Phase I, Open Label, Dose Escalation Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis
This study is currently recruiting participants.
Verified May 2013 by Onclave Therapeutics Limited, a wholly-owned subsidiary of Prothena Corporation plc
Sponsor:
Onclave Therapeutics Limited, a wholly-owned subsidiary of Prothena Corporation plc
Information provided by (Responsible Party):
Onclave Therapeutics Limited, a wholly-owned subsidiary of Prothena Corporation plc
ClinicalTrials.gov Identifier:
NCT01707264
First received: October 11, 2012
Last updated: May 1, 2013
Last verified: May 2013
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Purpose
Dose escalation study to determine the maximum tolerated dose of NEOD001 in approximately 30 subjects with AL amyloidosis. Expansion phase to evaluate safety, efficacy and pharmacokinetics of NEOD001 in 20 additional subjects at the maximum tolerated dose.
| Condition | Intervention | Phase |
|---|---|---|
|
Primary Amyloidosis |
Drug: NEOD001 |
Phase 1 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Phase I, Open Label, Dose Escalation Study of Intravenous Administration of Single Agent NEOD001 in Subjects With Light Chain (AL) Amyloidosis |
Resource links provided by NLM:
Further study details as provided by Onclave Therapeutics Limited, a wholly-owned subsidiary of Prothena Corporation plc:
Primary Outcome Measures:
- Safety and tolerability [ Time Frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator ] [ Designated as safety issue: Yes ]
- Adverse event profile
- Dose limiting toxicity and maximum tolerated dose
- Maximum tolerated dose [ Time Frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator ] [ Designated as safety issue: Yes ]
- Adverse event profile
- Dose Limiting Toxicity and maximum tolerated dose
Secondary Outcome Measures:
- Pharmacokinetics [ Time Frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator ] [ Designated as safety issue: No ]• Pharmacokinetic parameters including Cmax, Tmax, AUC, Cav, Cmin, t½, CL, and Vz
- Immunogenicity [ Time Frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator ] [ Designated as safety issue: Yes ]• Measurement of anti-NEOD001 antibodies
Other Outcome Measures:
- Hematologic Response [ Time Frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator ] [ Designated as safety issue: Yes ]• Hematologic response
- Organ response [ Time Frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator ] [ Designated as safety issue: Yes ]
- Organ response
- Changes in organ function markers
| Estimated Enrollment: | 50 |
| Study Start Date: | April 2013 |
| Estimated Study Completion Date: | September 2016 |
| Estimated Primary Completion Date: | May 2016 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: NEOD001
NEOD001 will be administered intravenously once every 28 days. The starting dose will be 0.5 mg/kg. Dose escalation will continue until the the maximum tolerated dose is determined for single agent NEOD001. Approximately 20 additional subjects will be treated with the maximum tolerated dose.
|
Drug: NEOD001
Monoclonal antibody administered by intravenous infusion every 28 days.
|
Detailed Description:
The purpose of the dose escalation phase of the study is to determine the maximum tolerated dose/Phase 2 recommended dose of NEOD001 when given as a single agent intravenously in approximately 30 subjects with AL amyloidosis.
The purpose of the expansion phase of the study is to evaluate the safety, preliminary efficacy and pharmacokinetics of single agent NEOD001 at the maximum tolerated dose/Phase 2 recommended dose in approximately 20 additional evaluable subjects.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Males and females aged ≥18 years;
- ECOG performance status (PS) 0-2;
- Diagnosis of systemic AL amyloidosis (subjects with non-AL amyloidosis are not eligible);
- Received at least one prior systemic therapy, which may include stem cell transplant, for AL amyloidosis;
- Have adequate organ function;
- Ability to understand and willingness to sign informed consent prior to initiation of any study procedures.
Exclusion Criteria:
- Secondary or familial amyloidosis;
- Life expectancy of < 3 months;
- Symptomatic multiple myeloma;
- Hypersensitivities to other monoclonal antibodies;
- Known HIV infection;
- Women who are lactating;
- Any other condition or prior therapy, which in the opinion of the PI, would make the subject unsuitable for the study.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01707264
Contacts
| Contact: Trinh Le | Trinh.le@onclavetherapeutics.com |
Locations
| United States, California | |
| Stanford University Cancer Center | Recruiting |
| Palo Alto, California, United States, 94305 | |
| Principal Investigator: Michaela Liedtke, MD | |
| United States, Massachusetts | |
| Tufts Medical Center | Recruiting |
| Boston, Massachusetts, United States, 02111 | |
| Contact: Jodi Jensen, RN 617-636-5558 jjensen@tuftsmedicalcenter.org | |
| Principal Investigator: Raymond Comenzo, MD | |
| United States, Minnesota | |
| Mayo Clinic | Recruiting |
| Rochester, Minnesota, United States, 55905 | |
| Contact: Ann Birgin, CRA 507-284-8828 | |
| Principal Investigator: Morris A Gertz, MD | |
Sponsors and Collaborators
Onclave Therapeutics Limited, a wholly-owned subsidiary of Prothena Corporation plc
Investigators
| Study Director: | Trinh Le | Onclave Therapeutics Ltd |
More Information
No publications provided
| Responsible Party: | Onclave Therapeutics Limited, a wholly-owned subsidiary of Prothena Corporation plc |
| ClinicalTrials.gov Identifier: | NCT01707264 History of Changes |
| Other Study ID Numbers: | NEOD001-001 |
| Study First Received: | October 11, 2012 |
| Last Updated: | May 1, 2013 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Onclave Therapeutics Limited, a wholly-owned subsidiary of Prothena Corporation plc:
|
AL amyloidosis Primary amyloidosis |
Additional relevant MeSH terms:
|
Amyloidosis Proteostasis Deficiencies Metabolic Diseases |
ClinicalTrials.gov processed this record on May 19, 2013