Lymphatic MalformationsPhase 2/3EPIK-L1NCT05948943
Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.
A Phase 2/3 study testing Alpelisib in people with Lymphatic Malformations, run by Novartis Pharmaceuticals at 61 sites in 12 countries.
Trial summary
The main purpose of this study in participants with PIK3CA-mutated LyM is to assess the change in radiological response and symptom severity upon treatment with alpelisib film-coated tablets (FCT) as compared to placebo. Read more about this study
- Who is it for?
- Ages 0 to 100 years
- Conditions the study is for
- Lymphatic Malformations
- What is being tested?
- Alpelisib
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 232 participants
- Where
- 61 sitesin 12 countries
- Study dates
- 24 Nov 2023to 2 May 2033
- Phase
- Phase 2/3
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 0 to 100 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Signed informed consent and assent (when applicable) from the participant, parent, legal authorized representative or guardian.
- Participant must be willing to remain at the clinical site as required by the protocol and be willing to adhere to study restrictions and examination schedules.
- Participant has a physician confirmed and documented diagnosis of a symptomatic LyM at the time of informed consent (Note: the physician must confirm that the LyM cannot be included under the PROS diagnostic criteria).
- Participant is not considered as a candidate for or is not willing to receive non-drug therapies including but not limited to sclerotherapy, embolization, and surgery until the completion of Week 24 in Stage 1 and 2.
- Participant has evidence of a somatic mutation(s) in the PIK3CA gene prior to randomization.
- Participant has at least one measurable LyM lesion confirmed by BIRC assessment prior to randomization.
- Participants must be able to ingest study drug (either in tablet form or as a drinkable suspension [Groups 1 to 4] or granules or as an oral suspension [Group 5]) as assessed within 7 days before study treatment start. Drug administration via feeding tubes is allowed.
Who cannot join
- Participant has a physician-confirmed and documented diagnosis of PROS at the time of informed consent.
- Participant has a physician-confirmed and documented diagnosis of a Central Conducting Lymphatic Anomaly, General Lymphatic Anomaly, Gorham-Stout disease, Kaposiform lymphangiomatosis at the time of informed consent.
- Participant has a known history of Stevens-Johnson syndrome, erythema multiforme, or toxic epidermal necrolysis at the time of informed consent.
- Participant has an established diagnosis of type I diabetes mellitus or uncontrolled type II diabetes mellitus at the time of informed consent.
- Participant had previous treatment with alpelisib and/or any other PI3K inhibitors with treatment duration longer than 2 weeks at the time of informed consent.
- Other inclusion/exclusion criteria may apply
Trial locations and contacts
61 sites in 12 countries. Contact the study team or a site near you to ask about taking part.
61 sites in 12 countries
United States18 sites, 18 recruiting
- Recruiting
Childrens Hosp Boston Dept of Heme
Boston, Massachusetts, 02115
Kelsey Deemer, 617-355-5526, kelsey.deemer@childrens.harvard.edu
Tishi Shah
- Recruiting
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
Miriam Davis, miriam.davis@unc.edu
Alexandra Borst
- Recruiting
Cinn Children Hosp Medical Center
Cincinnati, Ohio, 45229-3039
Sarah Price, 513-636-6090, Sarah.Price@cchmc.org
