NCT00965458

Brief Summary

The purpose of this trial is to test whether a drug called alefacept will slow or halt destruction of the beta cells in the pancreas. If the destruction of the beta cells is stopped, the patients might be able to produce insulin on their own longer, which could stop or slow the progression of their type 1 diabetes. This is a multi-center prospective, placebo-controlled, double-blind and randomized trial to investigate the ability of alefacept to protect residual beta cells from ongoing autoimmune destruction in adolescents and young adults with newly diagnosed Type 1 Diabetes Mellitus (T1DM).

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
49

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Mar 2011

Typical duration for phase_2

Geographic Reach
1 country

15 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 22, 2009

Completed
3 days until next milestone

First Posted

Study publicly available on registry

August 25, 2009

Completed
1.5 years until next milestone

Study Start

First participant enrolled

March 1, 2011

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2013

Completed
1.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2014

Completed
9 months until next milestone

Results Posted

Study results publicly available

January 7, 2015

Completed
Last Updated

July 6, 2017

Status Verified

June 1, 2017

Enrollment Period

2 years

First QC Date

August 22, 2009

Results QC Date

December 19, 2014

Last Update Submit

June 12, 2017

Conditions

Keywords

New-onset Type 1 Diabetes MellitusNew-onset T1DMNew-onset T1DAlefaceptAmevive®

Outcome Measures

Primary Outcomes (1)

  • 2-Hour C-peptide Area Under the Curve (AUC) Result in Response to Standardized Mixed Meal Tolerance Test (MMTT)

    C-peptide is a substance released by the pancreas into the bloodstream in equal amounts to insulin and reflects how much insulin pancreatic beta cells are making. The standardized MMTT evaluates whether beta cells are producing endogenous insulin. The MMTT was performed in the morning and blood samples for C-peptide collected at baseline (pre-meal) and 15, 30, 60, 90, 120, 150, 180, 210,and 240 minutes post-meal. Results of the stimulated 2-hour (e.g., 120 minutes) post-meal C-peptide AUC are provided. Larger numbers are preferable (better) in these AUC results: more insulin being produced reflects less severe disease. C-peptide levels in the serum (e.g., AUC following a standardized MMTT) compared to control group at 1 year post treatment initiation for the evaluation of investigational products intended to preserve endogenous beta-cell function in T1DM trials is recognized by the Center for Drug Evaluation and Research (CDER) at the FDA as a valid efficacy primary endpoint.

    Baseline (pre-treatment initiation), Week 52

Secondary Outcomes (5)

  • 4-Hour C-peptide Area Under the Curve (AUC) Result in Response to Standardized Mixed Meal Tolerance Test (MMTT)

    Baseline (Pre-treatment initiation), Week 52, and Week 104

  • 2-Hour C-peptide Area Under the Curve (AUC) Result in Response to Standardized Mixed Meal Tolerance Test (MMTT)

    Baseline (Pre-treatment initiation), Week 52, and Week 104

  • Insulin Use in Units Per Kilogram Body Weight Per Day

    Baseline (Pre-treatment initiation), Week 52, and Week 104

  • Major Hypoglycemic Events Occurring From Randomization

    Baseline to Week 52 and Week 52 to Week 104

  • Hemoglobin A1c

    Baseline (Pre-treatment initiation), Week 52, and Week 104

Study Arms (2)

Alefacept

EXPERIMENTAL

Subjects in this group receive weekly intramuscular injections of alefacept (15 mg) for 2 cycles of 12 weeks each, separated by a 12 week pause in treatment.

Biological: Alefacept

Placebo

PLACEBO COMPARATOR

Subjects in this group received weekly intramuscular injections of a placebo saline solution of equal volume to the alefacept group for 2 cycles of 12 weeks each, separated by a 12 week pause in treatment.

Drug: Placebo

Interventions

AlefaceptBIOLOGICAL

Weekly intramuscular injections of alefacept (15 mg) for 2 cycles of 12 weeks each, separated by a 12 week pause in treatment.

