Clinical trial • Phase III • Rare Disease|Musculoskeletal
ETEPLIRSEN for Duchenne muscular dystrophy
Phase III trial of ETEPLIRSEN for Duchenne muscular dystrophy.
Overview
- Trial Therapeutic Area
- Rare Disease|Musculoskeletal
- Trial Disease
- Duchenne muscular dystrophy
- Trial Stage
- Phase III
- Drug Modality
- Oligonucleotide
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 26-09-2024
- First CTIS Authorization Date
- 04-11-2024
Trial design
Randomised, open-label, eteplirsen 30 mg/kg iv weekly (comparator arm) compared with eteplirsen 100 mg/kg iv weekly and 200 mg/kg iv weekly; an open-label dose escalation evaluates weekly iv doses of 100 mg/kg and 200 mg/kg prior to the double-blind dose-finding/comparison.-controlled, adaptive Phase III trial in Italy, Denmark, Czechia and others.
- Randomised
- Yes
- Open Label
- Yes
- Comparator
- eteplirsen 30 mg/kg IV weekly (comparator arm) compared with eteplirsen 100 mg/kg IV weekly and 200 mg/kg IV weekly; an open-label dose escalation evaluates weekly IV doses of 100 mg/kg and 200 mg/kg prior to the double-blind dose-finding/comparison.
- Adaptive
- True, open-label dose-escalation design followed by double-blind dose-finding and dose-comparison; includes interim (conditional efficacy) analysis at Week 72 or 96 and dose-escalation safety/tolerability rules.
- Single Multiple Or Escalation Dose Combined
- Yes
- Target Sample Size
- 123
- Trial Duration For Participant
- 1008
Eligibility
Recruits 123 paediatric patients.
- Vulnerable Population
- Participants are minors (male pediatric patients aged 4–13). Informed assent from participants is required where applicable and informed consent must be provided by parent(s) or legal guardian(s). Age-specific assent forms are provided (examples: Assent Form for ages 4-6, Assent Form for ages 7-9, Assent Form for ages 10-14 and equivalents); parental/main informed consent forms are provided (parental ICFs). Documents and ICF/assent templates are provided in multiple country-specific languages.
Inclusion criteria
- {"criterion_text":"- 1. Be a male with an established clinical diagnosis of DMD and an out-of- frame deletion mutation of the DMD gene amenable to exon 51 skipping (for example, deletions of exons 45-50, 47-50, 48-50, 49-50, 50, 52, and 52-63)."}
- {"criterion_text":"- 2. Be aged 4 to 13 years, inclusive"}
- {"criterion_text":"- 3. Ambulatory patient, able to perform TTRISE in 10 seconds or less at the time of screening visit."}
- {"criterion_text":"- 4. Able to walk independently without assistive devices."}
- {"criterion_text":"- 5. Has intact right and left biceps muscles (the preferred biopsy site) or an alternative upper arm muscle group that will allow for sufficiently sized (1 cm3) muscle biopsies to be obtained prior to and on treatment (for patients in the double-blind part of the study)."}
- {"criterion_text":"- 6. Has been on a stable dose or dose equivalent of oral corticosteroids for at least 12 weeks prior to randomization, and the dose is expected to remain constant (except for modifications to accommodate changes in weight and stress-related needs as per the recently published guidelines [Birnkrant 2018, Kinnett 2017]) throughout the study)."}
- {"criterion_text":"- 7. For ages 7 years and older, has stable pulmonary function (forced vital capacity ≥50% of predicted and no requirement for nocturnal ventilation) that, in the Investigator's opinion, is unlikely to decompensate significantly over the duration of the study. For ages 4 to 6 years , does not require support from ventilator or non- invasive ventilation at time of screening."}
- {"criterion_text":"- 8. If sexually active, agree to use a male condom during such activity for the entire duration of the study and for 90 days after the last dose. The sexual partner must also use a medically acceptable form of contraceptive (ie, female oral contraceptives) during this timeframe. Acceptable methods of contraception include combined (estrogen and progesterone containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal); progesterone- only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable); intrauterine device; intra-uterine hormone-releasing system; bilateral tubal occlusion; vasectomized partner; sexual abstinence (True abstinence: When this is in line with the preferred and usual lifestyle of the patient. Periodic abstinence [such as calendar, ovulation, symptothermal, post-ovulation methods] and withdrawal are not acceptable methods of contraception.); or condom in combination with either cap, diaphragm, or sponge with spermicide (double-barrier contraception)."}
- {"criterion_text":"- 9. Has (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with all the study requirements."}
- {"criterion_text":"- 10. Is willing to provide informed assent (if applicable) and has (a) parent(s) or legal guardian(s) who is (are) willing to provide informed consent for the patient to participate in the study."}
Exclusion criteria
- {"criterion_text":"- 1. Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks prior to randomization that may have an effect on muscle strength or function. Growth hormone for short stature and testosterone for delayed puberty are permitted if physician has documented the diagnosis and medical necessity of treatment and if the patient has been on a stable dose for at least 24 weeks prior to randomization."}
- {"criterion_text":"- 2. Current or previous treatment with any other experimental pharmacologic treatment for DMD or any prior exposure to antisense oligonucleotide, gene therapy or gene editing; except the following: Ezutromid administered at least 12 weeks prior to first dose. Drisapersen administered at least 36 weeks prior to first dose. Suvodirsen administered at least 12 weeks prior to first dose. Vamorolone administered at least 12 weeks prior to first dose. Eteplirsen (previous or current use) Tamoxifen administered at least 4 weeks prior to first dose."}
- {"criterion_text":"- 3. Major surgery within 3 months prior to randomization or planned surgery for any time during this study, except for allowed protocol- specified surgery, as applicable."}
- {"criterion_text":"- 4. Presence of any significant neuromuscular or genetic disease other than DMD (eg, dwarfism)."}
- {"criterion_text":"- 5. Gamma-glutamyl transpeptidase (GGT) > 3 × the upper limit of normal (ULN) or serum bilirubin > ULN unexplained by Gilbert's Syndrome."}
- {"criterion_text":"- 6. Any known impairment of renal function (eg, estimated glomerular filtration rate [eGFR] ≤ 60 mL/min as assessed by the Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] cystatin C based equation [Inker, 2012, Filler 2012]), or dipstick protein result +2, or persistent and unexplained dipstick protein result +1"}
- {"criterion_text":"- 7. Platelet count < the lower limit of normal."}
- {"criterion_text":"- 8. Presence of other clinically significant illness including significant cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease or malignancy."}
- {"criterion_text":"- 9. Has evidence of cardiomyopathy, as defined by left ventricular ejection fraction <50% on the screening ECHO or the Fridericia's correction formula (QTcF) ≥450 milliseconds based on the screening ECGs."}
- {"criterion_text":"- 10. Prior or ongoing medical condition that could, in the Investigator's opinion, adversely affect the safety of the patient, make it unlikely that the course of treatment would be completed, or impair the assessment of study results."}
- {"criterion_text":"- 11. Known hypersensitivity to eteplirsen or any excipients of eteplirsen."}
- {"criterion_text":"- 12. Is, in the Investigator's opinion, unable or unwilling to comply with the study procedures."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Open-Label Dose Escalation: Incidence of AEs, Incidence of adverse events of special interest (AESIs), Abnormal changes from Baseline or worsening of vitals or physical examination findings, Incidence of SAEs, Safety laboratory assessments, Electrocardiograms (ECGs) and Echocardiograms (ECHO).","definition_or_measurement_approach":"Safety assessed by incidence rates of AEs, AESIs, SAEs; monitoring of abnormal changes from baseline in vital signs and physical examination; safety laboratory assessments; ECG and ECHO assessments."}
- {"endpoint_text":"- Double-Blind Dose Finding and Dose Comparison Part: -Change from Baseline at Week 72 or Week 96 in NSAA total score (for conditional efficacy interim analysis) -Change from Baseline at Week 144 in NSAA total score (for final analysis)","definition_or_measurement_approach":"Efficacy measured as change from baseline in North Star Ambulatory Assessment (NSAA) total score at specified timepoints (Week 72 or 96 for interim conditional analysis; Week 144 for final analysis)."}
Secondary endpoints
- {"endpoint_text":"- • Change from Baseline at Week 144 in o Time to rise (TTRISE) from floor o 10-meter walk/run time o 6-minute walk test (6MWT) o Timed 4-step stair ascend test o Forced vital capacity percent predicted (FVC%p)","definition_or_measurement_approach":"Functional and pulmonary measures: change from baseline at Week 144 in TTRISE, 10-meter walk/run time, 6MWT distance, timed 4-step stair ascend, and percent predicted FVC."}
- {"endpoint_text":"- • Time to Loss of Ambulation (LOA) through Week 144","definition_or_measurement_approach":"Time-to-event endpoint measuring time until loss of ambulation through study Week 144."}
- {"endpoint_text":"- • Change from Baseline at Week 24, Week 48, or Week 144 in skeletal muscle dystrophin expression by: o Western blot (quantitation) o Immunohistochemistry (IHC) fiber intensity by Immunofluorescence o Exon skipping quantitation by droplet digital polymerase chain reaction (ddPCR) o Percent dystrophin-positive fibers (PDPF) by Immunofluorescence","definition_or_measurement_approach":"Biomarker endpoints assessing dystrophin expression using Western blot quantitation, IHC/immunofluorescence fiber intensity, exon skipping quantitation by ddPCR, and percent dystrophin-positive fibers by immunofluorescence at specified timepoints."}
- {"endpoint_text":"- PK parameters (plasma and muscle biopsy)","definition_or_measurement_approach":"Pharmacokinetic parameters measured in plasma and muscle biopsy samples."}
- {"endpoint_text":"- • Incidence of AEs • Incidence of AESIs • Incidence of SAEs • Safety laboratory assessments","definition_or_measurement_approach":"Safety endpoints measured by incidence counts/rates of AEs, AESIs, SAEs and safety laboratory test results."}
- {"endpoint_text":"- • ECGs • ECHO • Abnormal changes from Baseline or worsening of vital signs and physical examination findings","definition_or_measurement_approach":"Cardiac and clinical safety assessments via ECGs, ECHO and monitoring of abnormal changes from baseline in vital signs and physical exam findings."}
Recruitment
- Digital Remote Recruitment
- True, recruitment-related digital/remote methods referenced include use of patient portal/site portals (site and patient porta), Greenphire ClinCard messaging/bank transfer templates/FAQs, and digital contact details for a sponsor patient recruitment hub (email and telephone).
- Planned Sample Size
- 123
- Recruitment Window Months
- 53
- Consent Approach
- Parental/legal guardian informed consent required for participants; informed assent required from participants when applicable. Age-specific assent forms and parental ICFs are provided (examples: Assent Form ages 4-6, 7-9, 10-14; Parental ICFs for dose finding/comparison). Consent/assent materials are available in multiple country-specific languages (documents present in English, Greek, German, Turkish, Dutch, Norwegian, Hungarian, Polish, Spanish, French, Italian, Czech, Romanian and others as per country submissions).
Methods
- Sponsor patient recruitment contact: functional contact 'Patient Recruitment' (SareptAlly@sarepta.com, telephone +18887273782) to support recruitment.
- Use of external vendors and third parties for recruitment and retention explicitly identified (examples: Elligo Health Research Inc. — 'Patient recruitment and retention'; 4g Clinical LLC listed as a third party).
- Country-specific recruitment arrangement documents were submitted (recruitment arrangement/K1 documents present for multiple countries).
- Patient travel reimbursement and related logistics via Greenphire ClinCard (duties: 'Patient travel reimbursement', messaging and bank transfer FAQs).
Geography
- Total Number Of Sites
- 16
- Total Number Of Participants
- 37
Italy
- Latest Decision Or Authorization Date
- 27-01-2026
- Number Of Sites
- 2
- Number Of Participants
- 3
Sites
- Site Name
- IRCCS Ospedale Policlinico San Martino
- Department Name
- U.O.S.D. Centro Traslazionale di Miologia e Patologie Neurodegenerative
- Contact Person Name
- Claudio Bruno
- Contact Person Email
- claudiobruno@ospedalegaslini.ge.it
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- Neuropsichiatria Infantile
- Contact Person Name
- Eugenio Mercuri
- Contact Person Email
- eumercuri@gmail.com
Denmark
- Latest Decision Or Authorization Date
- 04-11-2024
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Rigshospitalet
- Department Name
- Department of Pediatric Neurology
- Contact Person Name
- Malene Landbo Børresen
- Contact Person Email
- malene.landbo.02.boerresen@regionh.dk
Czechia
- Latest Decision Or Authorization Date
- 27-01-2026
- Number Of Sites
- 2
- Number Of Participants
- 3
Sites
- Site Name
- Fakultni Nemocnice Brno
- Department Name
- Klinika dětské neurologie LF MU a FN Brno Dětská nemocnice
- Contact Person Name
- Lenka Juříková
- Contact Person Email
- Mrazova.Lenka@fnbrno.cz
- Site Name
- Fakultni Nemocnice V Motole
- Department Name
- Klinika dětské neurologie 2.LF UK a FN Motol
- Contact Person Name
- Jana Haberlová
- Contact Person Email
- jana.haberlova@fnmotol.cz
Norway
- Latest Decision Or Authorization Date
- 28-01-2026
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Helse Stavanger HF
- Department Name
- Barneklinikk, Gerd-Ragna Bloch Thorsens gate 8, 4011 Stavanger
- Contact Person Name
- Katja Sara Holmefjord
- Contact Person Email
- katja.sara.brocke.holmefjord@sus.no
Hungary
- Latest Decision Or Authorization Date
- 05-11-2024
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Semmelweis University
- Department Name
- Genomikai Medicina és Ritka Betegségek Intézete
- Contact Person Name
- Mária Judit Molnár
- Contact Person Email
- molnarmj@gmail.com
Netherlands
- Latest Decision Or Authorization Date
- 02-02-2026
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Radboud universitair medisch centrum Stichting
- Department Name
- Medical Rehabilitation
- Contact Person Name
- Saskia Houwen
- Contact Person Email
- clinicaltrials.reval@radboudumc.nl
Spain
- Latest Decision Or Authorization Date
- 02-02-2026
- Number Of Sites
- 2
- Number Of Participants
- 3
Sites
- Site Name
- Hospital Sant Joan De Deu Barcelona
- Department Name
- Pediatric Neurology
- Contact Person Name
- Andrés Nascimento
- Contact Person Email
- andres.nascimento@sjd.es
- Site Name
- Hospital Universitario Y Politecnico La Fe
- Department Name
- Neurology
- Contact Person Name
- Nuria Muelas
- Contact Person Email
- muelas_nur@gva.es
France
- Latest Decision Or Authorization Date
- 30-01-2026
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Association Institut De Myologie
- Department Name
- I-Motion institute
- Contact Person Name
- Andreea SEFERIAN
- Contact Person Email
- a.seferian@institut-myologie.org
Germany
- Latest Decision Or Authorization Date
- 28-01-2026
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- Klinik für Pädiatrie mit Schwerpunkt Neurologie und Sozialpädiatrisches Zentrum der Charité
- Contact Person Name
- Claudia Weiss
- Contact Person Email
- claudia.weiss@charite.de
Greece
- Latest Decision Or Authorization Date
- 13-05-2025
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Iaso Private General Obstetrics Gynecological & Pediatric Clinic Diagnostic Therapeutic & Research Center S.A.
- Department Name
- Neurological Department of Pediatric Clinic
- Contact Person Name
- Antigoni E. Papavasileiou
- Contact Person Email
- theon@otenet.gr
Slovenia
- Latest Decision Or Authorization Date
- 30-01-2026
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- University Medical Center Ljubljana
- Department Name
- Department of Pediatric Neurology
- Contact Person Name
- Damjan Osredkar
- Contact Person Email
- damjan.osredkar@kclj.si
Poland
- Latest Decision Or Authorization Date
- 30-01-2026
- Number Of Sites
- 1
- Number Of Participants
- 7
Sites
- Site Name
- Uniwersyteckie Centrum Kliniczne
- Department Name
- Klinika Neurologii Rozwojowej
- Contact Person Name
- Maria Mazurkiewicz-Bełdzińska
- Contact Person Email
- mmazur@gumed.edu.pl
Romania
- Latest Decision Or Authorization Date
- 24-03-2026
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Centrul National Clinic De Recuperare Neuropsihomotorie Copii Dr. Nicolae Robanescu
- Department Name
- Sectia Neurologie Pediatrica
- Contact Person Name
- Madalina Cristina Leanca
- Contact Person Email
- mada_mada332@yahoo.com
Sponsor
Primary sponsor
- Full Name
- Sarepta Therapeutics Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Pharmaceutical Product Development LLC
- Responsibilities
- coagulation screening, urinalysis, viral detection, renal function blood test, and liver chemistry; code: 4
- Name
- PPD Development LP
- Responsibilities
- Project and Clinical Management, Biopsy kits Supply (GCS); project and clinical management roles
- Name
- PPD Global Ltd.