Adrienne Hammill
- Recruiting
Univ Hospital Of Cleveland
Cleveland, Ohio, 44106
Erika Waites, 216-844-7028, Erika.Waites@uhhospitals.org
Howard Wang
- Recruiting
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Natalie Mau, maun4@ccf.org
Michael Kelly
- Recruiting
Nationwide Children s Hospital
Columbus, Ohio, 43205
Star Seum, star.seum@nationwidechildrens.org
Bhuvana Setty
- Recruiting
Baylor College Of Medicine
Houston, Texas, 77030
Gaylon Stevenson, 713-798-3701, gnsteve1@texaschildrens.org
Ionela Iacobas
- Recruiting
U of TX Health Science Ct
Houston, Texas, 77030
Kelly Mulligan, 713-500-6852, kelly.mulligan@uth.tmc.edu
Autumn Atkinson
- Recruiting
Nemours Childrens Clinic
Jacksonville, Florida, 32207
Rosita Almira, 904-697-3674, rosita.almira@nemours.org
Anderson Collier
- Recruiting
UCSF Benioff Children s Hospital
Oakland, California, 94609
Sophie Shean, +1 510 428 3885 8227, sophie.shean@ucsf.edu
Beth Apsel Winger
- Recruiting
Childrens Hospital of Orange County
Orange, California, 92868
Eliud Espinal, 714-509-8810, eliud.espinal@choc.org
Maria Buethe
- Recruiting
Lucile Packard Childrens Hosp
Palo Alto, California, 94304
Thomas Buschbacher, tbusch@stanford.edu
Joyce Teng
- Recruiting
CHOP Abramson Pediatric Resch Ctr
Philadelphia, Pennsylvania, 19104
Melissa Casey, CASEYMA@chop.edu
Denise Adams
- Recruiting
Childrens Hosp Pittsburgh UPMC
Pittsburgh, Pennsylvania, 15224
Alex Berkebile, 412-692-5346, berkebileak@upmc.edu
Julia Segal
- Recruiting
Oregon Health Science University
Portland, Oregon, 97239
Annie Yang, yangann@ohsu.edu
Melinda Wu
- Recruiting
Childrens Hospital and Regional Medical Center
Seattle, Washington, 98105
Maya Younoszai, 206-526-2100, maya.younosza@seattlechildrens.org
Jonathan A Perkins
- Recruiting
WA Uni School Of Med
St Louis, Missouri, 63110
Alison Barnwell, abarnwell@wustl.edu
Bryan Sisk
- Recruiting
Childrens National Medical Center
Washington D.C., District of Columbia, 20010-2970
Regine Hyppolite, rhyppolite@childrensnational.org
Yaser Diab
France12 sites, 12 recruiting
- Recruiting
Novartis Investigative Site
Angers, 49933
- Recruiting
Novartis Investigative Site
Bordeaux, 33076
- Recruiting
Novartis Investigative Site
Bron, 69677
- Recruiting
Novartis Investigative Site
Caen, 14033
- Recruiting
Novartis Investigative Site
Dijon, 21000
- Recruiting
Novartis Investigative Site
Lille, 59000
- Recruiting
Novartis Investigative Site
Marseille, 13885
- Recruiting
Novartis Investigative Site
Montpellier, 34295
- Recruiting
Novartis Investigative Site
Paris, 75010
- Recruiting
Novartis Investigative Site
Paris, 75015
- Recruiting
Novartis Investigative Site
Toulouse, 31054
- Recruiting
Novartis Investigative Site
Tours, 37044
Spain7 sites, 7 recruiting
- Recruiting
Novartis Investigative Site
A Coruña, 15006
- Recruiting
Novartis Investigative Site
Barcelona, 08035
- Recruiting
Novartis Investigative Site
Esplugues, Barcelona, 08950
- Recruiting
Novartis Investigative Site
L'Hospitalet de Llobregat, Barcelona, 08907
- Recruiting
Novartis Investigative Site
Madrid, 28009
- Recruiting
Novartis Investigative Site
Madrid, 28046
- Recruiting
Novartis Investigative Site
Palma, Balearic Islands, 07120
Germany6 sites, 5 recruiting
- Recruiting
Novartis Investigative Site
Berlin, 13353
- Recruiting
Novartis Investigative Site
Cologne, North Rhine-Westphalia, 50937
- Recruiting
Novartis Investigative Site
Freiburg im Breisgau, Baden-Wurttemberg, 79106
- Recruiting
Novartis Investigative Site
Leipzig, Saxony, 04103
- Withdrawn
Novartis Investigative Site
Mannheim, Baden-Wurttemberg, 68305
- Recruiting
Novartis Investigative Site
Ulm, 89081
Italy6 sites, 6 recruiting
- Recruiting
Novartis Investigative Site
Bologna, BO, 40138
- Recruiting
Novartis Investigative Site
Milan, MI, 20122
- Recruiting
Novartis Investigative Site
Naples, 80122
- Recruiting
Novartis Investigative Site
Roma, RM, 00165
- Recruiting
Novartis Investigative Site
Roma, RM, 00168
- Recruiting
Novartis Investigative Site
Torino, TO, 10126
Argentina3 sites, 3 recruiting
- Recruiting
Novartis Investigative Site
CABA, Buenos Aires, C1181ACH
- Recruiting
Novartis Investigative Site
CABA, Buenos Aires, C1425BEA
- Recruiting
Novartis Investigative Site
Capital Federal, C1023AAB
Australia3 sites, 3 recruiting
- Recruiting
Novartis Investigative Site
Brisbane, Queensland, 4101
- Recruiting
Novartis Investigative Site
Sydney, New South Wales, 2010
- Recruiting
Novartis Investigative Site
Sydney, New South Wales, 2031
Netherlands2 sites, 2 recruiting
- Recruiting
Novartis Investigative Site
Nijmegen, 6525 GA
- Recruiting
Novartis Investigative Site
Rotterdam, South Holland, 3015 GD
Belgium1 site, 1 recruiting
- Recruiting
Novartis Investigative Site
Brussels, 1200
Czechia1 site, 1 recruiting
- Recruiting
Novartis Investigative Site
Brno, 625 00
Switzerland1 site, 1 recruiting
- Recruiting
Novartis Investigative Site
Lausanne, 1011
United Kingdom1 site, 1 recruiting
- Recruiting
Novartis Investigative Site
London, WC1N 3JH
About this study
Official title: A Two-stage Double-blind, Randomized, Placebo-controlled Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.
The main purpose of this study in participants with PIK3CA-mutated LyM is to assess the change in radiological response and symptom severity upon treatment with alpelisib film-coated tablets (FCT) as compared to placebo.
Read the detailed description
This is a phase II/III multi-center study with two stages:
- Stage 1 is designed to select the dose(s) for the confirmatory phase (DSCP) for alpelisib in Stage 2 and will comprise a 24-week open-label core phase in adult (≥18 years of age) and pediatric participants (6-17 years of age) with PIK3CA-mutated LyM, followed by an extension. After eligibility has been confirmed at screening, participants will be randomized in a 1:1 ratio to the different alpelisib doses according to their age. Depending on the results at the end of Stage 1 core phase, the Stage 2 will be opened to adult and/or pediatric participants or the study may be stopped.
- Stage 2 is designed to confirm the efficacy and assess safety of alpelisib at the DSCP in participants with PIK3CA-mutated LyM and will comprise a 24-week randomized, double blind, placebo-controlled confirmatory phase in adult (≥18 years of age) and pediatric participants 6-17 years of age followed by an open-label extension. After eligibility has been confirmed at screening participants will be randomized in a 2:1 ratio to alpelisib or placebo.
Additionally, in parallel, Stage 2 will include a 24-week open-label core phase in pediatric participants 0-5 years of age followed by an extension, if pediatric participants will be enrolling in Stage 2.
Based on the results of the 24-week open-label core phase of Stage 1, the dose(s) for Stage 2 will be selected by Novartis in consultation with the Steering Committee (SC). During the 24-week randomized, double blind, placebo-controlled core phase of Stage 2, an Independent Data Monitoring Committee (DMC) will conduct periodic safety and efficacy reviews to assess the risk benefit profile of the treatment.