Also known as: Amevive®
Alefacept

Weekly intramuscular injections of a placebo saline solution of equal volume to the alefacept group for 2 cycles of 12 weeks each, separated by a 12 week pause in treatment.

Also known as: Inactive drug (pharmacologically)
Placebo

Eligibility Criteria

Age12 Years - 35 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Recent diagnosis (within 100 days of enrollment) of T1DM
  • Positive for at least one diabetes autoantibody (Glutamate decarboxylase \[GAD-65GAD65\], IA2, ZnT8, ICA and Insulin, if obtained within 10 days of the onset of exogenous insulin therapy)
  • Peak stimulated C-peptide level \> 0.2 pmol/mL following a mixed-meal tolerance test (MMTT)
  • Willingness to provide written informed consent (either the subject or the subject's legally authorized representative).

You may not qualify if:

  • Severe reaction or anaphylaxis to human monoclonal antibodies
  • History of malignancy or significant cardiovascular disease (including history of myocardial infarction, angina, use of anti-anginal medicines (e.g., nitroglycerin), or abnormal stress test)
  • History of recent or ongoing uncontrolled bacterial, viral, fungal, or other opportunistic infections
  • Evidence of infection with hepatitis B virus (HBV) as defined by hepatitis B surface antigen, HBsAg; hepatitis C virus (HCV) defined by anti-HCV antibodies; human immunodeficiency virus (HIV); or toxoplasmosis
  • Positive tuberculin skin test (PPD)
  • Clinically active infection with Epstein-Barr virus (EBV)-EBV viral load ≥ 10,000 copies per 10\^6 PBMCs; cytomegalovirus (CMV) -CMV viral load ≥10,000 copies per mL whole blood; or tuberculosis (TB)
  • Diagnosis of liver disease or hepatic enzymes, as defined by ALT and/or AST ≥ 2 times the upper limit of normal
  • Prior or current treatment that is known to cause a significant, ongoing change in the course of T1DM or immunologic status, including high-dose inhaled, extensive topical or systemic glucocorticoids
  • Current or prior (within the last 30 days) use of metformin, sulfonylureas, glinides, thiazolidinediones, exenatide, liraglutide, DPP-IV inhibitors or amylin
  • Current use of any medication known to influence glucose tolerance (e.g., atypical antipsychotics, diphenylhydantoin, thiazide, or other potassium-depleting diuretics, β-adrenergic blockers, niacin)
  • Any of the following hematologic abnormalities, confirmed by repeat tests at least 1 week apart:
  • White blood count \<4000/μL or \>14,000/μL;
  • CD4+ count below the lower limit of normal;
  • Platelet count \<150,000 /μL; or
  • Hemoglobin \<10 g/dL.
  • +6 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (15)

University of Arizona

Tucson, Arizona, 85724, United States

Location

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

Location

University of California - San Francisco

San Francisco, California, 94143, United States

Location

Barbara Davis Center for Childhood Diabetes - University of Colorado

Aurora, Colorado, 80045, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Indiana University

Indianapolis, Indiana, 46202, United States

Location

University of Iowa Hospital & Clinics

Iowa City, Iowa, 52242, United States

Location

University of Maryland

Baltimore, Maryland, 21201, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Children's Mercy Hospitals and Clinics

Kansas City, Missouri, 64108, United States

Location

Creighton University

Omaha, Nebraska, 68131, United States

Location

University of North Carolina

Durham, North Carolina, 27713, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

Benaroya Research Institute at Virginia Mason

Seattle, Washington, 98101, United States

Location

Related Publications (4)

  • Herold KC. Restoring immune balance in type 1 diabetes. Lancet Diabetes Endocrinol. 2013 Dec;1(4):261-3. doi: 10.1016/S2213-8587(13)70123-2. Epub 2013 Sep 23. No abstract available.

