Down SyndromePhase 1NCT02432716
Investigation of the Safety of Intranasal Glulisine in Down Syndrome
A Phase 1 study testing Insulin glulisine and Saline in people with Down Syndrome, run by HealthPartners Institute at 1 site in United States.
Trial summary
This study is a single center, randomized, double-blind, placebo-controlled, cross-over pilot study designed to assess the safety of intranasally (IN) delivered glulisine versus placebo in patients with DS. Read more about this study
- Who is it for?
- Ages 35 to 80 years
- Conditions the study is for
- Down Syndrome
- What is being tested?
- Insulin glulisine and Saline
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2015to 18 Oct 2018
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 35 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female aged 35-80 years with a Down syndrome diagnosis that is confirmed by karyotype.
- Vital signs must be within normal limits for their age. (Medically treated hypertension will be allowed).
- Must have an electrocardiogram free of clinically significant findings.
- Must have an authorized representative to provide written informed consent.
- Level of speech and comprehension of verbal commands are sufficient to understand and to answer simple requests.
- Must have a reliable caregiver or family member who agrees to accompany the subject to all visits, provide information about the subject as required by this protocol.
- Must be independent for activities of daily living.
- Must tolerate the initial IN treatment of placebo and adhere to study procedures.
Who cannot join
- Any current psychiatric or neurologic diagnosis other than Down syndrome or Down syndrome with dementia that is judged to impact cognition.
- Subjects who currently meet or have within the past five years met DSM-IV (Diagnostic and Statistical Manual) criteria for drug or alcohol abuse or dependence.
- Subjects residing in a skilled nursing facility or subjects who are anticipated to enter a nursing home within the next 6 months. (Subjects may reside in group homes, assisted living, or other residential settings where they do not require 24 hour skilled nursing.)
- Subjects receiving any experimental drug for Down syndrome within the past 30 days of screening visit.
- Subjects with significant allergies to or other significant intolerance insulin.
- Presence of active seizure disorder.
Show all 10Show fewer
- Presence of significant aggression or agitation that may impact participation with testing and IN administration. All subjects must have NPI-C aggression and agitation subscore ≤ 4 (severity ≤ 2; frequency ≤ 2).
- Significant cerebrovascular disease with Modified Hachinski Score>4.
- Subjects who may not be able to comply with the protocol or perform the outcomes measures due to significant hearing or visual impairment or other issues judged relevant by the investigators.
- Subject has been diagnosed with any form of diabetes mellitus, actively takes insulin, or has HbA1c > 6.1% at screening.
Trial locations and contacts
1 site in 1 country.
United States1 site
HealthPartners Neuroscience Center
Saint Paul, Minnesota, 55130
About this study
Official title: A Double-Blind, Placebo-Controlled Pilot Investigation of the Safety of Intranasal Glulisine in Down Syndrome
This study is a single center, randomized, double-blind, placebo-controlled, cross-over pilot study designed to assess the safety of intranasally (IN) delivered glulisine versus placebo in patients with DS. Subjects will be randomized into this cross-over study and within subject comparisons conducted between single treatment of intranasal insulin glulisine and single treatment of intranasal placebo. All subjects will also receive a single treatment of placebo prior to randomization to ensure adherence to study procedures.
- Conditions
- Down Syndrome
- Being tested
- Insulin glulisine, Saline
- Medical subject headings
- Down Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Insulin glulisineDrug
Also called insulin, glulisine, Apidra
Insulin (glulisine) Glulisine 20 IU/IN (.1ml/10 units intranasal in each nostril), once per study
SalineDrug
Also called Placebo
Placebo Comparator: Placebo Sterile Normal Saline 20 IU/IN (.1ml intransal in each nostril), once per study
| Study group | Role | What they receive |
|---|---|---|
| Insulin (glulisine), then Placebo | Experimental | Participants first receive one dose of Glulisine 20 IU/IN (.1ml/10 units intranasal in each nostril). After a washout period of 2 weeks, they then received one dose of placebo, Sterile Normal Saline 20 IU/IN (.1ml intransal in each nostril). Receives: Drug: Insulin glulisine; Drug: Saline |
| Placebo, then Insulin (glulisine) | Experimental | Participants first receive one dose of placebo, Sterile Normal Saline 20 IU/IN (.1ml intransal in each nostril). After a washout period of 2 weeks, they then received one dose of Glulisine 20 IU/IN (.1ml/10 units intranasal in each nostril). Receives: Drug: Insulin glulisine; Drug: Saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety Measured by Adverse Events
Number of adverse and/or serious events
Measured: 1 year
Secondary outcomes (4)
Cognitive Change Measured by Fuld Object-Memory Evaluation (FOME)
During the examination, a patient is presented with ten common objects they are asked to identify by touch. The test uses distraction to test recall. For all, a higher score indicates a better outcome.