- Responsibilities
- Project management duties or monitoring/regulatory; project management and monitoring/regulatory duties
- Name
- 4g Clinical LLC
- Responsibilities
- sponsorDuties code: 3
- Name
- Elligo Health Research Inc.
- Responsibilities
- Patient recruitment and retention
- Name
- Pharpoint Research Inc.
- Responsibilities
- Biostatistics/statistical programming
- Name
- Marken LLP
- Responsibilities
- Home health care vendor
- Name
- Emvenio Clinical Research LLC
- Responsibilities
- Home Health Care
Third parties
- {"country":"United States","full_name":"4g Clinical LLC","duties_or_roles":"sponsorDuties codes: 3","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Sarepta Therapeutics Inc.","duties_or_roles":"Muscle biopsy analysis","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"coagulation screening, urinalysis, viral detection, renal function blood test, and liver chemistry; sponsorDuties code: 4","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"University Of Iowa Hospitals And Clinics","duties_or_roles":"sponsorDuties code: 4","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Patient travel reimbursement","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Elligo Health Research Inc.","duties_or_roles":"Patient recruitment and retention","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"Singapore","full_name":"Eurofins Central Laboratory Pte Ltd","duties_or_roles":"GLDH Testing","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Emvenio Clinical Research LLC","duties_or_roles":"Home Health Care","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"Singapore","full_name":"PPD Global Central Labs (S) Pte Ltd","duties_or_roles":"coagulation screening, urinalysis, viral detection, renal function blood test, and liver chemistry; sponsorDuties code: 4","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Pharpoint Research Inc.","duties_or_roles":"Biostatistics/statistical programming","organisation_type":"Pharmaceutical company"}
- {"country":"Greece","full_name":"PPD Global Ltd.","duties_or_roles":"Project management duties or monitoring/regulatory; sponsorDuties codes: 12, 5","organisation_type":"Pharmaceutical company"}
- {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"coagulation screening, urinalysis, viral detection, renal function blood test, and liver chemistry; sponsorDuties code: 4","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"Axolabs GmbH","duties_or_roles":"PK analysis","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Inotiv Inc.","duties_or_roles":"PK analysis","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Marken LLP","duties_or_roles":"Home health care vendor","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eclinical Solutions LLC","duties_or_roles":"sponsorDuties code: 6","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom (Northern Ireland)","full_name":"Almac Group Limited","duties_or_roles":"QP release and IMP storage, distribution and destruction","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"Project and Clinical Management, Biopsy kits Supply (GCS); project management and other duties (multiple codes)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Blueprint Genetics Inc.","duties_or_roles":"DMD genotype analysis","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"United Biosource LLC","duties_or_roles":"Review of safety information; sponsorDuties code: 8","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"eResearchTechnology GmbH","duties_or_roles":"Central review of PFT","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eurofins Central Laboratory LLC","duties_or_roles":"GLDH Testing","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"Netherlands","full_name":"Eurofins Central Laboratory B.V.","duties_or_roles":"GLDH Testing","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Biologics Development Services LLC","duties_or_roles":"immunogenicity analysis","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Chillibean Limited","duties_or_roles":"Functional assessment video storage for QC","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Central review of ECHO, ECG","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Longboat Clinical Limited","duties_or_roles":"Site and patient porta","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"ATOM International Limited","duties_or_roles":"Functional assessment training and QC of videos","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Flagship Biosciences Inc.","duties_or_roles":"sponsorDuties code: 4","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Q Squared Solutions Holdings LLC","duties_or_roles":"DMD genotype analysis","organisation_type":"Laboratory/Research/Testing facility"}
Investigational products
- Investigational Product Name
- ETEPLIRSEN (AVI-4658)
- Active Substance
- ETEPLIRSEN
- Modality
- Oligonucleotide
- Routes Of Administration
- Intravenous
- Route
- Intravenous
- Authorisation Status
- Authorised (prodAuthStatus 1; UK MIA 20377)
- Orphan Designation
- Yes
- Starting Dose
- 30 mg/kg (comparator) and investigational higher doses 100 mg/kg and 200 mg/kg; weekly IV
- Dose Levels
- 30 mg/kg; 100 mg/kg; 200 mg/kg
- Frequency
- Weekly
- Maximum Dose
- 200 mg/kg
- Dose Escalation Increase
- Initial escalation dose 100 mg/kg followed by 200 mg/kg
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