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A Research Trial of Aralast in New Onset Diabetes (RETAIN) (RETAIN)

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ClinicalTrials.gov Identifier: NCT01183468
Recruitment Status : Terminated (Due to lack of mechanistic signal and competing industry studies)
First Posted : August 17, 2010
Results First Posted : January 16, 2015
Last Update Posted : June 13, 2018
Sponsor:
Collaborators:
Immune Tolerance Network (ITN)
Juvenile Diabetes Research Foundation
Information provided by (Responsible Party):
National Institute of Allergy and Infectious Diseases (NIAID)

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Diabetes Mellitus, Type 1
Interventions Biological: Aralast NP 45 mg dose
Biological: Aralast NP 90 mg dose
Biological: Aralast NP 180 mg dose
Enrollment 17
Recruitment Details Twelve of the fifteen centers enrolled a total of 17 newly diagnosed (within 100 days of Visit 0 first low dose infusion) Type 1 Diabetes Mellitus participants.
Pre-assignment Details  
Arm/Group Title Part 1a(Aralast NP)-Subjects Aged 16-35 Yrs Part 1a (Aralast NP)-Subjects 8-15 Yrs Part 1b (Aralast NP)-Subjects Aged 18-35 Yrs Part 1b (Aralast NP)-Subjects 8-17 Yrs
Hide Arm/Group Description Subjects aged 16-35 years at enrollment with new-onset type 1 diabetes mellitus (T1DM) received Aralast NP 45 mg/kg by intravenous (IV) infusion once a week for 6 weeks. Following the Week 6 infusion, participants underwent a minimum 3-week washout period. After the washout period, each participant proceeded to a high dose of Aralast NP 90 mg/kg by IV infusion for the next 6 weeks, for a total of 12 infusions. Subjects aged 8-15 years at enrollment with new-onset type 1 diabetes mellitus (T1DM) received Aralast NP 45 mg/kg by IV infusion once a week for 6 weeks. Following the Week 6 infusion, participants underwent a minimum 3-week washout period. After the washout period, each participant proceeded to a high dose of Aralast NP 90 mg/kg by IV infusion for the next 6 weeks, for a total of 12 infusions.

Aralast NP 90 mg/kg/wk by IV infusion once a week for 6 weeks. Following the Week 6 infusion, participants undergo a minimum 3-wk washout period than then, each participant proceeds to high dose Aralast NP 180 mg/kg/wk by IV infusion for the next 6 weeks, for a total of 12 infusions.

Aralast NP 90 mg dose: - 90 mg/kg/week

Aralast NP 180 mg dose: - 180 mg/kg/week

Aralast NP 90 mg/kg/wk by IV infusion once a week for 6 weeks. Following the Week 6 infusion, participants undergo a minimum 3-wk washout period than then, each participant proceeds to high dose Aralast NP 180 mg/kg/wk by IV infusion for the next 6 weeks, for a total of 12 infusions.

Aralast NP 90 mg dose: - 90 mg/kg/week

Aralast NP 180 mg dose: - 180 mg/kg/week

Period Title: Overall Study
Started 8 9 0 0
Completed 5 7 0 0
Not Completed 3 2 0 0
Reason Not Completed
Lost to Follow-up             2             1             0             0
Physician Decision             1             0             0             0
Withdrawal by Subject             0             1             0             0
Arm/Group Title Part 1a(Aralast NP)-Subjects Aged 16-35 Yrs Part 1a (Aralast NP)-Subjects 8-15 Yrs Part 1b (Aralast NP)-Subjects Aged 18-35 Yrs Part 1b (Aralast NP)-Subjects 8-17 Yrs Total
Hide Arm/Group Description Subjects aged 16-35 years at enrollment with new-onset type 1 diabetes mellitus (T1DM) received Aralast NP 45 mg/kg by intravenous (IV) infusion once a week for 6 weeks. Following the Week 6 infusion, participants underwent a minimum 3-week washout period. After the washout period, each participant proceeded to a high dose of Aralast NP 90 mg/kg by IV infusion for the next 6 weeks, for a total of 12 infusions. Subjects aged 8-15 years at enrollment with new-onset type 1 diabetes mellitus (T1DM) received Aralast NP 45 mg/kg by IV infusion once a week for 6 weeks. Following the Week 6 infusion, participants underwent a minimum 3-week washout period. After the washout period, each participant proceeded to a high dose of Aralast NP 90 mg/kg by IV infusion for the next 6 weeks, for a total of 12 infusions.

Aralast NP 90 mg/kg/wk by IV infusion once a week for 6 weeks. Following the Week 6 infusion, participants undergo a minimum 3-wk washout period than then, each participant proceeds to high dose Aralast NP 180 mg/kg/wk by IV infusion for the next 6 weeks, for a total of 12 infusions.

Aralast NP 90 mg dose: - 90 mg/kg/week

Aralast NP 180 mg dose: - 180 mg/kg/week

Aralast NP 90 mg/kg/wk by IV infusion once a week for 6 weeks. Following the Week 6 infusion, participants undergo a minimum 3-wk washout period than then, each participant proceeds to high dose Aralast NP 180 mg/kg/wk by IV infusion for the next 6 weeks, for a total of 12 infusions.

Aralast NP 90 mg dose: - 90 mg/kg/week

Aralast NP 180 mg dose: - 180 mg/kg/week

Total of all reporting groups
Overall Number of Baseline Participants 8 9 0 0 17
Hide Baseline Analysis Population Description
Enrolled Sample
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 9 participants 0 participants 0 participants 17 participants
<=18 years
4
  50.0%
9
 100.0%
13
  76.5%
Between 18 and 65 years
4
  50.0%
0
   0.0%
4
  23.5%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 8 participants 9 participants 0 participants 0 participants 17 participants
20.6  (5.7) 10.7  (2.2) 15.4  (6.6)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 9 participants 0 participants 0 participants 17 participants
Female
3
  37.5%
3
  33.3%
6
  35.3%
Male
5
  62.5%
6
  66.7%
11
  64.7%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 8 participants 9 participants 0 participants 0 participants 17 participants
8 9 17
1.Primary Outcome
Title C-peptide 2-hour AUC in Response to a Mixed-meal Tolerance Test at Week 52
Hide Description No results for the primary outcome measure are available since the study was terminated prior to reaching the outcome measure time frame of 52 weeks.
Time Frame Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Enrolled Sample
Arm/Group Title Part 1a(Aralast NP)-Subjects Aged 16-35 Yrs Part 1a (Aralast NP)-Subjects 8-15 Yrs Part 1b (Aralast NP)-Subjects Aged 18-35 Yrs Part 1b (Aralast NP)-Subjects 8-17 Yrs
Hide Arm/Group Description:
Subjects aged 16-35 years at enrollment with new-onset type 1 diabetes mellitus (T1DM) received Aralast NP 45 mg/kg by intravenous (IV) infusion once a week for 6 weeks. Following the Week 6 infusion, participants underwent a minimum 3-week washout period. After the washout period, each participant proceeded to a high dose of Aralast NP 90 mg/kg by IV infusion for the next 6 weeks, for a total of 12 infusions.
Subjects aged 8-15 years at enrollment with new-onset type 1 diabetes mellitus (T1DM) received Aralast NP 45 mg/kg by IV infusion once a week for 6 weeks. Following the Week 6 infusion, participants underwent a minimum 3-week washout period. After the washout period, each participant proceeded to a high dose of Aralast NP 90 mg/kg by IV infusion for the next 6 weeks, for a total of 12 infusions.

Aralast NP 90 mg/kg/wk by IV infusion once a week for 6 weeks. Following the Week 6 infusion, participants undergo a minimum 3-wk washout period than then, each participant proceeds to high dose Aralast NP 180 mg/kg/wk by IV infusion for the next 6 weeks, for a total of 12 infusions.

