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A Study of Oral ARD-101 in Patients With Prader-Willi Syndrome

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ClinicalTrials.gov Identifier: NCT05153434
Recruitment Status : Recruiting
First Posted : December 10, 2021
Last Update Posted : July 19, 2022
Children's Hospital Colorado
Stanford University
Information provided by (Responsible Party):
Aardvark Therapeutics, Inc.

Brief Summary:
A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Efficacy of ARD-101 in Patients with Prader-Willi Syndrome

Condition or disease Intervention/treatment Phase
Prader-Willi Syndrome Drug: ARD-101 Phase 2

Detailed Description:
This is a Phase 2, open-label study to investigate the effects of ARD-101 in subjects with Prader-Willi Syndrome. This study has a planned enrollment of 12 subjects and will be conducted in a single center in the United States.The study will consist of a Screening Period (up to 28 days), a Treatment Period (28 days), and a Follow-up Period (End-of-Study Visit within 14 days after receiving the last dose of ARD-101). The screening procedures will be initiated upon completion of the informed consent process. Following completion of screening procedures and confirmation of eligibility, subjects will be enrolled to receive ARD-101 in an outpatient setting and will be instructed to visit the clinical center periodically for safety and efficacy assessments.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Efficacy of ARD-101 in Patients With Prader-Willi Syndrome
Actual Study Start Date : May 27, 2022
Estimated Primary Completion Date : March 31, 2023
Estimated Study Completion Date : May 31, 2023

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: ARD-101
Dose 200 mg of ARD-101, twice daily for 28 days
Drug: ARD-101
Twice Daily, Oral Administration

Primary Outcome Measures :
  1. Incidence of Treatment-Emergent Adverse Events (TEAE) [ Time Frame: Baseline to Day 28 ]
    The incidence of treatment-emergent adverse events (TEAE) during the treatment period

Secondary Outcome Measures :
  1. Efficacy Evaluation of Hyperphagia in Prader-Willi Syndrome [ Time Frame: Baseline, Day 15, Day 28 ]
    Quantitative evaluation of hyperphagia via the Hyperphagia Questionnaire for Clinical Trials (HQ-CT). Score will range from 0 (no hyperphagia behaviors) to 36 (most severe hyperphagia behaviors)

  2. Relative Change in Body Weight (%) [ Time Frame: Run-in Visit (baseline), Day 28 ]
    The percent total weight change at the end of treatment from baseline

Other Outcome Measures:
  1. Change in Insulin Resistance [ Time Frame: Run-in Visit (baseline), Day 28 ]
    Change in insulin resistance by determining homeostatic model assessment for insulin resistance (HOMA-IR) at the end of treatment compared to baseline

  2. Change in Blood Lipid Concentrations [ Time Frame: Run-in Visit (baseline), Day 28 ]
    The change in blood lipid concentrations (total cholesterol, triglyceride, high density lipoprotein cholesterol, and low- density lipoprotein cholesterol) at the end of treatment from the baseline

  3. Body Fat Percentage [ Time Frame: Run-in Visit (baseline), Day 28 ]
    Body fat percentage measured by bioelectrical impedance scale

  4. The Change in Body Composition [ Time Frame: Run-in Visit (baseline), Day 28 ]
    The change in body composition based on evaluation of dual-energy X-ray absorptiometry (DEXA) scans at the end of treatment compared to baseline

  5. Effect on Psychiatric Status [ Time Frame: Baseline to day 28 ]
    Effect on psychiatric status through screening presence of suicidal ideation and behavior in addition to screening for degree of depression.[Columbia-Suicide Severity Rating Scale (C-SSRS), as assessed by caregiver, and Patient Health Questionnaire (PHQ)-9 Questionnaires)

  6. The Change in Body-Mass Index (BMI) [ Time Frame: Run-in Visit (baseline), Day 28 ]
    The change in body-mass index (BMI) at the end of treatment from the baseline as well as 28 days after end of treatment

  7. The Change in Waist Circumference [ Time Frame: Run-in Visit (baseline), Day 28, Day 42 ]
    The change in waist circumference at the end of treatment from the baseline as well as 14 days after end of treatment

Information from the National Library of Medicine

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Ages Eligible for Study:   17 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Male and female subjects, 17-65 years of age
  • Provide voluntary, written informed consent (parent(s) / legal guardian(s) of participant); provide voluntary, written assent (participants, as appropriate)
  • PWS due to chromosome 15 micro-deletion, maternal uniparental disomy, or imprinting defect, confirmed by fluorescent in situ hybridization, chromosomal microarray, and/or methylation studies
  • Body weight of at least 50 kg with BMI ≥27 kg/m²
  • A HQ-CT score >10
  • If a subject has a diagnosis of type 2 diabetes, the following criteria must be met:

    1. Hemoglobin A1c (HbA1c) <7.5% not being managed with insulin. Patients taking glucagon-like peptide (GLP)-1 analogues (exenatide or liraglutide) must have been on stable dose for greater than 3 months.
    2. Fasting plasma glucose <140 mg/dL during the Screening Period
    3. No history of ketoacidosis or hyperosmolar coma
  • Stable or well-controlled blood pressure (BP) and vital signs. Specifically: Vital signs after 5 minutes resting in seated position (feet flat on floor, back supported):

    1. 95 mmHg <systolic blood pressure (SBP) <160 mmHg
    2. 45 mmHg <diastolic blood pressure (DBP) <100 mmHg
    3. 40 bpm <heart rate (HR) <100 bpm
  • Stable body weight for ~2 months (self or guardian-reported loss/gain within ± 10%) prior to enrollment
  • Standard 12-lead ECG parameters after 10 minutes resting in supine position in the following ranges; 120 ms <PR <220 ms, QRS <120 ms, QTc ≤430 ms if male, ≤450 ms if female and normal ECG tracing unless the Investigator considers an ECG abnormality to be not clinically relevant.
  • Parent or guardian is able to communicate well with the investigator, to understand and comply with the requirements of the study, and be able to understand and sign the written informed consent. Assent is to be provided for the patient who cannot consent for himself or herself
  • Results of screening clinical laboratory tests [complete blood count (CBC) with differential and platelets and chemistry profile] and vital signs must be within normal range or, if outside of the normal range, must be accepted by the investigator and sponsor as not clinically significant
  • Females of non-childbearing potential, defined as surgically sterile (status post hysterectomy, bilateral oophorectomy, or bilateral tubal ligation) or post-menopausal for at least 12 months (and confirmed with a screening FSH level in the post-menopausal lab range), do not require contraception during the study. This may also apply to subjects with documented hypogonadism with and without estrogen replacement therapy as per investigator judgement. All other females of child-bearing potential must agree to use contraception as outlined in the protocol
  • Males with female partners of childbearing potential must agree to a double barrier method if they become sexually active during the study and for 90 days following the study.
  • Patients must be on a stable dose of any allowed chronic concomitant medications while participating in the study, as described in protocol. This is defined as no changes in medication or dose for at least 30 days prior to Day 1 Stable concomitant usage (>3 months) of medications commonly used in PWS patients are allowed

Exclusion Criteria:

  • Use of weight loss agents, including herbal medication, within 3 months prior to enrollment
  • Diagnosis of schizophrenia, bipolar disorder, personality disorder, or other DSM-III disorders which the investigator believes will interfere significantly with study compliance
  • A PHQ-9 score of ≥10
  • Any suicidal ideation of type 4 or 5 on the C-SSRS
  • Clinically significant illness in the 8 weeks prior to enrollment
  • History of clinically significant bleeding disorders
  • Current, clinically significant liver, renal, pulmonary, cardiac, oncologic, or gastrointestinal (GI) disease
  • Diagnosis of type 1 diabetes mellitus or other active endocrine disorders (e.g., Cushing syndrome, or thyroid dysfunction except if on stable adequate thyroid or glucocorticoid replacement supplement)
  • Liver disease or liver injury as indicated by abnormal liver function tests, SGOT (aspartate aminotransferase (AST)), alkaline phosphatase, or serum bilirubin (> 1.5 x upper limit of normal (ULN) for any of these tests) or history of hepatic cirrhosis
  • History or presence of impaired renal function as indicated by clinically significantly abnormal creatinine, blood urea nitrogen (BUN), or urinary constituents (e.g., albuminuria) or moderate to severe renal dysfunction as defined by the Cockroft Gault equation (< 50 mL/min)
  • Significant history of abuse of drugs within 1 year prior to enrollment or a positive Drugs of Abuse (DOA) test at screening
  • History of alcohol abuse within 1 year prior to enrollment or currently drinks in excess of 21 units per week (3 servings or units/day)
  • Caffeine consumption exceeding 6 cups of caffeinated tea/coffee (or equivalent) per day
  • Participation in any clinical study with an investigational drug/device within 1 month prior to enrollment
  • Serious adverse reaction or significant hypersensitivity to any drug
  • Clinically significant blood loss or blood donation > 500 mL within 3 months prior to enrollment
  • Inadequate venous access
  • History of significant drug hypersensitivity or anaphylaxis
  • Any condition that the investigator or primary physician believes may not be appropriate for participating the study

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05153434

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Contact: Andreas Niethammer, MD, PhD 858-349-4820 AndreasNiethammer@aardvarktherapeutics.com

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United States, California
Stanford University Recruiting
Palo Alto, California, United States, 94304
Contact: Diane Stafford, MD    650-721-1811    dejs@stanford.edu   
United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Shawn E. McCandless, MD         
Sponsors and Collaborators
Aardvark Therapeutics, Inc.
Children's Hospital Colorado
Stanford University
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Responsible Party: Aardvark Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT05153434    
Other Study ID Numbers: AARD-203
First Posted: December 10, 2021    Key Record Dates
Last Update Posted: July 19, 2022
Last Verified: July 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Prader-Willi Syndrome
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Nutrition Disorders