    PMID: 24622404BACKGROUND
  • Rigby MR, DiMeglio LA, Rendell MS, Felner EI, Dostou JM, Gitelman SE, Patel CM, Griffin KJ, Tsalikian E, Gottlieb PA, Greenbaum CJ, Sherry NA, Moore WV, Monzavi R, Willi SM, Raskin P, Moran A, Russell WE, Pinckney A, Keyes-Elstein L, Howell M, Aggarwal S, Lim N, Phippard D, Nepom GT, McNamara J, Ehlers MR; T1DAL Study Team. Targeting of memory T cells with alefacept in new-onset type 1 diabetes (T1DAL study): 12 month results of a randomised, double-blind, placebo-controlled phase 2 trial. Lancet Diabetes Endocrinol. 2013 Dec;1(4):284-94. doi: 10.1016/S2213-8587(13)70111-6. Epub 2013 Sep 23.

  • Rigby MR, Harris KM, Pinckney A, DiMeglio LA, Rendell MS, Felner EI, Dostou JM, Gitelman SE, Griffin KJ, Tsalikian E, Gottlieb PA, Greenbaum CJ, Sherry NA, Moore WV, Monzavi R, Willi SM, Raskin P, Keyes-Elstein L, Long SA, Kanaparthi S, Lim N, Phippard D, Soppe CL, Fitzgibbon ML, McNamara J, Nepom GT, Ehlers MR. Alefacept provides sustained clinical and immunological effects in new-onset type 1 diabetes patients. J Clin Invest. 2015 Aug 3;125(8):3285-96. doi: 10.1172/JCI81722. Epub 2015 Jul 20.

  • Boyle KD, Keyes-Elstein L, Ehlers MR, McNamara J, Rigby MR, Gitelman SE, Weiner LJ, Much KL, Herold KC. Two- and Four-Hour Tests Differ in Capture of C-Peptide Responses to a Mixed Meal in Type 1 Diabetes. Diabetes Care. 2016 Jun;39(6):e76-8. doi: 10.2337/dc15-2077. Epub 2016 Apr 13. No abstract available.

Related Links

MeSH Terms

Interventions

Alefacept

Intervention Hierarchy (Ancestors)

CD58 AntigensMembrane GlycoproteinsGlycoproteinsGlycoconjugatesCarbohydratesImmunoglobulin GImmunoglobulin IsotypesAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulinsMembrane ProteinsRecombinant Fusion ProteinsRecombinant Proteins

Limitations and Caveats

Enrollment ended early with 49 subjects (instead of the planned 66) as a result of the decision, unrelated to safety reasons, made by Astellas Pharma US, Inc. to discontinue manufacturing Amevive® (alefacept).

Results Point of Contact

Title
Director, Clinical Research Operations Program
Organization
DAIT/NIAID

Study Officials

  • Mark R Rigby, MD, PhD

    Indiana University

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 22, 2009

First Posted

August 25, 2009

Study Start

March 1, 2011

Primary Completion

March 1, 2013

Study Completion

April 1, 2014

Last Updated

July 6, 2017

Results First Posted

January 7, 2015

Record last verified: 2017-06

Data Sharing

IPD Sharing
Will share

Participant level data access and additional relevant materials are available to the public in: 1.) the Immunology Database and Analysis Portal (ImmPort), a long-term archive of clinical and mechanistic data from DAIT-funded grants and contracts that also provides data analysis tools available to researchers; and 2.) TrialShare, the Immune Tolerance Network (ITN) Clinical Trials Research Portal that makes data from the consortium's clinical trials publicly available.

Available IPD Datasets

Individual Participant Data Set (SDY797)Access
Study Protocol (SDY797)Access
Study summary, -design, -adverse event(s), -demographics, -study files (SDY797)Access
Individual Participant Data Set (T1DAL ITN045AI)Access
Study Protocol (T1DAL ITN045AI)Access
Study overview, -data and reports, -schedule of assessments, -study design, -original article & abstracts et al. (T1DAL ITN045AI)Access

Locations