Current Research

CURRENTTLY ENROLLING STUDIES

Type 1 Studies

Dexcom Inc. / Post Approval Study for Non-Adjunctive Use of Dexcom G5® and G6® CGM System for Diabetes Management; Continuous Monitoring and Control of Hypoglycemia
Medtronic; Protocol: Multi-center, Randomized, Parallel, Adaptive, Controlled Trial in Adult and Pediatric Patients with Type 1 Diabetes Using Hybrid Closed Loop System and Control (CSII, MDI and SAP) at Home (“Study”) Study Product: MiniMed 670G insulin pump with hybrid closed loop algorithm (“Product”)
DOMPE LADAXARIN- TYPE 1 DIABETES (NEW ONSET) : The objective of this clinical trial is to assess whether ladarixin treatment is effective in preserving beta-cell function and delaying the progression of type 1 diabetes (T1D) in adolescent and adult patients. The safety of ladarixin in the specific clinical setting will be also evaluated.
Afrezza INHALE-1: A 26-week Primary Treatment Phase, with 26-week Extension, Open label, Randomized Clinical Trial Evaluating the Efficacy and Safety of Afrezza Versus Rapid-acting Insulin Analog Injections, Both in Combination with a Basal Insulin, in Pediatric Subjects with Type 1 or Type 2 Diabetes Mellitus (for ages 4-17yrs of age)

Type 2 Studies:

Eli Lilly: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study with an Open-Label Extension Assessing the Efficacy, Safety, and Pharmacokinetics/Pharmacodynamics of Tirzepatide in Pediatric and Adolescent Participants with Type 2 Diabetes Mellitus Inadequately Controlled with Metformin, or Basal Insulin, or Both (SURPASS-PEDS)”
Janssen Research & Development: A Randomized, Multicenter, Double-Blind, Parallel-Group, Placebo-Controlled Study to Investigate the Efficacy and Safety of Canagliflozin in Children and Adolescents (≥10 to <18 years) with Type 2 Diabetes Mellitus
Dexcom Inc. / Post Approval Study for Non-Adjunctive Use of Dexcom G5® and G6® CGM System for Diabetes Management ; Continuous Monitoring and Control of Hypoglycemia:
Afrezza INHALE-1: A 26-week Primary Treatment Phase, with 26-week Extension, Open label, Randomized Clinical Trial Evaluating the Efficacy and Safety of Afrezza Versus Rapid-acting Insulin Analog Injections, Both in Combination with a Basal Insulin, in Pediatric Subjects with Type 1 or Type 2 Diabetes Mellitus (for ages 4-17yrs of age)

Growth Hormone Studies:

Lumos Pharma: A Multicenter, 6-Month, Randomized, Open-Label, Active Control, Parallel Arm, Phase 2b Study of Daily Oral LUM-201 in Naïve-to-Treatment, Prepubertal Children with Growth Hormone Deficiency (GHD)”

CAH (CONGENITAL ADRENAL HYPERPLASIA)

Spruce Pharma: A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects with Classic Congenital Adrenal Hyperplasia (203)
Spruce Pharma: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Reducing Supraphysiologic Glucocorticoid Use in Adult Subjects with Classic Congenital Adrenal Hyperplasia (204)

ONGOING BUT NO LONGER ENROLLING

Type 1 Diabetes studies

Type 2 Diabetes Studies

Growth Hormone Studies

Ascendis Pharma TransCon hGH CT-301EXT enliGHten: A Multicenter, Phase 3, Long-term, Open-label Trial Investigating Safety and Efficacy of TransCon hGH Administered Once-Weekly in Children with Growth Hormone Deficiency (GHD) Who Have Completed a Prior TransCon hGH Clinical Trial February

Novo Nordisk: REAL A trial comparing the effect and safety of once weekly dosing of somapacitan with daily Norditropin® in children with growth hormone deficiency.

ALL AREAS CLOSED/NO LONGER ONGOING
Dexcom, Inc. Dexcom Inc. Assessing Non-adjunctive CGM Safety at Home and in New markets ANHSIN

Eli Lilly: GBGC A Randomized, Double-Blind Study with an Open-Label Extension Comparing the Effect of Once-Weekly Dulaglutide with Placebo in Pediatric Patients with Type 2 Diabetes Mellitus

AstraZeneca T2Now- AB- A 26 Week, Multicenter, Randomized, Placebo-Controlled, Double-Blind, Parallel Group, Phase 3 Trial with a 26 Week Safety Extension Period Evaluating the Safety and Efficacy of Dapagliflozin 5 and 10 mg, and Saxagliptin 2.5 and 5 mg in Pediatric Patients with Type 2 Diabetes Mellitus who are between 10 and below 18 years of age

