (for publication) | D4_Kyverna_KVY-101-006_Publication Of Licensed Patient Questionnaires_Public | N/A |
| Recruitment arrangements (for publication) | K1_DE_Recruitment arrangements_For publication | N/A |
| Recruitment arrangements (for publication) | K1_KYV101-006_Add-Recruitment-IC-Procedure_DEU_Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_KYV101-006_ Main-ICF_Phase-3_DEU_deu_Public | 9.2 |
| Subject information and informed consent form (for publication) | L1_KYV101-006_ICF_Main Phase 2_DEU_deu_clean_Public | 7.4 |
| Subject information and informed consent form (for publication) | L1_KYV101-006_optFutResearch-ICF_DEU_deu_Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_KYV101-006_PP-ICF_DEU_deu_clean_Public | 4.0 |
| Subject information and informed consent form (for publication) | L2_KYV101-006_Patient-card_DEU_deu_Public | 1.0.0 |
| Subject information and informed consent form (for publication) | L2_KYV101-006_Patient-diary_DEU_deu_clean_Public | 2.0 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_Kyverna_KYV-101-006_SmPC_Azathioprine_Public | N/A |
| Summary of Product Characteristics (SmPC) (for publication) | E2_Kyverna_KYV-101-006_SmPC_Cyclosporin_Public | N/A |
| Summary of Product Characteristics (SmPC) (for publication) | E2_Kyverna_KYV-101-006_SmPC_Eculizumab_Public | N/A |
| Summary of Product Characteristics (SmPC) (for publication) | E2_Kyverna_KYV-101-006_SmPC_Ivig_Public | N/A |
| Summary of Product Characteristics (SmPC) (for publication) | E2_Kyverna_KYV-101-006_SmPC_Methotrexate_Public | N/A |
| Summary of Product Characteristics (SmPC) (for publication) | E2_Kyverna_KYV-101-006_SmPC_Mycophenolate-mofetil_Public | N/A |
| Summary of Product Characteristics (SmPC) (for publication) | E2_Kyverna_KYV-101-006_SmPC_Prednisolone_Public | N/A |
| Summary of Product Characteristics (SmPC) (for publication) | E2_Kyverna_KYV-101-006_SmPC_Ravulizumab_Public | N/A |
| Summary of Product Characteristics (SmPC) (for publication) | E2_Kyverna_KYV-101-006_SmPC_Tacrolimus_Public | N/A |
| Summary of Product Characteristics (SmPC) (for publication) | E2_Kyverna_KYV-101-006_SmPC_Zilucoplan_Public | N/A |
| Synopsis of the protocol (for publication) | D1_Kyverna_KYV101-006_Protocol synopsis_2023-509892-17-00_EEA_Public | 8.2 |
Application history
10 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-01-31 | Germany | Acceptable 2024-05-08
|
2024-05-13 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2024-08-19 | Germany | Acceptable 2024-05-08
|
2024-08-19 |
| 3 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-09-24 | Germany | Acceptable 2024-10-24
|
2024-10-24 |
| 4 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2025-01-16 | Germany | Acceptable 2024-10-24
|
2025-01-16 |
| 5 | NON SUBSTANTIAL MODIFICATION | NSM-3 | 2025-05-21 | Germany | Acceptable 2024-10-24
|
2025-05-21 |
| 6 | SUBSTANTIAL MODIFICATION | SM-4 | 2025-05-26 | Germany | Acceptable | 2025-06-13 |
| 7 | SUBSTANTIAL MODIFICATION | SM-5 | 2025-07-03 | Germany | Acceptable 2025-07-18
|
2025-07-22 |
| 8 | NON SUBSTANTIAL MODIFICATION | NSM-4 | 2025-07-25 | Germany | Acceptable 2025-07-18
|
2025-07-25 |
| 9 | NON SUBSTANTIAL MODIFICATION | NSM-5 | 2025-08-07 | Germany | Acceptable 2025-07-18
|
2025-08-07 |
| 10 | SUBSTANTIAL MODIFICATION | SM-8 | 2025-12-05 | Germany | Acceptable 2026-02-04
|
2026-02-06 |