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A Phase I, Open Label, Dose Escalation Study of Intravenous Administration of Single Agent NEOD001 in Subjects With Light Chain (AL) Amyloidosis


Phase 1
18 Years
N/A
Open (Enrolling)
Both
Primary Amyloidosis

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Trial Information

A Phase I, Open Label, Dose Escalation Study of Intravenous Administration of Single Agent NEOD001 in Subjects With Light Chain (AL) Amyloidosis


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.


Inclusion Criteria:



1. Males and females aged ≥18 years;

2. ECOG performance status (PS) 0-2;

3. Diagnosis of systemic AL amyloidosis (subjects with non-AL amyloidosis are not
eligible);

4. Received at least one prior systemic therapy, which may include stem cell transplant,
for AL amyloidosis;

5. Have adequate organ function;

6. Ability to understand and willingness to sign informed consent prior to initiation of
any study procedures.

Exclusion Criteria:

1. Secondary or familial amyloidosis;

2. Life expectancy of < 3 months;

3. Symptomatic multiple myeloma;

4. Hypersensitivities to other monoclonal antibodies;

5. Known HIV infection;

6. Women who are lactating;

7. Any other condition or prior therapy, which in the opinion of the PI, would make the
subject unsuitable for the study.

Type of Study:

Interventional

Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Safety and tolerability

Outcome Description:

Adverse event profile Dose limiting toxicity and maximum tolerated dose

Outcome Time Frame:

28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator

Safety Issue:

Yes

Principal Investigator

Trinh Le

Investigator Role:

Study Director

Investigator Affiliation:

Onclave Therapeutics Ltd

Authority:

United States: Food and Drug Administration

Study ID:

NEOD001-001

NCT ID:

NCT01707264

Start Date:

April 2013

Completion Date:

September 2016

Related Keywords:

  • Primary Amyloidosis
  • AL amyloidosis
  • Primary amyloidosis
  • Amyloidosis

Name

Location

Mayo Clinic Rochester, Minnesota  55905
Memorial Sloan-Kettering Cancer Center New York, New York  10021
Tufts Medical Center Boston, Massachusetts  02111
Stanford University Cancer Center Stanford, California  94305