Allergan/Abbvie: A multicenter, randomized, double-blind, dose-conversion study to evaluate the safety and efficacy of hormone replacement therapy with Armour® Thyroid compared to synthetic T4 (levothyroxine) in previously hypothyroid participants, who are euthyroid on T4 replacement therapy

Eli Lilly and Co. ITSB Study- A Prospective, Randomized, Double-Blind Comparison of LY900014 to Humalog with an Open-Label Postprandial LY900014 Treatment Group in Children and Adolescents with Type 1 Diabetes (PRONTO-Peds)

GH Pfizer, Inc - C0311002, A PHASE 3, RANDOMIZED, MULTICENTER, OPEN-LABEL, CROSSOVER STUDY ASSESSING SUBJECT PERCEPTION OF TREATMENT BURDEN WITH USE OF WEEKLY GROWTH HORMONE (SOMATROGON)
VERSUS DAILY GROWTH HORMONE (GENOTROPIN®) INJECTIONS IN CHILDREN WITH GROWTH HORMONE DEFICIENCY (Pro00028035)

Ascendis Pharma Endocrinology Division A/S: fliGHt: A Multicenter, Phase 3, Open-Label, 26-Week Trial Investigating the Safety, Tolerability and Efficacy of TransCon hGH Administered Once Weekly in Children with Growth Hormone Deficiency (GHD)

Eli Lilly and Co. ITRO A Prospective, Randomized Double-Blind Comparison of LY900014 to Humalog in Adults with Type 1 Diabetes using Continuous Subcutaneous Insulin Infusion (PRONTO-Pump-2)
NIH- Trialnet : Pathway to Prevention – Type 1 diabetes trial to test family members of Type 1 for antibodies.

AstraZeneca, AB MB102138 T2GO (AZ study number: D1690C00017) A 24 Week, Multicenter, Randomized, Double-Blind, Parallel Group, Phase 3 Trial with a 28 Week Long Term Safety Extension Period Evaluating the Safety and Efficacy of Dapagliflozin 10 mg in T2DM Patients aged 10-24 years. November 2018 to October 2019 Data Lock schedule

Dexcom, Inc Protocol: Evaluation of the New Adhesive Material used with the Dexcom iCGM Sensor Pod
Dexcom Patch Study #2

(TIGER) Type 1 DM: SIMPONI® to Arrest ß-cell Loss in Type 1 Diabetes

Merck 170: A Phase III, Multicenter, Double-Blind, Randomized, Placebo-Controlled Clinical Trial to Evaluate the Safety and Efficacy of MK-0431A (A Fixed-Dose Combination Tablet of Sitagliptin and Metformin) in Pediatric Patients with Type 2 Diabetes Mellitus With Inadequate Glycemic Control on Metformin Therapy (Alone or in Combination with Insulin)

Merck 083 Type 2 DM: A Phase III, Multicenter, Double-Blind, Randomized, Placebo-Controlled Clinical Trial to Evaluate the Safety and Efficacy of Sitagliptin in Pediatric Patients with Type 2 Diabetes Mellitus with Inadequate Glycemic Control

CURRENT STUDIES At Our Center

Eli Lilly Type 2 Tirzepatide Once a week Injectable (Add-On therapy)
Study Using Tirzepatide in Pediatric and Adolescent Participants with Type 2 Diabetes Mellitus Inadequately Controlled with Metformin, or Basal Insulin, or Both (SURPASS-PEDS)
Inclusion

Participant must be 10 to <18 years of age, at the time of signing the informed consent/assent. Type of Participant and Disease Characteristics For T2DM diagnosis criteria see Appendix 8, Section 10.8. 2. Participants have T2DM treated at the time of randomization with lifestyle measures (standardized diet and exercise program), and a. Metformin, or b. Basal insulin, or c. Metformin and basal insulin. If metformin is used, the dose must be ≥1000 mg/day and not more than the locally approved dose a. Doses of metformin and basal insulin must have been stable (±15% for basal insulin) for at least 90 days prior to Visit 1 and until Visit 3 3. Have HbA1c >17 yrs of age and Dx w/Type 2 Diabetes
Current use of Metformin > 1000 mg/day with or without Basal insulin (for at least 3 months)
A1c of > 6.5% to < 11% at screening visit (determined by central laboratory)
Body weight ≥50 kg and BMI of >85th percentile of the general age and gender-matched population for that country or region

Exclusion

Use of Short Acting/Bolus Insulins
Positive antibodies (GAD, MIAA, etc.)
On any type of weight lowering medications