Aralast NP 90 mg dose: - 90 mg/kg/week

Aralast NP 180 mg dose: - 180 mg/kg/week

Aralast NP 90 mg/kg/wk by IV infusion once a week for 6 weeks. Following the Week 6 infusion, participants undergo a minimum 3-wk washout period than then, each participant proceeds to high dose Aralast NP 180 mg/kg/wk by IV infusion for the next 6 weeks, for a total of 12 infusions.

Aralast NP 90 mg dose: - 90 mg/kg/week

Aralast NP 180 mg dose: - 180 mg/kg/week

Overall Number of Participants Analyzed 0 0 0 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame Adverse events were collected for 2.5 years, from the start of the study in January 2011 until the last participant completed the study in July 2013.
Adverse Event Reporting Description Adverse events were collected from the time a participant signed informed consent until the time an event resolved or until 30 days after the participant completed study treatment, whichever came first.
 
Arm/Group Title Subjects Aged 16 - 35 Years Subjects Aged 8 - 15 Years
Hide Arm/Group Description Subjects aged 16-35 years at enrollment with new-onset type 1 diabetes mellitus (T1DM) received Aralast NP 45 mg/kg by IV infusion once a week for 6 weeks. Following the Week 6 infusion, participants underwent a minimum 3-week washout period. After the washout period, each participant proceeded to a high dose of Aralast NP 90 mg/kg by IV infusion for the next 6 weeks, for a total of 12 infusions. Subjects aged 8-15 years at enrollment with new-onset type 1 diabetes mellitus (T1DM) received Aralast NP 45 mg/kg by IV infusion once a week for 6 weeks. Following the Week 6 infusion, participants underwent a minimum 3-week washout period. After the washout period, each participant proceeded to a high dose of Aralast NP 90 mg/kg by IV infusion for the next 6 weeks, for a total of 12 infusions.
All-Cause Mortality
Subjects Aged 16 - 35 Years Subjects Aged 8 - 15 Years
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Hide Serious Adverse Events
Subjects Aged 16 - 35 Years Subjects Aged 8 - 15 Years
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/8 (25.00%)      1/9 (11.11%)    
Metabolism and nutrition disorders     
Diabetic ketoacidosis  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Hypoglycaemia  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Musculoskeletal and connective tissue disorders     
Musculoskeletal pain  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 11.1
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Subjects Aged 16 - 35 Years Subjects Aged 8 - 15 Years
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   8/8 (100.00%)      8/9 (88.89%)    
Endocrine disorders     
Goitre  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Eye disorders     
Blepharitis  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Conjunctivitis  1  0/8 (0.00%)  0 1/9 (11.11%)  2
Gastrointestinal disorders     
Abdominal discomfort  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Abdominal pain  1  0/8 (0.00%)  0 1/9 (11.11%)  4
Abdominal pain upper  1  0/8 (0.00%)  0 2/9 (22.22%)  12
Constipation  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Diarrhoea  1  1/8 (12.50%)  1 2/9 (22.22%)  3
Nausea  1  1/8 (12.50%)  1 4/9 (44.44%)  7
Vomiting  1  0/8 (0.00%)  0 4/9 (44.44%)  5
General disorders     
Chest pain  1  1/8 (12.50%)  1 2/9 (22.22%)  2
Influenza like illness  1  1/8 (12.50%)  1 1/9 (11.11%)  1
Infusion site haematoma  1  0/8 (0.00%)  0 1/9 (11.11%)  4
Infusion site pain  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Injection site haematoma  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Pyrexia  1  1/8 (12.50%)  1 3/9 (33.33%)  3
Infections and infestations     
Ear infection  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Fungal infection  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Gastroenteritis  1  0/8 (0.00%)  0 2/9 (22.22%)  2
Gastroenteritis viral  1  0/8 (0.00%)  0 3/9 (33.33%)  3
Influenza  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Kidney infection  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Nasopharyngitis  1  2/8 (25.00%)  2 2/9 (22.22%)  3
Otitis externa  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Pharyngitis streptococcal  1  1/8 (12.50%)  1 1/9 (11.11%)  1
Pneumonia  1  1/8 (12.50%)  1 1/9 (11.11%)  1
