Phase 1/2, Open Label, Dose Escalation Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis

Primary Amyloidosis Clinical Trial

Official title:

A Phase 1/2, Open Label, Dose Escalation Study of Intravenous Administration of Single Agent NEOD001 in Subjects With Light Chain (AL) Amyloidosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01707264
• Other study ID #: NEOD001-001
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: April 2013
• Est. completion date: August 9, 2016

Study information

• Verified date: August 2018
• Source: Prothena Therapeutics Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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 25 additional subjects at the maximum tolerated dose.

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 25 additional evaluable subjects.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 69
• Est. completion date: August 9, 2016
• Est. primary completion date: August 9, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Amyloidosis
• Primary Amyloidosis

Intervention

Drug:
• NEOD001
Monoclonal antibody administered by intravenous infusion every 28 days.

Locations

Country	Name	City	State
United States	Boston University School of Medicine	Boston	Massachusetts
United States	Tufts Medical Center	Boston	Massachusetts
United States	Karmanos Cancer Institute	Detroit	Michigan
United States	Memorial Sloan-Kettering Cancer Center	New York	New York
United States	Stanford University Cancer Center	Palo Alto	California
United States	Hospital of the University of Pennsylvania	Philadelphia	Pennsylvania
United States	Mayo Clinic	Rochester	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Prothena Therapeutics Ltd.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Hematologic Response	• Hematologic response	28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Other	Organ response	Organ response; Changes in organ function markers	28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Primary	Safety and tolerability	Adverse event profile; Dose limiting toxicity and maximum tolerated dose	28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Primary	Maximum tolerated dose	Adverse event profile; Dose Limiting Toxicity and maximum tolerated dose	28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Secondary	Pharmacokinetics	• Pharmacokinetic parameters including Cmax, Tmax, AUC, Cav, Cmin, t½, CL, and Vz	28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Secondary	Immunogenicity	• Measurement of anti-NEOD001 antibodies	28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator