Prader-Willi Syndrome
Conditions
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
Detailed description
This is a Phase 2, open-label study to investigate the effects of ARD-101 in subjects with Prader-Willi Syndrome. 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. ARD-101 will be provided as a fixed dose of 200 mg BID for 28 days (Group 1) and then in a dose escalation of 1 week at 400 mg BID, 1 week at 600 mg BID, then 2 weeks at 800 mg BID (Group 2).
Interventions
DRUGARD-101
Oral administration of ARD-101 taken BID (twice daily) for 28 days.
Sponsors
Aardvark Therapeutics, Inc.
Children's Hospital Colorado
Stanford University
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
17 Years to 65 Years
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 * BMI ≥ 18.5 kg/m² * Qualifying HQ-CT score
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 * Clinically significant illness in the 8 weeks prior to enrollment * 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) * 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)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Treatment Emergent Adverse Events (TEAE) | Adverse Events were collected from time of informed consent through end of study; approximately 70 days (28 days of screening, 28 days of treatment, 14 days post treatment follow up). | The incidence of treatment emergent adverse events (TEAE) reported through the 28 days of treatment and the 14 days post treatment phase. TEAEs were those events occurring after treatment began. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy Evaluation of Hyperphagia in Prader-Willi Syndrome | Baseline, Day 15, Day 28 | Quantitative evaluation of hyperphagia via the Hyperphagia Questionnaire for Clinical Trials (HQ-CT). The HQCT is a 9-item hyperphasia questionnaire which is validated for use in PWS clinical trials. Score will range from 0 (no hyperphagia behaviors) to 36 (most severe hyperphagia behaviors) |
| Change in Body Weight | Baseline to day 28 | Total weight change at the end of treatment (day 28) from baseline |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORShawn McCandless, MD
Children's Hospital Colorado
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Categorical <=18 years | 2 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 17 Participants |
| Age, Continuous | 23.8 years STANDARD_DEVIATION 7.81 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 6 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 1 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 16 Participants |
| Region of Enrollment United States | 6 participants |
| Sex: Female, Male Female | 12 Participants |
| Sex: Female, Male Male | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 13 | 0 / 6 |
| other Total, other adverse events | 8 / 13 | 5 / 6 |
| serious Total, serious adverse events | 0 / 13 | 0 / 6 |
Outcome results
None listed