Respiratory tract infection viral  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Sinusitis  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Streptococcal infection  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Tooth infection  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Upper respiratory tract infection  1  0/8 (0.00%)  0 4/9 (44.44%)  10
Urinary tract infection  1  2/8 (25.00%)  9 0/9 (0.00%)  0
Injury, poisoning and procedural complications     
Concussion  1  0/8 (0.00%)  0 1/9 (11.11%)  2
Contusion  1  1/8 (12.50%)  1 3/9 (33.33%)  3
Fibula fracture  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Foot fracture  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Hand fracture  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Procedural pain  1  1/8 (12.50%)  3 1/9 (11.11%)  1
Radius fracture  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Upper limb fracture  1  0/8 (0.00%)  0 1/9 (11.11%)  2
Investigations     
Fibrin D dimer increased  1  0/8 (0.00%)  0 3/9 (33.33%)  3
Glycosylated haemoglobin increased  1  0/8 (0.00%)  0 2/9 (22.22%)  2
Lymphocyte count decreased  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Lymphocyte count increased  1  1/8 (12.50%)  2 0/9 (0.00%)  0
Neutrophil count decreased  1  1/8 (12.50%)  1 0/9 (0.00%)  0
White blood cell count decreased  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Metabolism and nutrition disorders     
Hyperglycaemia  1  0/8 (0.00%)  0 1/9 (11.11%)  4
Hypoglycaemia  1  7/8 (87.50%)  69 8/9 (88.89%)  104
Vitamin D deficiency  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Musculoskeletal and connective tissue disorders     
Arthralgia  1  3/8 (37.50%)  3 0/9 (0.00%)  0
Back pain  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Muscle spasms  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Musculoskeletal pain  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Myalgia  1  0/8 (0.00%)  0 1/9 (11.11%)  4
Neck pain  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Pain in extremity  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Skin papilloma  1  0/8 (0.00%)  0 1/9 (11.11%)  2
Nervous system disorders     
Dizziness  1  0/8 (0.00%)  0 2/9 (22.22%)  2
Headache  1  2/8 (25.00%)  2 4/9 (44.44%)  77
Sinus headache  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Syncope  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Psychiatric disorders     
Panic attack  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Renal and urinary disorders     
Hypercalciuria  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Ketonuria  1  0/8 (0.00%)  0 2/9 (22.22%)  4
Nephrolithiasis  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Cough  1  3/8 (37.50%)  3 2/9 (22.22%)  4
Nasal congestion  1  3/8 (37.50%)  3 2/9 (22.22%)  2
Oropharyngeal pain  1  0/8 (0.00%)  0 2/9 (22.22%)  9
Respiratory tract congestion  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Rhinitis allergic  1  1/8 (12.50%)  1 1/9 (11.11%)  1
Upper respiratory tract congestion  1  0/8 (0.00%)  0 1/9 (11.11%)  2
Upper-airway cough syndrome  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Skin and subcutaneous tissue disorders     
Dry skin  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Eczema  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Rash  1  1/8 (12.50%)  3 3/9 (33.33%)  4
Rash papular  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Rash pruritic  1  0/8 (0.00%)  0 1/9 (11.11%)  2
Skin mass  1  0/8 (0.00%)  0 1/9 (11.11%)  1
Urticaria  1  1/8 (12.50%)  1 0/9 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 11.1
This (Part 1) was the drug dosing portion of the study; however, due to lack of a mechanistic signal and competing industry studies, the study was terminated and the trial portion (Part II, NCT01183455) was withdrawn.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Director, Clinical Research Operations Program
Organization: DAIT/NIAID
Phone: 301-594-7669
EMail: DAITClinicalTrialsGov@niaid.nih.gov
Layout table for additonal information
Responsible Party: National Institute of Allergy and Infectious Diseases (NIAID)
ClinicalTrials.gov Identifier: NCT01183468    
Other Study ID Numbers: DAIT ITN041AI Part 1
First Submitted: August 16, 2010
First Posted: August 17, 2010
Results First Submitted: January 6, 2015
Results First Posted: January 16, 2015
Last Update Posted: June 13, 2018