AFREEZA INHALED INSULIN
INHALE-1: This is a study using Afrezza® (an Inhaled Insulin) Versus Rapid-acting Insulin Analog Injections, Both in Combination with a Basal Insulin, in Pediatric Subjects with Type 1 or Type 2 DM
Inclusion

Subject ≥4 years and <18 years of age
Clinical diagnosis of T1DM or T2DM (per the Investigator) and have been using insulin for at least 6 months for T1DM, or at least 3 months for T2DM
Treatment with basal-bolus insulin therapy delivered by multiple daily injections for at least 2 weeks
HbA1c ≥7.0% and ≤11.0%

Exclusion

Site Staff can determine as there are many

LADAXARIN Oral Medication for use in NEW ONSET Type 1
Type 1 New onset using a twice a day oral ladarixin in patients with new-onset type 1 diabetes and a low residual β-cell function at baseline.
Inclusion

Male and female patients aged 14-45 years, inclusive;
New-onset T1D (1st IMP dose within 100 days from 1st insulin administration);
Positive for at least one diabetes-related auto-antibody (anti-GAD; IAA, if obtained within 10 days of the onset of insulin therapy; IA-2 antibody; ZnT8);
Require, or has required at some time, insulin therapy through multiple daily injections (MDI) or Continuous Subcutaneous Insulin Infusion (CSII).
Fasting C peptide < 0.205nmol/L;

CAH Studies Spruce 203 and 204

203 Protocol- A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects with Classic Congenital Adrenal Hyperplasia
Inclusion

Has been on a stable dose of GC replacement ≥15 mg/day and ≤50 mg/day in HCe (see Table 1 for GC dose conversion to HCe) that does not vary in total daily dose from day to day for ≥3 months before screening without any evidence of non-adherence to the GC regimen during this period (stress dosing is allowed)
For subjects with the salt-wasting form of CAH, subject has been on a stable dose of mineralocorticoid replacement for ≥3 months before screening

Exclusion

Has a known or suspected diagnosis of any other known form of classic CAH (not due to

21-hydroxylase deficiency)

Has a history that includes bilateral adrenalectomy or hypopituitarism
Current treatment with dexamethasone as GC therapy for CAH
Prior treatment with dexamethasone is allowed as long as the transition to an alternative GC regimen (e.g., HC, prednisone, or prednisolone) has resulted in a stable dose of GC replacement for ≥3 months before screening.
Shows clinical signs or symptoms of adrenal insufficiency

204 Protocol-A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Reducing Supraphysiologic Glucocorticoid Use in Adult Subjects with Classic Congenital Adrenal Hyperplasia
Inclusion

Male and female subjects 18 to 55 years old, inclusive
Has a documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or elevated 17-OHP
Has been on a stable, supraphysiologic dose of GC replacement (defined as ≥30 mg/day and ≤50 mg/day in HCe) for ≥3 months before screening without any evidence of non-adherence to the GC regimen during this period (stress dosing is allowed). See Section 4.1.4.2 for calculation of HCe and Section 4.1.4.1 for further details.
For subjects with the salt-wasting form of CAH, subject has been on a stable dose of mineralocorticoid replacement for ≥3 months before screening
Agrees to convert from his/her existing GC regimen to the Sponsor’s standardized GC regimen (Section 4.1.4.3) at the start of the Glucocorticoid Conversion Period

Exclusion

Has a known or suspected diagnosis of any other known form of classic CAH (not due to

21- hydroxylase deficiency)

Has a history that includes bilateral adrenalectomy or hypopituitarism
Is not adherent to GC dosing during the Glucocorticoid Conversion Period (defined as taking <80% of expected doses as directed; stress dosing of GC is allowed)
Shows clinical signs or symptoms of adrenal insufficiency

LUMOS Growth Hormone
A 12-Month, Randomized, Open-Label Study of Daily Oral LUM-201 in Naïve-to-Treatment, Prepubertal Children with Growth Hormone Deficiency (GHD)

Inclusion

Have an established diagnosis of PGHD. Eligible subjects must be naïve-to-treatment and be prepubertal.
maximal GH response < 10 ng/mL
Have a historical (within the past 12 months) FASTING cortisol response > 14 µg/dL on the glucagon, ACTH or insulin test OR RANDOM Cortisol <7 ug/dl
At Screening, be age ≥ 3.0 years and age < 11.0 years for girls and < 12.0 years for boys.
Have bone age delayed by > 6 months by chronological age for this < 9.0 years and at least 1.0 yr delayed for subject older than 9yrs .
Have HT-SDS < -2.0.OR we can check mean parental height

