Pilot Study of ALLN-177 in Adult and Pediatric (Child) Subjects Aged 12 Years or Older with Enteric (relates to the intestines) or Primary Hyperoxaluria (oxalate in the urine) and Hyperoxalemia (oxalate in the blood)
- Conditions
- Enteric or primary hyperoxaluria and hyperoxalemiaTherapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2017-003547-38-DE
- Lead Sponsor
- Allena Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 20
1. Signed and dated the Institutional Review Board (IRB)/Ethics Committee (EC) approved informed consent form (ICF) or Assent before undergoing any Screening
2. Aged 12 or older with body weight =35kg
3. Has diagnosis of primary hyperoxaluria determined by standard diagnostic methods, or a history of enteric hyperoxaluria (urinary oxalate excretion = 40 mg/24h [ = 0.45 mmol/24h] normalized for body surface area in children, that is associated with a known underlying enteric disorder associated with malabsorption such as bariatric surgery, Crohn’s disease, short bowel syndrome, or other malabsorption syndrome)
- Note: Subjects with estimated glomerular filtration rate (eGFR) >15 mL/min/1.73m2 who have suspected enteric hyperoxaluria but no elevated 24-hour UOx should obtain a pre-screening 24-hour urine collection to determine UOx level prior to undergoing full screening procedures after providing appropriate consent
- Note: Subjects with enteric hyperoxaluria on dialysis or with eGFR =15 mL/min/1.73m2 must have an elevated UOx level documented in the medical record.
- Note: 24-hour urine collections are not obtained in subjects on dialysis
4. In subjects with eGFR >15 mL/ min/1.73m2, UOx at Screening = 40 mg/24h ( = 0.45 mmol/24h; normalized to body surface area in children) on an adequate collection (i.e., appropriate ratio of creatinine [mg]/body weight [kg] for gender)
- Note: 24-hour urine collections are not obtained in subjects with eGFR =15 mL/min/1.73m2 or on dialysis
- Note: A pre-screen 24-hour UOx obtained within 6 weeks prior to Screening can be used to satisfy this screening UOx entry criterion
5. In subjects with enteric hyperoxaluria, has estimated glomerular filtration rate (eGFR) <45 mL/ min/1.73m2 at Screening, or has been on dialysis for greater than 3 months but less than 2 years. The number of subjects with eGFR <15 mL/ min/1.73m2 or on dialysis will be limited to 25% of total enrollment
6. In subjects with enteric hyperoxaluria, documented history of elevated plasma oxalate (>5 µmol/L)
- Note: Subjects with no documented elevated plasma oxalate who are interested in participating in this study should undergo a pre-screening plasma oxalate test prior to undergoing full screening procedures to determine whether they have elevated plasma oxalate, after providing appropriate consent. For subjects on hemodialysis, the plasma samples will be obtained immediately prior to dialysis, optimally following the longer interval between dialysis treatments.
7. In subjects with enteric hyperoxaluria, plasma oxalate >5 µmol/L at screening
- Note: A plasma oxalate obtained within 6 weeks prior to Screening can be used to satisfy this entry criterion
8. For subjects taking concomitant medication for management of kidney stone risk factors (e.g., pyridoxine, thiazides, citrate supplements, allopurinol): has been on a stable dose regimen for >8 weeks prior to Screening, with no changes in dosing (dose level or dosing frequency) anticipated during the remainder of the study
9. For female subjects: Is either medically incapable of pregnancy (e.g., has undergone hysterectomy or tubal ligation or has experienced prolonged [= 1 year prior to Screening] amenorrhea) or, if a woman of childbearing potential, has a negative Screening serum pregnancy test, is not pregnant or nursing at Screening, and agrees to use an effective (approved by the Investigator) method of birth control for the duration of the study
10. Subjects on dialysis, must be
1. Is unable or unwilling to discontinue Vitamin C supplementation at Screening and for the duration of the study
2. Any clinically significant findings during Screening, any ongoing clinically significant illness requiring changes in management within 1 month prior to or during Screening (e.g., flare of inflammatory bowel disease), or initiation of dialysis or any surgical/invasive procedure is planned during the study
3. Malignancy or treatment for malignancy within 12 months prior to screening with the exception of localized basal cell or squamous cell skin cancer.
- Note: Subjects in remission on stable doses of chronic suppressive or maintenance therapies are not excluded
4. Has active autoimmune disorder or other condition requiring therapy with high doses of systemic steroids (i.e., >10 mg/day prednisone or equivalent) or intensification of other immunosuppressant therapy within 1month prior to or during Screening
- Note: Subjects on stable chronic or maintenance doses of steroids or other immunosuppressant drugs, including transplant recipients, are not excluded
5. Has participated in any other ALLN-177 clinical study, or participation in another drug or device clinical trial within 30 days prior to or during Screening
6. Is not, per Investigator judgment, an ideal clinical trial candidate due to a personal issue (e.g., unwillingness/inability to comply with protocol) or medical condition (e.g., mental illness, laboratory abnormalities) that is likely to impede successful study completion
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method