- Conditions
- Lymphatic Malformations
- Being tested
- Alpelisib, Placebo
- Keywords
- Lymphatic malformations (LyM), Cystic lymphangioma, Cystic hygroma, Lymphangioma circumscriptum, Cavernous lymphangioma, Lymphangioma, Macrocystic lymphatic malformation, Microcystic lymphatic malformationn, Alpelisib
- Medical subject headings
- Lymphatic Abnormalities, Lymphangioma, Cystic, Lymphangioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AlpelisibDrug
Also called BYL719
In Stage 1: adult participants (≥18 years of age) will receive dose 1 or dose 2 of alpelisib; pediatric participants (6-17 years of age) will receive dose 2 or dose 3 of alpelisib.
In Stage 2: Adult participants will receive alpelisib at the dose selected for confirmatory phase in adult participants; pediatric participants (6-17 years of age) will will receive alpelisib at the dose selected for confirmatory phase in pediatric participants; and pediatric participants of 0-5 years of age will receive dose 3 of alpelisib
PlaceboDrug
In Stage 2, participants will receive matching placebo for 24 weeks of the study
| Study group | Role | What they receive |
|---|---|---|
| Adult participants, alpelisib dose 1 (Stage 1) | Experimental | Adult participants (≥18 years of age) who will receive dose 1 of alpelisib an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1) Receives: Drug: Alpelisib |
| Adult participants, alpelisib dose 2 (Stage 1) | Experimental | Adult participants (≥18 years of age) who will receive dose 2 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1). Receives: Drug: Alpelisib |
| Pediatric participants (6-17 years of age), alpelisib dose 2 (Stage 1) | Experimental | Pediatric participants 6-17 years of age who will receive dose 2 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1) Receives: Drug: Alpelisib |
| Pediatric participants (6-17 years of age), alpelisib dose 3 (Stage 1) | Experimental | Pediatric participants 6-17 years of age who will receive dose 3 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1). Receives: Drug: Alpelisib |
| Adult participants, alpelisib (Stage 2) | Experimental | Adult participants (≥18 years of age) who will receive alpelisib at the dose selected for confirmatory phase in adult participants (Stage 2) Receives: Drug: Alpelisib |
| Adult participants, placebo (Stage 2) | Placebo comparator | Adult participants (≥18 years of age) who will receive matching placebo Receives: Drug: Placebo |
| Pediatric participants (6-17 years of age), alpelisib (Stage 2) | Experimental | Pediatric participants (6-17 years of age) who will receive alpelisib at the dose selected for confirmatory phase in pediatric participants (Stage 2) Receives: Drug: Alpelisib |
| Pediatric participants (6-17 years of age), placebo (Stage 2) | Placebo comparator | Pediatric participants (6-17 years of age) who will receive matching placebo Receives: Drug: Placebo |
| Pediatric participants (0-5 years of age), alpelisib (Stage 2) | Experimental | Pediatric participants of 0-5 years who will dose 3 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier Receives: Drug: Alpelisib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Stage 2:Radiological response rate at Week 24 of Stage 2 (adult and pediatric (6 - 17 years of age) participants)
Radiological response defined by achieving at least 20% reduction in the sum of target lesion volumes (1 to 3 lesions), assessed by MRI by a BIRC at Week 24, provided that none of the individual target lesions has at least 20% increase from baseline and in absence of progression of non-target lesions and without new lesions. The percentage of participants with a radiological response at Week 24 of Stage 2 in adult and pediatric (6-17 years of age) groups will be assessed
Measured: Baseline, Week 24
Secondary outcomes (18)
Stage 2: Percentage of participants with at least a 1-point improvement compared to baseline based on patient global impression of severity (PGI-S) scale at Week 24 of Stage 2 (adult and pediatric (6 - 17 years of age) participants)
PGI-S is a single item measure to assess the overall severity of a patient's condition. This single item instrument uses a 5-point rating scale, which ranges from 1 (no symptoms) to 5 (very severe). Lower scores indicate better health status. The percentage of participants with at least a 1-point improvement compared to baseline at Week 24 of Stage 2 in adult and pediatric (6-17 years of age) groups will be assessed
Measured: Baseline, Week 24