Exclusion

Any medical or genetic condition which, in the opinion of the Investigator or MM, can be an independent cause of short stature and/or limit the response to exogenous growth factor treatment. (Examples: diabetes, named syndromes).
Use of any medication that, in the opinion of the Investigator and/or MM, can independently cause short stature or limit the response to exogenous growth factors (Example: glucocorticoids).
Evidence or history of an intracranial mass (e.g., pituitary tumor, craniopharyngioma).
SGA Diagnosis
Body weight < 5th % or gestational birth weight < 5th %

DEXCOM COACH Study
https://clinicaltrials.gov/ct2/show/NCT03340831?term=dexcom+coach&rank=1
Inclusion

Naïve to real-time CGM
Type 1 and/or insulin-requiring Type 2 diabetes
≥ 2 years old

Exclusion

Use of RT-CGM, within the past 12 months

Concomitant disease or condition that may compromise patient safety
Known (or suspected) significant allergy to medical grade adhesives
Dialysis

Medtronic CEP304 THIS STUDY REQUIRES A LOT OF INVOLVEMENT/WEEKLY UPLOADS/ETC. PLEASE TALK TO YOUR PYSICIAN TO LEARN MOREhttps://clinicaltrials.gov/ct2/show/NCT02748018?term=cep304&rank=1
Inclusion

Type 1 Diabetes patients aged 2-17 years of age diagnosed for at least 3 months
Pediatric Patients with Type 1 Diabetes Using Either Multiple Daily Injections or on Insulin Pump therapy with or without CGM
Using a minimum of 8 units of Insulin/day

Exclusion

Subject participated in any Closed Loop study in the past
Subject is unable to tolerate tape adhesive in the area of sensor placement
Subject is being treated for hyperthyroidism at time of screening

Janssen Type2 Canagliflozin Oral (Add-On therapy) https://clinicaltrials.gov/ct2/show/NCT03170518?term=28431754DIA3018&rank=1
Inclusion

Diagnosed with Type 2DM
HbA1c ≥ 6.5% and ≤ 10.5%
Diet & exercise alone
OR
Current 8-week stable dose of metformin alone (at least 1000 mg daily) or with Insulin
OR
Insulin alone for at least 8 weeks
Males and Females, 10-<18 years of age

Exclusion

Elevated LFT’S
Current use of Sulfonylureas, alpha glucosidase inhibitors, metiglinide, oral or injectable incretins or incretin mimetics or other antidiabetes, thiazolidinediones

A Research Study in Children With a Low Level of Hormone to Grow. Treatment is Somapacitan Once a Week Compared to Norditropin® Once a Day (REAL4)https://clinicaltrials.gov/ct2/show/NCT03811535?term=real+4&draw=2&rank=1
CriteriaInclusion Criteria:

Prepubertal children: a) Boys: Age more than or equal to 2 years and 26 weeks and less than 11.0 years at screening. Testis volume less than 4 ml. b) Girls: Age more than or equal to 2 years and 26 weeks and less than 10.0 years at screening. Tanner stage 1 for breast development (no palpable glandular breast tissue)
Confirmed diagnosis of growth hormone deficiency determined by two different growth hormone stimulation tests performed within 12 months prior to randomisation, defined as a peak growth hormone level of less than or equal to 10.0 ng/ml using the World Health Organisation (WHO) International Somatropin 98/574 standard
Impaired height defined as at least 2.0 standard deviations below the mean height for chronological age and gender at screening according to the standards of Center for Disease Control and Prevention
Impaired height velocity, defined as annualised height velocity below the 25th percentile for chronological age and gender according to the standards of Prader calculated over a time span of minimum 6 months and maximum 18 months prior to screening
Insulin-like Growth Factor-I (IGF-I) less than -1.0 SDS at screening, compared to age and gender normalized range measured at central laboratory
No prior exposure to growth hormone therapy or IGF-I treatment

Exclusion Criteria:

Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurements
Current inflammatory diseases requiring systemic corticosteroid treatment for longer than 2 consecutive weeks within the last 3 months prior to screening
Children requiring inhaled glucocorticoid therapy at a dose of greater than 400 μg/day of inhaled budesonide or equivalents for longer than 4 consecutive weeks within the last 12 months prior to screening
Diagnosis of attention deficit hyperactivity disorder
Concomitant administration of other treatments that may have an effect on growth, e.g. but not limited to methylphenidate for treatment of attention deficit hyperactivity disorder
Prior history or presence of malignancy including intracranial tumours

UCSF DIABETES RESEARCH
We partner and refer our children to UCSF for research
These are the current projects at UCSF: https://madisonclinic.ucsf.edu/research/study-participation-opportunities

PLEASE TALK TO ONE OF US IF YOU ARE INTERESTED IN PARTICIPATING IN A CLINICAL STUDY