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Phase 2 Study to Evaluate Safety & Efficacy of RM-131 Administered to Patients With Diabetic Gastroparesis

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Rhythm Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT01571297
First received: April 2, 2012
Last updated: November 6, 2014
Last verified: November 2014
  Purpose

The purpose of this study is to evaluate the effects of RM-131 on gastric emptying, gastroparesis symptoms, and the safety and tolerability of RM-131 compared to placebo in patients with Type 1 and Type 2 diabetes mellitus and gastroparesis. The study is designed to evaluate the efficacy and safety of multiple dose regimens of RM-131. Study drug (RM-131 and placebo) will be administered subcutaneously in a blinded fashion.


Condition Intervention Phase
Diabetes Mellitus
Diabetes Mellitus Complications
Gastroparesis
Drug: RM-131
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 2, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of RM-131 Administered to Patients With Diabetic Gastroparesis

Resource links provided by NLM:


Further study details as provided by Rhythm Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • Effect of RM-131 on gastric emptying time [ Time Frame: Screening and Day 28 ] [ Designated as safety issue: No ]
    Change from baseline in gastric half-emptying time (t½)


Secondary Outcome Measures:
  • Effect of RM-131 on symptoms of gastroparesis [ Time Frame: Baseline, daily for 28 days, and Day 35 ] [ Designated as safety issue: No ]
    Longitudinal change from baseline over time in daily symptom scores from a self administered daily symptom diary

  • Safety and tolerability of RM-131 [ Time Frame: From Screening through Day 35 ] [ Designated as safety issue: Yes ]
    Assessment of adverse events and clinical laboratory evaluations


Enrollment: 204
Study Start Date: April 2012
Study Completion Date: September 2013
Primary Completion Date: August 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: RM-131 Drug: RM-131
Double blind RM-131 will be studied with various doses and regimens from 10 to 100 μg for 35 days.
Placebo Comparator: Placebo Drug: Placebo
Placebo given subcutaneously for 35 days

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Able to provide written informed consent prior to any study procedures and be willing and able to comply with study procedures.
  • Type 1 or Type 2 diabetes mellitus with HbA1c ≤11% at screening.
  • Diabetic gastroparesis defined as at least 3 months history of symptoms suggestive of gastroparesis on an ongoing basis.
  • Average Gastroparesis Cardinal Symptom Index Daily Diary (GCSI-DD) > 2.6 during Visit 2.
  • History of nausea and/or vomiting/emesis at least once a week during the 2 weeks prior to Visit 1.
  • Delayed gastric emptying confirmed at screening by abnormal gastric emptying breath test (GEBT), defined as half-emptying time (t½) > 79 minutes.
  • Stable concomitant medications defined as no changes in regimen for at least 2 weeks prior to Visit 2.
  • No use of metoclopramide, erythromycin or anti-emetics for at least 2 weeks prior to Visit 2.
  • Body mass index > 18 kg/m2.
  • Female patients must have negative serum or urine pregnancy tests and must not be lactating. For females able to bear children, a hormonal (i.e., oral, implantable, or injectable) and single-barrier method, or a double-barrier method of birth control must be used throughout the study. Female patients unable to bear children must have this documented in the electronic case report form (eCRF) (i.e., tubal ligation, hysterectomy, or post-menopausal [defined as a minimum of one year since the last menstrual period]). Post-menopausal status will be confirmed by FSH.

Exclusion Criteria:

  • Currently receiving parenteral feeding; presence of a nasogastric or other enteral tube for feeding or decompression.
  • History of gastric surgery such as fundoplication, gastrectomy, gastric pacemaker placement, vagotomy, bariatric procedure.
  • History of pyloric injection of botulinum toxin within 6 months of screening.
  • Persistent daily vomiting.
  • Patients with clinical suspicion of upper gastrointestinal obstruction must have been evaluated per standard of care, and obstruction ruled out before screening.
  • Currently taking opiates.
  • Currently taking GLP-1 and amylin analogs.
  • Allergic or intolerant of egg, wheat, milk or algae, as these are components of the GEBT study meal.
  • History of anorexia nervosa, binge-eating or bulimia within 5 years.
  • ALT or AST > 2 X upper limit of normal during screening.
  • History of intestinal malabsorption or pancreatic exocrine disease.
  • Requires hemodialysis or has end-stage renal disease.
  • History of human immunodeficiency virus (HIV) infection.
  • Clinically significant neurologic or psychiatric disorders which are likely to impact compliance with protocol requirements.
  • Poor venous access or inability to tolerate venipuncture.
  • Participation in a clinical study within the 30 days prior to dosing in the present study.
  • Any other reason, which in the opinion of the Investigator, including renal, hepatic or cardiopulmonary disease, or significant acute ECG abnormalities that would confound proper interpretation of the study or expose a patient to unacceptable risk.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01571297

  Show 30 Study Locations
Sponsors and Collaborators
Rhythm Pharmaceuticals, Inc.
Investigators
Study Director: Chief Development Officer Rhythm Pharmaceuticals
  More Information

No publications provided

Responsible Party: Rhythm Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT01571297     History of Changes
Other Study ID Numbers: RM-131-004
Study First Received: April 2, 2012
Last Updated: November 6, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Rhythm Pharmaceuticals, Inc.:
Diabetes Mellitus
Delayed Gastric Emptying
Gastroparesis
Gastrointestinal Motility Disorder

Additional relevant MeSH terms:
Gastroparesis
Diabetes Complications
Diabetes Mellitus
Digestive System Diseases
Endocrine System Diseases
Gastrointestinal Diseases
Glucose Metabolism Disorders
Metabolic Diseases
Neurologic Manifestations
Paralysis
Signs and Symptoms
Stomach Diseases

ClinicalTrials.gov processed this record on November 27, 2014