Stage 2: Percentage of participants with a radiological response at Week 24 of Stage 2 (pediatric participants 0-5 years of age)
Radiological response defined by achieving at least 20% reduction in the sum of target lesion volumes (1 to 3 lesions), assessed by MRI by a BIRC at Week 24, provided that none of the individual target lesions has at least 20% increase from baseline and in absence of progression of non-target lesions and without new lesions. The percentage of participants with a radiological response at Week 24 of Stage 2 in pediatric participants 0-5 years of age will be assessed
Measured: Baseline, Week 24
Stage 2: Change from baseline in patient global impression of change (PGI-C) scale (adult and pediatric (6-17 years of age) participants)
PGI-C is a single item measure to assess the change in overall symptoms severity since the start of study. This single item instrument uses a 7-point rating scale, which ranges from 1 (very much improved) to 7 (very much worse). Lower scores indicate better health status. The change from baseline in PGI-C score will be assessed in adult and pediatric (6-17 years of age) participants
Measured: Up to approximately 8 years
Stage 2: Change from baseline in patient-reported outcomes measurement information system (PROMIS) profile domains(adult and pediatric (6-17 years of age) participants)
The PROMIS-29 plus 2 Profile v2.1 (completed by adult participant) includes 29 items across domains of depression, anxiety, physical function, pain interference, fatigue, sleep disturbance, ability to participant in social roles and activities, cognitive function abilities and pain intensity.
The PROMIS Pediatric-25 Profile v2.0 (completed by children over 8 years of age) and PROMIS Parent-Proxy-25 Profile v2.0 (completed by parents for children under 8 years of age) include 25 items across the domains of depressive symptoms, anxiety, physical function-mobility, pain interference, fatigue, and peer relationships and pain intensity All items (except the pain intensity item) use a 5-point Likert scale, which ranges from 1 (not at all) to 5 (very much). The pain intensity item is scored on a 0-10 numeric rating scale, where 0 represents "no pain" and 10 represents "worst imaginable pain".
The change from baseline in PROMIS domains will be assessed
Measured: Up to approximately 8 years
Stage 2: Change from baseline in investigator global impression of change (IGIC) scale (adult and pediatric (6-17 years of age) participants)
The IGIC involves a single question that asks the investigator to rate the change in the patient's condition since the start of treatment or intervention, using a 7-point scale ranging from 1 (Very much improved) to 7 (Very much worse). The change from baseline in IGIC score will be assessed in adult and pediatric (6-17 years of age) participants
Measured: Up to approximately 8 years
Stage 2: Change from baseline in health utilities of the EuroQol 5-dimension (EQ-5D) (adult and pediatric (6-17 years of age) participants)
The EQ-5D-5L (completed by adult participants) includes 5 items addressing dimensions of mobility, self-care, usual activities, pain/discomfort, and anxiety/depression), each with 5 response options, ranging from 1=no problems to 5=extreme problems The EQ-5D-Y (completed by children over 8 years of age) and EQ-5D-Y Proxy version (completed by parent for participants under 8 years of age or unable to record for themselves) includes 5 items addressing dimensions of mobility, self-care, usual activities, pain/discomfort, and anxiety/depression), each with 3 response options, ranging from 1= no problems to 3= a lot of problems
Measured: Up to approximately 8 years
Stage 1 and 2: Duration of response (DOR) in adult and pediatric participants who receive alpelisib
DOR is defined as the time from first documented response until progression of LyM lesions by BIRC or death. This analysis only applies to participants who are on treatment with alpelisib (Stage 1 and 2) and who achieve response.
Measured: Up to approximately 8 years
Stage 1: Radiological response rate of alpelisib in adult and pediatric (6-17 years of age) participants
Radiological response defined by achieving at least 20% reduction in the sum of target lesion volumes (1 to 3 lesions), assessed by MRI by a BIRC at Week 24, provided that none of the individual target lesions has at least 20% increase from baseline and in absence of progression of non-target lesions and without new lesions.