- Learning curve is the number of objects the difference in the number of items they are able to correctly identify from the greater of trials 4 or 5 compared to trial 1. Range: 0-10
- Total immediate recall is the number of objects recalled over all of the trials. Range: 0-50
- Total delayed recall is the number of objects recalled after 5 minutes. Range: 0-10
- Recognition memory is the number of items correct from a multiple choice list of three when unable to correctly identify items from delayed recall. Range: 0-10
- Retention estimate is the number of items recalled after 5 minutes or being reminded with multiple choice. Range: 0-10
Measured: 20 minutes
Memory Retention Measured by Fuld Object-Memory Evaluation (FOME)
Memory retention is the percentage of items correctly identify during the delayed recall trial compared storage trial 5. Range: 0-100 percent. A higher percentage indicates a better outcome.
Measured: 20 minutes
Cognitive Change Measured by Rivermead Behavioral Memory Test (RBMT-C)
The RBMT-C provides an objective measure of everyday memory problems reported and observed in subjects with memory difficulties. The test is standardized for use with children ranging in age from 5 to 10 years. Here, we used it for evaluation of Down Syndrome subjects. The story recall subtests involves immediate free recall, cued recall, and delayed recall of short story material which is presented orally to subjects by the examiner. The RBMT-C is appealing for use in this population because the task is engaging, simple, and has been shown in other studies to be an effective measure of memory functions. For all, a higher score means a better outcome.
- Immediate Recall is the number of story elements recalled right after the story is complete. Range: 0-31
- Delayed Recall is the number of story elements recalled after a delay. Range: 0-31
Measured: 20 minutes
Memory Retention Measured by Rivermead Behavioral Memory Test (RBMT-C).
The RBMT-C provides an objective measure of everyday memory problems reported and observed in subjects with memory difficulties. The test is standardized for use with children ranging in age from 5 to 10 years. Here, we used it for evaluation of Down Syndrome subjects. The story recall subtests involves immediate free recall, cued recall, and delayed recall of short story material which is presented orally to subjects by the examiner. The RBMT-C is appealing for use in this population because the task is engaging, simple, and has been shown in other studies to be an effective measure of memory functions. Memory retention is the percentage of story elements recalled after a delay compared to right after the story is complete. Range: 0-100. A higher score means a better outcome.
Measured: 20 minutes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 May 2015 (estimated)
- Study startApr 2015
- Primary completion18 Oct 2018
- Study completion18 Oct 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Down SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- HealthPartners Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Michael H Rosenbloom, MD, Principal investigator, HealthPartners Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02432716
- Sponsor's protocol ID
- HealthPartnersRF
- Status last verified
- Jun 2018
- First submitted
- 8 Apr 2015
- Last updated
- 6 Dec 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Rosenbloom M, Barclay T, Johnsen J, Erickson L, Svitak A, Pyle M, Frey W, Hanson LR. Double-Blind Placebo-Controlled Pilot Investigation of the Safety of a Single Dose of Rapid-Acting Intranasal Insulin in Down Syndrome. Drugs R D. 2020 Mar;20(1):11-15. doi: 10.1007/s40268-020-00296-2.
PubMed 32077057
Study at a glance
From the registry record, last updated by the sponsor on 6 Dec 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-Blind, Placebo-Controlled Pilot Investigation of the Safety of Intranasal Glulisine in Down Syndrome
- ClinicalTrials.gov ID
- NCT02432716
- Acronym / sponsor ID
- HealthPartnersRF
- Conditions
- Down Syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- HealthPartners InstituteUniversity, hospital or other
- Study start
- Apr 2015
- Primary completion
- 18 Oct 2018 (actual)
- Study completion
- 18 Oct 2018 (actual)
- First posted
- 4 May 2015 (estimated)
- Results
- Posted 6 Dec 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is a single center, randomized, double-blind, placebo-controlled, cross-over pilot study designed to assess the safety of intranasally (IN) delivered glulisine versus placebo in patients with DS. Subjects will be randomized into this cross-over study and within subject comparisons conducted between single treatment of intranasal insulin glulisine and single treatment of intranasal placebo. All subjects will also receive a single treatment of placebo prior to randomization to ensure adherence to study procedures.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Insulin (glulisine), then Placebo | Experimental | Drug: Insulin glulisine, Drug: SalineParticipants first receive one dose of Glulisine 20 IU/IN (.1ml/10 units intranasal in each nostril). After a washout period of 2 weeks, they then received one dose of placebo, Sterile Normal Saline 20 IU/IN (.1ml intransal in each nostril). |
| Placebo, then Insulin (glulisine) | Experimental | Drug: Insulin glulisine, Drug: SalineParticipants first receive one dose of placebo, Sterile Normal Saline 20 IU/IN (.1ml intransal in each nostril). After a washout period of 2 weeks, they then received one dose of Glulisine 20 IU/IN (.1ml/10 units intranasal in each nostril). |
Who may qualify
- Ages
- 35 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Male or female aged 35-80 years with a Down syndrome diagnosis that is confirmed by karyotype.
- Vital signs must be within normal limits for their age. (Medically treated hypertension will be allowed).
- Must have an electrocardiogram free of clinically significant findings.
- Must have an authorized representative to provide written informed consent.
- Level of speech and comprehension of verbal commands are sufficient to understand and to answer simple requests.
Key exclusion criteria (10 listed)
- Any current psychiatric or neurologic diagnosis other than Down syndrome or Down syndrome with dementia that is judged to impact cognition.
- Subjects who currently meet or have within the past five years met DSM-IV (Diagnostic and Statistical Manual) criteria for drug or alcohol abuse or dependence.
- Subjects residing in a skilled nursing facility or subjects who are anticipated to enter a nursing home within the next 6 months. (Subjects may reside in group homes, assisted living, or other residential settings where they do not require 24 hour skilled nursing.)
- Subjects receiving any experimental drug for Down syndrome within the past 30 days of screening visit.
- Subjects with significant allergies to or other significant intolerance insulin.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Insulin glulisineAlso: insulin, glulisine, ApidraInsulin (glulisine) Glulisine 20 IU/IN (.1ml/10 units intranasal in each nostril), once per study | Drug | Insulin (glulisine), then Placebo, Placebo, then Insulin (glulisine) |
| SalineAlso: PlaceboPlacebo Comparator: Placebo Sterile Normal Saline 20 IU/IN (.1ml intransal in each nostril), once per study | Drug | Insulin (glulisine), then Placebo, Placebo, then Insulin (glulisine) |
Primary outcome
Safety Measured by Adverse Events
Time frame: 1 year
Number of adverse and/or serious events
4 secondary outcomes
Cognitive Change Measured by Fuld Object-Memory Evaluation (FOME)
Time frame: 20 minutes
Memory Retention Measured by Fuld Object-Memory Evaluation (FOME)
Time frame: 20 minutes
Cognitive Change Measured by Rivermead Behavioral Memory Test (RBMT-C)
Time frame: 20 minutes
Memory Retention Measured by Rivermead Behavioral Memory Test (RBMT-C).
Time frame: 20 minutes
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Dec 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 32077057Rosenbloom M, Barclay T, Johnsen J, Erickson L, Svitak A, Pyle M, Frey W, Hanson LR. Double-Blind Placebo-Controlled Pilot Investigation of the Safety of a Single Dose of Rapid-Acting Intranasal Insulin in Down Syndrome. Drugs R D. 2020 Mar;20(1):11-15. doi: 10.1007/s40268-020-00296-2.
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Down Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michael H Rosenbloom, MD principal investigator
HealthPartners Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Insulin glulisine and Saline in people with Down Syndrome, run by HealthPartners Institute at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Dec 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 6 Dec 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety Measured by Adverse Events (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02432716, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Dec 2019.