The percentage of participants (adult and pediatric 6-17 years of age) with radiological response at Week 24 of Stage 1 will be assessed.
Measured: Baseline, Week 24
Stage 1 and 2: Radiological response rate of alpelisib in adult and pediatric participants
Radiological response defined by achieving at least 20% reduction in the sum of target lesion volumes (1 to 3 lesions), assessed by MRI by a BIRC, provided that none of the individual target lesions has at least 20% increase from baseline and in absence of progression of non-target lesions and without new lesions.
The percentage of participants who receive alpelisib (Stage 1 and 2) with radiological response will be assessed.
Measured: Up to approximately 8 years
Stage 1 and 2: Alpelisib plasma concentrations
Alpelisib plasma concentrations in adult and pediatric participants (Stage 1 and 2).
Measured: On Day 1 of Week 8, 16, 24, 48 and 120
Stage 1 and 2: Percentage of participants with of LyM-related symptoms, complications, and comorbidities on treatment with alpelisib in adult and pediatric participants at Week 24
Percentage of participants with of LyM-related symptoms, complications, and comorbidities on treatment with alpelisib in adult and pediatric participants at Week 24 (Stage 1 and 2)
Measured: Week 24
Stage 1 and 2: Percentage of participants with of LyM-related symptoms, complications, and comorbidities on treatment with alpelisib in adult and pediatric participants
Percentage of participants with of LyM-related symptoms, complications, and comorbidities on treatment with alpelisib in adult and pediatric participants (Stage 1 and 2)
Measured: Up to approximately 8 years
Stage 1 and 2: Change from baseline in LyM lesions in adult and pediatric participants at Week 24
Change from baseline in the sum of target lesion volumes, the sum of MRI-measurable non-target lesion (if applicable) volumes, and the sum of all MRI-measurable lesion (target and non-target) volumes as assessed by BIRC at Week 24 in adult and pediatric participants (Stage 1 and 2)
Measured: Baseline, Week 24
Stage 1 and 2: Change from baseline in LyM lesions in adult and pediatric participants
Change from baseline in the sum of target lesion volumes, the sum of MRI-measurable non-target lesion (if applicable) volumes, and the sum of all MRI-measurable lesion (target and non-target) volumes as assessed by BIRC in adult and pediatric participants (Stage 1 and 2)
Measured: Up to approximately 8 years
Stage 1 and 2: Percentage of participants with changes in non-target lesions in adult and pediatric participants at Week 24
Percentage of participants with changes in non-target lesions as assessed by BIRC at Week 24 in adult and pediatric participants (Stage 1 and 2)
Measured: Baseline, Week 24
Stage 1 and 2: Percentage of participants with changes in non-target lesions in adult and pediatric participants
Percentage of participants with changes in non-target lesions as assessed by BIRC in adult and pediatric participants (Stage 1 and 2)
Measured: Up to approximately 8 years
Stage 1 and 2: Percentage of participants with new lesions in adult and pediatric participants at Week 24
The percentage of participants with new lesions as assessed by BIRC at Week 24 in adult and pediatric participants (Stage 1 and 2)
Measured: Baseline, Week 24
Stage 1 and 2: Percentage of participants with new lesions in adult and pediatric participants
The percentage of participants with new lesions as assessed by BIRC in adult and pediatric participants (Stage 1 and 2)
Measured: Up to approximately 8 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jul 2023
- Study start24 Nov 2023
- Primary completion31 May 2028 (estimated)
- Study completion2 May 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lymphatic MalformationsStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 232 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05948943
- Sponsor's protocol ID
- CBYL719P12201
- Other IDs
- 2023-504146-60-00, 2023
- Acronym
- EPIK-L1
- Status last verified
- Oct 2026
- First submitted
- 4 Jul 2023
- Last updated
- 9 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
- Expanded access
- Available